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class="max-w-full"><time class="tex-sm " dateTime="2024-08-21T11:00:00.000">August 21, 2024</time><div class="pb-2"><div><span class="text-md "><span class="">By </span><a class="mr-1 text-author hover:text-primary" href="/authors/anjali-pradhan-ms">Anjali Pradhan, MS</a></span><span class="text-md "><span class=""><br/></span><a class="mr-1 text-author hover:text-primary" href="/authors/noah-stansfield">Noah Stansfield</a></span></div></div><div class="flex flex-wrap sm:flex-nowrap items-center w-fit my-2"><div class="flex items-center w-fit h-[22px] mr-4 px-2 bg-primary text-white text-xs"><em>Commentary</em></div><div class="flex items-center w-fit h-[22px] mr-4 px-2 bg-primary text-white text-xs"><em>Article</em></div></div><div class="mt-4"><div class="mt-2 flex items-center max-w-fit"><button title="Using Single Cell Sequencing for Gene Editing Quality Control" aria-label="facebook" class="react-share__ShareButton" 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c6.1,0,10.1,4.4,10.1,9.2c0,6.3-3.5,11-8.6,11c-1.7,0-3.4-0.9-3.9-2c0,0-0.9,3.7-1.1,4.4c-0.3,1.2-1,2.5-1.6,3.4 c1.4,0.4,3,0.7,4.5,0.7c8.8,0,16-7.2,16-16C48,23.2,40.8,16,32,16z" fill="white"></path></svg></button><button aria-label="email" class="react-share__ShareButton" style="background-color:transparent;border:none;padding:0;font:inherit;color:inherit;cursor:pointer"><svg viewBox="0 0 64 64" width="32" height="32"><circle cx="32" cy="32" r="31" fill="#7f7f7f"></circle><path d="M17,22v20h30V22H17z M41.1,25L32,32.1L22.9,25H41.1z M20,39V26.6l12,9.3l12-9.3V39H20z" fill="white"></path></svg></button><a class="print-wrap flex justify-center items-center cursor-pointer"><svg id="print" xmlns="http://www.w3.org/2000/svg" width="24" height="24" fill="currentColor" class="print bi bi-printer" viewBox="0 0 16 16"> <path d="M2.5 8a.5.5 0 1 0 0-1 .5.5 0 0 0 0 1z"></path> <path d="M5 1a2 2 0 0 0-2 2v2H2a2 2 0 0 0-2 2v3a2 2 0 0 0 2 2h1v1a2 2 0 0 0 2 2h6a2 2 0 0 0 2-2v-1h1a2 2 0 0 0 2-2V7a2 2 0 0 0-2-2h-1V3a2 2 0 0 0-2-2H5zM4 3a1 1 0 0 1 1-1h6a1 1 0 0 1 1 1v2H4V3zm1 5a2 2 0 0 0-2 2v1H2a1 1 0 0 1-1-1V7a1 1 0 0 1 1-1h12a1 1 0 0 1 1 1v3a1 1 0 0 1-1 1h-1v-1a2 2 0 0 0-2-2H5zm7 2v3a1 1 0 0 1-1 1H5a1 1 0 0 1-1-1v-3a1 1 0 0 1 1-1h6a1 1 0 0 1 1 1z"></path></svg></a></div><style> .print-wrap { width: 32px; height: 32px; background: #7F7F7F; border-radius: 100%; } .print { background: #7F7F7F; color: white; padding: 2px; border-radius: 100%; } </style></div></div><div class="flex justify-between gap-4"></div></div><p class="py-2 mb-2 text-sm italic text-gray-600">Anjali Pradhan, MS, the chief product officer at Mission Bio discussed the company’s Genome Editing Solution. </p><div class="py-2"><div class="blockText_blockContent__TbCXh"><div class=""><div style="width:auto%;float:left;max-width:525px;margin:0 1.5rem 1.5rem 0;clear:both;cursor:" class=" figure"><div class="flex-none relative text-center"><span style="box-sizing:border-box;display:inline-block;overflow:hidden;width:initial;height:initial;background:none;opacity:1;border:0;margin:0;padding:0;position:relative;max-width:100%"><span style="box-sizing:border-box;display:block;width:initial;height:initial;background:none;opacity:1;border:0;margin:0;padding:0;max-width:100%"><img style="display:block;max-width:100%;width:initial;height:initial;background:none;opacity:1;border:0;margin:0;padding:0" alt="" aria-hidden="true" src="data:image/svg+xml,%3csvg%20xmlns=%27http://www.w3.org/2000/svg%27%20version=%271.1%27%20width=%27300%27%20height=%27300%27/%3e"/></span><img alt="Anjali Pradhan, MS, the chief product officer at Mission Bio" title="Anjali Pradhan, MS, the chief product officer at Mission Bio" src="data:image/gif;base64,R0lGODlhAQABAIAAAAAAAP///yH5BAEAAAAALAAAAAABAAEAAAIBRAA7" decoding="async" data-nimg="intrinsic" style="position:absolute;top:0;left:0;bottom:0;right:0;box-sizing:border-box;padding:0;border:none;margin:auto;display:block;width:0;height:0;min-width:100%;max-width:100%;min-height:100%;max-height:100%;object-fit:contain"/><noscript><img alt="Anjali Pradhan, MS, the chief product officer at Mission Bio" title="Anjali Pradhan, MS, the chief product officer at Mission Bio" srcSet="/_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fgenetherapy%2F57aa15f32df153ae83c1b698050f97a0150c1355-300x300.jpg%3Ffit%3Dcrop%26auto%3Dformat&w=384&q=75 1x, /_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fgenetherapy%2F57aa15f32df153ae83c1b698050f97a0150c1355-300x300.jpg%3Ffit%3Dcrop%26auto%3Dformat&w=640&q=75 2x" src="/_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fgenetherapy%2F57aa15f32df153ae83c1b698050f97a0150c1355-300x300.jpg%3Ffit%3Dcrop%26auto%3Dformat&w=640&q=75" decoding="async" data-nimg="intrinsic" style="position:absolute;top:0;left:0;bottom:0;right:0;box-sizing:border-box;padding:0;border:none;margin:auto;display:block;width:0;height:0;min-width:100%;max-width:100%;min-height:100%;max-height:100%;object-fit:contain" loading="lazy"/></noscript></span></div><div id="image-caption" class="text-gray-500 italic"><div class="blockText_blockContent__TbCXh"><p class="pb-2">Anjali Pradhan, MS</p></div></div><div class="top-[-100%] block w-[1px] transition-opacity duration-500 ease-in-out opacity-0 overflow-hidden"><img class="m-auto absolute inset-0 max-w-[0%] max-h-[0%] border-[3px] border-solid border-white shadow-[0px_0px_8px_rgba(0,0,0,0.3)] box-border transition ease-in-out duration-500" src="https://cdn.sanity.io/images/0vv8moc6/genetherapy/57aa15f32df153ae83c1b698050f97a0150c1355-300x300.jpg?fit=crop&auto=format"/></div></div><style> #image-caption p{ font-size: 12px; max-width: 525px; margin: 0 auto; text-align: center; } </style></div><p class="pb-2">Off-target edits can pose safety risks during the use of gene editing therapies. Although, detecting these edits precisely and accurately can prove difficult with traditional bulk DNA sequencing.</p><p class="pb-2">Mission Bio's Tapestri Genome Editing Solution, which uses DNA sequencing to detect gene edits at a single cell level, is intended to overcome this challenge. Notably, the company presented on Tapestri at <a rel="nofollow" target="_self" href="https://www.cgtlive.com/conferences/american-society-for-gene-cell-therapy-asgct-">the American Society of Gene & Cell Therapy (ASGCT) 27th Annual Meeting</a>, held May 7 to 10, 2024, in Baltimore, MD. At the conference, <em>CGTLive® </em>interviewed Anjali Pradhan, MS, the chief product officer at Mission Bio, to learn more.</p><h3 class="text-xl pb-4 pt-2"><strong><em>CGTLive:</em> Can you give some background about what you presented at ASGCT this year?</strong></h3><p class="pb-2"><strong>Anjali Pradhan, MS:</strong> We were really excited that we have launched our genome editing solution. What this is, is a genome editing solution that helps you quality control your edits and your genes as you do a CRISPR edit or a prime edit, using various editing tools. Our solution is a unique solution because it's the only DNA single cell solution that allows you to do on-targets, off-targets, translocations, measures zygosity of edits, as well as—we're excited to introduce—looks at copy number variations. Our whole product solution is available for users who are using prime editing tools, etc. and looking at what the efficiency of gene editing is, so that they can safety control their product as it goes into a patient.</p><h3 class="text-xl pb-4 pt-2"><strong>What were the key points you presented?</strong></h3><p class="pb-2">Mission Bio is a DNA single cell company. Our tools are an instrument platform that is based on microfluidics. We have also worked with the National Institute of Standards and Technology (NIST) Consortium. Our collaboration has really helped us to emphasize the value of looking at these edits at a single cell level. Our presentation was focused on highlighting when they compared in an interlab study what the value was of our Tapestri platform, our instrument for measuring single cell edits. What it highlighted is looking at attributes like single cell zygosity, looking at translocation events, off-target edits, and on-target edits.</p><h3 class="text-xl pb-4 pt-2"><strong>How would you summarize the big picture implications that doctors and the healthcare community should take away from this?</strong></h3><p class="pb-2">What we've seen is in the industry today there's a lot of excitement with new editing technologies—there's various new technologies coming out. But what you are always concerned about is: What is the safety of those edits? How can you be sure that your edit is as it was intended to be and if it's going in the right place? That's a huge concern to doctors, especially with various editing efficiencies, etc. How do you make sure you're doing the right thing? The FDA also requires you to report out where those edits are and what's the frequency of these edits. So you need the right single cell level tool to measure that accuracy. That's where Mission Bio can really help you to do that quality control (QC), to make sure again that all the critical attributes are safe, your editing efficiency is safe, and you can feel much more confident about where those edits are made.</p><h3 class="text-xl pb-4 pt-2"><strong>Have there been any major challenges in this work so far?</strong></h3><p class="pb-2">I think the challenge continues to be in measuring the safety. But what is continually exciting for the community is as the various forms of editing continue to expand, there will always be checks and balances required to measure the safety. As such, I think as you think about more tools in the editing space, you can also see the scope of looking at those tools at a single cell level. The highlight is you have to use your cell as your most accurate level at a macro level. When you're using other tools like bulk sequencing or PCR, you're measuring at a bulk level, so you don't get the granularity when cells as we know are heterogeneous. The challenge is the heterogeneity of cells and this is why you should always use a single cell and then DNA single cell level methods to help measure the QC.</p><h3 class="text-xl pb-4 pt-2"><strong>Is there anything else you would like to share?</strong></h3><p class="pb-2">I think our goal as a company with Mission Bio is with our DNA single cell tools and technology, we are enabling the community to do safety and efficacy of gene editing. We also have solutions for characterizing cell therapeutic products. Our goal is to work with the community to really build out the right tools. Our vision is to become sort of more of a standardization practice, partnering with institutes like NIST and the FDA, where we can really help ensure safety and efficacy, especially because so many of these edits, if they're not done and measured correctly, can lead to genotoxicity. That's what we want to avoid and enables safety and more precision medicine.</p><p class="pb-2"><strong><em>This transcript has been edited for clarity.</em></strong></p><p class="pb-2"><a rel="nofollow" target="_self" href="https://www.cgtlive.com/conferences/american-society-for-gene-cell-therapy-asgct-"><strong><em>Click here to view more coverage of the 2024 ASGCT Annual Meeting.</em></strong></a></p><h6 class="text-sm pb-4 pt-2"></h6><p class="pb-2"></p><p class="pb-2"></p></div></div><div class="flex items-center lg:w-3/4 mb-4 pb-12"></div><div class="jsx-19ede9f0a5a45918 py-4 relative bg-primary md:px-8 -ml-6 xs:ml-0 w-screen xs:w-auto"><div class="jsx-19ede9f0a5a45918 px-4 sm:px-0"><div class="flex justify-between items-center py-1 space-x-4 border-0 select-none sm:border-b border-secondary"><div class="text-3xl text-white text-lg sm:text-3xl">Recent Videos</div></div></div><div style="scroll-snap-type:none" class="jsx-19ede9f0a5a45918 flex items-start overflow-x-auto space-x-4 py-4 relative mx-auto w-full pl-4"><a id="" class="w-[200px] h-fit space-y-3 flex-none select-none no-underline" style="scroll-snap-align:center;text-decoration:none" href="/view/schett-exploring-car-t-autoimmune-disease"><div class="w-full shadow-md shadow-gray-800 overflow-hidden relative bg-white aspect-video"><span style="box-sizing:border-box;display:block;overflow:hidden;width:initial;height:initial;background:none;opacity:1;border:0;margin:0;padding:0;position:absolute;top:0;left:0;bottom:0;right:0"><img alt="Georg Schett, MD, vice president research and chair of internal medicine at the University of Erlangen – Nuremberg" title="Georg Schett, MD, vice president research and chair of internal medicine at the University of Erlangen – Nuremberg" src="data:image/gif;base64,R0lGODlhAQABAIAAAAAAAP///yH5BAEAAAAALAAAAAABAAEAAAIBRAA7" decoding="async" data-nimg="fill" style="position:absolute;top:0;left:0;bottom:0;right:0;box-sizing:border-box;padding:0;border:none;margin:auto;display:block;width:0;height:0;min-width:100%;max-width:100%;min-height:100%;max-height:100%;object-fit:contain"/><noscript><img alt="Georg Schett, MD, vice president research and chair of internal medicine at the University of Erlangen – Nuremberg" title="Georg Schett, MD, vice president research and chair of internal medicine at the University of Erlangen – Nuremberg" sizes="100vw" srcSet="/_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fgenetherapy%2Fa9442bca6eabb1cffae5b9f82172d8d8b318c0dc-300x425.jpg%3Ffit%3Dcrop%26auto%3Dformat&w=640&q=30 640w, /_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fgenetherapy%2Fa9442bca6eabb1cffae5b9f82172d8d8b318c0dc-300x425.jpg%3Ffit%3Dcrop%26auto%3Dformat&w=750&q=30 750w, /_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fgenetherapy%2Fa9442bca6eabb1cffae5b9f82172d8d8b318c0dc-300x425.jpg%3Ffit%3Dcrop%26auto%3Dformat&w=828&q=30 828w, /_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fgenetherapy%2Fa9442bca6eabb1cffae5b9f82172d8d8b318c0dc-300x425.jpg%3Ffit%3Dcrop%26auto%3Dformat&w=1080&q=30 1080w, /_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fgenetherapy%2Fa9442bca6eabb1cffae5b9f82172d8d8b318c0dc-300x425.jpg%3Ffit%3Dcrop%26auto%3Dformat&w=1200&q=30 1200w, /_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fgenetherapy%2Fa9442bca6eabb1cffae5b9f82172d8d8b318c0dc-300x425.jpg%3Ffit%3Dcrop%26auto%3Dformat&w=1920&q=30 1920w, /_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fgenetherapy%2Fa9442bca6eabb1cffae5b9f82172d8d8b318c0dc-300x425.jpg%3Ffit%3Dcrop%26auto%3Dformat&w=2048&q=30 2048w, /_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fgenetherapy%2Fa9442bca6eabb1cffae5b9f82172d8d8b318c0dc-300x425.jpg%3Ffit%3Dcrop%26auto%3Dformat&w=3840&q=30 3840w" src="/_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fgenetherapy%2Fa9442bca6eabb1cffae5b9f82172d8d8b318c0dc-300x425.jpg%3Ffit%3Dcrop%26auto%3Dformat&w=3840&q=30" decoding="async" data-nimg="fill" style="position:absolute;top:0;left:0;bottom:0;right:0;box-sizing:border-box;padding:0;border:none;margin:auto;display:block;width:0;height:0;min-width:100%;max-width:100%;min-height:100%;max-height:100%;object-fit:contain" loading="lazy"/></noscript></span></div><div class="w-full flex-wrap text-center text-sm mt-4 font-light no-underline text-white"></div></a><a id="" class="w-[200px] h-fit space-y-3 flex-none select-none no-underline" style="scroll-snap-align:center;text-decoration:none" href="/view/townsend-tai-johnson-gene-therapy-apoe4-homozygous-alzheimer-disease"><div class="w-full shadow-md shadow-gray-800 overflow-hidden relative bg-white aspect-video"><span style="box-sizing:border-box;display:block;overflow:hidden;width:initial;height:initial;background:none;opacity:1;border:0;margin:0;padding:0;position:absolute;top:0;left:0;bottom:0;right:0"><img alt="R. 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/_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fgenetherapy%2F06e896e8fdad4ea58e0766e784b9513070d4dc28-300x300.jpg%3Ffit%3Dcrop%26auto%3Dformat&w=1920&q=75 1920w, /_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fgenetherapy%2F06e896e8fdad4ea58e0766e784b9513070d4dc28-300x300.jpg%3Ffit%3Dcrop%26auto%3Dformat&w=2048&q=75 2048w, /_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fgenetherapy%2F06e896e8fdad4ea58e0766e784b9513070d4dc28-300x300.jpg%3Ffit%3Dcrop%26auto%3Dformat&w=3840&q=75 3840w" src="/_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fgenetherapy%2F06e896e8fdad4ea58e0766e784b9513070d4dc28-300x300.jpg%3Ffit%3Dcrop%26auto%3Dformat&w=3840&q=75" decoding="async" data-nimg="responsive" style="border-top-left-radius:0rem;border-top-right-radius:0rem;border-bottom-left-radius:0rem;border-bottom-right-radius:0rem;position:absolute;top:0;left:0;bottom:0;right:0;box-sizing:border-box;padding:0;border:none;margin:auto;display:block;width:0;height:0;min-width:100%;max-width:100%;min-height:100%;max-height:100%" class="shrink-0" loading="lazy"/></noscript></span></div></a><div class="flex-auto w-[200%] md:w-auto ml-2 flex-1"><p class="font-bold text-[1rem] pl-4 text-undefined" style="font-size:1rem"><a href="/view/gene-therapy-apoe4-homozygous-alzheimer-disease?utm_source=www.cgtlive.com&utm_medium=relatedContent">Gene Therapy for APOE4 Homozygous Alzheimer Disease</a></p><div class=" pl-4"><a class="text-sm text-sky-800" href="/authors/kim-g-johnson-md">Kim G. Johnson, MD</a><span class="mr-1 ml-[1px]">;</span><a class="text-sm text-sky-800" href="/authors/r-nolan-townsend">R. Nolan Townsend</a><span class="mr-1 ml-[1px]">;</span><a class="text-sm text-sky-800" href="/authors/sandi-see-tai-md">Sandi See Tai, MD</a><span class="mr-1 ml-[1px]">;</span><a class="text-sm text-sky-800" href="/authors/isabella-ciccone-mph">Isabella Ciccone, MPH</a><span class="mr-1 ml-[1px]">;</span><a class="text-sm text-sky-800" href="/authors/noah-stansfield">Noah Stansfield</a></div><a href="/view/gene-therapy-apoe4-homozygous-alzheimer-disease?utm_source=www.cgtlive.com&utm_medium=relatedContent"><span class="text-sm text-gray-500 pl-4">November 6th 2024</span><div><span class="px-2 py-1 ml-4 text-xs text-white border bg-primary italic">Article</span></div><div class="mt-2 ml-4"></div><div class="flex flex-row gap-2"></div><p class=" mt-4 text-gray-800 pl-4">R. Nolan Townsend, Sandi See Tai, MD, and Kim G. Johnson, MD, discussed Lexeo Therapeutics’ LX1001 gene therapy trial that demonstrated promising safety and biomarker effects in patients with early-stage Alzheimer disease. </p><div class="pl-4"></div></a><div class="flex flex-col sm:flex-row pl-2 mt-4"></div></div></div></div></div></div><div class="mb-4 w-full h-full"><hr class="mt-1 w-full " style="border-top-width:1px;border-top-color:#F3F4F6"/><div class="w-full h-full" style="box-shadow:0px 0px 0 0 rgb(194, 194, 194, 1)"><div class="w-full md:w-auto md:flex md:flex-col md:items-center lg:items-start lg:flex-row mb-4 mt-3 p-4"><div class="flex flex-1 md:col-span-2 " style="background-color:transparent;border-color:#F3F4F6;border-width:0;border-top-left-radius:0rem;border-top-right-radius:0rem;border-bottom-left-radius:0rem;border-bottom-right-radius:0rem"><a class=" md:flex-none w-full md:w-48 mt-2" href="/view/capsida-biotherapeutics-cap-003-displays-properties-disease-modification-preclinical-parkinson-disease-research?utm_source=www.cgtlive.com&utm_medium=relatedContent"><div class=""><span style="box-sizing:border-box;display:block;overflow:hidden;width:initial;height:initial;background:none;opacity:1;border:0;margin:0;padding:0;position:relative"><span style="box-sizing:border-box;display:block;width:initial;height:initial;background:none;opacity:1;border:0;margin:0;padding:0;padding-top:56.25%"></span><img alt=" Capsida Biotherapeutics' CAP-003 Displays Properties of Disease-Modification in Preclinical Parkinson Disease Research" title=" Capsida Biotherapeutics' CAP-003 Displays Properties of Disease-Modification in Preclinical Parkinson Disease Research" src="data:image/gif;base64,R0lGODlhAQABAIAAAAAAAP///yH5BAEAAAAALAAAAAABAAEAAAIBRAA7" decoding="async" data-nimg="responsive" class="shrink-0" style="border-top-left-radius:0rem;border-top-right-radius:0rem;border-bottom-left-radius:0rem;border-bottom-right-radius:0rem;position:absolute;top:0;left:0;bottom:0;right:0;box-sizing:border-box;padding:0;border:none;margin:auto;display:block;width:0;height:0;min-width:100%;max-width:100%;min-height:100%;max-height:100%"/><noscript><img alt=" Capsida Biotherapeutics' CAP-003 Displays Properties of Disease-Modification in Preclinical Parkinson Disease Research" title=" Capsida Biotherapeutics' CAP-003 Displays Properties of Disease-Modification in Preclinical Parkinson Disease Research" sizes="100vw" srcSet="/_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fgenetherapy%2F7a9fdbe46c49ea22af071b1d56f16c1ff1bceda7-1200x675.jpg%3Ffit%3Dcrop%26auto%3Dformat&w=640&q=75 640w, /_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fgenetherapy%2F7a9fdbe46c49ea22af071b1d56f16c1ff1bceda7-1200x675.jpg%3Ffit%3Dcrop%26auto%3Dformat&w=750&q=75 750w, 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/_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fgenetherapy%2F7a9fdbe46c49ea22af071b1d56f16c1ff1bceda7-1200x675.jpg%3Ffit%3Dcrop%26auto%3Dformat&w=3840&q=75 3840w" src="/_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fgenetherapy%2F7a9fdbe46c49ea22af071b1d56f16c1ff1bceda7-1200x675.jpg%3Ffit%3Dcrop%26auto%3Dformat&w=3840&q=75" decoding="async" data-nimg="responsive" style="border-top-left-radius:0rem;border-top-right-radius:0rem;border-bottom-left-radius:0rem;border-bottom-right-radius:0rem;position:absolute;top:0;left:0;bottom:0;right:0;box-sizing:border-box;padding:0;border:none;margin:auto;display:block;width:0;height:0;min-width:100%;max-width:100%;min-height:100%;max-height:100%" class="shrink-0" loading="lazy"/></noscript></span></div></a><div class="flex-auto w-[200%] md:w-auto ml-2 flex-1"><p class="font-bold text-[1rem] pl-4 text-undefined" style="font-size:1rem"><a href="/view/capsida-biotherapeutics-cap-003-displays-properties-disease-modification-preclinical-parkinson-disease-research?utm_source=www.cgtlive.com&utm_medium=relatedContent"> Capsida Biotherapeutics' CAP-003 Displays Properties of Disease-Modification in Preclinical Parkinson Disease Research</a></p><div class=" pl-4"><a class="text-sm text-sky-800" href="/authors/marco-meglio">Marco Meglio</a></div><a href="/view/capsida-biotherapeutics-cap-003-displays-properties-disease-modification-preclinical-parkinson-disease-research?utm_source=www.cgtlive.com&utm_medium=relatedContent"><span class="text-sm text-gray-500 pl-4">November 5th 2024</span><div><span class="px-2 py-1 ml-4 text-xs text-white border bg-primary italic">Article</span></div><div class="mt-2 ml-4"></div><div class="flex flex-row gap-2"></div><p class=" mt-4 text-gray-800 pl-4">The noninvasive gene therapy exceeded the 30% efficacy threshold for normalizing GCase activity across all doses.</p><div class="pl-4"></div></a><div class="flex flex-col sm:flex-row pl-2 mt-4"></div></div></div></div></div></div><div class="mb-4 w-full h-full"><hr class="mt-1 w-full " style="border-top-width:1px;border-top-color:#F3F4F6"/><div class="w-full h-full" style="box-shadow:0px 0px 0 0 rgb(194, 194, 194, 1)"><div class="w-full md:w-auto md:flex md:flex-col md:items-center lg:items-start lg:flex-row mb-4 mt-3 p-4"><div class="flex flex-1 md:col-span-2 " style="background-color:transparent;border-color:#F3F4F6;border-width:0;border-top-left-radius:0rem;border-top-right-radius:0rem;border-bottom-left-radius:0rem;border-bottom-right-radius:0rem"><a class=" md:flex-none w-full md:w-48 mt-2" href="/view/lexeo-therapeutics-gene-therapy-lx1001-increases-apoe2-expression-patients-alzheimer-disease?utm_source=www.cgtlive.com&utm_medium=relatedContent"><div class=""><span style="box-sizing:border-box;display:block;overflow:hidden;width:initial;height:initial;background:none;opacity:1;border:0;margin:0;padding:0;position:relative"><span style="box-sizing:border-box;display:block;width:initial;height:initial;background:none;opacity:1;border:0;margin:0;padding:0;padding-top:56.25%"></span><img alt="Lexeo Therapeutics' Gene Therapy LX1001 Increases APOE2 Expression in Patients With Alzheimer Disease" title="Lexeo Therapeutics' Gene Therapy LX1001 Increases APOE2 Expression in Patients With Alzheimer Disease" src="data:image/gif;base64,R0lGODlhAQABAIAAAAAAAP///yH5BAEAAAAALAAAAAABAAEAAAIBRAA7" decoding="async" data-nimg="responsive" class="shrink-0" style="border-top-left-radius:0rem;border-top-right-radius:0rem;border-bottom-left-radius:0rem;border-bottom-right-radius:0rem;position:absolute;top:0;left:0;bottom:0;right:0;box-sizing:border-box;padding:0;border:none;margin:auto;display:block;width:0;height:0;min-width:100%;max-width:100%;min-height:100%;max-height:100%"/><noscript><img alt="Lexeo Therapeutics' Gene Therapy LX1001 Increases APOE2 Expression in Patients With Alzheimer Disease" title="Lexeo Therapeutics' Gene Therapy LX1001 Increases APOE2 Expression in Patients With Alzheimer Disease" sizes="100vw" srcSet="/_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fgenetherapy%2Fdb612581b6df174344b5c04187efb16204ecbe64-1200x675.jpg%3Ffit%3Dcrop%26auto%3Dformat&w=640&q=75 640w, /_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fgenetherapy%2Fdb612581b6df174344b5c04187efb16204ecbe64-1200x675.jpg%3Ffit%3Dcrop%26auto%3Dformat&w=750&q=75 750w, 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/_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fgenetherapy%2Fdb612581b6df174344b5c04187efb16204ecbe64-1200x675.jpg%3Ffit%3Dcrop%26auto%3Dformat&w=3840&q=75 3840w" src="/_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fgenetherapy%2Fdb612581b6df174344b5c04187efb16204ecbe64-1200x675.jpg%3Ffit%3Dcrop%26auto%3Dformat&w=3840&q=75" decoding="async" data-nimg="responsive" style="border-top-left-radius:0rem;border-top-right-radius:0rem;border-bottom-left-radius:0rem;border-bottom-right-radius:0rem;position:absolute;top:0;left:0;bottom:0;right:0;box-sizing:border-box;padding:0;border:none;margin:auto;display:block;width:0;height:0;min-width:100%;max-width:100%;min-height:100%;max-height:100%" class="shrink-0" loading="lazy"/></noscript></span></div></a><div class="flex-auto w-[200%] md:w-auto ml-2 flex-1"><p class="font-bold text-[1rem] pl-4 text-undefined" style="font-size:1rem"><a href="/view/lexeo-therapeutics-gene-therapy-lx1001-increases-apoe2-expression-patients-alzheimer-disease?utm_source=www.cgtlive.com&utm_medium=relatedContent">Lexeo Therapeutics' Gene Therapy LX1001 Increases APOE2 Expression in Patients With Alzheimer Disease</a></p><div class=" pl-4"><a class="text-sm text-sky-800" href="/authors/marco-meglio">Marco Meglio</a></div><a href="/view/lexeo-therapeutics-gene-therapy-lx1001-increases-apoe2-expression-patients-alzheimer-disease?utm_source=www.cgtlive.com&utm_medium=relatedContent"><span class="text-sm text-gray-500 pl-4">November 4th 2024</span><div><span class="px-2 py-1 ml-4 text-xs text-white border bg-primary italic">Article</span></div><div class="mt-2 ml-4"></div><div class="flex flex-row gap-2"></div><p class=" mt-4 text-gray-800 pl-4">LX1001's safety profile was characterized as well-tolerated with no amyloid-related imaging abnormalities reported.</p><div class="pl-4"></div></a><div class="flex flex-col sm:flex-row pl-2 mt-4"></div></div></div></div></div></div><div class="mb-4 w-full h-full"><hr class="mt-1 w-full " style="border-top-width:1px;border-top-color:#F3F4F6"/><div class="w-full h-full" style="box-shadow:0px 0px 0 0 rgb(194, 194, 194, 1)"><div class="w-full md:w-auto md:flex md:flex-col md:items-center lg:items-start lg:flex-row mb-4 mt-3 p-4"><div class="flex flex-1 md:col-span-2 " style="background-color:transparent;border-color:#F3F4F6;border-width:0;border-top-left-radius:0rem;border-top-right-radius:0rem;border-bottom-left-radius:0rem;border-bottom-right-radius:0rem"><a class=" md:flex-none w-full md:w-48 mt-2" href="/view/overcoming-aav-barriers-limitations-treat-duchenne-muscular-dystrophy?utm_source=www.cgtlive.com&utm_medium=relatedContent"><div class=""><span style="box-sizing:border-box;display:block;overflow:hidden;width:initial;height:initial;background:none;opacity:1;border:0;margin:0;padding:0;position:relative"><span style="box-sizing:border-box;display:block;width:initial;height:initial;background:none;opacity:1;border:0;margin:0;padding:0;padding-top:100%"></span><img alt="Overcoming AAV Barriers and Limitations to Treat Duchenne Muscular Dystrophy" title="Overcoming AAV Barriers and Limitations to Treat Duchenne Muscular Dystrophy" src="data:image/gif;base64,R0lGODlhAQABAIAAAAAAAP///yH5BAEAAAAALAAAAAABAAEAAAIBRAA7" decoding="async" data-nimg="responsive" class="shrink-0" style="border-top-left-radius:0rem;border-top-right-radius:0rem;border-bottom-left-radius:0rem;border-bottom-right-radius:0rem;position:absolute;top:0;left:0;bottom:0;right:0;box-sizing:border-box;padding:0;border:none;margin:auto;display:block;width:0;height:0;min-width:100%;max-width:100%;min-height:100%;max-height:100%"/><noscript><img alt="Overcoming AAV Barriers and Limitations to Treat Duchenne Muscular Dystrophy" title="Overcoming AAV Barriers and Limitations to Treat Duchenne Muscular Dystrophy" sizes="100vw" 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style="border-top-left-radius:0rem;border-top-right-radius:0rem;border-bottom-left-radius:0rem;border-bottom-right-radius:0rem;position:absolute;top:0;left:0;bottom:0;right:0;box-sizing:border-box;padding:0;border:none;margin:auto;display:block;width:0;height:0;min-width:100%;max-width:100%;min-height:100%;max-height:100%" class="shrink-0" loading="lazy"/></noscript></span></div></a><div class="flex-auto w-[200%] md:w-auto ml-2 flex-1"><p class="font-bold text-[1rem] pl-4 text-undefined" style="font-size:1rem"><a href="/view/gene-therapy-apoe4-homozygous-alzheimer-disease?utm_source=www.cgtlive.com&utm_medium=relatedContent">Gene Therapy for APOE4 Homozygous Alzheimer Disease</a></p><div class=" pl-4"><a class="text-sm text-sky-800" href="/authors/kim-g-johnson-md">Kim G. Johnson, MD</a><span class="mr-1 ml-[1px]">;</span><a class="text-sm text-sky-800" href="/authors/r-nolan-townsend">R. Nolan Townsend</a><span class="mr-1 ml-[1px]">;</span><a class="text-sm text-sky-800" href="/authors/sandi-see-tai-md">Sandi See Tai, MD</a><span class="mr-1 ml-[1px]">;</span><a class="text-sm text-sky-800" href="/authors/isabella-ciccone-mph">Isabella Ciccone, MPH</a><span class="mr-1 ml-[1px]">;</span><a class="text-sm text-sky-800" href="/authors/noah-stansfield">Noah Stansfield</a></div><a href="/view/gene-therapy-apoe4-homozygous-alzheimer-disease?utm_source=www.cgtlive.com&utm_medium=relatedContent"><span class="text-sm text-gray-500 pl-4">November 6th 2024</span><div><span class="px-2 py-1 ml-4 text-xs text-white border bg-primary italic">Article</span></div><div class="mt-2 ml-4"></div><div class="flex flex-row gap-2"></div><p class=" mt-4 text-gray-800 pl-4">R. Nolan Townsend, Sandi See Tai, MD, and Kim G. Johnson, MD, discussed Lexeo Therapeutics’ LX1001 gene therapy trial that demonstrated promising safety and biomarker effects in patients with early-stage Alzheimer disease. </p><div class="pl-4"></div></a><div class="flex flex-col sm:flex-row pl-2 mt-4"></div></div></div></div></div></div><div class="mb-4 w-full h-full"><hr class="mt-1 w-full " style="border-top-width:1px;border-top-color:#F3F4F6"/><div class="w-full h-full" style="box-shadow:0px 0px 0 0 rgb(194, 194, 194, 1)"><div class="w-full md:w-auto md:flex md:flex-col md:items-center lg:items-start lg:flex-row mb-4 mt-3 p-4"><div class="flex flex-1 md:col-span-2 " style="background-color:transparent;border-color:#F3F4F6;border-width:0;border-top-left-radius:0rem;border-top-right-radius:0rem;border-bottom-left-radius:0rem;border-bottom-right-radius:0rem"><a class=" md:flex-none w-full md:w-48 mt-2" href="/view/capsida-biotherapeutics-cap-003-displays-properties-disease-modification-preclinical-parkinson-disease-research?utm_source=www.cgtlive.com&utm_medium=relatedContent"><div class=""><span style="box-sizing:border-box;display:block;overflow:hidden;width:initial;height:initial;background:none;opacity:1;border:0;margin:0;padding:0;position:relative"><span style="box-sizing:border-box;display:block;width:initial;height:initial;background:none;opacity:1;border:0;margin:0;padding:0;padding-top:56.25%"></span><img alt=" Capsida Biotherapeutics' CAP-003 Displays Properties of Disease-Modification in Preclinical Parkinson Disease Research" title=" Capsida Biotherapeutics' CAP-003 Displays Properties of Disease-Modification in Preclinical Parkinson Disease Research" src="data:image/gif;base64,R0lGODlhAQABAIAAAAAAAP///yH5BAEAAAAALAAAAAABAAEAAAIBRAA7" decoding="async" data-nimg="responsive" class="shrink-0" style="border-top-left-radius:0rem;border-top-right-radius:0rem;border-bottom-left-radius:0rem;border-bottom-right-radius:0rem;position:absolute;top:0;left:0;bottom:0;right:0;box-sizing:border-box;padding:0;border:none;margin:auto;display:block;width:0;height:0;min-width:100%;max-width:100%;min-height:100%;max-height:100%"/><noscript><img alt=" Capsida Biotherapeutics' CAP-003 Displays Properties of Disease-Modification in Preclinical Parkinson Disease Research" title=" Capsida Biotherapeutics' CAP-003 Displays Properties of Disease-Modification in Preclinical Parkinson Disease Research" sizes="100vw" srcSet="/_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fgenetherapy%2F7a9fdbe46c49ea22af071b1d56f16c1ff1bceda7-1200x675.jpg%3Ffit%3Dcrop%26auto%3Dformat&w=640&q=75 640w, /_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fgenetherapy%2F7a9fdbe46c49ea22af071b1d56f16c1ff1bceda7-1200x675.jpg%3Ffit%3Dcrop%26auto%3Dformat&w=750&q=75 750w, 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/_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fgenetherapy%2F7a9fdbe46c49ea22af071b1d56f16c1ff1bceda7-1200x675.jpg%3Ffit%3Dcrop%26auto%3Dformat&w=3840&q=75 3840w" src="/_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fgenetherapy%2F7a9fdbe46c49ea22af071b1d56f16c1ff1bceda7-1200x675.jpg%3Ffit%3Dcrop%26auto%3Dformat&w=3840&q=75" decoding="async" data-nimg="responsive" style="border-top-left-radius:0rem;border-top-right-radius:0rem;border-bottom-left-radius:0rem;border-bottom-right-radius:0rem;position:absolute;top:0;left:0;bottom:0;right:0;box-sizing:border-box;padding:0;border:none;margin:auto;display:block;width:0;height:0;min-width:100%;max-width:100%;min-height:100%;max-height:100%" class="shrink-0" loading="lazy"/></noscript></span></div></a><div class="flex-auto w-[200%] md:w-auto ml-2 flex-1"><p class="font-bold text-[1rem] pl-4 text-undefined" style="font-size:1rem"><a href="/view/capsida-biotherapeutics-cap-003-displays-properties-disease-modification-preclinical-parkinson-disease-research?utm_source=www.cgtlive.com&utm_medium=relatedContent"> Capsida Biotherapeutics' CAP-003 Displays Properties of Disease-Modification in Preclinical Parkinson Disease Research</a></p><div class=" pl-4"><a class="text-sm text-sky-800" href="/authors/marco-meglio">Marco Meglio</a></div><a href="/view/capsida-biotherapeutics-cap-003-displays-properties-disease-modification-preclinical-parkinson-disease-research?utm_source=www.cgtlive.com&utm_medium=relatedContent"><span class="text-sm text-gray-500 pl-4">November 5th 2024</span><div><span class="px-2 py-1 ml-4 text-xs text-white border bg-primary italic">Article</span></div><div class="mt-2 ml-4"></div><div class="flex flex-row gap-2"></div><p class=" mt-4 text-gray-800 pl-4">The noninvasive gene therapy exceeded the 30% efficacy threshold for normalizing GCase activity across all doses.</p><div class="pl-4"></div></a><div class="flex flex-col sm:flex-row pl-2 mt-4"></div></div></div></div></div></div><div class="mb-4 w-full h-full"><hr class="mt-1 w-full " style="border-top-width:1px;border-top-color:#F3F4F6"/><div class="w-full h-full" style="box-shadow:0px 0px 0 0 rgb(194, 194, 194, 1)"><div class="w-full md:w-auto md:flex md:flex-col md:items-center lg:items-start lg:flex-row mb-4 mt-3 p-4"><div class="flex flex-1 md:col-span-2 " style="background-color:transparent;border-color:#F3F4F6;border-width:0;border-top-left-radius:0rem;border-top-right-radius:0rem;border-bottom-left-radius:0rem;border-bottom-right-radius:0rem"><a class=" md:flex-none w-full md:w-48 mt-2" href="/view/lexeo-therapeutics-gene-therapy-lx1001-increases-apoe2-expression-patients-alzheimer-disease?utm_source=www.cgtlive.com&utm_medium=relatedContent"><div class=""><span style="box-sizing:border-box;display:block;overflow:hidden;width:initial;height:initial;background:none;opacity:1;border:0;margin:0;padding:0;position:relative"><span style="box-sizing:border-box;display:block;width:initial;height:initial;background:none;opacity:1;border:0;margin:0;padding:0;padding-top:56.25%"></span><img alt="Lexeo Therapeutics' Gene Therapy LX1001 Increases APOE2 Expression in Patients With Alzheimer Disease" title="Lexeo Therapeutics' Gene Therapy LX1001 Increases APOE2 Expression in Patients With Alzheimer Disease" src="data:image/gif;base64,R0lGODlhAQABAIAAAAAAAP///yH5BAEAAAAALAAAAAABAAEAAAIBRAA7" decoding="async" data-nimg="responsive" class="shrink-0" style="border-top-left-radius:0rem;border-top-right-radius:0rem;border-bottom-left-radius:0rem;border-bottom-right-radius:0rem;position:absolute;top:0;left:0;bottom:0;right:0;box-sizing:border-box;padding:0;border:none;margin:auto;display:block;width:0;height:0;min-width:100%;max-width:100%;min-height:100%;max-height:100%"/><noscript><img alt="Lexeo Therapeutics' Gene Therapy LX1001 Increases APOE2 Expression in Patients With Alzheimer Disease" title="Lexeo Therapeutics' Gene Therapy LX1001 Increases APOE2 Expression in Patients With Alzheimer Disease" sizes="100vw" srcSet="/_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fgenetherapy%2Fdb612581b6df174344b5c04187efb16204ecbe64-1200x675.jpg%3Ffit%3Dcrop%26auto%3Dformat&w=640&q=75 640w, /_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fgenetherapy%2Fdb612581b6df174344b5c04187efb16204ecbe64-1200x675.jpg%3Ffit%3Dcrop%26auto%3Dformat&w=750&q=75 750w, 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/_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fgenetherapy%2Fdb612581b6df174344b5c04187efb16204ecbe64-1200x675.jpg%3Ffit%3Dcrop%26auto%3Dformat&w=3840&q=75 3840w" src="/_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fgenetherapy%2Fdb612581b6df174344b5c04187efb16204ecbe64-1200x675.jpg%3Ffit%3Dcrop%26auto%3Dformat&w=3840&q=75" decoding="async" data-nimg="responsive" style="border-top-left-radius:0rem;border-top-right-radius:0rem;border-bottom-left-radius:0rem;border-bottom-right-radius:0rem;position:absolute;top:0;left:0;bottom:0;right:0;box-sizing:border-box;padding:0;border:none;margin:auto;display:block;width:0;height:0;min-width:100%;max-width:100%;min-height:100%;max-height:100%" class="shrink-0" loading="lazy"/></noscript></span></div></a><div class="flex-auto w-[200%] md:w-auto ml-2 flex-1"><p class="font-bold text-[1rem] pl-4 text-undefined" style="font-size:1rem"><a href="/view/lexeo-therapeutics-gene-therapy-lx1001-increases-apoe2-expression-patients-alzheimer-disease?utm_source=www.cgtlive.com&utm_medium=relatedContent">Lexeo Therapeutics' Gene Therapy LX1001 Increases APOE2 Expression in Patients With Alzheimer Disease</a></p><div class=" pl-4"><a class="text-sm text-sky-800" href="/authors/marco-meglio">Marco Meglio</a></div><a href="/view/lexeo-therapeutics-gene-therapy-lx1001-increases-apoe2-expression-patients-alzheimer-disease?utm_source=www.cgtlive.com&utm_medium=relatedContent"><span class="text-sm text-gray-500 pl-4">November 4th 2024</span><div><span class="px-2 py-1 ml-4 text-xs text-white border bg-primary italic">Article</span></div><div class="mt-2 ml-4"></div><div class="flex flex-row gap-2"></div><p class=" mt-4 text-gray-800 pl-4">LX1001's safety profile was characterized as well-tolerated with no amyloid-related imaging abnormalities reported.</p><div class="pl-4"></div></a><div class="flex flex-col sm:flex-row pl-2 mt-4"></div></div></div></div></div></div><div class="mb-4 w-full h-full"><hr class="mt-1 w-full " style="border-top-width:1px;border-top-color:#F3F4F6"/><div class="w-full h-full" style="box-shadow:0px 0px 0 0 rgb(194, 194, 194, 1)"><div class="w-full md:w-auto md:flex md:flex-col md:items-center lg:items-start lg:flex-row mb-4 mt-3 p-4"><div class="flex flex-1 md:col-span-2 " style="background-color:transparent;border-color:#F3F4F6;border-width:0;border-top-left-radius:0rem;border-top-right-radius:0rem;border-bottom-left-radius:0rem;border-bottom-right-radius:0rem"><a class=" md:flex-none w-full md:w-48 mt-2" href="/view/overcoming-aav-barriers-limitations-treat-duchenne-muscular-dystrophy?utm_source=www.cgtlive.com&utm_medium=relatedContent"><div class=""><span style="box-sizing:border-box;display:block;overflow:hidden;width:initial;height:initial;background:none;opacity:1;border:0;margin:0;padding:0;position:relative"><span style="box-sizing:border-box;display:block;width:initial;height:initial;background:none;opacity:1;border:0;margin:0;padding:0;padding-top:100%"></span><img alt="Overcoming AAV Barriers and Limitations to Treat Duchenne Muscular Dystrophy" title="Overcoming AAV Barriers and Limitations to Treat Duchenne Muscular Dystrophy" src="data:image/gif;base64,R0lGODlhAQABAIAAAAAAAP///yH5BAEAAAAALAAAAAABAAEAAAIBRAA7" decoding="async" data-nimg="responsive" class="shrink-0" style="border-top-left-radius:0rem;border-top-right-radius:0rem;border-bottom-left-radius:0rem;border-bottom-right-radius:0rem;position:absolute;top:0;left:0;bottom:0;right:0;box-sizing:border-box;padding:0;border:none;margin:auto;display:block;width:0;height:0;min-width:100%;max-width:100%;min-height:100%;max-height:100%"/><noscript><img alt="Overcoming AAV Barriers and Limitations to Treat Duchenne Muscular Dystrophy" title="Overcoming AAV Barriers and Limitations to Treat Duchenne Muscular Dystrophy" sizes="100vw" srcSet="/_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fgenetherapy%2F2186783b212093b56503299ea1f33828b31bf0f2-300x300.png%3Ffit%3Dcrop%26auto%3Dformat&w=640&q=75 640w, /_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fgenetherapy%2F2186783b212093b56503299ea1f33828b31bf0f2-300x300.png%3Ffit%3Dcrop%26auto%3Dformat&w=750&q=75 750w, /_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fgenetherapy%2F2186783b212093b56503299ea1f33828b31bf0f2-300x300.png%3Ffit%3Dcrop%26auto%3Dformat&w=828&q=75 828w, /_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fgenetherapy%2F2186783b212093b56503299ea1f33828b31bf0f2-300x300.png%3Ffit%3Dcrop%26auto%3Dformat&w=1080&q=75 1080w, /_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fgenetherapy%2F2186783b212093b56503299ea1f33828b31bf0f2-300x300.png%3Ffit%3Dcrop%26auto%3Dformat&w=1200&q=75 1200w, /_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fgenetherapy%2F2186783b212093b56503299ea1f33828b31bf0f2-300x300.png%3Ffit%3Dcrop%26auto%3Dformat&w=1920&q=75 1920w, /_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fgenetherapy%2F2186783b212093b56503299ea1f33828b31bf0f2-300x300.png%3Ffit%3Dcrop%26auto%3Dformat&w=2048&q=75 2048w, /_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fgenetherapy%2F2186783b212093b56503299ea1f33828b31bf0f2-300x300.png%3Ffit%3Dcrop%26auto%3Dformat&w=3840&q=75 3840w" src="/_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fgenetherapy%2F2186783b212093b56503299ea1f33828b31bf0f2-300x300.png%3Ffit%3Dcrop%26auto%3Dformat&w=3840&q=75" decoding="async" data-nimg="responsive" style="border-top-left-radius:0rem;border-top-right-radius:0rem;border-bottom-left-radius:0rem;border-bottom-right-radius:0rem;position:absolute;top:0;left:0;bottom:0;right:0;box-sizing:border-box;padding:0;border:none;margin:auto;display:block;width:0;height:0;min-width:100%;max-width:100%;min-height:100%;max-height:100%" class="shrink-0" loading="lazy"/></noscript></span></div></a><div class="flex-auto w-[200%] md:w-auto ml-2 flex-1"><p class="font-bold text-[1rem] pl-4 text-undefined" style="font-size:1rem"><a href="/view/overcoming-aav-barriers-limitations-treat-duchenne-muscular-dystrophy?utm_source=www.cgtlive.com&utm_medium=relatedContent">Overcoming AAV Barriers and Limitations to Treat Duchenne Muscular Dystrophy</a></p><div class=" pl-4"><a class="text-sm text-sky-800" href="/authors/melissa-spencer-phd">Melissa Spencer, PhD</a><span class="mr-1 ml-[1px]">;</span><a class="text-sm text-sky-800" href="/authors/cgtlive-staff">CGTLive Staff</a></div><a href="/view/overcoming-aav-barriers-limitations-treat-duchenne-muscular-dystrophy?utm_source=www.cgtlive.com&utm_medium=relatedContent"><span class="text-sm text-gray-500 pl-4">October 29th 2024</span><div><span class="px-2 py-1 ml-4 text-xs text-white border bg-primary italic">Article</span></div><div class="mt-2 ml-4"></div><div class="flex flex-row gap-2"></div><p class=" mt-4 text-gray-800 pl-4">Melissa Spencer, PhD, spoke about the emerging role of adeno-associated virus as a delivery system for gene therapies targeting muscular dystrophies, and the challenbges associated with them.</p><div class="pl-4"></div></a><div class="flex flex-col sm:flex-row pl-2 mt-4"></div></div></div></div></div></div></div></div></div><div class="pb-24"></div></div><script type="application/ld+json">{"@context":"https://schema.org","@type":"NewsArticle","headline":"Using Single Cell Sequencing for Gene Editing Quality Control","datePublished":"2024-08-21T11:00:00.000Z","dateModified":"2024-08-21T10:04:10Z","inLanguage":"en-US","image":"https://cdn.sanity.io/images/0vv8moc6/genetherapy/57aa15f32df153ae83c1b698050f97a0150c1355-300x300.jpg?fit=crop&auto=format","mainEntityOfPage":{"@type":"WebPage","@id":"https://www.cgtlive.com/view/single-cell-sequencing-gene-editing-quality-control"},"publisher":{"@type":"Organization","name":"CGTlive™","logo":{"@type":"ImageObject","url":"https://www.cgtlive.com/CGT_Color_NoTagline.png"}},"keywords":"gene therapy","articleBody":"\n\nOff-target edits can pose safety risks during the use of gene editing therapies. Although, detecting these edits precisely and accurately can prove difficult with traditional bulk DNA sequencing.\n\nMission Bio's Tapestri Genome Editing Solution, which uses DNA sequencing to detect gene edits at a single cell level, is intended to overcome this challenge. Notably, the company presented on Tapestri at the American Society of Gene & Cell Therapy (ASGCT) 27th Annual Meeting, held May 7 to 10, 2024, in Baltimore, MD. At the conference, CGTLive® interviewed Anjali Pradhan, MS, the chief product officer at Mission Bio, to learn more.\n\nCGTLive: Can you give some background about what you presented at ASGCT this year?\n\nAnjali Pradhan, MS: We were really excited that we have launched our genome editing solution. What this is, is a genome editing solution that helps you quality control your edits and your genes as you do a CRISPR edit or a prime edit, using various editing tools. Our solution is a unique solution because it's the only DNA single cell solution that allows you to do on-targets, off-targets, translocations, measures zygosity of edits, as well as—we're excited to introduce—looks at copy number variations. Our whole product solution is available for users who are using prime editing tools, etc. and looking at what the efficiency of gene editing is, so that they can safety control their product as it goes into a patient.\n\nWhat were the key points you presented?\n\nMission Bio is a DNA single cell company. Our tools are an instrument platform that is based on microfluidics. We have also worked with the National Institute of Standards and Technology (NIST) Consortium. Our collaboration has really helped us to emphasize the value of looking at these edits at a single cell level. Our presentation was focused on highlighting when they compared in an interlab study what the value was of our Tapestri platform, our instrument for measuring single cell edits. What it highlighted is looking at attributes like single cell zygosity, looking at translocation events, off-target edits, and on-target edits.\n\nHow would you summarize the big picture implications that doctors and the healthcare community should take away from this?\n\nWhat we've seen is in the industry today there's a lot of excitement with new editing technologies—there's various new technologies coming out. But what you are always concerned about is: What is the safety of those edits? How can you be sure that your edit is as it was intended to be and if it's going in the right place? That's a huge concern to doctors, especially with various editing efficiencies, etc. How do you make sure you're doing the right thing? The FDA also requires you to report out where those edits are and what's the frequency of these edits. So you need the right single cell level tool to measure that accuracy. That's where Mission Bio can really help you to do that quality control (QC), to make sure again that all the critical attributes are safe, your editing efficiency is safe, and you can feel much more confident about where those edits are made.\n\nHave there been any major challenges in this work so far?\n\nI think the challenge continues to be in measuring the safety. But what is continually exciting for the community is as the various forms of editing continue to expand, there will always be checks and balances required to measure the safety. As such, I think as you think about more tools in the editing space, you can also see the scope of looking at those tools at a single cell level. The highlight is you have to use your cell as your most accurate level at a macro level. When you're using other tools like bulk sequencing or PCR, you're measuring at a bulk level, so you don't get the granularity when cells as we know are heterogeneous. The challenge is the heterogeneity of cells and this is why you should always use a single cell and then DNA single cell level methods to help measure the QC.\n\nIs there anything else you would like to share?\n\nI think our goal as a company with Mission Bio is with our DNA single cell tools and technology, we are enabling the community to do safety and efficacy of gene editing. We also have solutions for characterizing cell therapeutic products. Our goal is to work with the community to really build out the right tools. Our vision is to become sort of more of a standardization practice, partnering with institutes like NIST and the FDA, where we can really help ensure safety and efficacy, especially because so many of these edits, if they're not done and measured correctly, can lead to genotoxicity. That's what we want to avoid and enables safety and more precision medicine.\n\nThis transcript has been edited for clarity.\n\nClick here to view more coverage of the 2024 ASGCT Annual Meeting.\n\n\n\n\n\n","description":"Anjali Pradhan, MS, the chief product officer at Mission Bio discussed the company’s Genome Editing Solution.\n\n","author":[{"@type":"Person","name":"Anjali Pradhan, MS"},{"@type":"Person","name":"Noah Stansfield"}]}</script></div></div><div class="flex-none w-[300px] z-[9999] relative hidden md:block"><div style="top:70px;margin-top:44px" class="sticky custom-spacing"></div></div></div><div id="div-gpt-ad-pixel" style="width:1px;height:1px" class=""></div><noscript><iframe src="https://www.googletagmanager.com/ns.html?id=GTM-MBPSSPT" height="0" width="0" style="display:none;visibility:hidden"></iframe></noscript><div id="footerOuterWrap" class="w-full bg-primary flex flex-col items-center justify-center"><div class="container w-[1340px]"><div id="footerInnerWrap" 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Although, detecting these edits precisely and accurately can prove difficult with traditional bulk DNA sequencing.","_key":"b947ecd5c97d0"}],"_type":"block","style":"normal","_key":"f888a5cad26d","upload_doc":null,"uploadAudio":null,"medias":null},{"_type":"block","style":"normal","upload_doc":null,"uploadAudio":null,"medias":null,"_key":"b436f97c0c78","markDefs":[{"_key":"f325ea23ff95","_type":"link","href":"https://www.cgtlive.com/conferences/american-society-for-gene-cell-therapy-asgct-"}],"children":[{"_type":"span","marks":[],"text":"Mission Bio's Tapestri Genome Editing Solution, which uses DNA sequencing to detect gene edits at a single cell level, is intended to overcome this challenge. Notably, the company presented on Tapestri at ","_key":"16620482b347"},{"_type":"span","marks":["f325ea23ff95"],"text":"the American Society of Gene \u0026 Cell Therapy (ASGCT) 27th Annual Meeting","_key":"a11787b86a99"},{"marks":[],"text":", held May 7 to 10, 2024, in Baltimore, MD. At the conference, ","_key":"54bae4a3c2a9","_type":"span"},{"marks":["em"],"text":"CGTLive® ","_key":"fc2b62840d11","_type":"span"},{"_type":"span","marks":[],"text":"interviewed Anjali Pradhan, MS, the chief product officer at Mission Bio, to learn more.","_key":"c9eb224f3a34"}]},{"upload_doc":null,"uploadAudio":null,"medias":null,"children":[{"_type":"span","marks":["strong","em"],"text":"CGTLive:","_key":"8877a82e07af0"},{"marks":["strong"],"text":" Can you give some background about what you presented at ASGCT this year?","_key":"24a5415d083b","_type":"span"}],"_type":"block","style":"h3","_key":"95131aaf3f41","markDefs":[]},{"style":"normal","_key":"2078bc759d0e","upload_doc":null,"uploadAudio":null,"medias":null,"markDefs":[],"children":[{"marks":["strong"],"text":"Anjali Pradhan, MS:","_key":"e8df5fc3b93f","_type":"span"},{"_key":"2478108e53f0","_type":"span","marks":[],"text":" We were really excited that we have launched our genome editing solution. What this is, is a genome editing solution that helps you quality control your edits and your genes as you do a CRISPR edit or a prime edit, using various editing tools. Our solution is a unique solution because it's the only DNA single cell solution that allows you to do on-targets, off-targets, translocations, measures zygosity of edits, as well as—we're excited to introduce—looks at copy number variations. Our whole product solution is available for users who are using prime editing tools, etc. and looking at what the efficiency of gene editing is, so that they can safety control their product as it goes into a patient."}],"_type":"block"},{"uploadAudio":null,"medias":null,"children":[{"_type":"span","marks":["strong"],"text":"What were the key points you presented?","_key":"9d809ea179490"}],"_type":"block","style":"h3","_key":"9b62cdaa996f","markDefs":[],"upload_doc":null},{"upload_doc":null,"uploadAudio":null,"medias":null,"markDefs":[],"children":[{"text":"Mission Bio is a DNA single cell company. Our tools are an instrument platform that is based on microfluidics. We have also worked with the National Institute of Standards and Technology (NIST) Consortium. Our collaboration has really helped us to emphasize the value of looking at these edits at a single cell level. Our presentation was focused on highlighting when they compared in an interlab study what the value was of our Tapestri platform, our instrument for measuring single cell edits. What it highlighted is looking at attributes like single cell zygosity, looking at translocation events, off-target edits, and on-target edits.","_key":"1794205b90f60","_type":"span","marks":[]}],"_type":"block","style":"normal","_key":"161c4eb09341"},{"markDefs":[],"upload_doc":null,"uploadAudio":null,"medias":null,"children":[{"text":"How would you summarize the big picture implications that doctors and the healthcare community should take away from this?","_key":"157bad1b7fcf","_type":"span","marks":["strong"]}],"_type":"block","style":"h3","_key":"6b94691ec07e"},{"upload_doc":null,"uploadAudio":null,"medias":null,"markDefs":[],"children":[{"_type":"span","marks":[],"text":"What we've seen is in the industry today there's a lot of excitement with new editing technologies—there's various new technologies coming out. But what you are always concerned about is: What is the safety of those edits? How can you be sure that your edit is as it was intended to be and if it's going in the right place? That's a huge concern to doctors, especially with various editing efficiencies, etc. How do you make sure you're doing the right thing? The FDA also requires you to report out where those edits are and what's the frequency of these edits. So you need the right single cell level tool to measure that accuracy. That's where Mission Bio can really help you to do that quality control (QC), to make sure again that all the critical attributes are safe, your editing efficiency is safe, and you can feel much more confident about where those edits are made.","_key":"23cfaa223a94"}],"_type":"block","style":"normal","_key":"d0684cd067ff"},{"uploadAudio":null,"medias":null,"_type":"block","style":"h3","_key":"00eabdc68b9a","markDefs":[],"children":[{"_key":"9521d43e5972","_type":"span","marks":["strong"],"text":"Have there been any major challenges in this work so far?"}],"upload_doc":null},{"uploadAudio":null,"medias":null,"markDefs":[],"children":[{"_key":"49f84a78b6f70","_type":"span","marks":[],"text":"I think the challenge continues to be in measuring the safety. But what is continually exciting for the community is as the various forms of editing continue to expand, there will always be checks and balances required to measure the safety. As such, I think as you think about more tools in the editing space, you can also see the scope of looking at those tools at a single cell level. The highlight is you have to use your cell as your most accurate level at a macro level. When you're using other tools like bulk sequencing or PCR, you're measuring at a bulk level, so you don't get the granularity when cells as we know are heterogeneous. The challenge is the heterogeneity of cells and this is why you should always use a single cell and then DNA single cell level methods to help measure the QC."}],"_type":"block","style":"normal","_key":"79a17260da1e","upload_doc":null},{"style":"h3","_key":"bf8d64d47cea","markDefs":[],"upload_doc":null,"uploadAudio":null,"medias":null,"children":[{"_type":"span","marks":["strong"],"text":"Is there anything else you would like to share?","_key":"b1a96b2a4282"}],"_type":"block"},{"_key":"2ff6e4637c9e","markDefs":[],"upload_doc":null,"uploadAudio":null,"medias":null,"children":[{"_type":"span","marks":[],"text":"I think our goal as a company with Mission Bio is with our DNA single cell tools and technology, we are enabling the community to do safety and efficacy of gene editing. We also have solutions for characterizing cell therapeutic products. Our goal is to work with the community to really build out the right tools. Our vision is to become sort of more of a standardization practice, partnering with institutes like NIST and the FDA, where we can really help ensure safety and efficacy, especially because so many of these edits, if they're not done and measured correctly, can lead to genotoxicity. That's what we want to avoid and enables safety and more precision medicine.","_key":"7c7e784403890"}],"_type":"block","style":"normal"},{"_key":"1e27ae9fbd54","markDefs":[],"children":[{"_type":"span","marks":["em","strong"],"text":"This transcript has been edited for clarity.","_key":"090e000f56cf0"}],"_type":"block","style":"normal","upload_doc":null,"uploadAudio":null,"medias":null},{"medias":null,"children":[{"_type":"span","marks":["135dc348e511","strong","em"],"text":"Click here to view more coverage of the 2024 ASGCT Annual Meeting.","_key":"297856ce074e0"}],"_type":"block","style":"normal","_key":"a6163508246b","markDefs":[{"_type":"link","href":"https://www.cgtlive.com/conferences/american-society-for-gene-cell-therapy-asgct-","_key":"135dc348e511"}],"upload_doc":null,"uploadAudio":null},{"style":"h6","_key":"d784d107d6d9","markDefs":[],"upload_doc":null,"uploadAudio":null,"medias":null,"children":[{"_type":"span","marks":[],"text":"","_key":"423394b02ade0"}],"_type":"block"},{"upload_doc":null,"uploadAudio":null,"medias":null,"markDefs":[],"children":[{"_type":"span","marks":[],"text":"","_key":"08d1e30b91e50"}],"_type":"block","style":"normal","_key":"68c7b9db0d9c"},{"style":"normal","upload_doc":null,"uploadAudio":null,"medias":null,"_key":"08507dbc0e28","markDefs":[],"children":[{"_type":"span","marks":[],"text":"","_key":"271de6c127c10"}],"_type":"block"}],"authorMapping":[{"_createdAt":"2024-05-30T21:41:40Z","_rev":"svK6BkgZ7y7yN8MUYdrUXl","_type":"author","_id":"1763e0d0-2cd2-4a27-b3cb-5bfe9722046c","_updatedAt":"2024-05-30T21:41:40Z","url":{"current":"anjali-pradhan-ms","_type":"slug"},"displayName":"Anjali Pradhan, MS"},{"_rev":"yD3aK4wI9vtjJazfpEZYwZ","firstName":"Noah","lastName":"Stansfield","displayName":"Noah Stansfield","_createdAt":"2022-06-14T20:44:15Z","_type":"author","_id":"f9e9001f-7a9c-46a2-9e9c-318ee31c1923","_updatedAt":"2022-06-14T20:44:15Z","url":{"current":"noah-stansfield","_type":"slug"}}],"issueGroup":null,"factCheckAuthorMapping":null,"contentCategory":{"_id":"54fdc3d6-b776-4653-9c81-26acd95a8a7e","name":"Articles"},"display_summary":true,"_id":"8efd2b46-33ec-41eb-b622-052cae117488","thumbnail":{"caption":"Anjali Pradhan, MS","asset":{"_ref":"image-57aa15f32df153ae83c1b698050f97a0150c1355-300x300-jpg","_type":"reference"},"_type":"mainImage","alt":"Anjali Pradhan, MS, the chief product officer at Mission Bio"},"is_visible":true,"documentGroup":null,"pdfUrl":null,"disableAds":false,"disableRelatedContent":false,"relatedArticles":[{"ExcludeFromPubMedXML":false,"internalTag":["Deborah Phippard","Precision for Medicine","Precision","gene therapy","CNS","central nervous system","AAV","neurological indications","neurology indications","future","Alzheimer disease","Parkinson disease","Alzheimer's disease","Parkinson's disease","delivery","diagnostic tools","artificial intelligence","AI","AI tools"],"body":[{"_key":"3caedbae470c","alignment":"left","asset":{"_ref":"image-966ec042ea0449de0fc2f4a9fff69360426657e0-300x300-jpg","_type":"reference"},"disableTextWrap":false,"disableLightBox":true,"_type":"figure","alt":"Bhagirathbhai R. Dholaria, MD, an associate professor of medicine in malignant hematology \u0026 stem cell transplantation at Vanderbilt University Medical Center","imgcaption":[{"style":"normal","_key":"6dc2efa8afd2","markDefs":[],"children":[{"text":"Bhagirathbhai R. Dholaria, MD","_key":"2de253161ea70","_type":"span","marks":[]}],"_type":"block"}]},{"children":[{"marks":[],"text":"Poseida Therapeutics is currently evaluating P-BCMA-ALLO1, an allogeneic BCMA-directed chimeric antigen receptor T-cell (CAR-T) therapy, in an ongoing phase 1/1b clinical trial (NCT04960579) for the treatment of relapsed/refractory (r/r) multiple myeloma (MM). 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Dholaria, MD, an associate professor of medicine in malignant hematology \u0026 stem cell transplantation at Vanderbilt University Medical Center, after the conference to interview him about the findings presented. Dholaria discussed what makes P-BCMA-ALLO1 unique and went over the key safety and efficacy results presented and their potential implications.","_key":"5636408d128f"}]},{"markDefs":[],"children":[{"text":"CGTLive:","_key":"8877a82e07af0","_type":"span","marks":["strong","em"]},{"_type":"span","marks":["strong"],"text":" Can you give some background context about P-BCMA-ALLO1 and what it is?","_key":"24a5415d083b"}],"_type":"block","style":"h3","_key":"95131aaf3f41"},{"_key":"2078bc759d0e","markDefs":[],"children":[{"_type":"span","marks":["strong"],"text":"Bhagirathbhai R. Dholaria, MD: ","_key":"649a575870dc"},{"marks":[],"text":"P-BCMA-ALLO1 is an off-the-shelf allogenic CAR T-cell therapy manufactured from healthy donor T-cells. The way it is different than other myeloma CAR-T therapies is that it is manufactured using a nonviral gene editing platform, as opposed to cilta-cel or ide-cel, which use virus-based vectors. [But] it also targets the BCMA protein, similar to other CAR T-cell therapies.","_key":"ab222ad51385","_type":"span"}],"_type":"block","style":"normal"},{"_type":"block","style":"h3","_key":"a78fffcdefb4","markDefs":[],"children":[{"_type":"span","marks":["strong"],"text":"Can you give an overview of the recently announced data from the phase 1/1b clinical trial?","_key":"3a13757030a2"}]},{"markDefs":[],"children":[{"_type":"span","marks":[],"text":"At the recent IMS meeting, we presented the data on a phase 1 trial using P-BCMA-ALLO1 at a different cell dose and a different lymphodepletion regimen. Overall, this looked at the data on 72 patients. These are heavily pretreated MM patients. Half of them had high-risk cytogenetics, and around 43% of the patients actually had prior other BCMA-targeted therapy or talquetamab, which is a GPRC5D-targeted bispecific antibody therapy. Several of these patients also had other CAR T-cell therapy before coming on this trial. ","_key":"500c589033950"}],"_type":"block","style":"normal","_key":"14952092c40b"},{"_key":"fd7e8d63ac40","markDefs":[],"children":[{"marks":[],"text":"Overall, 100% of the intention-to-treat patients actually ended up receiving the P-BCMA-ALLO1 infusion. The primary end point of this trial was the safety of P-BCMA-ALLO1. Looking at the safety aspect of the trial results, no graft versus host disease was reported. It is important, given that this is an off-the-shelf allogeneic T-cell being infused in the recipient. The risk of cytokine release syndrome (CRS) was relatively low. Only around 27% of the patients had this event. Most of the events were grade 1 or grade 2. No patient actually had grade 3 or higher CRS or neurotoxicity or immune effector cell-associated neurotoxicity syndrome. There were some infections seen, but the risk of grade 3 or higher infection was relatively low. ","_key":"9efb4802fb65","_type":"span"}],"_type":"block","style":"normal"},{"markDefs":[],"children":[{"_type":"span","marks":[],"text":"Looking at the in vivo kinetics, we noticed that with higher lymphodepletion we noticed better in vivo expansion and persistence of P-BCMA-ALLO1. That actually correlated with the antimyeloma activity of this agent compared to the standard lymphodepletion, where overall response rate (ORR) was only 21%. We noticed that in arms with enhanced lymphodepletion, such as arms A, B, and C, we noticed increasing ORR of 42%, 72%, and 91% respectively. Arm C, which is using cyclophosphamide 750 mg/m2/day is currently selected for phase 1b expansion, where we saw a 91% ORR, even in heavily pretreated MM patients.","_key":"9692c4614c03"}],"_type":"block","style":"normal","_key":"4909e183ce60"},{"style":"h3","_key":"56f82d62e8d9","markDefs":[],"children":[{"_key":"486fce2afd82","_type":"span","marks":["strong"],"text":"How would you summarize the big-picture implications that doctors and the broader healthcare community should take away from these findings?"}],"_type":"block"},{"markDefs":[],"children":[{"text":"This is still a very early study. I think we definitely need a larger patient cohort with longer follow-up to accurately understand the safety and more importantly the efficacy of this agent. However, in current era of bispecific antibodies and CAR T-cell therapies, there is still a huge unmet need of patients who have failed other bispecific antibody therapies, or patients who have failed or are not eligible for standard-of-care CAR T-cell therapy such as cilta-cel or ide-cel. Among those patients, I think this treatment can offer a good option if it turns out to be good enough and safe enough. Given the fact that this is an allogenic product, it's readily available. There is no leukapheresis needed. There is no waiting for manufacturing, which is a huge issue in autologous BCMA-targeted CAR T-cell therapy, which is takes anywhere from 4 to 6 weeks from the time of leukapheresis to actual cell infusion. Many of our high-risk patients are not able to wait that long, and they end up requiring bridging therapy, which none of the patients on this trial needed because P-BCMA-ALLO1 was available right away. So I think that this agent has potential to address that unmet need in high-risk, aggressively-progressing, extramedullary MM patients or high-risk MM patients who had failed other standard-of-care myeloma therapies.","_key":"e7cf7c6a0dfe0","_type":"span","marks":[]}],"_type":"block","style":"normal","_key":"1ba97d171ccb"},{"style":"h3","_key":"c048425fff7d","markDefs":[],"children":[{"text":"Have there been any challenges in the study and/or are there any areas of interest for further research?","_key":"9d03c4e4edf4","_type":"span","marks":["strong"]}],"_type":"block"},{"_type":"block","style":"normal","_key":"6a65969d84d5","markDefs":[],"children":[{"_key":"c7534f3a20780","_type":"span","marks":[],"text":"In terms of challenges, of course very early on, like I said, we realized that the standard lymphodepletion was just not good enough to have a better expansion, persistence, and efficacy of this drug. But we appear to have overcome that now. We actually did a very systemic dose escalation of the Cytoxan dose along with the cell dose expansion also, and we learned that there is a sweet spot—not the highest lymphodepletion, but we picked kind of the intermediate dose lymphodepletion, which is cyclophosphamide, cyclophosphamide 750 mg/m2/day, where we saw kind of the right balance of safety and efficacy. That's what we are currently expanding, both in patients who had prior BCMA-targeted therapies and patients who had no prior BCMA-targeted therapies. We are also exploring multiple cell dose infusions to improve the durability of the responses."}]},{"style":"normal","_key":"1e27ae9fbd54","markDefs":[],"children":[{"_type":"span","marks":["em","strong"],"text":"This transcript has been edited for clarity.","_key":"090e000f56cf0"}],"_type":"block"},{"_type":"block","style":"h6","_key":"3a5ccb5a7eed","markDefs":[],"children":[{"_type":"span","marks":[],"text":"REFERENCES\n1. Poseida Therapeutics garners FDA RMAT designation for allogeneic CAR-T P-BCMA-ALLO1 in r/r multiple myeloma. News release. Poseida Therapeutics, Inc. September 27, 2024. Accessed October 10, 2024. https://investors.poseida.com/news-releases/news-release-details/poseida-therapeutics-reports-positive-interim-phase-1-results","_key":"cb653088ad930"}]},{"_key":"68c7b9db0d9c","markDefs":[],"children":[{"_type":"span","marks":[],"text":"","_key":"08d1e30b91e50"}],"_type":"block","style":"normal"},{"children":[{"text":"","_key":"271de6c127c10","_type":"span","marks":[]}],"_type":"block","style":"normal","_key":"08507dbc0e28","markDefs":[]}],"documentGroup":null,"display_summary":true,"_updatedAt":"2024-11-22T10:38:53Z","_type":"article","contentCategory":{"_type":"contentCategory","name":"Articles","_id":"54fdc3d6-b776-4653-9c81-26acd95a8a7e","_updatedAt":"2022-07-01T12:33:15Z","_createdAt":"2020-12-08T16:21:29Z","_rev":"yD3aK4wI9vtjJazfqNRs7r"},"authors":[{"displayName":"Bhagirathbhai Dholaria, MBBS","url":"bhagirathbhai-dholaria-mbbs"},{"displayName":"Noah Stansfield","url":"noah-stansfield"}],"factCheckAuthorMapping":null,"factCheckAuthors":null,"documentGroupMapping":null,"seoTag":["gene therapy"],"summary":"Bhagirathbhai R. 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One of these is immunogenicity, which prevents redosing if the initial dose is insufficient or the effect of the therapy wears off over time. ","_key":"6c82da9de699"}],"_type":"block","style":"normal","_key":"34f33c1e6874"},{"_key":"b040ef347cf4","markDefs":[{"_type":"link","href":"https://www.cgtlive.com/conferences/american-society-for-gene-cell-therapy-asgct-","_key":"7933d246685f"}],"children":[{"_type":"span","marks":[],"text":"Ring Therapeutics is seeking to use anellovectors as potential alternatives to AAV vectors for gene therapy in order to address this issue. At ","_key":"6575c7e9699c0"},{"_key":"a8481a3c4c4e","_type":"span","marks":["7933d246685f"],"text":"the American Society of Gene \u0026 Cell Therapy (ASGCT) 27th Annual Meeting"},{"_type":"span","marks":[],"text":", held May 7 to 10, 2024, the company gave several presentations on its research. At the conference, ","_key":"b9139a2bc1f2"},{"_type":"span","marks":["em"],"text":"CGTLive®","_key":"81b0f6476f0f"},{"_key":"c2e68d297f3d","_type":"span","marks":[],"text":" interviewed Chris Wright, MD, PhD, the chief medical officer and head of translational research at Ring Therapeutics, to learn more."}],"_type":"block","style":"normal"},{"_type":"block","style":"h3","_key":"95131aaf3f41","markDefs":[],"children":[{"_type":"span","marks":["strong","em"],"text":"CGTLive:","_key":"8877a82e07af0"},{"_type":"span","marks":["strong"],"text":" Can you tell us about Ring's platform and its potential advantages?","_key":"24a5415d083b"}]},{"_type":"block","style":"normal","_key":"2078bc759d0e","markDefs":[],"children":[{"_type":"span","marks":["strong"],"text":"Chris Wright, MD, PhD: ","_key":"d88f4bbeb696"},{"_key":"51d159af8971","_type":"span","marks":[],"text":"There's a lot of great potential for gene therapy. You're here at the gene therapy conference, there's a lot of people, and everybody is excited about making a difference for patients that really need therapies. One of the challenges with current gene therapy, while it seems to be working in some areas, is that there's problems with it being immunogenic. You get safety problems because people recognize it as foreign, and then they have a reaction. That can lead to problems from the safety perspective, with side effects and the like. It also makes it challenging to give it to everybody because many people already have antibodies to the viruses that are used for gene therapy for the most part these days. So ideally, you'd like to find a virus that wouldn't have those properties, that you could give maybe even repeat doses of if you need to, and that wouldn't cause any side effects and could be given to a broader population of people. That's one of the main areas that Ring is working on. "}]},{"markDefs":[],"children":[{"_type":"span","marks":[],"text":"In order to try to understand, the viruses that might be suitable—that maybe have better properties—the company thought that maybe the best virus around is actually inside of us. What I mean by that is that it's known that the body has a bunch of viruses that just kind of hang out there and aren't doing anything harmful. People have heard about the microbiome, which is all the kind of bacteria in your body, but there's also a human virome, which are all the different viruses that are in your body. We were looking in the body for different viruses that are persisting there, but not causing any problems. We did find a number of viruses, and those are the anelloviruses. Anelloviruses actually make up about 80% of all the viruses in a person's body. And it turns out that that they've been there throughout all of the person's life, pretty much shortly after birth. So, they persist for a long period of time, and they don't appear to cause any problems, they don't induce an immune reaction, and they don't seem to have any associations with diseases. So, we thought that could be a great virus to use to try to deliver gene therapy because it should be a safe approach.","_key":"71453e0b953f"}],"_type":"block","style":"normal","_key":"f84d8e548dbf"},{"style":"h3","_key":"ccfa93d2e0f4","markDefs":[],"children":[{"marks":["strong"],"text":"Can you give an overview of Ring's presentations at the conference?","_key":"eaebcdb15127","_type":"span"}],"_type":"block"},{"_key":"30dbb818c4ba","markDefs":[],"children":[{"marks":[],"text":"We're fortunate to have 3 different abstracts accepted. We have 2 abstracts that were posters, and then I was able to have a talk that I'll be giving tomorrow. The posters are around some of the science. One thing about viral capsids is that sometimes they can only contain a certain amount of therapy. The idea is the more therapy you can get inside of it, the bigger the gene [can be], the more likely you're going to be able to treat many different diseases. We had one poster that showed that we could increase the size of the therapeutic gene. That was kind of a great advancement... The other poster is looking at how we're able to transduce different tissues, particularly in nonhuman primates (NHPs), in the eye. Getting to an NHP study is one of those studies that's very close to getting into humans eventually. So if you can take your new gene therapy and show that you can safely dose NHPs, that gives you a higher level of confidence that you can get into a human study sooner rather than later. That poster showed when we injected in the eye, we could get expression there. It was also the first time we demonstrated that you could put a gene in that is a therapeutic gene, and that you can have expression of that, as well. We showed that DNA and RNA was expressed in the NHP eyes, and that's sort of along the lines of the therapies that we want to develop. That was exciting to see. Redosability is also very important because right now, gene therapy is one-and-done. If you give it once, you can't ever give it again. And so what happens is sometimes maybe you underdose, and the person doesn't get the adequate response that you'd like them to have, but you can't do anything about that, or you might want to increase the duration of an effect—so sometimes gene therapies wear off, and then if you can't redose, then you're also kind of stuck. ","_key":"891cda568f180","_type":"span"}],"_type":"block","style":"normal"},{"markDefs":[],"children":[{"text":"This gives us the possibility to redose or to dose multiple times up front to get to the right therapeutic levels... It turns out that the outside of the capsid has pieces that are very variable, and so that means they're not really easily recognized by the immune system, and the parts of the capsid that are less variable are kind of hidden on the inside, so they can't be accessed by antibodies. So it's probably one of the reasons why it's immune silent. If you look at AAV, it actually has all of the more constant and nonvariable regions of the protein on the outside of the capsid. That kind of explains a little bit why it might be immune reactive, and why anello vectors might not be... This is all foundational knowledge that Ring discovered along with collaborators; in this case, it was at Johns Hopkins that collaborated to understand if there's any antibodies already in the body that recognize proteins on the capsid of the anello vector. If you look at all these capsid proteins and you try to find antibodies in human serum, you actually really almost find none. But if you look at other human viruses like HIV or HSV or even AAV, you see a lot of reactivity. That sort of also proved the point that anello viruses are sort of immune silent because they don't produce antibodies in people... ","_key":"44263b11f2e4","_type":"span","marks":[]}],"_type":"block","style":"normal","_key":"794b85a61169"},{"markDefs":[],"children":[{"marks":[],"text":"As I mentioned before, redosing is important because gene therapies can wear off, or you might not get the dose right the first time and you can't give it again. We did some experiments where we showed we could inject an anello vector that produced a marker that we could measure in the animals, and we showed that when you redose it, you actually get increased levels of the DNA, and when you redose AAV you actually got decreased levels. So that kind of speaks to the fact that with AAV, you're probably inducing an immune reaction, which means when you try to dose it a second time, you're not getting quite as much of the virus in the system, whereas with the anello vectors we saw actually a little bit of an increase in the liver in particular, and that's one of the organs where gene therapies tend to wear off. ","_key":"2dae13d71e9f","_type":"span"}],"_type":"block","style":"normal","_key":"7df6b52dcc12"},{"_type":"block","style":"normal","_key":"b9fc749651af","markDefs":[],"children":[{"_type":"span","marks":[],"text":"We also did another study looking at redosing in the eye in NHPs and in that experiment, we actually used a aflibbercept, which is our disease gene. The first one [we did] was kind of a marker gene, just like a proof-of-concept to show that we could do redosing. Then the second study was in NHPs, where we were injecting in the eye with a therapeutic payload to see whether we could redose there. We were able to show that we can actually redose there, which was really nice. If you give 2 doses, you get a lot higher DNA and RNA. In particular with eye diseases, some of the current gene therapies that are AAV-based sort of are starting to run out of steam. Their efficacy goes down after about 6 to 12 months. That's not a great situation to be in if you can't redose. So in our case, we could give a gene therapy and if the efficacy wears off, we could treat again to prolong the efficacy, and help patients for longer.","_key":"10aee32cd288"}]},{"_type":"block","style":"normal","_key":"1e27ae9fbd54","markDefs":[],"children":[{"text":"This transcript has been edited for clarity.","_key":"090e000f56cf0","_type":"span","marks":["em","strong"]}]},{"style":"normal","_key":"a6163508246b","markDefs":[{"_type":"link","href":"https://www.cgtlive.com/conferences/american-society-for-gene-cell-therapy-asgct-","_key":"135dc348e511"}],"children":[{"_key":"297856ce074e0","_type":"span","marks":["135dc348e511","strong","em"],"text":"Click here to view more coverage of the 2024 ASGCT Annual Meeting."}],"_type":"block"},{"style":"h6","_key":"5c86d428481e","markDefs":[],"children":[{"_type":"span","marks":[],"text":"REFERENCES\n1. Bounoutas GS, Pozsgai R, Gold I, et al. Anellovectors, a Gene Delivery Platform Based on Commensal Human Anelloviruses, Have the Potential to Evade the Immune System and Deliver DNA Payloads to a Broad Range of Tissues in a Redosable Manner. Presented at: ASGCT 27th Annual Meeting, May 7-10; Baltimore, Maryland. Abstract #203","_key":"42da5d9bbaf80"}],"_type":"block"},{"children":[{"_type":"span","marks":[],"text":"","_key":"08d1e30b91e50"}],"_type":"block","style":"normal","_key":"68c7b9db0d9c","markDefs":[]},{"_type":"block","style":"normal","_key":"08507dbc0e28","markDefs":[],"children":[{"marks":[],"text":"","_key":"271de6c127c10","_type":"span"}]}],"factCheckAuthorMapping":null,"targeting":{"content_placement":["topic/neurology","conferences","news"],"document_url":["anelloviruses-potential-alternative-aav-gene-therapy"],"document_group":{"identifier":{"current":"american-society-for-gene-cell-therapy-asgct-","_type":"slug"},"_rev":"0XSEuRf1Tr20DJpKe4dB71","_type":"documentGroup","_id":"35821c62-f2b7-4f15-8ec5-becad3bdf6b8","pixelTrackingCode":null,"parent":{"_id":"bae95326-2090-4312-85b8-c56d8cd5f294","_updatedAt":"2022-03-03T07:30:12Z","identifier":{"current":"conferences","_type":"slug"},"_createdAt":"2021-06-04T17:10:51Z","parent":null,"_rev":"33TqzEVq9GPvfh5kXjm7SH","_type":"documentGroup","name":"Conferences"},"_createdAt":"2021-06-24T19:03:21Z","name":"American Society for Gene \u0026 Cell Therapy (ASGCT)","_updatedAt":"2021-06-24T19:04:23Z"},"rootDocumentGroup":["conferences"],"issue_url":"","publication_url":""},"relatedArticles":[{"title":"CGTLive®’s Weekly Rewind – November 22, 2024 ","url":{"current":"cgtlive-weekly-rewind-november-22-2024","_type":"slug"},"thumbnail":{"_type":"mainImage","alt":"CGTLive®’s Weekly Rewind","caption":"CGTLive®’s Weekly Rewind","asset":{"_ref":"image-1feeab70bc7d00036bee4f6bc891982276d46bc0-500x500-jpg","_type":"reference"}},"published":"2024-11-22T20:00:00.000Z"},{"title":"David Barrett, JD, on Future Trends for Genomic Medicines","url":{"current":"barrett-future-trends-genomic-medicines","_type":"slug"},"thumbnail":{"_type":"mainImage","alt":"David Barrett, JD, the chief executive officer of ASGCT","caption":"David Barrett, JD","asset":{"_ref":"image-73c396ac9ec1c25274e5148785e53cc12aad6577-755x426-png","_type":"reference"}},"published":"2024-11-22T12:00:05.483Z"},{"title":"Evaluating Allogeneic CAR-T P-BCMA-ALLO1 in R/R Multiple Myeloma","url":{"current":"evaluating-allogeneic-car-t-p-bcma-allo1-multiple-myeloma","_type":"slug"},"thumbnail":{"alt":"Bhagirathbhai R. 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Nolan Townsend, Sandi See Tai, MD, and Kim G. Johnson, MD, discussed Lexeo Therapeutics’ LX1001 gene therapy trial that demonstrated promising safety and biomarker effects in patients with early-stage Alzheimer disease. ","body":[{"_key":"fac7a2eb68b2","markDefs":[],"children":[{"_type":"span","marks":[],"text":"","_key":"9c5dff2f12a7"}],"_type":"block","style":"normal"},{"asset":{"_ref":"image-06e896e8fdad4ea58e0766e784b9513070d4dc28-300x300-jpg","_type":"reference"},"disableTextWrap":false,"disableLightBox":true,"_type":"figure","alt":"Sandi See Tai, MD, chief development officer at Lexeo","imgcaption":[{"children":[{"_type":"span","marks":[],"text":"Sandi See Tai, MD","_key":"42c2728cd07f0"}],"_type":"block","style":"normal","_key":"9e7d28369cea","markDefs":[]}],"_key":"22743fa4c361","alignment":"left"},{"style":"normal","_key":"b9dd9d12b2f3","markDefs":[{"_type":"link","href":"https://www.neurologylive.com/conferences/ctad","_key":"abcd9dae0dc5"}],"children":[{"_type":"span","marks":[],"text":"Interim safety and efficacy data regarding Lexeo Therapeutics' LX1001, an investigational adeno-associated virus (AAV) vector-based gene therapy, were recently gathered in a phase 1/2, open-label clinical trial (NCT03634007) in patients with ","_key":"162b63ff5b8d"},{"marks":["em"],"text":"APOE4","_key":"d5ab57e3d34a","_type":"span"},{"_type":"span","marks":[],"text":" homozygote Alzheimer disease (AD). The data were presented at the ","_key":"8aecc6163e10"},{"_type":"span","marks":["abcd9dae0dc5"],"text":"2024 Clinical Trials on Alzheimer’s Disease (CTAD) conference","_key":"1551be93cd41"},{"_key":"c78964524d57","_type":"span","marks":[],"text":", held October 29 to November 1, in Madrid, Spain."}],"_type":"block"},{"children":[{"_type":"span","marks":[],"text":"Shortly before the presentation, ","_key":"0d4caad442dc"},{"marks":["em"],"text":"CGTLive®","_key":"3ca79fa7f8c7","_type":"span"},{"_type":"span","marks":[],"text":"'s sister site ","_key":"81ef57820079"},{"text":"NeurologyLive® ","_key":"2c5c8f5ededf","_type":"span","marks":["em"]},{"marks":[],"text":"interviewed","_key":"152f48bccd64","_type":"span"},{"_type":"span","marks":["em"],"text":" ","_key":"97a5367b4ad3"},{"_type":"span","marks":[],"text":"R. Nolan Townsend, chief executive officer at Lexeo, coauthor Sandi See Tai, MD, chief development officer at Lexeo, and lead author Kim G. Johnson, MD, the division chief of memory disorders at Duke University, to get some more background about the gene therapy and the trial. The 3 experts explained the rationale behind the approach and discussed the early findings.","_key":"e95ce5699910"}],"_type":"block","style":"normal","_key":"ea7ef3e0bb1d","markDefs":[]},{"markDefs":[],"children":[{"_type":"span","marks":["strong"],"text":"Can you speak about the presentation from your perspectives?","_key":"1f17a6955a12"}],"_type":"block","style":"h3","_key":"69bd67090263"},{"markDefs":[],"children":[{"marks":["strong"],"text":"R. Nolan Townsend:","_key":"ece631de82080","_type":"span"},{"_type":"span","marks":[],"text":" Lexeo is a clinical stage gene therapy company focused in genetic cardiac disease and AD. The program that we'll be discussing in the presentation tomorrow is LX1001, it's the company's most advanced clinical stage program focused on APOE4 homozygous AD, where we're delivering the APOE2 gene to the CNS of APOE4 homozygotes. Taking a step back, we looked at the overall AD treatment landscape and found that there were not sufficient treatment options for E4 homozygotes—in a disease with very complex biology, the thinking from a gene therapy company is to go upstream—treat the genetics of the disease, which we would expect to have a downstream impact on multiple different pathogenic mechanisms simultaneously. This is the study which we've advanced over the last couple of years and this is a data which we'll be reporting at this conference. We think it's exciting that a gene therapy has been introduced to the AD population and we're excited to present the results from the study in the conference.","_key":"ba9bda2f1361"}],"_type":"block","style":"normal","_key":"b6c6275e764f"},{"markDefs":[],"children":[{"_type":"span","marks":["strong"],"text":"Sandi See Tai, MD:","_key":"a3ff9a0bf9ae0"},{"_type":"span","marks":[],"text":" I can give a brief overview of the study. We included APOE4 homozygotes with mild cognitive impairment and also mild to moderate dementia due to AD. We enrolled 15 patients across 4 dose escalation cohorts. The vector we're using is an AAVrh10, and we administer the APOE2 gene, as Nolan mentioned. We administer that intrathecally, or intracisternal. We followed the patients out through 1 year initially, and then they can move over to long-term follow up study. Across the 15 patients, what we've been able to demonstrate and we were really happy to see is whether you can actually achieve transduction and expression of the protein. We've been able to show APOE2 protein expression in the CSF across all the patients that were dosed. That has translated to seeing some stabilization in terms of amyloid biomarkers, and then also we're particularly encouraged at seeing reductions across multiple tau biomarkers—whether it's t tau, p tau, but also on our tau PET—and so we look at that in combination with the safety profile that we've observed, which is one that has shown LX1001 to be generally well-tolerated. We had some CSF pleocytosis that was observed, but it was transient and not associated with any adverse events. Most importantly, we did not see any events of amyloid related imaging abnormalities, which was to us a key concern, given that we were going specifically into E4 homozygotes. We felt confident in terms of looking at what we're seeing so far in terms of a benefit risk profile here.","_key":"50b6d50d7ba0"}],"_type":"block","style":"normal","_key":"a7fb4022f514"},{"_type":"block","style":"normal","_key":"4c958eb9c3c1","markDefs":[],"children":[{"_type":"span","marks":["strong"],"text":"Kim G. Johnson, MD:","_key":"f5e8563c25150"},{"_type":"span","marks":[],"text":" I'll speak a little bit about the participant experience, since that's the population that I have the most interaction with as one of the primary investigators. I would say that APOE4/APOE4s have no good treatment options right now and this trial is a potential treatment option for this population that they're very hopeful about. I found that working with participants, participants not only wanted to do this trial for themselves and see how the disease affects them, but also for their children and their grandchildren, because they know that there's a genetic variant to the disease. They also know that recent research shows that all E4/4s will develop amyloid pathology by about 65 years of age, and if they end up developing cognitive symptoms, they often have the fastest progression in the disease. I think that for an E4/4, knowing that can be a little hopeless. ","_key":"61422d6afe3e"}]},{"_type":"block","style":"normal","_key":"8eba2172e1a6","markDefs":[],"children":[{"_type":"span","marks":[],"text":"We do know also with research that people with ","_key":"eae490e0475b"},{"_type":"span","marks":["em"],"text":"APOE2","_key":"0597b64d5087"},{"marks":[],"text":" have less rates of AD. If they do get AD (for example, if they are an ","_key":"8f317ecd89ee","_type":"span"},{"_type":"span","marks":["em"],"text":"APOE2/4","_key":"c949aeb641c4"},{"marks":[],"text":") the disease progresses slower. So I think introducing an ","_key":"55d58f060b25","_type":"span"},{"_type":"span","marks":["em"],"text":"APOE2 ","_key":"36c3f9bbe03e"},{"_type":"span","marks":[],"text":"as","_key":"03227eceb6b3"},{"_type":"span","marks":["em"],"text":" ","_key":"634bf58860b1"},{"marks":[],"text":"a protective factor gives participants who are E4/4s hope. That was really important and one of the things that I took away from the trial working with people: that this treatment could offer people hope. I think, as Sandy was saying, it's really important that we did a trial in only 15 people to just see if it was safe. That was the primary reason to do the trial, and we found out that it was basically safe. Out of the 15 people it was well-tolerated in the patient population, with 3 serious adverse events and 1 serious adverse event that involved some mild to moderate hearing loss. So it was pretty well-tolerated in the patient population and as Sandy mentioned, also affected key biomarkers of tau.","_key":"4576ef5fb6af","_type":"span"}]},{"_type":"block","style":"normal","_key":"00d4040c369b","markDefs":[],"children":[{"text":"This transcript has been edited for clarity.","_key":"1d62fdf665340","_type":"span","marks":["strong","em"]}]},{"_type":"block","style":"h6","_key":"a8243a899eab","markDefs":[],"children":[{"_key":"51c923734d4e0","_type":"span","marks":[],"text":"REFERENCES\n1. Johnson K, et al. Safety and Preliminary Efficacy of AAV Gene Therapy (LX1001) in Patients with APOE4 Homozygote Alzheimer’s Disease – Interim Data from a Phase 1/2, Open-Label, 52-Week, Multicenter Study. Presented at: 2024 CTAD; October 29-November 1; Madrid, Spain. Abstract 486.\n2. Lexeo Therapeutics Announces Positive Interim Data for LX1001, First-Ever Gene Therapy to Impact the Underlying Genetic Cause of APOE4-Associated Alzheimer’s Disease, at the Clinical Trials on Alzheimer’s Disease (CTAD) Conference. News Release. Lexeo Therapeutics. Published October 30, 2024. Accessed October 20, 2024."}]}],"display_summary":true,"_type":"article","_rev":"DnHvGJh1KCyBWsJCLXPbpl","factCheckAuthorMapping":null,"factCheckAuthors":null,"url":"gene-therapy-apoe4-homozygous-alzheimer-disease","_updatedAt":"2024-11-06T11:15:22Z","internalTag":["AAV","gene therapy","Seattle Children’s Research Institute","hemophilia","hemophilia A","gene editing","gene delivery","nonviral","delivery methods","preclinical","preclinical research","clinical","safety"],"articleType":"Commentary","targeting":{"content_placement":["topic/neurology","conferences","news"],"document_url":["gene-therapy-apoe4-homozygous-alzheimer-disease"],"document_group":null,"rootDocumentGroup":[],"issue_url":"","publication_url":""},"relatedArticles":[{"title":"CGTLive®’s Weekly Rewind – November 22, 2024 ","url":{"current":"cgtlive-weekly-rewind-november-22-2024","_type":"slug"},"thumbnail":{"_type":"mainImage","alt":"CGTLive®’s Weekly Rewind","caption":"CGTLive®’s Weekly Rewind","asset":{"_ref":"image-1feeab70bc7d00036bee4f6bc891982276d46bc0-500x500-jpg","_type":"reference"}},"published":"2024-11-22T20:00:00.000Z"},{"title":"David Barrett, JD, on Future Trends for Genomic Medicines","url":{"current":"barrett-future-trends-genomic-medicines","_type":"slug"},"thumbnail":{"_type":"mainImage","alt":"David Barrett, JD, the chief executive officer of ASGCT","caption":"David Barrett, JD","asset":{"_ref":"image-73c396ac9ec1c25274e5148785e53cc12aad6577-755x426-png","_type":"reference"}},"published":"2024-11-22T12:00:05.483Z"},{"title":"Evaluating Allogeneic CAR-T P-BCMA-ALLO1 in R/R Multiple Myeloma","url":{"current":"evaluating-allogeneic-car-t-p-bcma-allo1-multiple-myeloma","_type":"slug"},"thumbnail":{"alt":"Bhagirathbhai R. 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It also contains up-to-date clinical news coverage in the field of gene and cell therapy for central nervous system disorders.","_key":"40352ebad809","_type":"span"}],"_type":"block"}],"perKeywordMapping":["Cardiology","Neurology","Health Equity Diversity \u0026 Inclusion"],"name":"Neurology","pixelTrackingCode":null,"_rev":"USRhz6Ofj9athfco75TZJx","_type":"taxonomy","identifier":"neurology","cmeType":"per","_id":"721f499d-3ed3-4310-94c7-f646bad56dc2","_updatedAt":"2023-11-27T17:07:43Z","parent":{"identifier":"topic","_createdAt":"2020-12-08T16:06:37Z","_rev":"60drdzXgFuuRxJsEyE6his","_id":"8ea01556-869e-480b-83a7-58eca140415e","_updatedAt":"2023-01-19T14:52:23Z","isMainTopic":true,"_type":"taxonomy","name":"Topic","parent":null},"_createdAt":"2020-12-08T16:07:23Z"},{"_createdAt":"2021-06-04T17:45:47Z","_rev":"GN79vWlZ4wTFBK5Yzyy182","_type":"taxonomy","pixelTrackingCode":null,"identifier":"conferences","name":"Conferences","_id":"53e0f868-dd0a-4d39-934f-6f8217803a2f","_updatedAt":"2022-03-03T07:15:17Z","parent":null},{"_type":"taxonomy","parent":null,"name":"News","_id":"bdd20025-c759-4afc-b2d4-7e727e4253cd","_updatedAt":"2022-03-03T06:51:18Z","identifier":"news","_createdAt":"2020-12-08T16:06:16Z","_rev":"2PyInWQFspcTNAL4XcZkLr","pixelTrackingCode":null}],"summary":"The noninvasive gene therapy exceeded the 30% efficacy threshold for normalizing GCase activity across all doses.","display_summary":true,"is_visible":true,"_rev":"DnHvGJh1KCyBWsJCLTWlLd","body":[{"markDefs":[{"href":"https://www.neurologylive.com/view/cap-003-demonstrates-disease-modifying-properties-preclinical-study-gba-related-parkinson","_key":"cbcdc47493f9","nofollow":true,"blank":true,"_type":"link"}],"children":[{"_type":"span","marks":["em","strong"],"text":"This article originally appeared on our sister site, ","_key":"496ac11fb78e"},{"text":"NeurologyLive®.","_key":"962c5a270a26","_type":"span","marks":["em","strong","cbcdc47493f9"]}],"_type":"block","style":"h6","_key":"b7892056bc34"},{"children":[{"_type":"span","marks":[],"text":"Capsida Therapeutics' CAP-003, an intravenously (IV) delivered gene therapy intended to treat Parkinson disease associated with ","_key":"e04a976a36760"},{"_type":"span","marks":["em"],"text":"GBA","_key":"ecab46bdc57a"},{"text":" mutations (PD-GBA), produced a brainwide RNA expression in nonhuman primates (NHPs) that was more than 200-fold greater than IV administered adeno-associated virus serotype 9 (AAV9) along with substantial increases in GCase protein and enzyme activity compared with untreated NHPs.","_key":"352c2b9d0c02","_type":"span","marks":[]},{"marks":["superscript"],"text":"1","_key":"9d885dc5d896","_type":"span"},{"_type":"span","marks":[],"text":" The data were presented in a late-breaking poster presented at the Society for Neuroscience annual meeting, held October 5-9, in Chicago, Illinois.","_key":"14643a8d0995"}],"_type":"block","style":"normal","_key":"04742d844f4d","markDefs":[]},{"children":[{"_type":"span","marks":[],"text":"\"Capsida's wholly owned novel gene therapy offers the potential to normalize GCase activity in patients with a single IV infusion safely, enabling the potential for long-term disease modification and substantial slowing of disease progression,\" Peter Anastasiou, MBA, the chief executive officer of Capsida, said in statement.","_key":"a4f13e0a8b760"},{"_type":"span","marks":["superscript"],"text":"1","_key":"a29017805c10"},{"_type":"span","marks":[],"text":" \"These data give us confidence that we are on track to enter the clinic with CAP-003 in the first half of 2025.\"","_key":"2691c6fc6984"}],"_type":"block","style":"normal","_key":"ecdc5275b25a","markDefs":[]},{"_type":"block","style":"normal","_key":"a4b0a125d875","markDefs":[],"children":[{"_key":"4470c31915f50","_type":"span","marks":[],"text":"CAP-003 is intended to enable production of the GCase enzyme, the gene for which is mutated in PD-GBA, with the aim of slowing disease progression via restoration of normal enzyme activity. The findings in the NHPs indicated that all CAP-003 doses exceeded the 30% efficacy threshold that would be expected to be required for normalizing GCase activity in human patients. Capsida noted that GCase activity in the NHPs, including withinin the substantial nigra, was 2-8-fold higher than that threshold."}]},{"markDefs":[],"children":[{"_type":"span","marks":[],"text":"CAP-003 delivered at a dose of 5.5x10","_key":"673abad8ffba0"},{"_type":"span","marks":["superscript"],"text":"13","_key":"a83a2306df07"},{"marks":[],"text":" vg/kg effected a 488% increase in GCase brain protein level, which was 8 to 24-times higher than that effected by ICM AAV9 in a previous NHP study. Furthermore, a 2.2x10","_key":"3f5f54b58ddf","_type":"span"},{"text":"13","_key":"ed008392980f","_type":"span","marks":["superscript"]},{"_type":"span","marks":[],"text":" vg/kg dose of the gene therapy produced a 335% increase in GCase protein. ","_key":"54c00d44a20b"}],"_type":"block","style":"normal","_key":"e83cf30475dc"},{"markDefs":[],"children":[{"_key":"19f69789a41d","_type":"span","marks":[],"text":"In addition, levels of glucosylsphingosine (GluSph) were reduced in the terminal plasma of NHPs treated with CAP-003, suggesting lysosomal activity and target engagement. It was noted that the 2.2x10"},{"_type":"span","marks":["superscript"],"text":"13","_key":"dbdc45367ffd"},{"_type":"span","marks":[],"text":" vg/kg dose reduced GluSph levels by 59% and the 5.5x10","_key":"5ac29617a585"},{"_type":"span","marks":["superscript"],"text":"13","_key":"dc3e44196e64"},{"marks":[],"text":" vg/kg dose reduced GluSph levels by 79%.","_key":"a8ff29794ab2","_type":"span"}],"_type":"block","style":"normal","_key":"38bc9199da13"},{"_key":"7f63f06f093f","markDefs":[],"children":[{"_key":"eb4622ed69180","_type":"span","marks":[],"text":"In the analysis, the average GCase activity in the brain of treated NHPs showed significant positive correlation with GCase protein levels in the cerebrospinal fluid (CSF) and trend of positive correlation with GCase activity in the CSF. In the CSF of NHPs that received CAP-003 at a dose of 5.5x10"},{"_type":"span","marks":["superscript"],"text":"13","_key":"d18e2ac4a0ec"},{"_type":"span","marks":[],"text":" vg/kg, a 1068% increase in GCase protein was observed along with a 103% increase in GCase activity. Overall, these data raise confidence in the use of CSG GCase biomarkers in the clinic.","_key":"89ab8c584e2a"}],"_type":"block","style":"normal"},{"markDefs":[],"children":[{"_type":"span","marks":[],"text":"NHPs treated with CAP-003 also showed significantly lessened DNA and RNA biodistribution to the liver and DRGs in comparison to historical IV-delivered AAV9 (4-week in-life, non-GBA cargo); specifically, a 19-fold lower liver biodistribution and a 17-fold lower expression in DRGs were reported. Notably, no adverse histopathology resulted.","_key":"8d6ff69054940"}],"_type":"block","style":"normal","_key":"28cb306c442c"},{"children":[{"_type":"span","marks":[],"text":"Approximately 5-15% of patients with PD have mutations in the ","_key":"7f1de32f1d8d0"},{"marks":["em"],"text":"GBA","_key":"7f1de32f1d8d1","_type":"span"},{"_type":"span","marks":[],"text":" gene, making it numerically the most important genetic risk factor for PD. Clinically, ","_key":"7f1de32f1d8d2"},{"_type":"span","marks":["em"],"text":"GBA","_key":"7f1de32f1d8d3"},{"_type":"span","marks":[],"text":"-PD is identical to sporadic PD, aside from the earlier age at onset, more frequent cognitive impairment, and more rapid progression. Mutations in the ","_key":"7f1de32f1d8d4"},{"_type":"span","marks":["em"],"text":"GBA","_key":"7f1de32f1d8d5"},{"_key":"7f1de32f1d8d6","_type":"span","marks":[],"text":" gene may lead to loss of GCase activity and lysosomal dysfunction, which may impair alpha-synuclein metabolism."}],"_type":"block","style":"normal","_key":"3a536863f966","markDefs":[]},{"_key":"5c1762b67596","markDefs":[],"children":[{"_type":"span","marks":[],"text":"In recent years, there has been greater industry interest in identifying effective treatments for GBA-PD, with several agents currently in low-level clinical trials right now. In August 2023, BIAL R\u0026D announced the first patients dosed in their phase 2 trial, ACTIVATE (NCT05819359), to investigate BIA 28-6156, an allosteric activator of GCase, as a treatment for patients with GBA-PD. The multicenter, randomized, double-blind, placebo-controlled study will assess the efficacy, safety, pharmacokinetics, and pharmacodynamics of 2 fixed dose levels of BIA 28-6156 (10 mg and 60 mg/day).","_key":"3d43f6af138e0"},{"_type":"span","marks":["superscript"],"text":"3","_key":"c9c77f230682"}],"_type":"block","style":"normal"},{"_type":"block","style":"normal","_key":"44813e95375f","markDefs":[{"_key":"1181fa85a908","nofollow":true,"_type":"link","href":"https://www.neurologylive.com/conferences/mds"},{"nofollow":true,"_type":"link","href":"https://www.neurologylive.com/view/understanding-therapeutic-potential-gt-02287-gba-parkinson-disease","_key":"59e40033afe0"}],"children":[{"_type":"span","marks":[],"text":"More recently, at the ","_key":"9e670b35e28a0"},{"_key":"9e670b35e28a1","_type":"span","marks":["1181fa85a908"],"text":"2024 International Congress of Parkinson’s Disease and Movement Disorders"},{"_type":"span","marks":[],"text":", data from a phase 1 first-in-human trial demonstrated the early efficacy and safety of ","_key":"9e670b35e28a2"},{"text":"GT-02287 in healthy volunteers","_key":"9e670b35e28a3","_type":"span","marks":["59e40033afe0"]},{"_type":"span","marks":[],"text":". GT-02287, an orally-bioavailable, brain-penetrant molecule, is also in development for patients with PD with or without a ","_key":"9e670b35e28a4"},{"_key":"e8966ca4981c","_type":"span","marks":["em"],"text":"GBA1"},{"_type":"span","marks":[],"text":" mutation. All told, the therapy was safe and generally well tolerated at single (2.4, 4.8, 7.7, 10.0, and 15.0 mg/kg) and multiple (4.8, 7.7, 10.0, and 13.5 mg/kg) doses, with nausea and headache as the most common AEs.","_key":"fb4a927c3096"},{"_type":"span","marks":["superscript"],"text":"4","_key":"9d1770c4eb17"}]},{"markDefs":[],"children":[{"_type":"span","marks":[],"text":"REFERENCES\n1. New Data Demonstrate Substantial Therapeutic Potential of Capsida's IV Gene Therapy for Parkinson's Disease Associated with GBA Mutations. News release. Capsida. October 7, 2024. Accessed October 25, 2024. https://www.prnewswire.com/news-releases/new-data-demonstrate-substantial-therapeutic-potential-of-capsidas-iv-gene-therapy-for-parkinsons-disease-associated-with-gba-mutations-302267777.html\n2. Unlocking the Potential of Gene Therapy for All. Capsida. October 2024. Accessed October 25, 2024. https://capsida.com/wp-content/uploads/Capsida-Non-Confidential-Corporate-Deck-10042024.pdf\n3. BIAL R\u0026D Announces First Patient Dosed in its Phase 2 Clinical Trial of BIA 28-6156 to Treat Parkinson's Disease Patients With a Pathogenic Variant in the Glucocerebrosidase (GBA1) Gene. News Release. Bial. Published May 25, 2023. Accessed October 25, 2024. https://prnmedia.prnewswire.com/news-releases/bial-rd-announces-first-patient-dosed-in-its-phase-2-clinical-trial-of-bia-28-6156-to-treat-parkinsons-disease-patients-with-a-pathogenic-variant-in-the-glucocerebrosidase-gba1-gene-301833969.html\n4. Gain Therapeutics Announces Positive Topline Results from the Phase 1 Clinical Trial of GT-02287, a Novel GCase-Targeting Small Molecule Therapy for Parkinson’s Disease. News release. Gain Therapeutics. August 29, 2024. 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Fifteen patients were dosed in the trial across 3 ascending single-dose cohorts: 1.4x10"},{"_type":"span","marks":["superscript"],"text":"10","_key":"6322277e8c2d"},{"_type":"span","marks":[],"text":" gc/ml (C1; n = 5), 4.4x10","_key":"b76a354720b7"},{"_type":"span","marks":["superscript"],"text":"10","_key":"8fea9c4b90d3"},{"_key":"5f6410c5ef23","_type":"span","marks":[],"text":" gc/ml (C2; n = 4), 1.4x10"},{"marks":["superscript"],"text":"11","_key":"e040476291a0","_type":"span"},{"_type":"span","marks":[],"text":" gc/ml (C3; n = 3). A single fixed-dose cohort was also included, treating patients at a dose of 1.4x10","_key":"ba6c33a43eec"},{"_type":"span","marks":["superscript"],"text":"14","_key":"d75d1f8bdf4a"},{"text":" gc (C4; n = 3). Participants in the 52-week study were administered prednisone for 8 weeks after treatment with LX1001. APOE4 homozygous status, an age of at least 50 years, positive amyloid PET, CSF biomarkers consistent with AD, and mild cognitive impairment or mild to moderate dementia due to AD were requirements for inclusion in the study.","_key":"d3049490f63c","_type":"span","marks":[]}]},{"markDefs":[],"children":[{"_type":"span","marks":[],"text":"In terms of safety, the gene therapy was deemed safe and well-tolerated, with no cases of amyloid-related imaging abnormalities (ARIA) reported. Expression of APOE2 in the CSF was observed in all of the patients, with a dose- and time-dependent increase in APOE2e4 expression noted. A decrease in CSF t-tau and phosphorylated-tau181 in 9 of the 13 participants was observed, although no directional trend in CSF amyloid-ß42/40 or amyloid PET was recorded. Notably, a decrease in CSF p-tau217 and p-tau231 was also seen in the 9 aforementioned patients.","_key":"1ae604ac3c9e0"}],"_type":"block","style":"normal","_key":"c61fd53bb6ef"},{"markDefs":[],"children":[{"_key":"82d9b49e72320","_type":"span","marks":[],"text":"\"In light of the rapid progression of AD in this population, these data highlight the therapeutic potential of delivering APOE2, which can impact multiple mechanisms of AD upstream of any specific pathway and thereby meaningfully alter the devastating course of this complex disease,\" Sandi See Tai, MD, the chief development officer at Lexeo, said in a statement."},{"_type":"span","marks":["superscript"],"text":"1","_key":"b56e7aa35aca"},{"_key":"92791f99bae1","_type":"span","marks":[],"text":" \"These data are highly encouraging and provide clinical evidence of the unique and targeted mechanism of LX1001 to potentially treat AD.\""}],"_type":"block","style":"normal","_key":"07bfc24b6fc2"},{"style":"normal","_key":"ea1500798186","markDefs":[],"children":[{"text":"Previous research has revealed that people who are homozygous for ","_key":"9269ce6bd44e0","_type":"span","marks":[]},{"text":"APOE4","_key":"9b22548765ad","_type":"span","marks":["em"]},{"_type":"span","marks":[],"text":" have a 14.5-fold higher risk of late-onset AD than people homozygous for ","_key":"d772fc38e170"},{"_type":"span","marks":["em"],"text":"APOE3","_key":"f2b619b15721"},{"_type":"span","marks":[],"text":", and that patients heterozygous for ","_key":"d31a86c3575e"},{"_type":"span","marks":["em"],"text":"APOE2/E4","_key":"7ea29cdb757f"},{"_type":"span","marks":[],"text":" face a 2.6-fold increased risk, suggesting that ","_key":"29f14b7952ff"},{"_key":"cb3c0e17cafc","_type":"span","marks":["em"],"text":"APOE2 "},{"_type":"span","marks":[],"text":"may play a protective role. LX1001 is intended to deliver the ","_key":"6b20fb0a011f"},{"marks":["em"],"text":"APOE2","_key":"9269ce6bd44e1","_type":"span"},{"text":" gene into the central nervous system of patients with AD who are homozygous for ","_key":"9269ce6bd44e2","_type":"span","marks":[]},{"_type":"span","marks":["em"],"text":"APOE4","_key":"7096dbdf05e6"},{"_type":"span","marks":[],"text":", with the aim of converting the profile of their cells to ","_key":"f529968fc167"},{"_type":"span","marks":["em"],"text":"APOE2/E4","_key":"ea3707851342"},{"text":" heterozygosity and thus potentially slowing progression of the disease. No therapies that targeted specifically at treating the population of patients with AD who are homozygous for ","_key":"809298e8d92d","_type":"span","marks":[]},{"_key":"1f1506c53e5b","_type":"span","marks":["em"],"text":"APOE4"},{"_key":"2770c967d422","_type":"span","marks":[],"text":" are currently approved by the FDA."}],"_type":"block"},{"markDefs":[],"children":[{"_key":"c0b67127d09b0","_type":"span","marks":[],"text":"The analysis presented at CTAD 2024 included 12-month data from patients in cohorts C1-C3 and 6-month data for those in C4. In the study, investigators observed transient CSF pleocytosis (\u003e5 cells/ul), predominantly lymphocytic, in 12 patients, with no significant associated adverse events. In addition, tau PET evaluated in C3 and C4 demonstrated a decrease in global update in 5 of the 6 participants assessed."}],"_type":"block","style":"normal","_key":"dde90011b9e2"},{"children":[{"_type":"span","marks":[],"text":"\"With research, we know that people with ","_key":"5c65a7c04dbb0"},{"text":"APOE2","_key":"1258635d7fc5","_type":"span","marks":["em"]},{"_type":"span","marks":[],"text":" have less rates of getting AD,\" Kim G. Johnson, MD, primary investigator of the trial, told ","_key":"88f6d40558dc"},{"_type":"span","marks":["em"],"text":"NeurologyLive","_key":"62b594b2cd56"},{"_type":"span","marks":[],"text":"®. \"If they do get AD, but with ","_key":"6554781f5771"},{"marks":["em"],"text":"APOE2/4","_key":"fd56fa83da2e","_type":"span"},{"text":", the disease progresses slower. Introducing an [","_key":"bda0abaede4d","_type":"span","marks":[]},{"marks":["em"],"text":"APOE","_key":"1256f1f52e0a","_type":"span"},{"text":"] 2 factor gives participants who are ","_key":"20f99c4d5dcd","_type":"span","marks":[]},{"_type":"span","marks":["em"],"text":"APOE4","_key":"0b1e4295f0ab"},{"_type":"span","marks":[],"text":" [carriers] hope. That was really important.\"","_key":"3ee6487d4af0"}],"_type":"block","style":"normal","_key":"c748507bccfc","markDefs":[]},{"children":[{"_type":"span","marks":[],"text":"Johnson, who serves as the division chief of memory disorders at Duke University, added that, \"one of the things I took away from this trial was that this treatment could offer people hope. It’s important that we did a trial in only 15 people to see if it was safe. That was the primary reason to do the trial, and we found that [LX1001] was safe.\"","_key":"f8797711e18a0"}],"_type":"block","style":"normal","_key":"09aa959dfd9f","markDefs":[]},{"_key":"b55783d6eb98","markDefs":[],"children":[{"_type":"span","marks":[],"text":"Lexeo originally released topline results from the lowest-dose group of the study in March 2022. All told, the APOEe2 protein was detectable in CSF after 3 months in all 4 participants in the cohort, and after 1 year in the 2 who reached that time point. In these 2, total tau and p-tau reportedly declined from baseline at 1 year with no serious adverse events reported.","_key":"52e43bcfa9eb0"},{"text":"3","_key":"77c5c5577d33","_type":"span","marks":["superscript"]}],"_type":"block","style":"normal"},{"style":"normal","_key":"09a515d1f1bf","markDefs":[{"blank":true,"_type":"link","href":"https://www.cgtlive.com/view/townsend-tai-johnson-gene-therapy-apoe4-homozygous-alzheimer-disease","_key":"42258814e2f0","nofollow":true}],"children":[{"_type":"span","marks":[],"text":"During CTAD 2024, Johnson, Tai, and R. 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Johnson K, Kaplitt M, Kaminsky S, et al. Safety and preliminary efficacy of AAV gene therapy (LX1001) in patients with APOE4 homozygote Alzheimer disease – interim data from a phase 1/2, open-label, 52-week, multicenter study. Presented at: Clinical Trials on Alzheimer’s Disease conference; October 29-November 1, 2024; Madrid, Spain. ABSTRACT LB04\n3. LEXEO Therapeutics Announces Positive Initial Data from Ongoing Phase 1/2 Clinical Trial of AAV-based Gene Therapy Candidate LX1001 in Patients with Alzheimer’s Disease. News Release. Published March 2, 2022. Accessed October 31, 2024. https://www.lexeotx.com/post/lexeo-therapeutics-announces-positive-initial-data-from-ongoing-phase-1-2-clinical-trial-of-aav-based-gene-therapy-candidate-lx1001-in-patients-with-alzheimers-disease/","_key":"81e3019875810"}],"_type":"block","style":"h6","_key":"122f3d4eb838"}],"factCheckAuthors":null,"url":"lexeo-therapeutics-gene-therapy-lx1001-increases-apoe2-expression-patients-alzheimer-disease","is_visible":true,"ExcludeFromPubMedXML":false,"authors":[{"displayName":"Marco Meglio","url":"marco-meglio"}],"factCheckAuthorMapping":null,"documentGroupMapping":null,"display_summary":true,"_id":"16eee2e2-499e-496b-9122-e7b7da76dbb9","summary":"LX1001's safety profile was characterized as well-tolerated with no amyloid-related imaging abnormalities reported.","title":"Lexeo Therapeutics' Gene Therapy LX1001 Increases APOE2 Expression in Patients With Alzheimer Disease","documentGroup":null,"published":"2024-11-04T20:00:00.408Z","_createdAt":"2024-11-04T22:25:13Z","targeting":{"content_placement":["topic/neurology","conferences","news"],"document_url":["lexeo-therapeutics-gene-therapy-lx1001-increases-apoe2-expression-patients-alzheimer-disease"],"document_group":null,"rootDocumentGroup":[],"issue_url":"","publication_url":""},"relatedArticles":[{"title":"CGTLive®’s Weekly Rewind – November 22, 2024 ","url":{"current":"cgtlive-weekly-rewind-november-22-2024","_type":"slug"},"thumbnail":{"_type":"mainImage","alt":"CGTLive®’s Weekly Rewind","caption":"CGTLive®’s Weekly Rewind","asset":{"_ref":"image-1feeab70bc7d00036bee4f6bc891982276d46bc0-500x500-jpg","_type":"reference"}},"published":"2024-11-22T20:00:00.000Z"},{"title":"David Barrett, JD, on Future Trends for Genomic Medicines","url":{"current":"barrett-future-trends-genomic-medicines","_type":"slug"},"thumbnail":{"_type":"mainImage","alt":"David Barrett, JD, the chief executive officer of ASGCT","caption":"David Barrett, JD","asset":{"_ref":"image-73c396ac9ec1c25274e5148785e53cc12aad6577-755x426-png","_type":"reference"}},"published":"2024-11-22T12:00:05.483Z"},{"title":"Evaluating Allogeneic CAR-T P-BCMA-ALLO1 in R/R Multiple Myeloma","url":{"current":"evaluating-allogeneic-car-t-p-bcma-allo1-multiple-myeloma","_type":"slug"},"thumbnail":{"alt":"Bhagirathbhai R. 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Much of her and her lab’s work has been focused on questions pertaining to the immune system’s contribution to Duchenne muscular dystrophy (DMD), specifically.","_key":"c1fcc5a7c4d9"}],"_type":"block","style":"normal","_key":"6a2a6be4ba4a"},{"_type":"block","style":"normal","_key":"1ad934ec7032","markDefs":[],"children":[{"_type":"span","marks":[],"text":"In the conversation, Spencer delved into the complexities of adeno-associated virus (AAV) as a gene delivery vehicle, highlighting its current role as the primary means to introduce therapeutic nucleic acids into muscle tissue. However, although AAV has shown efficacy, it presents some challenges related to the broad tissue distribution and potential adverse effects of therapy delivery, including liver toxicity and complement activation.","_key":"df50478a4149"}]},{"children":[{"_type":"span","marks":[],"text":"Spencer offered her perspective on the nuanced decision-making required for treating DMD, and the dilemma of balancing early intervention with the difficulty of assessing functional improvement over time. She also emphasized the need for innovation in gene therapy vectors, particularly those with increased specificity for skeletal muscle and stem cells, which could lead to more durable, targeted treatments.","_key":"9f7ac50630020"}],"_type":"block","style":"normal","_key":"94b5d647c4db","markDefs":[]},{"style":"h3","_key":"1db857ed4c5b","markDefs":[],"children":[{"_type":"span","marks":[],"text":"Can you describe your talk in detail and why this was a topic of conversation for you?","_key":"3e043ed2493e0"}],"_type":"block"},{"style":"normal","_key":"ef89b7fff50c","markDefs":[],"children":[{"_key":"5dcd0ed119400","_type":"span","marks":["strong"],"text":"Melissa Spencer, PhD:"},{"text":" My talk focused on adeno-associated virus, or AAV, which is the delivery vehicle used to transport nucleic acids into the body for gene therapies. This can be for gene replacement or gene editing. You can package any nucleic acid into AAV, and muscle is a particularly difficult target because there's so much of it and it's distributed throughout the body. AAV is really the only efficient way to deliver genes to muscle for muscle-based diseases, although there have been some recent reports of intrathecal administration reaching the muscle, which is exciting. That could mean using lower doses, less AAV, and potentially avoiding some of the adverse events like liver toxicity and complement activation.","_key":"5dcd0ed119401","_type":"span","marks":[]}],"_type":"block"},{"_type":"block","style":"normal","_key":"a428504272fc","markDefs":[],"children":[{"_key":"3d550dcac8540","_type":"span","marks":[],"text":"I also discussed the difficulty of assessing function or functional benefits in Duchenne muscular dystrophy, which is a degenerative disease. There's this paradox where you want to treat very early while they still have a lot of muscle, but the disease is already causing muscle loss, yet the patients are still growing and gaining strength up until around age seven. Most trials start from age seven because that's when you can clearly see functional decline, but by then, they don’t have as much muscle to treat. So, it’s a real challenge—how do you assess function after gene therapy and treat early without losing durability too soon?"}]},{"children":[{"text":"Do you feel the community is aware of the limitations of AAV delivery?","_key":"cffb841ae3c10","_type":"span","marks":[]}],"_type":"block","style":"h3","_key":"ba714c324f69","markDefs":[]},{"children":[{"_type":"span","marks":[],"text":"Well, we can only address these problems one at a time, so I’m not sure how aware the broader community is. Certainly, we’re dealing with the best outcome measures we can right now. We also have challenges in standardizing processes across different companies, like AAV production and how to assess pre-existing immunity. Whether the community is fully aware or not, I can't say. Ultimately, we want to develop treatments and get them to patients as soon as possible. We can test things in mice, but the real test is in patients. So, we learn with each trial. I don’t want to put a negative spin on it—we work with the best tools we have and apply them as safely as possible to avoid harm, and then we learn and move forward.","_key":"b386bb49f0a50"}],"_type":"block","style":"normal","_key":"688322fe003b","markDefs":[]},{"markDefs":[],"children":[{"marks":[],"text":"Are there specific gene therapy vehicles you believe hold more promise?","_key":"6eb3b24de3010","_type":"span"}],"_type":"block","style":"h3","_key":"cc69538ac906"},{"style":"normal","_key":"bbcdae483910","markDefs":[],"children":[{"_type":"span","marks":[],"text":"I think we need better vectors that are more myotropic, meaning they have higher tropism for skeletal muscle. There are a few promising ones, like MyoAAV and AAVMyo—they’re very similar in name. These work well at lower doses and are less likely to target the liver. Some are specifically de-targeted from the liver, which is important because the liver is a major sink for AAV. The liver just absorbs AAV, so if you can de-target it, more is available for muscle. These vectors show a lot of promise, and I hope more people start using them. We are considering them for our gene therapy trials for limb girdle muscular dystrophy type 2A, and the collaboration is going well.","_key":"dc2d8b8d0ba30"}],"_type":"block"},{"_key":"5504893eb109","markDefs":[],"children":[{"_key":"34f26fe4ddd60","_type":"span","marks":[],"text":"Where we need improvement is in AAVs that target specific cell types, like muscle stem cells for CRISPR therapies. If you can target and edit muscle stem cells, you could make a permanent change. These stem cells regenerate muscle, so editing them could lead to a long-lasting therapy. Right now, there aren’t any AAV vectors that specifically target microglia, but that would be helpful for certain applications. We're also screening for AAVs that target microglia and other cell types."}],"_type":"block","style":"normal"},{"style":"h3","_key":"eb2a25f7b357","markDefs":[],"children":[{"_type":"span","marks":[],"text":"Have there been issues identifying the right patients for gene therapy trials?","_key":"fdfdee05632f0"}],"_type":"block"},{"style":"normal","_key":"192c2caae988","markDefs":[],"children":[{"_type":"span","marks":[],"text":"We definitely need more standardized assays to determine who has pre-existing immunity, and we need more than just screening for neutralizing and binding antibodies. We should be looking at memory responses because, if someone was exposed to wild-type AAV long ago, they might not have circulating antibodies now, but their immune system could still react. We also need to understand how polymorphisms affect the complement system—some people may have a predisposition for quick activation. There’s also some emerging data suggesting that coincident viral infections, like human herpesvirus, might tip the balance and cause things like thrombotic microangiopathy (TMA). We still need to study this more, but it shows how much we don’t fully understand yet. We need better communication and collaboration across the community, sharing samples and data so we don’t expose patients to unnecessary risks.","_key":"bd129c1855ed0"}],"_type":"block"},{"_key":"01d58b1b6afb","markDefs":[],"children":[{"_type":"span","marks":[],"text":"In terms of research, where should future efforts be focused?","_key":"bf4ce63b43230"}],"_type":"block","style":"h3"},{"children":[{"_type":"span","marks":[],"text":"We need better micro-dystrophins and also larger dystrophins, like the split-inteins Jeff Chamberlain developed. Better AAV vectors are critical, not just for muscle but for stem cells as well. Understanding the immune response is also crucial. It’s challenging to study this in mice in a way that translates well to humans. We’re constantly trying to validate what we see in mice with human data, and as we gather more samples, we’ll get a better understanding. One key finding is that if you give two doses of AAV to mice—not just one—you start to see some of the effects that occur in patients. Mice in labs live in sterile environments and are never exposed to AAV, whereas humans are exposed to varying degrees of AAV throughout their lives, even if it’s not detectable in current assays. If we could develop a mouse model that reflects the human immune response to AAV more accurately, it would be a very valuable tool.","_key":"609f33c262100"}],"_type":"block","style":"normal","_key":"1a687d6db866","markDefs":[]},{"markDefs":[],"children":[{"_key":"f3083b2602690","_type":"span","marks":["em"],"text":"Transcript edited for clarity."}],"_type":"block","style":"normal","_key":"884490e64687"}],"canonicalUrl":"https://www.neurologylive.com/view/aav-vectors-gene-therapy-overcoming-barriers-muscular-dystrophy-treatment","is_visible":true,"contentCategory":{"name":"Articles","_id":"54fdc3d6-b776-4653-9c81-26acd95a8a7e","_updatedAt":"2022-07-01T12:33:15Z","_createdAt":"2020-12-08T16:21:29Z","_rev":"yD3aK4wI9vtjJazfqNRs7r","_type":"contentCategory"},"display_summary":true,"factCheckAuthors":null,"_updatedAt":"2024-10-28T21:05:32Z","url":"overcoming-aav-barriers-limitations-treat-duchenne-muscular-dystrophy","targeting":{"content_placement":["topic/neurology","conferences","news"],"document_url":["overcoming-aav-barriers-limitations-treat-duchenne-muscular-dystrophy"],"document_group":null,"rootDocumentGroup":[],"issue_url":"","publication_url":""},"relatedArticles":[{"title":"CGTLive®’s Weekly Rewind – November 22, 2024 ","url":{"current":"cgtlive-weekly-rewind-november-22-2024","_type":"slug"},"thumbnail":{"asset":{"_ref":"image-1feeab70bc7d00036bee4f6bc891982276d46bc0-500x500-jpg","_type":"reference"},"_type":"mainImage","alt":"CGTLive®’s Weekly Rewind","caption":"CGTLive®’s Weekly Rewind"},"published":"2024-11-22T20:00:00.000Z"},{"title":"David Barrett, JD, on Future Trends for Genomic Medicines","url":{"current":"barrett-future-trends-genomic-medicines","_type":"slug"},"thumbnail":{"_type":"mainImage","alt":"David Barrett, JD, the chief executive officer of ASGCT","caption":"David Barrett, JD","asset":{"_ref":"image-73c396ac9ec1c25274e5148785e53cc12aad6577-755x426-png","_type":"reference"}},"published":"2024-11-22T12:00:05.483Z"},{"title":"Evaluating Allogeneic CAR-T P-BCMA-ALLO1 in R/R Multiple Myeloma","url":{"current":"evaluating-allogeneic-car-t-p-bcma-allo1-multiple-myeloma","_type":"slug"},"thumbnail":{"asset":{"_ref":"image-966ec042ea0449de0fc2f4a9fff69360426657e0-300x300-jpg","_type":"reference"},"_type":"mainImage","alt":"Bhagirathbhai R. 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