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md:justify-between"><div class="max-w-full"><time class="tex-sm " dateTime="2024-10-29T18:00:00.000">October 29, 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/melissa-spencer-phd">Melissa Spencer, PhD</a></span><span class="text-md "><span class=""><br/></span><a class="mr-1 text-author hover:text-primary" href="/authors/cgtlive-staff">CGTLive Staff</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="Overcoming AAV Barriers and Limitations to Treat Duchenne Muscular Dystrophy" 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">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="py-2"><div class="blockText_blockContent__TbCXh"><div class=""><div style="width:33%;float:right;max-width:525px;margin:0 0 1.5rem 1.5rem;clear:both;cursor:pointer" 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="Melissa Spencer, PhD, professor in residence at UCLA Health" title="Melissa Spencer, PhD, professor in residence at UCLA Health" 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="Melissa Spencer, PhD, professor in residence at UCLA Health" title="Melissa Spencer, PhD, professor in residence at UCLA Health" srcSet="/_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fgenetherapy%2F2186783b212093b56503299ea1f33828b31bf0f2-300x300.png%3Ffit%3Dcrop%26auto%3Dformat&w=384&q=75 1x, /_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fgenetherapy%2F2186783b212093b56503299ea1f33828b31bf0f2-300x300.png%3Ffit%3Dcrop%26auto%3Dformat&w=640&q=75 2x" src="/_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fgenetherapy%2F2186783b212093b56503299ea1f33828b31bf0f2-300x300.png%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">Melissa Spencer, PhD</p><p class="pb-2">(Credit: UCLA Health)</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/2186783b212093b56503299ea1f33828b31bf0f2-300x300.png?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">At the recent American Association of Neuromuscular & Electrodiagnostic Medicine annual meeting in Savannah, Georgia, <em>CGTLive<sup class="text-inherit">®</sup></em>'s sister site, <em>NeurologyLive<sup class="text-inherit">®</sup></em>, spoke with Melissa Spencer, PhD, a professor in residence at UCLA Health and an expert in neuromuscular gene therapy who has primarily spent her time trying to understand the pathogenic mechanisms that occur in muscular dystrophies to develop therapeutic interventions. 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.</p><p class="pb-2">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.</p><p class="pb-2">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.</p><h3 class="text-xl pb-4 pt-2">Can you describe your talk in detail and why this was a topic of conversation for you?</h3><p class="pb-2"><strong>Melissa Spencer, PhD:</strong> 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.</p><p class="pb-2">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?</p><h3 class="text-xl pb-4 pt-2">Do you feel the community is aware of the limitations of AAV delivery?</h3><p class="pb-2">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.</p><h3 class="text-xl pb-4 pt-2">Are there specific gene therapy vehicles you believe hold more promise?</h3><p class="pb-2">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.</p><p class="pb-2">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.</p><h3 class="text-xl pb-4 pt-2">Have there been issues identifying the right patients for gene therapy trials?</h3><p class="pb-2">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.</p><h3 class="text-xl pb-4 pt-2">In terms of research, where should future efforts be focused?</h3><p class="pb-2">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.</p><p class="pb-2"><em>Transcript edited for clarity.</em></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 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Landscape Report. </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/genethon-gene-therapy-gnt0004-stabilizes-improves-motor-functions-patients-duchenne-muscular-dystrophy?utm_source=www.cgtlive.com&utm_medium=relatedContent"><div class=""><span 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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%"/><noscript><img alt="Genethon’s Gene Therapy GNT0004 Stabilizes or Improves Motor Functions in Patients With Duchenne Muscular Dystrophy" title="Genethon’s Gene Therapy GNT0004 Stabilizes or Improves Motor Functions in Patients With Duchenne Muscular Dystrophy" sizes="100vw" srcSet="/_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fgenetherapy%2Fe292d9fc799af049ac1bd0109419772a7b6fbdd4-1000x563.jpg%3Ffit%3Dcrop%26auto%3Dformat&w=640&q=75 640w, /_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fgenetherapy%2Fe292d9fc799af049ac1bd0109419772a7b6fbdd4-1000x563.jpg%3Ffit%3Dcrop%26auto%3Dformat&w=750&q=75 750w, 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href="/view/genethon-gene-therapy-gnt0004-stabilizes-improves-motor-functions-patients-duchenne-muscular-dystrophy?utm_source=www.cgtlive.com&utm_medium=relatedContent">Genethon’s Gene Therapy GNT0004 Stabilizes or Improves Motor Functions in Patients With Duchenne Muscular Dystrophy</a></p><div class=" pl-4"><a class="text-sm text-sky-800" href="/authors/noah-stansfield">Noah Stansfield</a></div><a href="/view/genethon-gene-therapy-gnt0004-stabilizes-improves-motor-functions-patients-duchenne-muscular-dystrophy?utm_source=www.cgtlive.com&utm_medium=relatedContent"><span class="text-sm text-gray-500 pl-4">November 25th 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">All 3 patients who received the high dose showed stabilization of motor function, as assessed by the 34-point North Star Ambulatory Assessment.</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="flex md:hidden justify-center items-center"></div><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/exploring-car-t-autoimmune-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:100%"></span><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="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="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%2F6808278d4a529c2b98af3c3297a83a2440fa06a2-300x300.png%3Ffit%3Dcrop%26auto%3Dformat&w=640&q=75 640w, /_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fgenetherapy%2F6808278d4a529c2b98af3c3297a83a2440fa06a2-300x300.png%3Ffit%3Dcrop%26auto%3Dformat&w=750&q=75 750w, /_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fgenetherapy%2F6808278d4a529c2b98af3c3297a83a2440fa06a2-300x300.png%3Ffit%3Dcrop%26auto%3Dformat&w=828&q=75 828w, /_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fgenetherapy%2F6808278d4a529c2b98af3c3297a83a2440fa06a2-300x300.png%3Ffit%3Dcrop%26auto%3Dformat&w=1080&q=75 1080w, /_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fgenetherapy%2F6808278d4a529c2b98af3c3297a83a2440fa06a2-300x300.png%3Ffit%3Dcrop%26auto%3Dformat&w=1200&q=75 1200w, /_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fgenetherapy%2F6808278d4a529c2b98af3c3297a83a2440fa06a2-300x300.png%3Ffit%3Dcrop%26auto%3Dformat&w=1920&q=75 1920w, /_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fgenetherapy%2F6808278d4a529c2b98af3c3297a83a2440fa06a2-300x300.png%3Ffit%3Dcrop%26auto%3Dformat&w=2048&q=75 2048w, /_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fgenetherapy%2F6808278d4a529c2b98af3c3297a83a2440fa06a2-300x300.png%3Ffit%3Dcrop%26auto%3Dformat&w=3840&q=75 3840w" src="/_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fgenetherapy%2F6808278d4a529c2b98af3c3297a83a2440fa06a2-300x300.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/exploring-car-t-autoimmune-disease?utm_source=www.cgtlive.com&utm_medium=relatedContent">Exploring CAR-T for Autoimmune Disease</a></p><div class=" pl-4"><a class="text-sm text-sky-800" href="/authors/georg-schett-md">Georg Schett, MD</a><span class="mr-1 ml-[1px]">;</span><a class="text-sm text-sky-800" href="/authors/noah-stansfield">Noah Stansfield</a><span class="mr-1 ml-[1px]">;</span><a class="text-sm text-sky-800" href="/authors/victoria-johnson">Victoria Johnson</a></div><a href="/view/exploring-car-t-autoimmune-disease?utm_source=www.cgtlive.com&utm_medium=relatedContent"><span class="text-sm text-gray-500 pl-4">November 25th 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">Georg Schett, MD, vice president research and chair of internal medicine at the University of Erlangen – Nuremberg, discussed findings from 2 early studies of CD19 CAR T-cell therapy.</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/alzheimer-disease-awareness-month-2024-looking-back-year-progress-cell-gene-therapy?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:56.166666666666664%"></span><img alt="Alzheimer Disease Awareness Month 2024: Looking Back at a Year of Progress in Cell and Gene Therapy" title="Alzheimer Disease Awareness Month 2024: Looking Back at a Year of Progress in Cell and Gene Therapy" 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="Alzheimer Disease Awareness Month 2024: Looking Back at a Year of Progress in Cell and Gene Therapy" title="Alzheimer Disease Awareness Month 2024: Looking Back at a Year of Progress in Cell and Gene Therapy" sizes="100vw" srcSet="/_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fgenetherapy%2Fbcf8b7384dbe616cc22cb250d8946d9b6758e997-1200x674.jpg%3Ffit%3Dcrop%26auto%3Dformat&w=640&q=75 640w, /_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fgenetherapy%2Fbcf8b7384dbe616cc22cb250d8946d9b6758e997-1200x674.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%2Fbcf8b7384dbe616cc22cb250d8946d9b6758e997-1200x674.jpg%3Ffit%3Dcrop%26auto%3Dformat&w=3840&q=75 3840w" src="/_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fgenetherapy%2Fbcf8b7384dbe616cc22cb250d8946d9b6758e997-1200x674.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/alzheimer-disease-awareness-month-2024-looking-back-year-progress-cell-gene-therapy?utm_source=www.cgtlive.com&utm_medium=relatedContent">Alzheimer Disease Awareness Month 2024: Looking Back at a Year of Progress in Cell and Gene Therapy</a></p><div class=" pl-4"><a class="text-sm text-sky-800" href="/authors/cgtlive-staff">CGTLive Staff</a></div><a href="/view/alzheimer-disease-awareness-month-2024-looking-back-year-progress-cell-gene-therapy?utm_source=www.cgtlive.com&utm_medium=relatedContent"><span class="text-sm text-gray-500 pl-4">November 24th 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">In observance of Alzheimer Disease Awareness Month, held annually in November, we took a look back at the past year's news and expert insights in cell and gene therapy for 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/cgtlive-weekly-rewind-november-22-2024?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="CGTLive®’s Weekly Rewind" title="CGTLive®’s Weekly Rewind" 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="CGTLive®’s Weekly Rewind" title="CGTLive®’s Weekly Rewind" sizes="100vw" srcSet="/_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fgenetherapy%2F1feeab70bc7d00036bee4f6bc891982276d46bc0-500x500.jpg%3Ffit%3Dcrop%26auto%3Dformat&w=640&q=75 640w, 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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/cgtlive-weekly-rewind-november-22-2024?utm_source=www.cgtlive.com&utm_medium=relatedContent">CGTLive®’s Weekly Rewind – November 22, 2024 </a></p><div class=" pl-4"><a class="text-sm text-sky-800" href="/authors/cgtlive-staff">CGTLive Staff</a></div><a href="/view/cgtlive-weekly-rewind-november-22-2024?utm_source=www.cgtlive.com&utm_medium=relatedContent"><span class="text-sm text-gray-500 pl-4">November 22nd 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">Review top news and interview highlights from the week ending November 22, 2024. </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/evaluating-allogeneic-car-t-p-bcma-allo1-multiple-myeloma?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="Bhagirathbhai R. Dholaria, MD, an associate professor of medicine in malignant hematology & stem cell transplantation at Vanderbilt University Medical Center" title="Bhagirathbhai R. Dholaria, MD, an associate professor of medicine in malignant hematology & stem cell transplantation at Vanderbilt University Medical Center" 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="Bhagirathbhai R. Dholaria, MD, an associate professor of medicine in malignant hematology & stem cell transplantation at Vanderbilt University Medical Center" title="Bhagirathbhai R. Dholaria, MD, an associate professor of medicine in malignant hematology & stem cell transplantation at Vanderbilt University Medical Center" sizes="100vw" srcSet="/_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fgenetherapy%2F966ec042ea0449de0fc2f4a9fff69360426657e0-300x300.jpg%3Ffit%3Dcrop%26auto%3Dformat&w=640&q=75 640w, /_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fgenetherapy%2F966ec042ea0449de0fc2f4a9fff69360426657e0-300x300.jpg%3Ffit%3Dcrop%26auto%3Dformat&w=750&q=75 750w, /_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fgenetherapy%2F966ec042ea0449de0fc2f4a9fff69360426657e0-300x300.jpg%3Ffit%3Dcrop%26auto%3Dformat&w=828&q=75 828w, /_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fgenetherapy%2F966ec042ea0449de0fc2f4a9fff69360426657e0-300x300.jpg%3Ffit%3Dcrop%26auto%3Dformat&w=1080&q=75 1080w, /_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fgenetherapy%2F966ec042ea0449de0fc2f4a9fff69360426657e0-300x300.jpg%3Ffit%3Dcrop%26auto%3Dformat&w=1200&q=75 1200w, /_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fgenetherapy%2F966ec042ea0449de0fc2f4a9fff69360426657e0-300x300.jpg%3Ffit%3Dcrop%26auto%3Dformat&w=1920&q=75 1920w, /_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fgenetherapy%2F966ec042ea0449de0fc2f4a9fff69360426657e0-300x300.jpg%3Ffit%3Dcrop%26auto%3Dformat&w=2048&q=75 2048w, /_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fgenetherapy%2F966ec042ea0449de0fc2f4a9fff69360426657e0-300x300.jpg%3Ffit%3Dcrop%26auto%3Dformat&w=3840&q=75 3840w" src="/_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fgenetherapy%2F966ec042ea0449de0fc2f4a9fff69360426657e0-300x300.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/evaluating-allogeneic-car-t-p-bcma-allo1-multiple-myeloma?utm_source=www.cgtlive.com&utm_medium=relatedContent">Evaluating Allogeneic CAR-T P-BCMA-ALLO1 in R/R Multiple Myeloma</a></p><div class=" pl-4"><a class="text-sm text-sky-800" href="/authors/bhagirathbhai-dholaria-mbbs">Bhagirathbhai Dholaria, MBBS</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/evaluating-allogeneic-car-t-p-bcma-allo1-multiple-myeloma?utm_source=www.cgtlive.com&utm_medium=relatedContent"><span class="text-sm text-gray-500 pl-4">November 21st 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">Bhagirathbhai R. 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Landscape Report. </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/genethon-gene-therapy-gnt0004-stabilizes-improves-motor-functions-patients-duchenne-muscular-dystrophy?utm_source=www.cgtlive.com&utm_medium=relatedContent"><div class=""><span 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href="/view/genethon-gene-therapy-gnt0004-stabilizes-improves-motor-functions-patients-duchenne-muscular-dystrophy?utm_source=www.cgtlive.com&utm_medium=relatedContent">Genethon’s Gene Therapy GNT0004 Stabilizes or Improves Motor Functions in Patients With Duchenne Muscular Dystrophy</a></p><div class=" pl-4"><a class="text-sm text-sky-800" href="/authors/noah-stansfield">Noah Stansfield</a></div><a href="/view/genethon-gene-therapy-gnt0004-stabilizes-improves-motor-functions-patients-duchenne-muscular-dystrophy?utm_source=www.cgtlive.com&utm_medium=relatedContent"><span class="text-sm text-gray-500 pl-4">November 25th 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">All 3 patients who received the high dose showed stabilization of motor function, as assessed by the 34-point North Star Ambulatory Assessment.</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="flex md:hidden justify-center items-center"></div><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/exploring-car-t-autoimmune-disease?utm_source=www.cgtlive.com&utm_medium=relatedContent"><div class=""><span 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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%"/><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%2F6808278d4a529c2b98af3c3297a83a2440fa06a2-300x300.png%3Ffit%3Dcrop%26auto%3Dformat&w=640&q=75 640w, /_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fgenetherapy%2F6808278d4a529c2b98af3c3297a83a2440fa06a2-300x300.png%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%2F6808278d4a529c2b98af3c3297a83a2440fa06a2-300x300.png%3Ffit%3Dcrop%26auto%3Dformat&w=3840&q=75 3840w" src="/_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fgenetherapy%2F6808278d4a529c2b98af3c3297a83a2440fa06a2-300x300.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/exploring-car-t-autoimmune-disease?utm_source=www.cgtlive.com&utm_medium=relatedContent">Exploring CAR-T for Autoimmune Disease</a></p><div class=" pl-4"><a class="text-sm text-sky-800" href="/authors/georg-schett-md">Georg Schett, MD</a><span class="mr-1 ml-[1px]">;</span><a class="text-sm text-sky-800" href="/authors/noah-stansfield">Noah Stansfield</a><span class="mr-1 ml-[1px]">;</span><a class="text-sm text-sky-800" href="/authors/victoria-johnson">Victoria Johnson</a></div><a href="/view/exploring-car-t-autoimmune-disease?utm_source=www.cgtlive.com&utm_medium=relatedContent"><span class="text-sm text-gray-500 pl-4">November 25th 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">Georg Schett, MD, vice president research and chair of internal medicine at the University of Erlangen – Nuremberg, discussed findings from 2 early studies of CD19 CAR T-cell therapy.</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/alzheimer-disease-awareness-month-2024-looking-back-year-progress-cell-gene-therapy?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:56.166666666666664%"></span><img alt="Alzheimer Disease Awareness Month 2024: Looking Back at a Year of Progress in Cell and Gene Therapy" title="Alzheimer Disease Awareness Month 2024: Looking Back at a Year of Progress in Cell and Gene Therapy" 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="Alzheimer Disease Awareness Month 2024: Looking Back at a Year of Progress in Cell and Gene Therapy" title="Alzheimer Disease Awareness Month 2024: Looking Back at a Year of Progress in Cell and Gene Therapy" sizes="100vw" srcSet="/_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fgenetherapy%2Fbcf8b7384dbe616cc22cb250d8946d9b6758e997-1200x674.jpg%3Ffit%3Dcrop%26auto%3Dformat&w=640&q=75 640w, /_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fgenetherapy%2Fbcf8b7384dbe616cc22cb250d8946d9b6758e997-1200x674.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%2Fbcf8b7384dbe616cc22cb250d8946d9b6758e997-1200x674.jpg%3Ffit%3Dcrop%26auto%3Dformat&w=3840&q=75 3840w" src="/_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fgenetherapy%2Fbcf8b7384dbe616cc22cb250d8946d9b6758e997-1200x674.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/alzheimer-disease-awareness-month-2024-looking-back-year-progress-cell-gene-therapy?utm_source=www.cgtlive.com&utm_medium=relatedContent">Alzheimer Disease Awareness Month 2024: Looking Back at a Year of Progress in Cell and Gene Therapy</a></p><div class=" pl-4"><a class="text-sm text-sky-800" href="/authors/cgtlive-staff">CGTLive Staff</a></div><a href="/view/alzheimer-disease-awareness-month-2024-looking-back-year-progress-cell-gene-therapy?utm_source=www.cgtlive.com&utm_medium=relatedContent"><span class="text-sm text-gray-500 pl-4">November 24th 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">In observance of Alzheimer Disease Awareness Month, held annually in November, we took a look back at the past year's news and expert insights in cell and gene therapy for 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/cgtlive-weekly-rewind-november-22-2024?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="CGTLive®’s Weekly Rewind" title="CGTLive®’s Weekly Rewind" 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="CGTLive®’s Weekly Rewind" title="CGTLive®’s Weekly Rewind" sizes="100vw" srcSet="/_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fgenetherapy%2F1feeab70bc7d00036bee4f6bc891982276d46bc0-500x500.jpg%3Ffit%3Dcrop%26auto%3Dformat&w=640&q=75 640w, 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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/cgtlive-weekly-rewind-november-22-2024?utm_source=www.cgtlive.com&utm_medium=relatedContent">CGTLive®’s Weekly Rewind – November 22, 2024 </a></p><div class=" pl-4"><a class="text-sm text-sky-800" href="/authors/cgtlive-staff">CGTLive Staff</a></div><a href="/view/cgtlive-weekly-rewind-november-22-2024?utm_source=www.cgtlive.com&utm_medium=relatedContent"><span class="text-sm text-gray-500 pl-4">November 22nd 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">Review top news and interview highlights from the week ending November 22, 2024. </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/evaluating-allogeneic-car-t-p-bcma-allo1-multiple-myeloma?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="Bhagirathbhai R. Dholaria, MD, an associate professor of medicine in malignant hematology & stem cell transplantation at Vanderbilt University Medical Center" title="Bhagirathbhai R. Dholaria, MD, an associate professor of medicine in malignant hematology & stem cell transplantation at Vanderbilt University Medical Center" 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="Bhagirathbhai R. Dholaria, MD, an associate professor of medicine in malignant hematology & stem cell transplantation at Vanderbilt University Medical Center" title="Bhagirathbhai R. Dholaria, MD, an associate professor of medicine in malignant hematology & stem cell transplantation at Vanderbilt University Medical Center" sizes="100vw" srcSet="/_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fgenetherapy%2F966ec042ea0449de0fc2f4a9fff69360426657e0-300x300.jpg%3Ffit%3Dcrop%26auto%3Dformat&w=640&q=75 640w, /_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fgenetherapy%2F966ec042ea0449de0fc2f4a9fff69360426657e0-300x300.jpg%3Ffit%3Dcrop%26auto%3Dformat&w=750&q=75 750w, /_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fgenetherapy%2F966ec042ea0449de0fc2f4a9fff69360426657e0-300x300.jpg%3Ffit%3Dcrop%26auto%3Dformat&w=828&q=75 828w, /_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fgenetherapy%2F966ec042ea0449de0fc2f4a9fff69360426657e0-300x300.jpg%3Ffit%3Dcrop%26auto%3Dformat&w=1080&q=75 1080w, /_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fgenetherapy%2F966ec042ea0449de0fc2f4a9fff69360426657e0-300x300.jpg%3Ffit%3Dcrop%26auto%3Dformat&w=1200&q=75 1200w, /_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fgenetherapy%2F966ec042ea0449de0fc2f4a9fff69360426657e0-300x300.jpg%3Ffit%3Dcrop%26auto%3Dformat&w=1920&q=75 1920w, /_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fgenetherapy%2F966ec042ea0449de0fc2f4a9fff69360426657e0-300x300.jpg%3Ffit%3Dcrop%26auto%3Dformat&w=2048&q=75 2048w, /_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fgenetherapy%2F966ec042ea0449de0fc2f4a9fff69360426657e0-300x300.jpg%3Ffit%3Dcrop%26auto%3Dformat&w=3840&q=75 3840w" src="/_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fgenetherapy%2F966ec042ea0449de0fc2f4a9fff69360426657e0-300x300.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/evaluating-allogeneic-car-t-p-bcma-allo1-multiple-myeloma?utm_source=www.cgtlive.com&utm_medium=relatedContent">Evaluating Allogeneic CAR-T P-BCMA-ALLO1 in R/R Multiple Myeloma</a></p><div class=" pl-4"><a class="text-sm text-sky-800" href="/authors/bhagirathbhai-dholaria-mbbs">Bhagirathbhai Dholaria, MBBS</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/evaluating-allogeneic-car-t-p-bcma-allo1-multiple-myeloma?utm_source=www.cgtlive.com&utm_medium=relatedContent"><span class="text-sm text-gray-500 pl-4">November 21st 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">Bhagirathbhai R. Dholaria, MD, an associate professor of medicine in malignant hematology & stem cell transplantation at Vanderbilt University Medical Center, discussed interim data from the phase 1/1b clinical trial evaluating Poseida's CAR-T.</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":"Overcoming AAV Barriers and Limitations to Treat Duchenne Muscular Dystrophy","datePublished":"2024-10-29T18:00:00.000Z","dateModified":"2024-10-28T21:05:32Z","inLanguage":"en-US","image":"https://cdn.sanity.io/images/0vv8moc6/genetherapy/2186783b212093b56503299ea1f33828b31bf0f2-300x300.png?fit=crop&auto=format","mainEntityOfPage":{"@type":"WebPage","@id":"https://www.cgtlive.com/view/overcoming-aav-barriers-limitations-treat-duchenne-muscular-dystrophy"},"publisher":{"@type":"Organization","name":"CGTlive™","logo":{"@type":"ImageObject","url":"https://www.cgtlive.com/CGT_Color_NoTagline.png"}},"articleBody":"\n\nAt the recent American Association of Neuromuscular & Electrodiagnostic Medicine annual meeting in Savannah, Georgia, CGTLive®'s sister site, NeurologyLive®, spoke with Melissa Spencer, PhD, a professor in residence at UCLA Health and an expert in neuromuscular gene therapy who has primarily spent her time trying to understand the pathogenic mechanisms that occur in muscular dystrophies to develop therapeutic interventions. 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.\n\nIn 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.\n\nSpencer 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.\n\nCan you describe your talk in detail and why this was a topic of conversation for you?\n\nMelissa Spencer, PhD: 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.\n\nI 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?\n\nDo you feel the community is aware of the limitations of AAV delivery?\n\nWell, 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.\n\nAre there specific gene therapy vehicles you believe hold more promise?\n\nI 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.\n\nWhere 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.\n\nHave there been issues identifying the right patients for gene therapy trials?\n\nWe 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.\n\nIn terms of research, where should future efforts be focused?\n\nWe 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.\n\nTranscript edited for clarity.","description":"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.","author":[{"@type":"Person","name":"Melissa Spencer, PhD"},{"@type":"Person","name":"CGTLive Staff"}]}</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 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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","upload_doc":null,"uploadAudio":null,"medias":null,"style":"normal","_key":"6a2a6be4ba4a"},{"upload_doc":null,"uploadAudio":null,"medias":null,"style":"normal","_key":"1ad934ec7032","markDefs":[],"children":[{"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","_type":"span"}],"_type":"block"},{"children":[{"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":"span","marks":[]}],"_type":"block","style":"normal","_key":"94b5d647c4db","markDefs":[],"upload_doc":null,"uploadAudio":null,"medias":null},{"markDefs":[],"upload_doc":null,"uploadAudio":null,"medias":null,"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":"h3","_key":"1db857ed4c5b"},{"_type":"block","upload_doc":null,"uploadAudio":null,"medias":null,"style":"normal","_key":"ef89b7fff50c","markDefs":[],"children":[{"_type":"span","marks":["strong"],"text":"Melissa Spencer, PhD:","_key":"5dcd0ed119400"},{"_key":"5dcd0ed119401","_type":"span","marks":[],"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."}]},{"medias":null,"markDefs":[],"children":[{"_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?","_key":"3d550dcac8540"}],"_type":"block","style":"normal","_key":"a428504272fc","upload_doc":null,"uploadAudio":null},{"_type":"block","style":"h3","_key":"ba714c324f69","upload_doc":null,"uploadAudio":null,"medias":null,"markDefs":[],"children":[{"marks":[],"text":"Do you feel the community is aware of the limitations of AAV delivery?","_key":"cffb841ae3c10","_type":"span"}]},{"medias":null,"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","upload_doc":null,"uploadAudio":null},{"style":"h3","_key":"cc69538ac906","markDefs":[],"upload_doc":null,"uploadAudio":null,"medias":null,"children":[{"_type":"span","marks":[],"text":"Are there specific gene therapy vehicles you believe hold more promise?","_key":"6eb3b24de3010"}],"_type":"block"},{"_type":"block","style":"normal","_key":"bbcdae483910","markDefs":[],"children":[{"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":"span","marks":[]}],"upload_doc":null,"uploadAudio":null,"medias":null},{"medias":null,"_key":"5504893eb109","markDefs":[],"children":[{"_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.","_key":"34f26fe4ddd60"}],"_type":"block","style":"normal","upload_doc":null,"uploadAudio":null},{"_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":"h3","upload_doc":null,"uploadAudio":null,"medias":null},{"_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","upload_doc":null,"uploadAudio":null,"medias":null,"style":"normal"},{"children":[{"_type":"span","marks":[],"text":"In terms of research, where should future efforts be focused?","_key":"bf4ce63b43230"}],"_type":"block","style":"h3","upload_doc":null,"uploadAudio":null,"medias":null,"_key":"01d58b1b6afb","markDefs":[]},{"medias":null,"_key":"1a687d6db866","markDefs":[],"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","upload_doc":null,"uploadAudio":null},{"_key":"884490e64687","markDefs":[],"upload_doc":null,"uploadAudio":null,"medias":null,"children":[{"text":"Transcript edited for clarity.","_key":"f3083b2602690","_type":"span","marks":["em"]}],"_type":"block","style":"normal"}],"published":"2024-10-29T18:00:00.000Z","title":"Overcoming AAV Barriers and Limitations to Treat Duchenne Muscular Dystrophy","_createdAt":"2024-10-28T20:50:39Z","display_summary":true,"documentGroup":null,"filter":null,"thumbnail":{"_type":"mainImage","asset":{"_ref":"image-2186783b212093b56503299ea1f33828b31bf0f2-300x300-png","_type":"reference"}},"_type":"article","articleType":"Commentary","ExcludeFromPubMedXML":false,"contentGroup":null,"issueSection":null,"_updatedAt":"2024-10-28T21:05:32Z","authorMapping":[{"_createdAt":"2024-10-28T21:00:13Z","_rev":"aJwyNk574RjbDSo1lbafc7","biography":[{"_key":"4a9f6f0a93c5","markDefs":[],"children":[{"_type":"span","marks":[],"text":"Professor in residence at UCLA Health","_key":"c51862c6f2b20"}],"_type":"block","style":"normal"}],"_updatedAt":"2024-10-28T21:00:13Z","url":{"current":"melissa-spencer-phd","_type":"slug"},"firstName":"Melissa ","lastName":"Spencer, PhD","displayName":"Melissa Spencer, PhD","_type":"author","_id":"4ffda529-0026-40cb-b919-d1b1c87a3337"},{"displayName":"CGTLive Staff","_createdAt":"2022-04-14T19:52:01Z","_rev":"hbv0rKeAdHbRrx7o9Ci0eY","_type":"author","_id":"f4c3492e-f219-4e4b-a311-6a75d85e2e40","_updatedAt":"2022-04-14T19:52:01Z","url":{"current":"cgtlive-staff","_type":"slug"}}],"_rev":"aJwyNk574RjbDSo1lbbzPc","disableAds":false,"disableRelatedContent":false,"relatedArticles":[{"documentGroupMapping":null,"display_summary":true,"seoTag":["gene therapy"],"body":[{"_type":"figure","alt":"David Barrett, JD, the chief executive officer of ASGCT","imgcaption":[{"_type":"block","style":"normal","_key":"0b37a68f8e95","markDefs":[],"children":[{"_type":"span","marks":[],"text":"David Barrett, JD","_key":"e3ef2bf3b5240"}]}],"_key":"3caedbae470c","alignment":"left","asset":{"_ref":"image-76e90a956bc67a67f1f41881008e7e2c47774079-300x300-jpg","_type":"reference"},"disableTextWrap":false,"disableLightBox":true},{"_type":"block","style":"normal","_key":"34f33c1e6874","markDefs":[],"children":[{"_type":"span","marks":[],"text":"Each quarter, the American Society of Gene \u0026 Cell Therapy (ASGCT) puts out its “Gene, Cell, \u0026 RNA Therapy Landscape Report”, which is intended to capture statistics related to the rapid evolution of this field of medicine. Following the publication of the most recent report, for quarter 3 (Q3) of 2024, ","_key":"6c82da9de699"},{"_type":"span","marks":["em"],"text":"CGTLive®","_key":"1cfcdf420925"},{"_type":"span","marks":[],"text":" reached out to David Barrett, JD, the chief executive officer of ASGCT, to learn more about its highlights.","_key":"23c21bdc7696"}]},{"markDefs":[{"_type":"link","href":"https://www.cgtlive.com/view/fda-approves-adaptimmune-tcr-t-cell-therapy-afami-cel-synovial-sarcoma","_key":"81084131991e"}],"children":[{"_type":"span","marks":[],"text":"Barret gave some background about the quarterly report and then went over the latest report's key findings. Notably, he touched on the ","_key":"6575c7e9699c0"},{"_type":"span","marks":["81084131991e"],"text":"approval of Adaptimmune Therapeutics’ cell therapy afamitresgene autoleuce","_key":"742034d82d60"},{"_type":"span","marks":[],"text":"l (afami-cel, marketed as Tecelra) for the treatment of synovial sarcoma and data pointing to the continued steady growth of the field as a whole.","_key":"91ac74e4aabe"}],"_type":"block","style":"normal","_key":"b040ef347cf4"},{"markDefs":[],"children":[{"_type":"span","marks":["strong","em"],"text":"CGTLive:","_key":"8877a82e07af0"},{"_type":"span","marks":["strong"],"text":" Can you give some background context about the Landscape Report and what it is?","_key":"24a5415d083b"}],"_type":"block","style":"h3","_key":"95131aaf3f41"},{"style":"normal","_key":"2078bc759d0e","markDefs":[],"children":[{"_type":"span","marks":["strong"],"text":"David Barrett, JD: ","_key":"6b72d7754b96"},{"marks":[],"text":"The landscape report is a report that we produce every quarter where we relay what has happened over the course of the past quarter in the field of cell and gene therapy. Some of the things that we track are the number of approvals—both in the United States and outside of the United States—we track the clinical pipeline, so we're looking at the clinical trials in cell and gene therapy. We're also looking at not just what we might think of as more traditional gene therapies—gene edition, gene replacement, gene editing, and genetically modified cell therapies—but we're also looking at RNA mediated therapies as well as vaccines, and we are also looking at nongenetically modified cell therapies and reporting on that entire pipeline. ","_key":"0d0d7577b91b","_type":"span"}],"_type":"block"},{"markDefs":[],"children":[{"_type":"span","marks":[],"text":"In addition to the total number of trials and total number of clinical programs that that we're aware of through the research, we are also reporting on what some of those targets are. So we're looking at the the most common diseases that are being targeted by these therapies; we're looking at as much information as we have on what the therapeutic type or therapeutic modality is. So we report on gene therapies that are delivered via viral vector versus gene therapies that are in a CAR-T versus other therapeutic modalities like RNA or nongenetically modified cell therapies.","_key":"ba66929afe96"}],"_type":"block","style":"normal","_key":"c8b882d28985"},{"markDefs":[],"children":[{"_type":"span","marks":["strong"],"text":"What stand out as some of the major highlights or key items from the Q3 Landscape Report?","_key":"141ee9f5a8af"}],"_type":"block","style":"h3","_key":"696b9a674282"},{"_type":"block","style":"normal","_key":"436448baf090","markDefs":[],"children":[{"_type":"span","marks":[],"text":"One of the big key takeaways was the approval of to Tecelra, which was approved during the quarter. One of the other things that we noted was the consistent trend of an increasing proportion of trials that are targeting nononcology indications. What I what I mean to say there is the the split of newly-initiated trials that are focusing on cancer versus focusing on noncancer targets is shifting slightly towards noncancer targets, which we think shows a maturation in the space and it shows one of the things that we that we thought would happen as the field evolves—which had been focused on cancer in many spaces—to an expansion of indications that are targeted through gene therapy. Now they are still primarily all rare disease, but that shift, I think, is showing some of the exciting changes that are in the space. What we are seeing is an increase in the proportion of disease targets that are nononcology-related. We are not seeing a reduction in oncology-related drugs and trials, but an increase in nononcology-related trials and new programs. ","_key":"cd54daa3bf0f"}]},{"style":"normal","_key":"43f805a9c1c5","markDefs":[],"children":[{"_key":"f4af8bcfda72","_type":"span","marks":[],"text":"The other thing that we've seen that we think is a really interesting and positive trend in cell and gene therapy is that while the volume remains somewhat flat, the startup financing has rebounded, so we're seeing that number increase. When we look over the course of the last several quarters, we see the total number of new programs that are being spun out and funded has stayed relatively stable. So in Q3 of 2023 there were 17 programs. In Q3 of 2024 there were 19 programs. While we saw a dip in Q4 of 2023, subsequent dips in Q1 of 2024 and Q2 of 2024, overall, the number of new programs that are being funded is finding some stability, and we are starting to see an increase in the amount of money that is being invested into launching those programs."}],"_type":"block"},{"_type":"block","style":"h3","_key":"652b3cbc9ffe","markDefs":[],"children":[{"_type":"span","marks":["strong"],"text":"What can we learn about development trends and patient/therapy needs from the report?","_key":"d0781c23b6da"}]},{"_key":"16fbc56f8dcb","markDefs":[],"children":[{"_type":"span","marks":[],"text":"One of the things that we are seeing is a continuation of trials that are moving through phase 1 into phase 2 and phase 3. So while the total number of new programs is increasing slightly, we're continuing to see that increase in clinical trials. So these trials are moving forward, mostly successfully. In Q3 of 2024, there were 35 phase 3 trials in cell and gene therapy, and 289 phase 2 trials, as well as 318 phase 1 trials. What we are seeing now is a continuation of the robust pipeline that we've been witnessing for the past several years. Despite some fluctuations in overall market performance, what remains is the steady drumbeat of cell therapies and gene therapies moving through the clinical pipeline. I think we will continue to see that trend for quite some time into the future. So for patients and caregivers, we are seeing the continuation of approvals in gene therapy, and we will likely see the continuation, hopefully, of successful trials and more approvals in the space.","_key":"31a89b3f29da0"}],"_type":"block","style":"normal"},{"style":"normal","_key":"1e27ae9fbd54","markDefs":[],"children":[{"_type":"span","marks":["em","strong"],"text":"This transcript has been edited for clarity.","_key":"090e000f56cf0"}],"_type":"block"},{"children":[{"_type":"span","marks":[],"text":"REFERENCE\n1. Gene, Cell, \u0026 RNA Therapy Landscape Report, Q3 2024 Quarterly Data Report. Presentation. ASGCT and Citeline. Accessed November 19, 2024. https://www.asgct.org/global/documents/asgct-citeline-q3-2024-report.aspx","_key":"a79e47f8b0370"}],"_type":"block","style":"h6","_key":"1c5252ecfa89","markDefs":[]},{"children":[{"_type":"span","marks":[],"text":"","_key":"08d1e30b91e50"}],"_type":"block","style":"normal","_key":"68c7b9db0d9c","markDefs":[]},{"_type":"block","style":"normal","_key":"08507dbc0e28","markDefs":[],"children":[{"_type":"span","marks":[],"text":"","_key":"271de6c127c10"}]}],"_type":"article","published":"2024-11-26T12:00:45.838Z","thumbnail":{"asset":{"_ref":"image-76e90a956bc67a67f1f41881008e7e2c47774079-300x300-jpg","_type":"reference"},"_type":"mainImage","alt":"David Barrett, JD, the chief executive officer of ASGCT","caption":"David Barrett, JD"},"authors":[{"displayName":"David Barrett, JD","url":"david-barrett-jd"},{"displayName":"Noah Stansfield","url":"noah-stansfield"}],"documentGroup":null,"authorMapping":[{"_createdAt":"2024-11-20T10:53:56Z","authorType":"expert","_rev":"7xPE9R64LNdWAAsgIBQgkd","_type":"author","_id":"10169e1e-d52b-4808-813b-60c253107faa","biography":[{"_type":"block","style":"normal","_key":"ce61b3046454","markDefs":[],"children":[{"_type":"span","marks":[],"text":"David Barrett, JD, the chief executive officer of ASGCT","_key":"a3d9af23709f0"}]}],"_updatedAt":"2024-11-20T10:53:56Z","url":{"current":"david-barrett-jd","_type":"slug"},"displayName":"David Barrett, JD"},{"url":{"current":"noah-stansfield","_type":"slug"},"displayName":"Noah Stansfield","_id":"f9e9001f-7a9c-46a2-9e9c-318ee31c1923","_updatedAt":"2022-06-14T20:44:15Z","_rev":"yD3aK4wI9vtjJazfpEZYwZ","_type":"author","firstName":"Noah","lastName":"Stansfield","_createdAt":"2022-06-14T20:44:15Z"}],"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"],"_id":"63407d72-8248-468e-a949-b9d968a7c4f1","taxonomyMapping":[{"_updatedAt":"2023-11-27T17:07:43Z","parent":{"_createdAt":"2020-12-08T16:06:37Z","_type":"taxonomy","_updatedAt":"2023-01-19T14:52:23Z","isMainTopic":true,"parent":null,"_rev":"60drdzXgFuuRxJsEyE6his","name":"Topic","_id":"8ea01556-869e-480b-83a7-58eca140415e","identifier":"topic"},"perKeywordMapping":["Cardiology","Neurology","Health Equity Diversity \u0026 Inclusion"],"_createdAt":"2020-12-08T16:07:23Z","identifier":"neurology","_type":"taxonomy","_id":"721f499d-3ed3-4310-94c7-f646bad56dc2","summary":[{"style":"normal","_key":"5869c0483365","markDefs":[],"children":[{"_type":"span","marks":[],"text":"The ","_key":"8bdd6ce5e667"},{"text":"CGTLive","_key":"d7e39fed968f","_type":"span","marks":["em"]},{"text":"™","_key":"5761d91435ea","_type":"span","marks":["superscript"]},{"marks":[],"text":" Neurology specialty topic page houses video interviews with key opinion leaders in the field of neurology about the latest relevant FDA actions, clinical guideline updates, and clinical trial findings related to cell therapies, gene therapies, and engineered and regenerative medicines developed for neurologic disorders and diseases. 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The patients were aged between 6 and 10 years old.","_key":"14574d964219","_type":"span","marks":[]}],"_type":"block","style":"normal"},{"style":"normal","_key":"487a699bd233","markDefs":[],"children":[{"_type":"span","marks":[],"text":"All 3 patients who received the high dose showed stabilization of motor function, as assessed by the 34-point North Star Ambulatory Assessment, at 1 to 2 years posttreatment. Notably, 1 of these patients showed improvement with a maximum score of 34 recorded 12 months after dosing. This improvement was later confirmed at the 18 month time point. By contrast, untreated patients followed in a natural history studied being carried out by Genethon showed a rapid decline in motor function over the same course of time.","_key":"d0fdbd98f9270"}],"_type":"block"},{"children":[{"_type":"span","marks":[],"text":"Furthermore, in the patients treated at the high dose in the trial, 15% to 85% (mean, 54%) of muscle fibers showed expression of microdystrophin as measured via immunohistochemistry at 8 weeks posttreatment. In addition, 0.4 to 2.4 (mean, 1.2) vector genome copies per muscle fiber nuclei were reported at the same time point. At 12 weeks posttreatment, levels of the biomarker creatine phosphokinase had dropped by 50% to 87% (mean, 74%). This drop was sustained at up to 18 months posttreatment for the first 2 patients treated in the higher dose group.","_key":"c4f96a0c4ff00"}],"_type":"block","style":"normal","_key":"693ca978bd10","markDefs":[]},{"_type":"block","style":"normal","_key":"4d08f995176e","markDefs":[],"children":[{"text":"With regard to safety, GNT0004, which was administered in conjunction with transient immunological prophylaxis, was deemed to be tolerated well. In light of the promising safety and efficacy results, Genethon plans to enroll patients in the trial’s pivotal portion in the United States and Europe in the middle of next year.","_key":"16c1971c78150","_type":"span","marks":[]}]},{"_type":"block","style":"normal","_key":"67005de6f2e6","markDefs":[],"children":[{"_type":"span","marks":[],"text":"“The results of treatment with our GNT0004 gene therapy are very positive in patients treated at the higher of 2 doses, both in terms of microdystrophin expression and clinical improvement,” Frederic Revah, PhD, the chief executive officer of Genethon, said in a statement.","_key":"fe6fdf4a4c410"},{"marks":["superscript"],"text":"1","_key":"36f2015e37b4","_type":"span"},{"text":" “In addition, the strength of our product lies in this selected dose, which is lower than those used in other gene therapy trials for DMD. GNT0004 has the potential to be the best-in-class curative gene therapy for DMD. These clinical resultsdemonstrate that gene therapy can provide solutions to one of the most complex genetic diseases. Our aim is to start the confirmatory (pivotal) phase in more than 60 children in Europe in the second quarter of 2025, followed by the US.”","_key":"e71c4a40f747","_type":"span","marks":[]}]},{"children":[{"_type":"span","marks":[],"text":"Four of the 5 patients treated in the study thus far were treated in France, and 1 patient was treated in the United Kingdom.","_key":"301b9a500b320"},{"_type":"span","marks":["superscript"],"text":"2","_key":"f19a20233af3"},{"text":" The study, which originally launched in 2021, resumed in 2022 after having been paused in relation to a serious adverse event (SAE) that occurred in the first patient dosed. The resumption of the trial commenced after the SAE resolved.","_key":"5d2c7bca1a90","_type":"span","marks":[]}],"_type":"block","style":"normal","_key":"eb56b44e6d86","markDefs":[]},{"markDefs":[{"_type":"link","href":"https://www.cgtlive.com/view/investigational-aav8-gene-therapy-gnt0003-safety-crigler-najjar-syndrome","_key":"428c07677a93"}],"children":[{"_type":"span","marks":[],"text":"Besides GNT0004, Genethon is also developing ","_key":"a96b05ba1a000"},{"text":"GNT-003 (GNT0003)","_key":"a96b05ba1a001","_type":"span","marks":["428c07677a93"]},{"text":", an investigational AAV vector-based gene therapy for the treatment of Crigler-Najjar syndrome.","_key":"a96b05ba1a002","_type":"span","marks":[]},{"_type":"span","marks":["superscript"],"text":"3","_key":"484e7621c697"},{"_type":"span","marks":[],"text":" Results from a phase 1/2 clinical trial (NCT03466463) reported in ","_key":"2d76d5fd6da8"},{"marks":["em"],"text":"The New England Journal of Medicine","_key":"6a1a1cd3246f","_type":"span"},{"marks":[],"text":" in August 2023 showed that patients with the rare liver disease who received the trial’s higher dose (n = 3; 5×10","_key":"4caa02fd101d","_type":"span"},{"_type":"span","marks":["superscript"],"text":"12","_key":"c0639fa7b26f"},{"_type":"span","marks":[],"text":" vg/kg) experienced a decrease in bilirubin levels and were not receiving phototherapy at least 78 weeks after vector administration.","_key":"10961594e28a"}],"_type":"block","style":"normal","_key":"d181748c3157"},{"style":"normal","_key":"52876caa4a28","markDefs":[],"children":[{"marks":[],"text":"“Although our study is small, among the patients who received the dose of 5×10","_key":"2f874329d9720","_type":"span"},{"_type":"span","marks":["superscript"],"text":"12","_key":"8e74454955f1"},{"marks":[],"text":" vg/kg, GNT0003 restored UGT1A1 activity to levels that permitted suspension of phototherapy, and the efficacy persisted at 18 months after the treatment,” study author Lorenzo D’Antiga, MD, of the Department of Pediatric Hepatology, Gastroenterology, and Transplantation at Hospital Papa Giovanni XXIII, in Bergamo, Italy, and colleagues wrote.","_key":"30813f6b3fe6","_type":"span"},{"_type":"span","marks":["superscript"],"text":"4","_key":"4a9a8a93b1df"},{"_type":"span","marks":[],"text":" “A test of replication in a larger, well-characterized cohort of patients will be important.”","_key":"5f71b46a3c34"}],"_type":"block"},{"children":[{"_type":"span","marks":[],"text":"REFERENCES\n1. Genethon presents positive initial results from a phase 1/2/3 trial of its gene therapy (GNT0004) for Duchenne muscular dystrophy at ASGCT breakthroughs in muscular dystrophy in Chicago. News release. Genethon. November 19, 2024. Accessed November 25, 2024. https://www.genethon.com/genethon-presents-positive-initial-results-from-a-phase-1-2-3-trial-of-its-gene-therapy-gnt0004-for-duchenne-muscular-dystrophy-at-asgct-breakthroughs-in-muscular-dystrophy-in-chicago/\n2. First clinical trial results of gene therapy (GNT0004) for Duchenne muscular dystrophy presented at international myology 2024 congress. News release. Genethon. April 23, 2024. Accessed June 21, 2024. https://www.genethon.com/first-clinical-trial-results-of-gene-therapy-gnt0004-for-duchenne-muscular-dystrophy-presented-at-international-myology-2024-congress/\n3. D’Antinga L, Beuers U, Ronzitti G, et al. Gene Therapy in Patients with the Crigler–Najjar Syndrome. N Engl J Med. 2023; 389:620-631. doi:10.1056/NEJMoa2214084\n4. Positive Phase 1/2 Clinical Trial Results of Genethon’s Gene Therapy for Crigler-Najjar Syndrome, a Rare Liver Disease, Published in The New England Journal of Medicine. News release. Genethon. August 22, 2023. Accessed August 29, 2023. https://www.businesswire.com/news/home/20230822525314/en/Positive-Phase-12-Clinical-Trial-Results-of-Genethon%E2%80%99s-Gene-Therapy-for-Crigler-Najjar-Syndrome-a-Rare-Liver-Disease-Published-in-The-New-England-Journal-of-Medicine\n","_key":"25ad67181d74"}],"_type":"block","style":"h6","_key":"845ff701b8db","markDefs":[]}],"authorMapping":[{"displayName":"Noah Stansfield","_id":"f9e9001f-7a9c-46a2-9e9c-318ee31c1923","_updatedAt":"2022-06-14T20:44:15Z","url":{"current":"noah-stansfield","_type":"slug"},"_type":"author","firstName":"Noah","lastName":"Stansfield","_createdAt":"2022-06-14T20:44:15Z","_rev":"yD3aK4wI9vtjJazfpEZYwZ"}],"authors":[{"displayName":"Noah Stansfield","url":"noah-stansfield"}],"thumbnail":{"_type":"mainImage","asset":{"_ref":"image-e292d9fc799af049ac1bd0109419772a7b6fbdd4-1000x563-jpg","_type":"reference"}},"seoTag":["Duchenne Muscular Dystrophy","DMD","gene therapy"],"title":"Genethon’s Gene Therapy GNT0004 Stabilizes or Improves Motor 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","_key":"6c82da9de699"},{"marks":["ce7edc147077"],"text":"Ameri","_key":"5dc4b06c9b30","_type":"span"},{"_key":"ba30640d8323","_type":"span","marks":["b309d0463db9"],"text":"can College of Rheumatology (ACR) Convergence 2024"},{"_type":"span","marks":[],"text":" in Washington, DC, Georg Schett, MD, vice president research and chair of internal medicine at the University of Erlangen – Nuremberg, presented data from 2 studies evaluating CD19-directed chimeric antigen receptor T-cell (CAR-T) therapies in patients with a variety of refractory autoimmune and rheumatic diseases. Specifically, these studies were the CASTLE basket study and a phase 1 clinical trial for CC-97540 (Bristol Myers Squibb).","_key":"e8c4c176be4d"}],"_type":"block"},{"style":"normal","_key":"b040ef347cf4","markDefs":[],"children":[{"_type":"span","marks":["em"],"text":"CGTLive®","_key":"2121c7ddda8d"},{"_type":"span","marks":[],"text":"'s sister site ","_key":"282fce5d674b"},{"_type":"span","marks":["em"],"text":"HCPLive® ","_key":"da8f94f164a6"},{"_type":"span","marks":[],"text":"interviewed Schett on the conference floor to learn more about the promising safety and efficacy findings from these studies. He went over the data and also discussed the advantages of a new manufacturing process used to produce CC-97540.","_key":"13009863cd4a"}],"_type":"block"},{"markDefs":[],"children":[{"_type":"span","marks":["strong","em"],"text":"HCPLive:","_key":"8877a82e07af0"},{"_type":"span","marks":["strong"],"text":" Can you discuss the safety findings of CC-97540 CAR-T therapy?","_key":"24a5415d083b"}],"_type":"block","style":"h3","_key":"95131aaf3f41"},{"style":"normal","_key":"2078bc759d0e","markDefs":[],"children":[{"_key":"caf461466dee","_type":"span","marks":["strong"],"text":"Georg Schett, MD: "},{"marks":[],"text":"That's a phase 1 study, and it's a safety study, so safety is the key end point in this study. Basically the study recapitulated very well what we know—that cytokine release syndrome (CRS) is mild to moderate—so there were no higher grade CRS cases in the study, which is very reassuring. There were no major infections in this study, which is also very reassuring. The bone marrow toxicity was also only very short because of the lymphodepletion, but there was no long term bone marrow toxicity. There was 1 grade 3 neurotoxicity, but that resolved within 24 hours and that's, I think, very okay. There was no other neurotoxicity. In fact, I think the bottom line is the safety of this phase 1 study so far looks very good.","_key":"069d4c3eb958","_type":"span"}],"_type":"block"},{"_key":"f4edf59ebae5","markDefs":[],"children":[{"_type":"span","marks":["strong"],"text":"How does next-generation manufacturing differentiate CC-97540?","_key":"21012ee0dd56"}],"_type":"block","style":"h3"},{"markDefs":[],"children":[{"_type":"span","marks":[],"text":"Well, I think that's very exciting, in fact... Development of new technologies in CAR T-cells is very important, and this fast manufacturing is very exciting because it's only 5 days, and you actually need much fewer cells to be infused into a patient with this fast manufacturing method, with a similar expansion of the cells as with the original method. So I think that's very exciting and it's obviously also important if there's a shorter duration in the turnaround time and actually less need for production space, because it's just shorter. That's a big advantage for the field, I would say. ","_key":"6f31487431a20"}],"_type":"block","style":"normal","_key":"3c6b21e88ef0"},{"markDefs":[],"children":[{"_type":"span","marks":["strong"],"text":"Can you discuss the CASTLE basket study and its findings?","_key":"486fce2afd82"}],"_type":"block","style":"h3","_key":"56f82d62e8d9"},{"markDefs":[],"children":[{"_type":"span","marks":[],"text":"CASTLE is an academic study and it's a basket study. It's a phase 1/2a study. CASTLE recruits lupus, myositis, and systemic sclerosis. CASTLE had actually recruited 8 patients and after 8 patients, when there was good efficacy and safety, it moved to the second phase, and that actually turned out to be positive. There was no major toxicity signal and a very good safety signal. This is essentially, at the moment, a positive study. We will see in total a 24 patient study. It's close to finishing off recruitment. Basically, we will see how at the end the data look, but everything at the moment looks good. I think what is really amazing is that all the patients who had the efficacy analysis after 6 months met the high quality efficacy end point. That means remission, major improvement in the myositis, or no progression with systemic sclerosis. All the 8 patients who had efficacy analysis, possible after 6 months, met the efficacy analysis and that was very exciting I would say.","_key":"92334345790a0"}],"_type":"block","style":"normal","_key":"da8a5dcab1b2"},{"markDefs":[],"children":[{"_type":"span","marks":["strong"],"text":"What has been your experience investigating CAR-T therapies with autoimmune diseases?","_key":"9d03c4e4edf4"}],"_type":"block","style":"h3","_key":"c048425fff7d"},{"_type":"block","style":"normal","_key":"633ba12c521e","markDefs":[],"children":[{"_key":"1cdedbaac4e20","_type":"span","marks":[],"text":"We have now in our center treated more than 40 patients, and we have a single relapse so the relapse rate is very low. There have been occasional relapses reported in the field by other studies, but also with low dose CAR T-cell therapy because that was the starting dose. So this has to be interpreted in the right way, and I would say that relapses occur, of course, right? Nothing 100%. But so far, it looks that most of the people enjoy a drug-free remission for a long time and that's very exciting."}]},{"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","_key":"bcb4af2819f3","markDefs":[],"children":[{"_type":"span","marks":["strong","em"],"text":"Relevant disclosures for Schett include Bristol-Myers Squibb, Cabaletta, Janssen, Kyverna Therapeutics, and Novartis.","_key":"270f47a4fea40"}],"_type":"block"},{"markDefs":[],"children":[{"_key":"5fc1dcfdb05b0","_type":"span","marks":[],"text":"REFERENCES\n1. Schett G, Littlejohn E, Kramer N, et al. A Phase 1, Multicenter, Open-Label Study to Establish the Preliminary Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of CC-97540 (BMS-986353), a CD19-directed CAR T Cell Therapy Manufactured Using a Next-generation Process, for Severe, Refractory Autoimmune Diseases. Presented at: ACR Convergence 2024; November 14-19; Washington, DC. Abstract 1753\n2. Schett G, Müller F, Hagen M, et al. Safety and Preliminary Efficacy of CD19 CAR-T Cell Treatment in Rheumatic Disease – Data from the First Part of the Phase I/II CASTLE Basket Study. Presented at: ACR Convergence 2024; November 14-19; Washington, DC. 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Therapy","url":{"_type":"slug","current":"alzheimer-disease-awareness-month-2024-looking-back-year-progress-cell-gene-therapy"},"thumbnail":{"_type":"mainImage","asset":{"_ref":"image-bcf8b7384dbe616cc22cb250d8946d9b6758e997-1200x674-jpg","_type":"reference"}},"published":"2024-11-24T14:00:00.000Z"},{"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"}]},{"is_visible":true,"title":"Alzheimer Disease Awareness Month 2024: Looking Back at a Year of Progress in Cell and Gene Therapy","display_summary":true,"ExcludeFromPubMedXML":false,"_type":"article","url":"alzheimer-disease-awareness-month-2024-looking-back-year-progress-cell-gene-therapy","internalTag":["GM1 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Fifteen patients were dosed in the trial across 3 ascending single-dose cohorts: 1.4x10","_key":"436f441789642"},{"marks":["superscript"],"text":"10","_key":"dfa05c48b3f2","_type":"span"},{"_key":"56f666ab715f","_type":"span","marks":[],"text":" gc/ml (C1; n = 5), 4.4x10"},{"_type":"span","marks":["superscript"],"text":"10","_key":"2395fc78c7db"},{"_type":"span","marks":[],"text":" gc/ml (C2; n = 4), 1.4x10","_key":"9598e12ebcc5"},{"_type":"span","marks":["superscript"],"text":"11","_key":"51db13af6eaf"},{"_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":"7e2e46ca5fb9"},{"text":"14","_key":"a152de76ff56","_type":"span","marks":["superscript"]},{"_type":"span","marks":[],"text":" gc (C4; n = 3). Participants in the 52-week study were administered prednisone for 8 weeks after treatment with LX1001. ","_key":"d0e8e5330480"},{"_type":"span","marks":["em"],"text":"APOE4","_key":"b49845762ae7"},{"_type":"span","marks":[],"text":" 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":"f9ae015331ec"}],"_type":"block","style":"normal","_key":"c63c6ea0a74f","markDefs":[{"_type":"link","href":"https://www.neurologylive.com/conferences/ctad","_key":"039663a5b30e"}]},{"style":"normal","_key":"16a88ed3f187","markDefs":[],"children":[{"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. 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The announcement comes after a safety review committee reviewed data from the phase 1 portion of the trial and an internal review board cleared the program to continue.","_key":"7c1575f6d3c3","_type":"span"}]},{"_type":"block","style":"normal","_key":"7ea0469c3e26","markDefs":[],"children":[{"_type":"span","marks":[],"text":"“I am encouraged by the continued promise of NKGen’s SNK01 NK cell therapy in a difficult to treat disease such as AD,” Jesse Carr, MD, Medical Director, Behavioral Research Specialists, Glendale, California, who is independently overseeing the trial site, said in a statement. “We are seeing remarkable preliminary clinical benefit in all patients without treatment-related adverse events, including apparent improvements in cognitive function, increases in daily living activities and overall quality of life. I look forward to the potential that this novel drug candidate holds as we continue to progress the trial.”","_key":"63f3401659a80"}]},{"markDefs":[],"children":[{"_type":"span","marks":[],"text":"SNK01 is an autologous NK cell therapy that is not genetically engineered but has enhanced cytotoxicity and activating receptor expression. The therapy has previously been assessed in the proof-of-concept phase 1 ASK-AD trial (NCT04678453) with a different manufacturing process. The study was an open label, 3+3 dose escalation study which demonstrated that 4 doses of 1, 2, or 4 billion SNK01 cells administered intravenously was safe and crossed the blood brain barrier to reduce amyloid, tau, and alpha-synuclein proteins. 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The trial will evaluate safety and seek to establish a recommended dose for a possible phase 2 trial, with AD clinical assessments and evaluations of biochemical and anatomical biomarkers serving as secondary outcome measures. The study is recruiting patients aged 45 to 80 years of age with mild-to-moderate AD, with plans to follow patients for up to 12 months posttreatment.","_key":"4dbb8c9961aa0","_type":"span"}]},{"markDefs":[],"children":[{"_type":"span","marks":[],"text":"“The first patient in this trial was dosed in March 2024, and no side effects or adverse events have been reported to date,” Gustavo Alva, MD, the principal investigator of the trial, said in a statement. “Enrollment is ongoing and we look forward to completing the trial and sharing its results. 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As such, patients with other forms of epilepsy are unable to participate in the first-in-human phase 1/2 clinical trial (NCT05135091). Although, the company has plans to eventually evaluate NRTX-1001 in other epilepsy indications in future clinical trials, pending regulatory clearance.","_key":"da9f8e1abcc5","_type":"span"}]},{"markDefs":[{"href":"https://www.cgtlive.com/conferences/american-epilepsy-society-aes","_key":"7fcdd9cd5a6d","_type":"link"}],"children":[{"_type":"span","marks":[],"text":"In December 2023, following the presentation of data from the trial at the Annual Meeting of ","_key":"37b27e1115530"},{"_type":"span","marks":["7fcdd9cd5a6d"],"text":"the American Epilepsy Society (AES)","_key":"37b27e1115531"},{"_type":"span","marks":[],"text":", held December 1-5, 2023, in Orlando, Florida, ","_key":"37b27e1115532"},{"_type":"span","marks":["em"],"text":"CGTLive","_key":"37b27e1115533"},{"marks":[],"text":" interviewed Cory R. Nicholas, PhD, the cofounder and CEO of Neurona Therapeutics, about challenges in the trial and future plans for the cell therapy product. Nicholas spoke potential future trials that could include patients with bilateral temporal lobe epilepsy, focal cortical dysplasias, and even AD that has epileptic-type activity.","_key":"37b27e1115534","_type":"span"}],"_type":"block","style":"normal","_key":"2b770c34c52d"},{"_key":"b48181141577","alignment":"center","asset":{"_ref":"image-10cc6e8348d63bae9563ddb8e47f06ca6dee3512-150x75-png","_type":"reference"},"blank":true,"disableTextWrap":false,"disableLightBox":true,"_type":"figure","link":"https://www.cgtlive.com/view/epilepsy-cell-therapy-nrtx-1001-eventually-evaluated-alzheimer-disease"},{"markDefs":[],"children":[{"_type":"span","marks":[],"text":"REFEENCE\n1. Facts About Alzheimer's Disease. Alzheimer's Foundation of America. Website. 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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","markDefs":[]},{"children":[{"_type":"span","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":"block","style":"normal","_key":"fd7e8d63ac40","markDefs":[]},{"_key":"4909e183ce60","markDefs":[],"children":[{"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":"span","marks":[]}],"_type":"block","style":"normal"},{"style":"h3","_key":"56f82d62e8d9","markDefs":[],"children":[{"_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?","_key":"486fce2afd82"}],"_type":"block"},{"children":[{"_type":"span","marks":[],"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":"block","style":"normal","_key":"1ba97d171ccb","markDefs":[]},{"markDefs":[],"children":[{"_key":"9d03c4e4edf4","_type":"span","marks":["strong"],"text":"Have there been any challenges in the study and/or are there any areas of interest for further research?"}],"_type":"block","style":"h3","_key":"c048425fff7d"},{"_key":"6a65969d84d5","markDefs":[],"children":[{"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.","_key":"c7534f3a20780","_type":"span"}],"_type":"block","style":"normal"},{"markDefs":[],"children":[{"_type":"span","marks":["em","strong"],"text":"This transcript has been edited for clarity.","_key":"090e000f56cf0"}],"_type":"block","style":"normal","_key":"1e27ae9fbd54"},{"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"}],"_type":"block"},{"style":"normal","_key":"68c7b9db0d9c","markDefs":[],"children":[{"marks":[],"text":"","_key":"08d1e30b91e50","_type":"span"}],"_type":"block"},{"markDefs":[],"children":[{"marks":[],"text":"","_key":"271de6c127c10","_type":"span"}],"_type":"block","style":"normal","_key":"08507dbc0e28"}],"taxonomyMapping":[{"_createdAt":"2020-12-08T16:07:23Z","name":"Neurology","parent":{"_type":"taxonomy","name":"Topic","_updatedAt":"2023-01-19T14:52:23Z","isMainTopic":true,"_createdAt":"2020-12-08T16:06:37Z","parent":null,"_id":"8ea01556-869e-480b-83a7-58eca140415e","identifier":"topic","_rev":"60drdzXgFuuRxJsEyE6his"},"perKeywordMapping":["Cardiology","Neurology","Health Equity Diversity \u0026 Inclusion"],"_type":"taxonomy","_id":"721f499d-3ed3-4310-94c7-f646bad56dc2","identifier":"neurology","cmeType":"per","_updatedAt":"2023-11-27T17:07:43Z","pixelTrackingCode":null,"summary":[{"children":[{"_type":"span","marks":[],"text":"The ","_key":"8bdd6ce5e667"},{"text":"CGTLive","_key":"d7e39fed968f","_type":"span","marks":["em"]},{"text":"™","_key":"5761d91435ea","_type":"span","marks":["superscript"]},{"marks":[],"text":" Neurology specialty topic page houses video interviews with key opinion leaders in the field of neurology about the latest relevant FDA actions, clinical guideline updates, and clinical trial findings related to cell therapies, gene therapies, and engineered and regenerative medicines developed for neurologic disorders and diseases. 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