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md:justify-between"><div class="max-w-full"><time class="tex-sm " dateTime="2024-09-30T19:00:50.223">September 30, 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/arun-upadhyay-phd">Arun Upadhyay, PhD</a></span><span class="text-md "><span class=""><br/></span><a class="mr-1 text-author hover:text-primary" href="/authors/noah-stansfield">Noah Stansfield</a></span></div></div><div class="flex flex-wrap sm:flex-nowrap items-center w-fit my-2"><div class="flex items-center w-fit h-[22px] mr-4 px-2 bg-primary text-white text-xs"><em>Commentary</em></div><div class="flex items-center w-fit h-[22px] mr-4 px-2 bg-primary text-white text-xs"><em>Video</em></div></div><div class="mt-4"><div class="mt-2 flex items-center max-w-fit"><button title="Arun Upadhyay, PhD, on Results from IRD Gene Therapy OCU400’s Phase 1/2 Trial" 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">The chief scientific officer and head of research, development, and Medical at Ocugen, also discussed future plans for the gene therapy, which is now in a phase 3 study.</p><div class="py-2"><div class="blockText_blockContent__TbCXh"><div class="relative"><div class="brightcove-fluid" autoplay=""></div></div><blockquote><div style="text-align:center"><strong><em>“I would like to highlight that currently we are enrolling patients in our OCU400 phase 3 study... It is for a broader indication, and this is the first of its own kind of trial in this space. No other company to our knowledge is running a broader RP designation trial.”</em></strong></div></blockquote><p class="pb-2"></p><p class="pb-2">Currently, substantial unmet need remains for patients with inherited retinal diseases (IRDs) such as retinitis pigmentosa (RP). Because mutations in a wide range of genes can cause IRDs like RP, developing targeted gene therapy products with the traditional “1 therapy for 1 gene” approach is impractical. As such, Ocugen has decided upon pursuing a gene-agnostic approach to treating IRDs with its Modifier Gene Therapy Platform. OCU400, a gene therapy product based on this platform, is currently being evaluated in a phase 1/2 clinical trial (NCT05203939) for the treatment of RP and Leber congenital amaurosis (LCA), as well as a more recently-initiated phase 3 clinical trial (liMeliGhT; NCT06388200), which dosed its first patient <a rel="nofollow" target="_self" href="https://www.cgtlive.com/view/ocugen-doses-first-patient-retinitis-pigmentosa-gene-therapy-ocu400-phase-3">in June</a> of this year. Notably, data from the earlier phase 1/2 trial was recently presented at the 24th EURETINA Congress held September 19 to 22, 2024, in Barcelona, Spain.</p><p class="pb-2">Shortly after the conference, <em>CGTLive®</em> spoke with Arun Upadhyay, PhD, the chief scientific officer and head of research, development, and Medical at Ocugen, to learn more about the updated data. Upadhyay pointed out that across 18 patients treated in the study, OCU400 was generally safe and well-tolerated. In terms of efficacy, he emphasized that close to 60% of the intent-to-treat population responded to OCU400 with regard to a luminance dependent navigation assessment, which constitutes a primary end point in the phase 3 trial. Upadhyay also noted that OCU400 has received orphan drug designation from both the FDA and the European Medicines Agency for RP and LCA, and that it has received regenerative medicines advanced therapy designation from the FDA, as well as an expanded access protocol from the FDA in RP.</p><h6 class="text-sm pb-4 pt-2">REFERENCE<br/>1. Ocugen, Inc. Announces First Patient Dosed in Phase 3 liMeliGhT Clinical Trial for OCU400—First Gene Therapy in Phase 3 with a Broad Retinitis Pigmentosa Indication. June 20, 2024. Accessed September 30, 2024. https://www.globenewswire.com/news-release/2024/06/20/2901513/0/en/Ocugen-Inc-Announces-First-Patient-Dosed-in-Phase-3-liMeliGhT-Clinical-Trial-for-OCU400-First-Gene-Therapy-in-Phase-3-with-a-Broad-Retinitis-Pigmentosa-Indication.html</h6></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" 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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/around-the-helix-cell-and-gene-therapy-company-updates-november-13-2024?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 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srcSet="/_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fgenetherapy%2F9b1b40bc81fc00eeb21179fe80ec2a22d6af7e25-800x500.jpg%3Ffit%3Dcrop%26auto%3Dformat&w=640&q=75 640w, /_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fgenetherapy%2F9b1b40bc81fc00eeb21179fe80ec2a22d6af7e25-800x500.jpg%3Ffit%3Dcrop%26auto%3Dformat&w=750&q=75 750w, /_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fgenetherapy%2F9b1b40bc81fc00eeb21179fe80ec2a22d6af7e25-800x500.jpg%3Ffit%3Dcrop%26auto%3Dformat&w=828&q=75 828w, /_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fgenetherapy%2F9b1b40bc81fc00eeb21179fe80ec2a22d6af7e25-800x500.jpg%3Ffit%3Dcrop%26auto%3Dformat&w=1080&q=75 1080w, /_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fgenetherapy%2F9b1b40bc81fc00eeb21179fe80ec2a22d6af7e25-800x500.jpg%3Ffit%3Dcrop%26auto%3Dformat&w=1200&q=75 1200w, 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style="border-top-left-radius:0rem;border-top-right-radius:0rem;border-bottom-left-radius:0rem;border-bottom-right-radius:0rem;position:absolute;top:0;left:0;bottom:0;right:0;box-sizing:border-box;padding:0;border:none;margin:auto;display:block;width:0;height:0;min-width:100%;max-width:100%;min-height:100%;max-height:100%" class="shrink-0" loading="lazy"/></noscript></span></div></a><div class="flex-auto w-[200%] md:w-auto ml-2 flex-1"><p class="font-bold text-[1rem] pl-4 text-undefined" style="font-size:1rem"><a href="/view/around-the-helix-cell-and-gene-therapy-company-updates-november-13-2024?utm_source=www.cgtlive.com&utm_medium=relatedContent">Around the Helix: Cell and Gene Therapy Company Updates – November 13, 2024</a></p><div class=" pl-4"><a class="text-sm text-sky-800" href="/authors/noah-stansfield">Noah Stansfield</a></div><a href="/view/around-the-helix-cell-and-gene-therapy-company-updates-november-13-2024?utm_source=www.cgtlive.com&utm_medium=relatedContent"><span class="text-sm text-gray-500 pl-4">November 13th 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">Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies. </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/anelloviruses-potential-alternative-aav-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:100%"></span><img alt="Chris Wright, MD, PhD, the chief medical officer and head of translational research at Ring Therapeutics" title="Chris Wright, MD, PhD, the chief medical officer and head of translational research at Ring Therapeutics" 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="Chris Wright, MD, PhD, the chief medical officer and head of translational research at Ring Therapeutics" title="Chris Wright, MD, PhD, the chief medical officer and head of translational research at Ring Therapeutics" sizes="100vw" srcSet="/_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fgenetherapy%2F581bc40993a4b19fb0c5a525d43fe83492ef1b89-300x300.jpg%3Ffit%3Dcrop%26auto%3Dformat&w=640&q=75 640w, 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translational research at Ring Therapeutics, discussed research presented at ASGCT 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/around-the-helix-cell-and-gene-therapy-company-updates-november-6-2024?utm_source=www.cgtlive.com&utm_medium=relatedContent"><div class=""><span 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and Gene Therapy Company Updates – November 6, 2024" title="Around the Helix: Cell and Gene Therapy Company Updates – November 6, 2024" sizes="100vw" srcSet="/_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fgenetherapy%2F9b1b40bc81fc00eeb21179fe80ec2a22d6af7e25-800x500.jpg%3Ffit%3Dcrop%26auto%3Dformat&w=640&q=75 640w, /_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fgenetherapy%2F9b1b40bc81fc00eeb21179fe80ec2a22d6af7e25-800x500.jpg%3Ffit%3Dcrop%26auto%3Dformat&w=750&q=75 750w, /_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fgenetherapy%2F9b1b40bc81fc00eeb21179fe80ec2a22d6af7e25-800x500.jpg%3Ffit%3Dcrop%26auto%3Dformat&w=828&q=75 828w, /_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fgenetherapy%2F9b1b40bc81fc00eeb21179fe80ec2a22d6af7e25-800x500.jpg%3Ffit%3Dcrop%26auto%3Dformat&w=1080&q=75 1080w, 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style="border-top-left-radius:0rem;border-top-right-radius:0rem;border-bottom-left-radius:0rem;border-bottom-right-radius:0rem;position:absolute;top:0;left:0;bottom:0;right:0;box-sizing:border-box;padding:0;border:none;margin:auto;display:block;width:0;height:0;min-width:100%;max-width:100%;min-height:100%;max-height:100%" class="shrink-0" loading="lazy"/></noscript></span></div></a><div class="flex-auto w-[200%] md:w-auto ml-2 flex-1"><p class="font-bold text-[1rem] pl-4 text-undefined" style="font-size:1rem"><a href="/view/around-the-helix-cell-and-gene-therapy-company-updates-november-6-2024?utm_source=www.cgtlive.com&utm_medium=relatedContent">Around the Helix: Cell and Gene Therapy Company Updates – November 6, 2024</a></p><div class=" pl-4"><a class="text-sm text-sky-800" href="/authors/noah-stansfield">Noah Stansfield</a></div><a href="/view/around-the-helix-cell-and-gene-therapy-company-updates-november-6-2024?utm_source=www.cgtlive.com&utm_medium=relatedContent"><span class="text-sm text-gray-500 pl-4">November 6th 2024</span><div><span class="px-2 py-1 ml-4 text-xs text-white border bg-primary italic">Article</span></div><div class="mt-2 ml-4"></div><div class="flex flex-row gap-2"></div><p class=" mt-4 text-gray-800 pl-4">Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies. </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="relative hidden sm:block"><div class="mt-4 overflow-hidden"><div class="flex items-center clear-both pt-4 text-xl font-bold">Related Content </div><div class="w-full mb-2 border border-secondary"></div><div class="flex flex-wrap items-center"></div><div class="flex flex-wrap w-full"><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 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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" 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style="border-top-left-radius:0rem;border-top-right-radius:0rem;border-bottom-left-radius:0rem;border-bottom-right-radius:0rem;position:absolute;top:0;left:0;bottom:0;right:0;box-sizing:border-box;padding:0;border:none;margin:auto;display:block;width:0;height:0;min-width:100%;max-width:100%;min-height:100%;max-height:100%" class="shrink-0" loading="lazy"/></noscript></span></div></a><div class="flex-auto w-[200%] md:w-auto ml-2 flex-1"><p class="font-bold text-[1rem] pl-4 text-undefined" style="font-size:1rem"><a href="/view/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/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. 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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 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/around-the-helix-cell-and-gene-therapy-company-updates-november-20-2024?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:62.5%"></span><img alt="Around the Helix: Cell and Gene Therapy Company Updates – November 20, 2024" title="Around the Helix: Cell and Gene Therapy Company Updates – November 20, 2024" 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="Around the Helix: Cell and Gene Therapy Company Updates – November 20, 2024" title="Around the Helix: Cell and Gene Therapy Company Updates – November 20, 2024" sizes="100vw" srcSet="/_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fgenetherapy%2F9b1b40bc81fc00eeb21179fe80ec2a22d6af7e25-800x500.jpg%3Ffit%3Dcrop%26auto%3Dformat&w=640&q=75 640w, /_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fgenetherapy%2F9b1b40bc81fc00eeb21179fe80ec2a22d6af7e25-800x500.jpg%3Ffit%3Dcrop%26auto%3Dformat&w=750&q=75 750w, 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href="/view/around-the-helix-cell-and-gene-therapy-company-updates-november-20-2024?utm_source=www.cgtlive.com&utm_medium=relatedContent">Around the Helix: Cell and Gene Therapy Company Updates – November 20, 2024</a></p><div class=" pl-4"><a class="text-sm text-sky-800" href="/authors/noah-stansfield">Noah Stansfield</a></div><a href="/view/around-the-helix-cell-and-gene-therapy-company-updates-november-20-2024?utm_source=www.cgtlive.com&utm_medium=relatedContent"><span class="text-sm text-gray-500 pl-4">November 20th 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">Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies. </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/around-the-helix-cell-and-gene-therapy-company-updates-november-13-2024?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 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style="border-top-left-radius:0rem;border-top-right-radius:0rem;border-bottom-left-radius:0rem;border-bottom-right-radius:0rem;position:absolute;top:0;left:0;bottom:0;right:0;box-sizing:border-box;padding:0;border:none;margin:auto;display:block;width:0;height:0;min-width:100%;max-width:100%;min-height:100%;max-height:100%" class="shrink-0" loading="lazy"/></noscript></span></div></a><div class="flex-auto w-[200%] md:w-auto ml-2 flex-1"><p class="font-bold text-[1rem] pl-4 text-undefined" style="font-size:1rem"><a href="/view/around-the-helix-cell-and-gene-therapy-company-updates-november-13-2024?utm_source=www.cgtlive.com&utm_medium=relatedContent">Around the Helix: Cell and Gene Therapy Company Updates – November 13, 2024</a></p><div class=" pl-4"><a class="text-sm text-sky-800" href="/authors/noah-stansfield">Noah Stansfield</a></div><a href="/view/around-the-helix-cell-and-gene-therapy-company-updates-november-13-2024?utm_source=www.cgtlive.com&utm_medium=relatedContent"><span class="text-sm text-gray-500 pl-4">November 13th 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">Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies. </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/anelloviruses-potential-alternative-aav-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:100%"></span><img alt="Chris Wright, MD, PhD, the chief medical officer and head of translational research at Ring Therapeutics" title="Chris Wright, MD, PhD, the chief medical officer and head of translational research at Ring Therapeutics" 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="Chris Wright, MD, PhD, the chief medical officer and head of translational research at Ring Therapeutics" title="Chris Wright, MD, PhD, the chief medical officer and head of translational research at Ring Therapeutics" sizes="100vw" srcSet="/_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fgenetherapy%2F581bc40993a4b19fb0c5a525d43fe83492ef1b89-300x300.jpg%3Ffit%3Dcrop%26auto%3Dformat&w=640&q=75 640w, 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translational research at Ring Therapeutics, discussed research presented at ASGCT 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/around-the-helix-cell-and-gene-therapy-company-updates-november-6-2024?utm_source=www.cgtlive.com&utm_medium=relatedContent"><div class=""><span 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and Gene Therapy Company Updates – November 6, 2024" title="Around the Helix: Cell and Gene Therapy Company Updates – November 6, 2024" sizes="100vw" srcSet="/_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fgenetherapy%2F9b1b40bc81fc00eeb21179fe80ec2a22d6af7e25-800x500.jpg%3Ffit%3Dcrop%26auto%3Dformat&w=640&q=75 640w, /_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fgenetherapy%2F9b1b40bc81fc00eeb21179fe80ec2a22d6af7e25-800x500.jpg%3Ffit%3Dcrop%26auto%3Dformat&w=750&q=75 750w, /_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fgenetherapy%2F9b1b40bc81fc00eeb21179fe80ec2a22d6af7e25-800x500.jpg%3Ffit%3Dcrop%26auto%3Dformat&w=828&q=75 828w, /_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fgenetherapy%2F9b1b40bc81fc00eeb21179fe80ec2a22d6af7e25-800x500.jpg%3Ffit%3Dcrop%26auto%3Dformat&w=1080&q=75 1080w, 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style="border-top-left-radius:0rem;border-top-right-radius:0rem;border-bottom-left-radius:0rem;border-bottom-right-radius:0rem;position:absolute;top:0;left:0;bottom:0;right:0;box-sizing:border-box;padding:0;border:none;margin:auto;display:block;width:0;height:0;min-width:100%;max-width:100%;min-height:100%;max-height:100%" class="shrink-0" loading="lazy"/></noscript></span></div></a><div class="flex-auto w-[200%] md:w-auto ml-2 flex-1"><p class="font-bold text-[1rem] pl-4 text-undefined" style="font-size:1rem"><a href="/view/around-the-helix-cell-and-gene-therapy-company-updates-november-6-2024?utm_source=www.cgtlive.com&utm_medium=relatedContent">Around the Helix: Cell and Gene Therapy Company Updates – November 6, 2024</a></p><div class=" pl-4"><a class="text-sm text-sky-800" href="/authors/noah-stansfield">Noah Stansfield</a></div><a href="/view/around-the-helix-cell-and-gene-therapy-company-updates-november-6-2024?utm_source=www.cgtlive.com&utm_medium=relatedContent"><span class="text-sm text-gray-500 pl-4">November 6th 2024</span><div><span class="px-2 py-1 ml-4 text-xs text-white border bg-primary italic">Article</span></div><div class="mt-2 ml-4"></div><div class="flex flex-row gap-2"></div><p class=" mt-4 text-gray-800 pl-4">Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies. </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":"Arun Upadhyay, PhD, on Results from IRD Gene Therapy OCU400’s Phase 1/2 Trial","datePublished":"2024-09-30T19:00:50.223Z","dateModified":"2024-10-06T23:05:23Z","inLanguage":"en-US","image":"https://cdn.sanity.io/images/0vv8moc6/genetherapy/5e6e60da06f90de53f590214c70716aaf6f7e3b9-665x373.png?fit=crop&auto=format","mainEntityOfPage":{"@type":"WebPage","@id":"https://www.cgtlive.com/view/upadhyay-results-ird-gene-therapy-ocu400-phase-trial"},"publisher":{"@type":"Organization","name":"CGTlive™","logo":{"@type":"ImageObject","url":"https://www.cgtlive.com/CGT_Color_NoTagline.png"}},"keywords":"gene therapy","articleBody":"\n\n“I would like to highlight that currently we are enrolling patients in our OCU400 phase 3 study... It is for a broader indication, and this is the first of its own kind of trial in this space. No other company to our knowledge is running a broader RP designation trial.”\n\n\n\nCurrently, substantial unmet need remains for patients with inherited retinal diseases (IRDs) such as retinitis pigmentosa (RP). Because mutations in a wide range of genes can cause IRDs like RP, developing targeted gene therapy products with the traditional “1 therapy for 1 gene” approach is impractical. As such, Ocugen has decided upon pursuing a gene-agnostic approach to treating IRDs with its Modifier Gene Therapy Platform. OCU400, a gene therapy product based on this platform, is currently being evaluated in a phase 1/2 clinical trial (NCT05203939) for the treatment of RP and Leber congenital amaurosis (LCA), as well as a more recently-initiated phase 3 clinical trial (liMeliGhT; NCT06388200), which dosed its first patient in June of this year. Notably, data from the earlier phase 1/2 trial was recently presented at the 24th EURETINA Congress held September 19 to 22, 2024, in Barcelona, Spain.\n\nShortly after the conference, CGTLive® spoke with Arun Upadhyay, PhD, the chief scientific officer and head of research, development, and Medical at Ocugen, to learn more about the updated data. Upadhyay pointed out that across 18 patients treated in the study, OCU400 was generally safe and well-tolerated. In terms of efficacy, he emphasized that close to 60% of the intent-to-treat population responded to OCU400 with regard to a luminance dependent navigation assessment, which constitutes a primary end point in the phase 3 trial. Upadhyay also noted that OCU400 has received orphan drug designation from both the FDA and the European Medicines Agency for RP and LCA, and that it has received regenerative medicines advanced therapy designation from the FDA, as well as an expanded access protocol from the FDA in RP.\n\nREFERENCE\n1. Ocugen, Inc. Announces First Patient Dosed in Phase 3 liMeliGhT Clinical Trial for OCU400—First Gene Therapy in Phase 3 with a Broad Retinitis Pigmentosa Indication. June 20, 2024. Accessed September 30, 2024. https://www.globenewswire.com/news-release/2024/06/20/2901513/0/en/Ocugen-Inc-Announces-First-Patient-Dosed-in-Phase-3-liMeliGhT-Clinical-Trial-for-OCU400-First-Gene-Therapy-in-Phase-3-with-a-Broad-Retinitis-Pigmentosa-Indication.html","description":"The chief scientific officer and head of research, development, and Medical at Ocugen, also discussed future plans for the gene therapy, which is now in a phase 3 study.","author":[{"@type":"Person","name":"Arun Upadhyay, PhD"},{"@type":"Person","name":"Noah Stansfield"}]}</script></div></div><div class="flex-none w-[300px] z-[9999] relative hidden md:block"><div style="top:70px;margin-top:44px" class="sticky custom-spacing"></div></div></div><div id="div-gpt-ad-pixel" style="width:1px;height:1px" class=""></div><noscript><iframe src="https://www.googletagmanager.com/ns.html?id=GTM-MBPSSPT" height="0" width="0" style="display:none;visibility:hidden"></iframe></noscript><div id="footerOuterWrap" class="w-full 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It is for a broader indication, and this is the first of its own kind of trial in this space. No other company to our knowledge is running a broader RP designation trial.”"}],"_type":"block","upload_doc":null,"uploadAudio":null,"medias":null,"style":"blockquote","_key":"2c632e0a5e19","markDefs":[]},{"_type":"block","style":"normal","_key":"50ee1ebc61c7","upload_doc":null,"uploadAudio":null,"medias":null,"markDefs":[],"children":[{"_type":"span","marks":[],"text":"","_key":"bf97a60429070"}]},{"children":[{"marks":[],"text":"Currently, substantial unmet need remains for patients with inherited retinal diseases (IRDs) such as retinitis pigmentosa (RP). Because mutations in a wide range of genes can cause IRDs like RP, developing targeted gene therapy products with the traditional “1 therapy for 1 gene” approach is impractical. As such, Ocugen has decided upon pursuing a gene-agnostic approach to treating IRDs with its Modifier Gene Therapy Platform. OCU400, a gene therapy product based on this platform, is currently being evaluated in a phase 1/2 clinical trial (NCT05203939) for the treatment of RP and Leber congenital amaurosis (LCA), as well as a more recently-initiated phase 3 clinical trial (liMeliGhT; NCT06388200), which dosed its first patient ","_key":"faa68b3c30230","_type":"span"},{"_type":"span","marks":["1cb80a208330"],"text":"in June","_key":"faa68b3c30231"},{"text":" of this year. Notably, data from the earlier phase 1/2 trial was recently presented at the 24th EURETINA Congress held September 19 to 22, 2024, in Barcelona, Spain.","_key":"faa68b3c30232","_type":"span","marks":[]}],"_type":"block","upload_doc":null,"uploadAudio":null,"medias":null,"style":"normal","_key":"be2da99368c5","markDefs":[{"_type":"link","href":"https://www.cgtlive.com/view/ocugen-doses-first-patient-retinitis-pigmentosa-gene-therapy-ocu400-phase-3","_key":"1cb80a208330"}]},{"uploadAudio":null,"medias":null,"_key":"ebbb4574c352","markDefs":[],"children":[{"_key":"0479c065e0770","_type":"span","marks":[],"text":"Shortly after the conference, "},{"_type":"span","marks":["em"],"text":"CGTLive®","_key":"b47819ce3146"},{"marks":[],"text":" spoke with Arun Upadhyay, PhD, the chief scientific officer and head of research, development, and Medical at Ocugen, to learn more about the updated data. Upadhyay pointed out that across 18 patients treated in the study, OCU400 was generally safe and well-tolerated. In terms of efficacy, he emphasized that close to 60% of the intent-to-treat population responded to OCU400 with regard to a luminance dependent navigation assessment, which constitutes a primary end point in the phase 3 trial. Upadhyay also noted that OCU400 has received orphan drug designation from both the FDA and the European Medicines Agency for RP and LCA, and that it has received regenerative medicines advanced therapy designation from the FDA, as well as an expanded access protocol from the FDA in RP.","_key":"112189dce739","_type":"span"}],"_type":"block","style":"normal","upload_doc":null},{"medias":null,"markDefs":[],"children":[{"_type":"span","marks":[],"text":"REFERENCE\n1. Ocugen, Inc. Announces First Patient Dosed in Phase 3 liMeliGhT Clinical Trial for OCU400—First Gene Therapy in Phase 3 with a Broad Retinitis Pigmentosa Indication. June 20, 2024. Accessed September 30, 2024. https://www.globenewswire.com/news-release/2024/06/20/2901513/0/en/Ocugen-Inc-Announces-First-Patient-Dosed-in-Phase-3-liMeliGhT-Clinical-Trial-for-OCU400-First-Gene-Therapy-in-Phase-3-with-a-Broad-Retinitis-Pigmentosa-Indication.html","_key":"82533fed9acf0"}],"_type":"block","style":"h6","_key":"586e68f9477a","upload_doc":null,"uploadAudio":null}],"_type":"article","url":{"current":"upadhyay-results-ird-gene-therapy-ocu400-phase-trial","_type":"slug"},"documentGroup":null,"seoTag":["gene therapy"],"_id":"b7d5fd00-b374-483e-8c2a-f5352c58d666","internalTag":["AAV","gene therapy","Seattle Children’s Research Institute","hemophilia","hemophilia A","gene editing","gene delivery","nonviral","delivery methods","preclinical","preclinical research","clinical","safety"],"ExcludeFromPubMedXML":false,"articleType":"Commentary","disableAds":false,"disableRelatedContent":false,"relatedArticles":[{"_id":"d58f1559-9176-41f3-818b-d667d5e49009","_updatedAt":"2024-11-25T12:14:17Z","taxonomyMapping":[{"_id":"53e0f868-dd0a-4d39-934f-6f8217803a2f","_updatedAt":"2022-03-03T07:15:17Z","_rev":"GN79vWlZ4wTFBK5Yzyy182","name":"Conferences","pixelTrackingCode":null,"identifier":"conferences","_createdAt":"2021-06-04T17:45:47Z","_type":"taxonomy","parent":null},{"_createdAt":"2020-12-08T16:06:16Z","_rev":"2PyInWQFspcTNAL4XcZkLr","_id":"bdd20025-c759-4afc-b2d4-7e727e4253cd","pixelTrackingCode":null,"identifier":"news","_type":"taxonomy","name":"News","_updatedAt":"2022-03-03T06:51:18Z","parent":null}],"url":"exploring-car-t-autoimmune-disease","authors":[{"displayName":"Georg Schett, MD","url":"georg-schett-md"},{"displayName":"Noah Stansfield","url":"noah-stansfield"},{"displayName":"Victoria Johnson","url":"victoria-johnson"}],"documentGroupMapping":null,"is_visible":true,"_rev":"BArp2x2qbybJfBIFoucDR4","contentCategory":{"_updatedAt":"2022-07-01T12:33:15Z","_createdAt":"2020-12-08T16:21:29Z","_rev":"yD3aK4wI9vtjJazfqNRs7r","_type":"contentCategory","name":"Articles","_id":"54fdc3d6-b776-4653-9c81-26acd95a8a7e"},"authorMapping":[{"url":{"current":"georg-schett-md","_type":"slug"},"authorType":"expert","displayName":"Georg Schett, MD","_createdAt":"2024-11-20T11:09:59Z","_rev":"7xPE9R64LNdWAAsgIBUAV9","_type":"author","biography":[{"markDefs":[],"children":[{"_type":"span","marks":[],"text":"Georg Schett, MD, Vice President Research and Chair of Internal Medicine, University of Erlangen – Nuremberg","_key":"bd84357b2a8f0"}],"_type":"block","style":"normal","_key":"d9828833c8d4"}],"_updatedAt":"2024-11-20T11:09:59Z","_id":"9fdc3c86-37f4-4b6a-afad-ea2963cee05f"},{"firstName":"Noah","_createdAt":"2022-06-14T20:44:15Z","_rev":"yD3aK4wI9vtjJazfpEZYwZ","_type":"author","_id":"f9e9001f-7a9c-46a2-9e9c-318ee31c1923","lastName":"Stansfield","displayName":"Noah Stansfield","_updatedAt":"2022-06-14T20:44:15Z","url":{"current":"noah-stansfield","_type":"slug"}},{"firstName":"Victoria","_createdAt":"2021-01-06T18:30:31Z","_type":"author","_id":"YxXi5DCehknTuOjGHyH0kO","_updatedAt":"2021-01-06T18:30:31Z","lastName":"Johnson","displayName":"Victoria Johnson","_rev":"YxXi5DCehknTuOjGHyH0fl","url":{"current":"victoria-johnson","_type":"slug"}}],"body":[{"disableLightBox":true,"_type":"figure","alt":"Georg Schett, MD, vice president research and chair of internal medicine at the University of Erlangen – Nuremberg","imgcaption":[{"children":[{"_type":"span","marks":[],"text":"Georg Schett, MD","_key":"8b007d386a090"}],"_type":"block","style":"normal","_key":"5a033256da34","markDefs":[]}],"_key":"3caedbae470c","alignment":"left","asset":{"_ref":"image-6808278d4a529c2b98af3c3297a83a2440fa06a2-300x300-png","_type":"reference"},"disableTextWrap":false},{"children":[{"text":"At the ","_key":"6c82da9de699","_type":"span","marks":[]},{"_key":"5dc4b06c9b30","_type":"span","marks":["ce7edc147077"],"text":"Ameri"},{"_type":"span","marks":["b309d0463db9"],"text":"can College of Rheumatology (ACR) Convergence 2024","_key":"ba30640d8323"},{"_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":"34f33c1e6874","markDefs":[{"_key":"ce7edc147077","_type":"link","href":"https://www.hcplive.com/conference/acr"},{"_key":"b309d0463db9","_type":"link","href":"https://www.hcplive.com/conference/acr"}]},{"style":"normal","_key":"b040ef347cf4","markDefs":[],"children":[{"_key":"2121c7ddda8d","_type":"span","marks":["em"],"text":"CGTLive®"},{"_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"},{"_key":"95131aaf3f41","markDefs":[],"children":[{"text":"HCPLive:","_key":"8877a82e07af0","_type":"span","marks":["strong","em"]},{"_type":"span","marks":["strong"],"text":" Can you discuss the safety findings of CC-97540 CAR-T therapy?","_key":"24a5415d083b"}],"_type":"block","style":"h3"},{"children":[{"_key":"caf461466dee","_type":"span","marks":["strong"],"text":"Georg Schett, MD: "},{"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","marks":[]}],"_type":"block","style":"normal","_key":"2078bc759d0e","markDefs":[]},{"children":[{"_type":"span","marks":["strong"],"text":"How does next-generation manufacturing differentiate CC-97540?","_key":"21012ee0dd56"}],"_type":"block","style":"h3","_key":"f4edf59ebae5","markDefs":[]},{"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"},{"_type":"block","style":"h3","_key":"56f82d62e8d9","markDefs":[],"children":[{"_type":"span","marks":["strong"],"text":"Can you discuss the CASTLE basket study and its findings?","_key":"486fce2afd82"}]},{"_type":"block","style":"normal","_key":"da8a5dcab1b2","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"}]},{"style":"h3","_key":"c048425fff7d","markDefs":[],"children":[{"_key":"9d03c4e4edf4","_type":"span","marks":["strong"],"text":"What has been your experience investigating CAR-T therapies with autoimmune diseases?"}],"_type":"block"},{"style":"normal","_key":"633ba12c521e","markDefs":[],"children":[{"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.","_key":"1cdedbaac4e20","_type":"span"}],"_type":"block"},{"_key":"1e27ae9fbd54","markDefs":[],"children":[{"_type":"span","marks":["em","strong"],"text":"This transcript has been edited for clarity.","_key":"090e000f56cf0"}],"_type":"block","style":"normal"},{"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"},{"style":"h6","_key":"3569dec1f866","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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Dholaria discussed what makes P-BCMA-ALLO1 unique and went over the key safety and efficacy results presented and their potential implications.","_key":"5636408d128f","_type":"span","marks":[]}],"_type":"block","style":"normal","_key":"b040ef347cf4","markDefs":[]},{"_type":"block","style":"h3","_key":"95131aaf3f41","markDefs":[],"children":[{"_type":"span","marks":["strong","em"],"text":"CGTLive:","_key":"8877a82e07af0"},{"_key":"24a5415d083b","_type":"span","marks":["strong"],"text":" Can you give some background context about P-BCMA-ALLO1 and what it is?"}]},{"children":[{"_type":"span","marks":["strong"],"text":"Bhagirathbhai R. Dholaria, MD: ","_key":"649a575870dc"},{"_type":"span","marks":[],"text":"P-BCMA-ALLO1 is an off-the-shelf allogenic CAR T-cell therapy manufactured from healthy donor T-cells. The way it is different than other myeloma CAR-T therapies is that it is manufactured using a nonviral gene editing platform, as opposed to cilta-cel or ide-cel, which use virus-based vectors. [But] it also targets the BCMA protein, similar to other CAR T-cell therapies.","_key":"ab222ad51385"}],"_type":"block","style":"normal","_key":"2078bc759d0e","markDefs":[]},{"_key":"a78fffcdefb4","markDefs":[],"children":[{"text":"Can you give an overview of the recently announced data from the phase 1/1b clinical trial?","_key":"3a13757030a2","_type":"span","marks":["strong"]}],"_type":"block","style":"h3"},{"_key":"14952092c40b","markDefs":[],"children":[{"_type":"span","marks":[],"text":"At the recent IMS meeting, we presented the data on a phase 1 trial using P-BCMA-ALLO1 at a different cell dose and a different lymphodepletion regimen. Overall, this looked at the data on 72 patients. These are heavily pretreated MM patients. Half of them had high-risk cytogenetics, and around 43% of the patients actually had prior other BCMA-targeted therapy or talquetamab, which is a GPRC5D-targeted bispecific antibody therapy. Several of these patients also had other CAR T-cell therapy before coming on this trial. ","_key":"500c589033950"}],"_type":"block","style":"normal"},{"_key":"fd7e8d63ac40","markDefs":[],"children":[{"_key":"9efb4802fb65","_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. "}],"_type":"block","style":"normal"},{"markDefs":[],"children":[{"_type":"span","marks":[],"text":"Looking at the in vivo kinetics, we noticed that with higher lymphodepletion we noticed better in vivo expansion and persistence of P-BCMA-ALLO1. That actually correlated with the antimyeloma activity of this agent compared to the standard lymphodepletion, where overall response rate (ORR) was only 21%. We noticed that in arms with enhanced lymphodepletion, such as arms A, B, and C, we noticed increasing ORR of 42%, 72%, and 91% respectively. Arm C, which is using cyclophosphamide 750 mg/m2/day is currently selected for phase 1b expansion, where we saw a 91% ORR, even in heavily pretreated MM patients.","_key":"9692c4614c03"}],"_type":"block","style":"normal","_key":"4909e183ce60"},{"children":[{"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":"span","marks":["strong"]}],"_type":"block","style":"h3","_key":"56f82d62e8d9","markDefs":[]},{"markDefs":[],"children":[{"text":"This is still a very early study. I think we definitely need a larger patient cohort with longer follow-up to accurately understand the safety and more importantly the efficacy of this agent. However, in current era of bispecific antibodies and CAR T-cell therapies, there is still a huge unmet need of patients who have failed other bispecific antibody therapies, or patients who have failed or are not eligible for standard-of-care CAR T-cell therapy such as cilta-cel or ide-cel. Among those patients, I think this treatment can offer a good option if it turns out to be good enough and safe enough. Given the fact that this is an allogenic product, it's readily available. There is no leukapheresis needed. There is no waiting for manufacturing, which is a huge issue in autologous BCMA-targeted CAR T-cell therapy, which is takes anywhere from 4 to 6 weeks from the time of leukapheresis to actual cell infusion. Many of our high-risk patients are not able to wait that long, and they end up requiring bridging therapy, which none of the patients on this trial needed because P-BCMA-ALLO1 was available right away. So I think that this agent has potential to address that unmet need in high-risk, aggressively-progressing, extramedullary MM patients or high-risk MM patients who had failed other standard-of-care myeloma therapies.","_key":"e7cf7c6a0dfe0","_type":"span","marks":[]}],"_type":"block","style":"normal","_key":"1ba97d171ccb"},{"markDefs":[],"children":[{"_type":"span","marks":["strong"],"text":"Have there been any challenges in the study and/or are there any areas of interest for further research?","_key":"9d03c4e4edf4"}],"_type":"block","style":"h3","_key":"c048425fff7d"},{"style":"normal","_key":"6a65969d84d5","markDefs":[],"children":[{"_type":"span","marks":[],"text":"In terms of challenges, of course very early on, like I said, we realized that the standard lymphodepletion was just not good enough to have a better expansion, persistence, and efficacy of this drug. But we appear to have overcome that now. We actually did a very systemic dose escalation of the Cytoxan dose along with the cell dose expansion also, and we learned that there is a sweet spot—not the highest lymphodepletion, but we picked kind of the intermediate dose lymphodepletion, which is cyclophosphamide, cyclophosphamide 750 mg/m2/day, where we saw kind of the right balance of safety and efficacy. That's what we are currently expanding, both in patients who had prior BCMA-targeted therapies and patients who had no prior BCMA-targeted therapies. We are also exploring multiple cell dose infusions to improve the durability of the responses.","_key":"c7534f3a20780"}],"_type":"block"},{"markDefs":[],"children":[{"_type":"span","marks":["em","strong"],"text":"This transcript has been edited for clarity.","_key":"090e000f56cf0"}],"_type":"block","style":"normal","_key":"1e27ae9fbd54"},{"children":[{"_key":"cb653088ad930","_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"}],"_type":"block","style":"h6","_key":"3a5ccb5a7eed","markDefs":[]},{"markDefs":[],"children":[{"text":"","_key":"08d1e30b91e50","_type":"span","marks":[]}],"_type":"block","style":"normal","_key":"68c7b9db0d9c"},{"_type":"block","style":"normal","_key":"08507dbc0e28","markDefs":[],"children":[{"_type":"span","marks":[],"text":"","_key":"271de6c127c10"}]}],"authors":[{"displayName":"Bhagirathbhai Dholaria, MBBS","url":"bhagirathbhai-dholaria-mbbs"},{"displayName":"Noah Stansfield","url":"noah-stansfield"}],"title":"Evaluating Allogeneic CAR-T P-BCMA-ALLO1 in R/R Multiple Myeloma","articleType":"Commentary","authorMapping":[{"displayName":"Bhagirathbhai Dholaria, MBBS","_createdAt":"2023-02-21T02:13:07Z","_type":"author","_id":"c38a63ef-9e1c-49f8-b233-756591d914ed","_updatedAt":"2023-02-21T02:13:07Z","url":{"current":"bhagirathbhai-dholaria-mbbs","_type":"slug"},"firstName":"Bhagirathbhai","lastName":"Dholaria, MBBS","_rev":"2bK8HvKx77F17Qx7cczZjG"},{"_id":"f9e9001f-7a9c-46a2-9e9c-318ee31c1923","firstName":"Noah","_createdAt":"2022-06-14T20:44:15Z","_rev":"yD3aK4wI9vtjJazfpEZYwZ","_type":"author","lastName":"Stansfield","displayName":"Noah Stansfield","_updatedAt":"2022-06-14T20:44:15Z","url":{"current":"noah-stansfield","_type":"slug"}}],"ExcludeFromPubMedXML":false,"taxonomyMapping":[{"identifier":"neurology","perKeywordMapping":["Cardiology","Neurology","Health Equity Diversity \u0026 Inclusion"],"parent":{"identifier":"topic","_rev":"60drdzXgFuuRxJsEyE6his","_type":"taxonomy","parent":null,"name":"Topic","_id":"8ea01556-869e-480b-83a7-58eca140415e","_createdAt":"2020-12-08T16:06:37Z","_updatedAt":"2023-01-19T14:52:23Z","isMainTopic":true},"_rev":"USRhz6Ofj9athfco75TZJx","name":"Neurology","_updatedAt":"2023-11-27T17:07:43Z","summary":[{"style":"normal","_key":"5869c0483365","markDefs":[],"children":[{"_type":"span","marks":[],"text":"The ","_key":"8bdd6ce5e667"},{"_type":"span","marks":["em"],"text":"CGTLive","_key":"d7e39fed968f"},{"_type":"span","marks":["superscript"],"text":"™","_key":"5761d91435ea"},{"_type":"span","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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One of these is immunogenicity, which prevents redosing if the initial dose is insufficient or the effect of the therapy wears off over time. ","_key":"6c82da9de699"}],"_type":"block"},{"style":"normal","_key":"b040ef347cf4","markDefs":[{"_type":"link","href":"https://www.cgtlive.com/conferences/american-society-for-gene-cell-therapy-asgct-","_key":"7933d246685f"}],"children":[{"_type":"span","marks":[],"text":"Ring Therapeutics is seeking to use anellovectors as potential alternatives to AAV vectors for gene therapy in order to address this issue. At ","_key":"6575c7e9699c0"},{"_type":"span","marks":["7933d246685f"],"text":"the American Society of Gene \u0026 Cell Therapy (ASGCT) 27th Annual Meeting","_key":"a8481a3c4c4e"},{"text":", held May 7 to 10, 2024, the company gave several presentations on its research. At the conference, ","_key":"b9139a2bc1f2","_type":"span","marks":[]},{"_type":"span","marks":["em"],"text":"CGTLive®","_key":"81b0f6476f0f"},{"_type":"span","marks":[],"text":" interviewed Chris Wright, MD, PhD, the chief medical officer and head of translational research at Ring Therapeutics, to learn more.","_key":"c2e68d297f3d"}],"_type":"block"},{"markDefs":[],"children":[{"_type":"span","marks":["strong","em"],"text":"CGTLive:","_key":"8877a82e07af0"},{"marks":["strong"],"text":" Can you tell us about Ring's platform and its potential advantages?","_key":"24a5415d083b","_type":"span"}],"_type":"block","style":"h3","_key":"95131aaf3f41"},{"style":"normal","_key":"2078bc759d0e","markDefs":[],"children":[{"text":"Chris Wright, MD, PhD: ","_key":"d88f4bbeb696","_type":"span","marks":["strong"]},{"text":"There's a lot of great potential for gene therapy. You're here at the gene therapy conference, there's a lot of people, and everybody is excited about making a difference for patients that really need therapies. One of the challenges with current gene therapy, while it seems to be working in some areas, is that there's problems with it being immunogenic. You get safety problems because people recognize it as foreign, and then they have a reaction. That can lead to problems from the safety perspective, with side effects and the like. It also makes it challenging to give it to everybody because many people already have antibodies to the viruses that are used for gene therapy for the most part these days. So ideally, you'd like to find a virus that wouldn't have those properties, that you could give maybe even repeat doses of if you need to, and that wouldn't cause any side effects and could be given to a broader population of people. That's one of the main areas that Ring is working on. ","_key":"51d159af8971","_type":"span","marks":[]}],"_type":"block"},{"markDefs":[],"children":[{"_type":"span","marks":[],"text":"In order to try to understand, the viruses that might be suitable—that maybe have better properties—the company thought that maybe the best virus around is actually inside of us. What I mean by that is that it's known that the body has a bunch of viruses that just kind of hang out there and aren't doing anything harmful. People have heard about the microbiome, which is all the kind of bacteria in your body, but there's also a human virome, which are all the different viruses that are in your body. We were looking in the body for different viruses that are persisting there, but not causing any problems. We did find a number of viruses, and those are the anelloviruses. Anelloviruses actually make up about 80% of all the viruses in a person's body. And it turns out that that they've been there throughout all of the person's life, pretty much shortly after birth. So, they persist for a long period of time, and they don't appear to cause any problems, they don't induce an immune reaction, and they don't seem to have any associations with diseases. So, we thought that could be a great virus to use to try to deliver gene therapy because it should be a safe approach.","_key":"71453e0b953f"}],"_type":"block","style":"normal","_key":"f84d8e548dbf"},{"markDefs":[],"children":[{"_type":"span","marks":["strong"],"text":"Can you give an overview of Ring's presentations at the conference?","_key":"eaebcdb15127"}],"_type":"block","style":"h3","_key":"ccfa93d2e0f4"},{"_type":"block","style":"normal","_key":"30dbb818c4ba","markDefs":[],"children":[{"_type":"span","marks":[],"text":"We're fortunate to have 3 different abstracts accepted. We have 2 abstracts that were posters, and then I was able to have a talk that I'll be giving tomorrow. The posters are around some of the science. One thing about viral capsids is that sometimes they can only contain a certain amount of therapy. The idea is the more therapy you can get inside of it, the bigger the gene [can be], the more likely you're going to be able to treat many different diseases. We had one poster that showed that we could increase the size of the therapeutic gene. That was kind of a great advancement... The other poster is looking at how we're able to transduce different tissues, particularly in nonhuman primates (NHPs), in the eye. Getting to an NHP study is one of those studies that's very close to getting into humans eventually. So if you can take your new gene therapy and show that you can safely dose NHPs, that gives you a higher level of confidence that you can get into a human study sooner rather than later. That poster showed when we injected in the eye, we could get expression there. It was also the first time we demonstrated that you could put a gene in that is a therapeutic gene, and that you can have expression of that, as well. We showed that DNA and RNA was expressed in the NHP eyes, and that's sort of along the lines of the therapies that we want to develop. That was exciting to see. Redosability is also very important because right now, gene therapy is one-and-done. If you give it once, you can't ever give it again. And so what happens is sometimes maybe you underdose, and the person doesn't get the adequate response that you'd like them to have, but you can't do anything about that, or you might want to increase the duration of an effect—so sometimes gene therapies wear off, and then if you can't redose, then you're also kind of stuck. ","_key":"891cda568f180"}]},{"markDefs":[],"children":[{"marks":[],"text":"This gives us the possibility to redose or to dose multiple times up front to get to the right therapeutic levels... It turns out that the outside of the capsid has pieces that are very variable, and so that means they're not really easily recognized by the immune system, and the parts of the capsid that are less variable are kind of hidden on the inside, so they can't be accessed by antibodies. So it's probably one of the reasons why it's immune silent. If you look at AAV, it actually has all of the more constant and nonvariable regions of the protein on the outside of the capsid. That kind of explains a little bit why it might be immune reactive, and why anello vectors might not be... This is all foundational knowledge that Ring discovered along with collaborators; in this case, it was at Johns Hopkins that collaborated to understand if there's any antibodies already in the body that recognize proteins on the capsid of the anello vector. If you look at all these capsid proteins and you try to find antibodies in human serum, you actually really almost find none. But if you look at other human viruses like HIV or HSV or even AAV, you see a lot of reactivity. That sort of also proved the point that anello viruses are sort of immune silent because they don't produce antibodies in people... ","_key":"44263b11f2e4","_type":"span"}],"_type":"block","style":"normal","_key":"794b85a61169"},{"style":"normal","_key":"7df6b52dcc12","markDefs":[],"children":[{"_key":"2dae13d71e9f","_type":"span","marks":[],"text":"As I mentioned before, redosing is important because gene therapies can wear off, or you might not get the dose right the first time and you can't give it again. We did some experiments where we showed we could inject an anello vector that produced a marker that we could measure in the animals, and we showed that when you redose it, you actually get increased levels of the DNA, and when you redose AAV you actually got decreased levels. So that kind of speaks to the fact that with AAV, you're probably inducing an immune reaction, which means when you try to dose it a second time, you're not getting quite as much of the virus in the system, whereas with the anello vectors we saw actually a little bit of an increase in the liver in particular, and that's one of the organs where gene therapies tend to wear off. "}],"_type":"block"},{"style":"normal","_key":"b9fc749651af","markDefs":[],"children":[{"marks":[],"text":"We also did another study looking at redosing in the eye in NHPs and in that experiment, we actually used a aflibbercept, which is our disease gene. The first one [we did] was kind of a marker gene, just like a proof-of-concept to show that we could do redosing. Then the second study was in NHPs, where we were injecting in the eye with a therapeutic payload to see whether we could redose there. We were able to show that we can actually redose there, which was really nice. If you give 2 doses, you get a lot higher DNA and RNA. In particular with eye diseases, some of the current gene therapies that are AAV-based sort of are starting to run out of steam. Their efficacy goes down after about 6 to 12 months. That's not a great situation to be in if you can't redose. So in our case, we could give a gene therapy and if the efficacy wears off, we could treat again to prolong the efficacy, and help patients for longer.","_key":"10aee32cd288","_type":"span"}],"_type":"block"},{"style":"normal","_key":"1e27ae9fbd54","markDefs":[],"children":[{"_type":"span","marks":["em","strong"],"text":"This transcript has been edited for clarity.","_key":"090e000f56cf0"}],"_type":"block"},{"_type":"block","style":"normal","_key":"a6163508246b","markDefs":[{"href":"https://www.cgtlive.com/conferences/american-society-for-gene-cell-therapy-asgct-","_key":"135dc348e511","_type":"link"}],"children":[{"_type":"span","marks":["135dc348e511","strong","em"],"text":"Click here to view more coverage of the 2024 ASGCT Annual Meeting.","_key":"297856ce074e0"}]},{"_type":"block","style":"h6","_key":"5c86d428481e","markDefs":[],"children":[{"_type":"span","marks":[],"text":"REFERENCES\n1. Bounoutas GS, Pozsgai R, Gold I, et al. Anellovectors, a Gene Delivery Platform Based on Commensal Human Anelloviruses, Have the Potential to Evade the Immune System and Deliver DNA Payloads to a Broad Range of Tissues in a Redosable Manner. Presented at: ASGCT 27th Annual Meeting, May 7-10; Baltimore, Maryland. 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