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md:justify-between"><div class="max-w-full"><time class="tex-sm " dateTime="2024-10-24T15:00:23.918">October 24, 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/sharon-hesterlee-phd">Sharon Hesterlee, PhD</a></span><span class="text-md "><span class=""><br/></span><a class="mr-1 text-author hover:text-primary" href="/authors/isabella-ciccone-mph">Isabella Ciccone, MPH</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="Sharon Hesterlee, PhD, on MDA 2025 as a Mixer for Strategies and Collaboration" aria-label="facebook" class="react-share__ShareButton" 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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=%27217.89310344827584%27%20height=%2751%27/%3e"/></span><img src="data:image/gif;base64,R0lGODlhAQABAIAAAAAAAP///yH5BAEAAAAALAAAAAABAAEAAAIBRAA7" decoding="async" data-nimg="intrinsic" class="shrink-0" 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%"/><noscript><img srcSet="/_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fgenetherapy%2F36d33c9aadb61ef83a8583bb7d1006db4d52083f-1239x290.png%3Ffit%3Dcrop%26auto%3Dformat&w=256&q=75 1x, /_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fgenetherapy%2F36d33c9aadb61ef83a8583bb7d1006db4d52083f-1239x290.png%3Ffit%3Dcrop%26auto%3Dformat&w=640&q=75 2x" src="/_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fgenetherapy%2F36d33c9aadb61ef83a8583bb7d1006db4d52083f-1239x290.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%" class="shrink-0" loading="lazy"/></noscript></span></div></a></div></div></div><p class="py-2 mb-2 text-sm italic text-gray-600">The chief research officer of the Muscular Dystrophy Association discussed the upcoming MDA Clinical and Scientific Conference, which will be held March 16-19, in Dallas, Texas.</p><div class="py-2"><div class="blockText_blockContent__TbCXh"><p class="pb-2"><strong><em>This interview originally appeared on our sister site, <a target="_blank" href="https://www.neurologylive.com/view/melting-pot-strategies-collaboration-2025-mda-conference-sharon-hesterlee">NeurologyLive®.</a></em></strong></p><div class="relative"><div class="brightcove-fluid" autoplay=""></div></div><blockquote><strong><em>"Our goal is really about community—bringing together all these different facets, from clinical care to research, to create incredible momentum."</em></strong></blockquote><p class="pb-2"></p><p class="pb-2">The Muscular Dystrophy Association (MDA) will hold the annual MDA Clinical and Scientific Conference for 2025 from March 16 to 19 in Dallas, Texas. The 4-day event will gather scientific and medical professionals, patients, and advocacy groups to discuss and share the latest advances in the field of neuromuscular disorders. Research pertaining to the range of neuromuscular disorders, from amyotrophic lateral sclerosis (ALS), to Duchenne muscular dystrophy, to spinal muscular atrophy, to limb-girdle muscular dystrophy, to myasthenia gravis, to Charcot-Marie-Tooth disease, and more will be represented at the meeting.</p><p class="pb-2">Furthermore, there will be session tracks featured on topics including strategies for regenerating muscle and nerve tissue, new insights into disease mechanisms, clinical experience in gene therapy, access and reimbursement, and clinical trial readouts. Notably, MDA's 2024 Clinical and Scientific Conference was the largest global gathering of the neuromuscular disease community, with 1492 in-person and 567 virtual attendees from over 30 countries. Those who are registered with MDA may partake in the virtual meeting in 2025 at no-cost or may register in-person at the patient/caregiver rate until allotted spots are filled.</p><p class="pb-2">In anticipation of the upcoming meeting, <em>CGTLive®</em>'s sister site, <em>NeurologyLive®</em>, reached out to Sharon Hesterlee, PhD, the chief research officer of the Muscular Dystrophy Association, to discuss the primary focus areas of the 2025 conference, particularly in muscle regeneration and ALS research. Hesterlee also spoke about how the conference addresses the challenges of workforce diversity and training in neuromuscular specialties. She also talked about the roles of advocacy groups and allied health professionals in shaping the discussions at the upcoming meeting.</p><p class="pb-2"><strong><em><a rel="nofollow noreferrer noopener" target="_blank" href="https://www.mdaconference.org/">Click here to register for the upcoming 2025 MDA Conference</a>.</em></strong></p><h6 class="text-sm pb-4 pt-2">REFERENCE<br/>1. Registration Now Open for the 2025 MDA Clinical & Scientific Conference Featuring the Latest Innovations in Neuromuscular Disease Research and Care with Global Leaders, March 16-19 in Dallas, TX. News Release. MDA. July 30, 2024. Accessed October 24, 2024. https://www.mda.org/press-releases/2024/registration-now-open-for-the-2025-mda-clinical-and-scientific-conference-featuring-the-latest-innovations-in-neuromuscular-disease-research-and-care-with-global-leaders-march-16-19-in-dallas-tx</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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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 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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/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="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/cgtlive-weekly-rewind-november-22-2024?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%" 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/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 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-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, /_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, /_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fgenetherapy%2F9b1b40bc81fc00eeb21179fe80ec2a22d6af7e25-800x500.jpg%3Ffit%3Dcrop%26auto%3Dformat&w=1920&q=75 1920w, /_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fgenetherapy%2F9b1b40bc81fc00eeb21179fe80ec2a22d6af7e25-800x500.jpg%3Ffit%3Dcrop%26auto%3Dformat&w=2048&q=75 2048w, /_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fgenetherapy%2F9b1b40bc81fc00eeb21179fe80ec2a22d6af7e25-800x500.jpg%3Ffit%3Dcrop%26auto%3Dformat&w=3840&q=75 3840w" src="/_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fgenetherapy%2F9b1b40bc81fc00eeb21179fe80ec2a22d6af7e25-800x500.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/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/atamyo-therapeutics-gene-therapy-ata-200-cleared-trial-lgmd-type-2c-r5?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="Sophie Olivier, MD, the chief medical officer of Atamyo" title="Sophie Olivier, MD, the chief medical officer of Atamyo" 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="Sophie Olivier, MD, the chief medical officer of Atamyo" title="Sophie Olivier, MD, the chief medical officer of Atamyo" sizes="100vw" srcSet="/_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fgenetherapy%2F0ad104c0cc1fd9a47e3e9a1b13dafff2d2418ed5-300x300.jpg%3Ffit%3Dcrop%26auto%3Dformat&w=640&q=75 640w, /_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fgenetherapy%2F0ad104c0cc1fd9a47e3e9a1b13dafff2d2418ed5-300x300.jpg%3Ffit%3Dcrop%26auto%3Dformat&w=750&q=75 750w, /_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fgenetherapy%2F0ad104c0cc1fd9a47e3e9a1b13dafff2d2418ed5-300x300.jpg%3Ffit%3Dcrop%26auto%3Dformat&w=828&q=75 828w, /_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fgenetherapy%2F0ad104c0cc1fd9a47e3e9a1b13dafff2d2418ed5-300x300.jpg%3Ffit%3Dcrop%26auto%3Dformat&w=1080&q=75 1080w, /_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fgenetherapy%2F0ad104c0cc1fd9a47e3e9a1b13dafff2d2418ed5-300x300.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/atamyo-therapeutics-gene-therapy-ata-200-cleared-trial-lgmd-type-2c-r5?utm_source=www.cgtlive.com&utm_medium=relatedContent">Atamyo Therapeutics' Gene Therapy ATA-200 Cleared for US Trial in LGMD Type 2C/R5</a></p><div class=" pl-4"><a class="text-sm text-sky-800" href="/authors/noah-stansfield">Noah Stansfield</a></div><a href="/view/atamyo-therapeutics-gene-therapy-ata-200-cleared-trial-lgmd-type-2c-r5?utm_source=www.cgtlive.com&utm_medium=relatedContent"><span class="text-sm text-gray-500 pl-4">November 19th 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 addition to the IND clearance, the FDA also granted ATA-200 orphan drug designation.</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 rgb(194, 194, 194, 1)"><div class="w-full md:w-auto 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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, 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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/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" 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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 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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, /_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fgenetherapy%2F1feeab70bc7d00036bee4f6bc891982276d46bc0-500x500.jpg%3Ffit%3Dcrop%26auto%3Dformat&w=750&q=75 750w, /_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fgenetherapy%2F1feeab70bc7d00036bee4f6bc891982276d46bc0-500x500.jpg%3Ffit%3Dcrop%26auto%3Dformat&w=828&q=75 828w, /_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fgenetherapy%2F1feeab70bc7d00036bee4f6bc891982276d46bc0-500x500.jpg%3Ffit%3Dcrop%26auto%3Dformat&w=1080&q=75 1080w, /_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fgenetherapy%2F1feeab70bc7d00036bee4f6bc891982276d46bc0-500x500.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/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 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-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, /_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, /_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fgenetherapy%2F9b1b40bc81fc00eeb21179fe80ec2a22d6af7e25-800x500.jpg%3Ffit%3Dcrop%26auto%3Dformat&w=1920&q=75 1920w, /_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fgenetherapy%2F9b1b40bc81fc00eeb21179fe80ec2a22d6af7e25-800x500.jpg%3Ffit%3Dcrop%26auto%3Dformat&w=2048&q=75 2048w, /_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fgenetherapy%2F9b1b40bc81fc00eeb21179fe80ec2a22d6af7e25-800x500.jpg%3Ffit%3Dcrop%26auto%3Dformat&w=3840&q=75 3840w" src="/_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fgenetherapy%2F9b1b40bc81fc00eeb21179fe80ec2a22d6af7e25-800x500.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/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/atamyo-therapeutics-gene-therapy-ata-200-cleared-trial-lgmd-type-2c-r5?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="Sophie Olivier, MD, the chief medical officer of Atamyo" title="Sophie Olivier, MD, the chief medical officer of Atamyo" 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="Sophie Olivier, MD, the chief medical officer of Atamyo" title="Sophie Olivier, MD, the chief medical officer of Atamyo" sizes="100vw" srcSet="/_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fgenetherapy%2F0ad104c0cc1fd9a47e3e9a1b13dafff2d2418ed5-300x300.jpg%3Ffit%3Dcrop%26auto%3Dformat&w=640&q=75 640w, /_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fgenetherapy%2F0ad104c0cc1fd9a47e3e9a1b13dafff2d2418ed5-300x300.jpg%3Ffit%3Dcrop%26auto%3Dformat&w=750&q=75 750w, /_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fgenetherapy%2F0ad104c0cc1fd9a47e3e9a1b13dafff2d2418ed5-300x300.jpg%3Ffit%3Dcrop%26auto%3Dformat&w=828&q=75 828w, /_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fgenetherapy%2F0ad104c0cc1fd9a47e3e9a1b13dafff2d2418ed5-300x300.jpg%3Ffit%3Dcrop%26auto%3Dformat&w=1080&q=75 1080w, /_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fgenetherapy%2F0ad104c0cc1fd9a47e3e9a1b13dafff2d2418ed5-300x300.jpg%3Ffit%3Dcrop%26auto%3Dformat&w=1200&q=75 1200w, /_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fgenetherapy%2F0ad104c0cc1fd9a47e3e9a1b13dafff2d2418ed5-300x300.jpg%3Ffit%3Dcrop%26auto%3Dformat&w=1920&q=75 1920w, /_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fgenetherapy%2F0ad104c0cc1fd9a47e3e9a1b13dafff2d2418ed5-300x300.jpg%3Ffit%3Dcrop%26auto%3Dformat&w=2048&q=75 2048w, /_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fgenetherapy%2F0ad104c0cc1fd9a47e3e9a1b13dafff2d2418ed5-300x300.jpg%3Ffit%3Dcrop%26auto%3Dformat&w=3840&q=75 3840w" src="/_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fgenetherapy%2F0ad104c0cc1fd9a47e3e9a1b13dafff2d2418ed5-300x300.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/atamyo-therapeutics-gene-therapy-ata-200-cleared-trial-lgmd-type-2c-r5?utm_source=www.cgtlive.com&utm_medium=relatedContent">Atamyo Therapeutics' Gene Therapy ATA-200 Cleared for US Trial in LGMD Type 2C/R5</a></p><div class=" pl-4"><a class="text-sm text-sky-800" href="/authors/noah-stansfield">Noah Stansfield</a></div><a href="/view/atamyo-therapeutics-gene-therapy-ata-200-cleared-trial-lgmd-type-2c-r5?utm_source=www.cgtlive.com&utm_medium=relatedContent"><span class="text-sm text-gray-500 pl-4">November 19th 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 addition to the IND clearance, the FDA also granted ATA-200 orphan drug designation.</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":"Sharon Hesterlee, PhD, on MDA 2025 as a Mixer for Strategies and Collaboration","datePublished":"2024-10-24T15:00:23.918Z","dateModified":"2024-10-25T15:53:39Z","inLanguage":"en-US","image":"https://cdn.sanity.io/images/0vv8moc6/genetherapy/42597e8b37c8de52ea7e6ce6bea50f84b2ec06de-1280x720.png?fit=crop&auto=format","mainEntityOfPage":{"@type":"WebPage","@id":"https://www.cgtlive.com/view/hesterlee-mda-2025-mixer-strategies-collaboration"},"publisher":{"@type":"Organization","name":"CGTlive™","logo":{"@type":"ImageObject","url":"https://www.cgtlive.com/CGT_Color_NoTagline.png"}},"keywords":"mda,gene therapy,muscular dystrophy association,Sharon Hesterlee,Duchenne muscular dystrophy,Clinical & Scientific Conference,conference preview,neuromuscular disorders,neuromuscular disease,ALS,cell therapy","articleBody":"This interview originally appeared on our sister site, NeurologyLive®.\n\n\n\n\"Our goal is really about community—bringing together all these different facets, from clinical care to research, to create incredible momentum.\"\n\n\n\nThe Muscular Dystrophy Association (MDA) will hold the annual MDA Clinical and Scientific Conference for 2025 from March 16 to 19 in Dallas, Texas. The 4-day event will gather scientific and medical professionals, patients, and advocacy groups to discuss and share the latest advances in the field of neuromuscular disorders. Research pertaining to the range of neuromuscular disorders, from amyotrophic lateral sclerosis (ALS), to Duchenne muscular dystrophy, to spinal muscular atrophy, to limb-girdle muscular dystrophy, to myasthenia gravis, to Charcot-Marie-Tooth disease, and more will be represented at the meeting.\n\nFurthermore, there will be session tracks featured on topics including strategies for regenerating muscle and nerve tissue, new insights into disease mechanisms, clinical experience in gene therapy, access and reimbursement, and clinical trial readouts. Notably, MDA's 2024 Clinical and Scientific Conference was the largest global gathering of the neuromuscular disease community, with 1492 in-person and 567 virtual attendees from over 30 countries. Those who are registered with MDA may partake in the virtual meeting in 2025 at no-cost or may register in-person at the patient/caregiver rate until allotted spots are filled.\n\nIn anticipation of the upcoming meeting, CGTLive®'s sister site, NeurologyLive®, reached out to Sharon Hesterlee, PhD, the chief research officer of the Muscular Dystrophy Association, to discuss the primary focus areas of the 2025 conference, particularly in muscle regeneration and ALS research. Hesterlee also spoke about how the conference addresses the challenges of workforce diversity and training in neuromuscular specialties. She also talked about the roles of advocacy groups and allied health professionals in shaping the discussions at the upcoming meeting.\n\nClick here to register for the upcoming 2025 MDA Conference.\n\nREFERENCE\n1. Registration Now Open for the 2025 MDA Clinical & Scientific Conference Featuring the Latest Innovations in Neuromuscular Disease Research and Care with Global Leaders, March 16-19 in Dallas, TX. News Release. MDA. July 30, 2024. Accessed October 24, 2024. https://www.mda.org/press-releases/2024/registration-now-open-for-the-2025-mda-clinical-and-scientific-conference-featuring-the-latest-innovations-in-neuromuscular-disease-research-and-care-with-global-leaders-march-16-19-in-dallas-tx","description":"The chief research officer of the Muscular Dystrophy Association discussed the upcoming MDA Clinical and Scientific Conference, which will be held March 16-19, in Dallas, Texas.","author":[{"@type":"Person","name":"Sharon Hesterlee, PhD"},{"@type":"Person","name":"Isabella Ciccone, MPH"}]}</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" 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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":[]},{"_key":"c048425fff7d","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"},{"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","style":"normal","_key":"633ba12c521e"},{"markDefs":[],"children":[{"_type":"span","marks":["em","strong"],"text":"This transcript has been edited for clarity.","_key":"090e000f56cf0"}],"_type":"block","style":"normal","_key":"1e27ae9fbd54"},{"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":"normal","_key":"bcb4af2819f3"},{"_type":"block","style":"h6","_key":"3569dec1f866","markDefs":[],"children":[{"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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In addition, over 11 million Americans currently act as caregivers for people with AD, with estimates of time spent providing unpaid care totaling over 18.4 billion hours.","_key":"6cc1bc464ff0","_type":"span"}],"_type":"block","style":"normal","_key":"e4051d92f3c8"},{"markDefs":[],"children":[{"_type":"span","marks":[],"text":"Cell therapy and gene therapy constitute an emerging area of interest for the potential treatment of AD, and a number of companies and academic institutions are now pursuing the development of a range of these advanced therapeutics for the disease. In honor of Alzheimer Disease Awareness Month, observed annually in November, ","_key":"3f6c7c592a25"},{"_type":"span","marks":["em"],"text":"CGTLive","_key":"a303a6e9259c"},{"_key":"3183d2fd72cd","_type":"span","marks":[],"text":"® is taking a look back at some of the progress that has been made for cell therapy and gene therapy candidates in AD over the past year. Click the \"READ MORE\" buttons for more details and information about each item."}],"_type":"block","style":"normal","_key":"7c78a33deed3"},{"_type":"block","style":"h3","_key":"37eff1557922","markDefs":[],"children":[{"text":"Lexeo Therapeutics' Gene Therapy LX1001 Increases APOE2 Expression in Patients With Alzheimer Disease","_key":"dbe8ca881f0b0","_type":"span","marks":["strong"]}]},{"children":[{"_type":"span","marks":["em"],"text":"November 4, 2024","_key":"d8dace4edd22"},{"_key":"e2c4f91cde60","_type":"span","marks":[],"text":" — Lexeo Therapeutics' LX1001, an investigational adeno-associated virus (AAV) vector-based gene therapy, has demonstrated a dose- and time-dependent impact on apolipoprotein (APOE2) expression in patients with "},{"_type":"span","marks":["em"],"text":"APOE4","_key":"7eb26105ac2c"},{"_type":"span","marks":[],"text":" homozygote AD in interim data from a phase 1/2, open-label clinical trial (NCT03634007). In addition, a decrease in several cerebrospinal fluid (CSF) AD tau biomarkers and changes on tau PET were reported.","_key":"64d257c5e9a1"}],"_type":"block","style":"normal","_key":"fda0427c9908","markDefs":[]},{"_type":"block","style":"normal","_key":"c63c6ea0a74f","markDefs":[{"_type":"link","href":"https://www.neurologylive.com/conferences/ctad","_key":"039663a5b30e"}],"children":[{"text":"The data were announced in a late-breaking presentation at the ","_key":"436f441789640","_type":"span","marks":[]},{"_type":"span","marks":["039663a5b30e"],"text":"2024 Clinical Trials on Alzheimer’s Disease (CTAD) conference","_key":"436f441789641"},{"_type":"span","marks":[],"text":", held October 29 to November 1, in Madrid, Spain. Fifteen patients were dosed in the trial across 3 ascending single-dose cohorts: 1.4x10","_key":"436f441789642"},{"_key":"dfa05c48b3f2","_type":"span","marks":["superscript"],"text":"10"},{"_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"},{"text":" gc/ml (C3; n = 3). A single fixed-dose cohort was also included, treating patients at a dose of 1.4x10","_key":"7e2e46ca5fb9","_type":"span","marks":[]},{"marks":["superscript"],"text":"14","_key":"a152de76ff56","_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"},{"_key":"f9ae015331ec","_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":"16a88ed3f187","markDefs":[],"children":[{"marks":[],"text":"In terms of safety, the gene therapy was deemed safe and well-tolerated, with no cases of amyloid-related imaging abnormalities (ARIA) reported. Expression of APOE2 in the CSF was observed in all of the patients, with a dose- and time-dependent increase in APOE2e4 expression noted. A decrease in CSF t-tau and phosphorylated-tau181 in 9 of the 13 participants was observed, although no directional trend in CSF amyloid-ß42/40 or amyloid PET was recorded. Notably, a decrease in CSF p-tau217 and p-tau231 was also seen in the 9 aforementioned patients.","_key":"4f4b0880d2ac0","_type":"span"}],"_type":"block","style":"normal"},{"asset":{"_ref":"image-10cc6e8348d63bae9563ddb8e47f06ca6dee3512-150x75-png","_type":"reference"},"blank":true,"disableTextWrap":false,"disableLightBox":true,"_type":"figure","link":"https://www.cgtlive.com/view/lexeo-therapeutics-gene-therapy-lx1001-increases-apoe2-expression-patients-alzheimer-disease","_key":"a5ca2a3ff40d"},{"children":[{"_type":"span","marks":[],"text":"","_key":"9a1fec425cdd"}],"_type":"block","style":"normal","_key":"b82c94511fbe","markDefs":[]},{"children":[{"_type":"span","marks":["strong"],"text":"Lomecel-B Safe, Seems Promising for Alzheimer Disease Measures","_key":"8967ac5a9bac0"}],"_type":"block","style":"h3","_key":"c5b3670604e5","markDefs":[]},{"markDefs":[],"children":[{"marks":["em"],"text":"July 29, 2024","_key":"92a2c1e85933","_type":"span"},{"text":" — Lomecel-B was well-tolerated in patients with mild AD receiving up to 4 infusions of the cell therapy and demonstrated some improvements in cognitive function and other disease measures, according to new data from the phase 2a CLEAR MIND trial (NCT05233774).","_key":"eec03c4f81b7","_type":"span","marks":[]}],"_type":"block","style":"normal","_key":"44c0a1af6cb9"},{"children":[{"_type":"span","marks":[],"text":"These data were presented at the 2024 Alzheimer’s Association International Conference (AAIC), held July 28-August 2nd in Philadelphia, Pennsylvania, by Brian G. 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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"}]},{"_key":"7ea0469c3e26","markDefs":[],"children":[{"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. 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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":"block","style":"normal","_key":"78b21ae8deb3","markDefs":[]},{"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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Dholaria discussed what makes P-BCMA-ALLO1 unique and went over the key safety and efficacy results presented and their potential implications."}],"_type":"block"},{"style":"h3","_key":"95131aaf3f41","markDefs":[],"children":[{"_type":"span","marks":["strong","em"],"text":"CGTLive:","_key":"8877a82e07af0"},{"_type":"span","marks":["strong"],"text":" Can you give some background context about P-BCMA-ALLO1 and what it is?","_key":"24a5415d083b"}],"_type":"block"},{"_type":"block","style":"normal","_key":"2078bc759d0e","markDefs":[],"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"}]},{"style":"h3","_key":"a78fffcdefb4","markDefs":[],"children":[{"_type":"span","marks":["strong"],"text":"Can you give an overview of the recently announced data from the phase 1/1b clinical trial?","_key":"3a13757030a2"}],"_type":"block"},{"_type":"block","style":"normal","_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":[{"marks":[],"text":"Overall, 100% of the intention-to-treat patients actually ended up receiving the P-BCMA-ALLO1 infusion. The primary end point of this trial was the safety of P-BCMA-ALLO1. Looking at the safety aspect of the trial results, no graft versus host disease was reported. It is important, given that this is an off-the-shelf allogeneic T-cell being infused in the recipient. The risk of cytokine release syndrome (CRS) was relatively low. Only around 27% of the patients had this event. Most of the events were grade 1 or grade 2. No patient actually had grade 3 or higher CRS or neurotoxicity or immune effector cell-associated neurotoxicity syndrome. There were some infections seen, but the risk of grade 3 or higher infection was relatively low. ","_key":"9efb4802fb65","_type":"span"}]},{"markDefs":[],"children":[{"_type":"span","marks":[],"text":"Looking at the in vivo kinetics, we noticed that with higher lymphodepletion we noticed better in vivo expansion and persistence of P-BCMA-ALLO1. That actually correlated with the antimyeloma activity of this agent compared to the standard lymphodepletion, where overall response rate (ORR) was only 21%. We noticed that in arms with enhanced lymphodepletion, such as arms A, B, and C, we noticed increasing ORR of 42%, 72%, and 91% respectively. Arm C, which is using cyclophosphamide 750 mg/m2/day is currently selected for phase 1b expansion, where we saw a 91% ORR, even in heavily pretreated MM patients.","_key":"9692c4614c03"}],"_type":"block","style":"normal","_key":"4909e183ce60"},{"style":"h3","_key":"56f82d62e8d9","markDefs":[],"children":[{"_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"},{"style":"normal","_key":"1ba97d171ccb","markDefs":[],"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"},{"_type":"block","style":"h3","_key":"c048425fff7d","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"}]},{"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","_key":"6a65969d84d5"},{"style":"normal","_key":"1e27ae9fbd54","markDefs":[],"children":[{"_type":"span","marks":["em","strong"],"text":"This transcript has been edited for clarity.","_key":"090e000f56cf0"}],"_type":"block"},{"_key":"3a5ccb5a7eed","markDefs":[],"children":[{"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":"span","marks":[]}],"_type":"block","style":"h6"},{"_type":"block","style":"normal","_key":"68c7b9db0d9c","markDefs":[],"children":[{"_type":"span","marks":[],"text":"","_key":"08d1e30b91e50"}]},{"children":[{"_type":"span","marks":[],"text":"","_key":"271de6c127c10"}],"_type":"block","style":"normal","_key":"08507dbc0e28","markDefs":[]}],"title":"Evaluating Allogeneic CAR-T P-BCMA-ALLO1 in R/R Multiple Myeloma","_id":"058af847-c612-4ee6-b670-0b2f5ca31e06","authors":[{"displayName":"Bhagirathbhai Dholaria, MBBS","url":"bhagirathbhai-dholaria-mbbs"},{"displayName":"Noah Stansfield","url":"noah-stansfield"}],"_updatedAt":"2024-11-22T10:38:53Z","targeting":{"content_placement":["topic/neurology","conferences","news"],"document_url":["evaluating-allogeneic-car-t-p-bcma-allo1-multiple-myeloma"],"document_group":null,"rootDocumentGroup":[],"issue_url":"","publication_url":""},"relatedArticles":[{"title":"Exploring CAR-T for Autoimmune Disease","url":{"current":"exploring-car-t-autoimmune-disease","_type":"slug"},"thumbnail":{"_type":"mainImage","alt":"Georg Schett, MD, vice president research and chair of internal medicine at the University of Erlangen – Nuremberg","caption":"Georg Schett, MD","asset":{"_ref":"image-6808278d4a529c2b98af3c3297a83a2440fa06a2-300x300-png","_type":"reference"}},"published":"2024-11-25T12:00:57.366Z"},{"title":"Alzheimer Disease Awareness Month 2024: Looking Back at a Year of Progress in Cell and Gene Therapy","url":{"current":"alzheimer-disease-awareness-month-2024-looking-back-year-progress-cell-gene-therapy","_type":"slug"},"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"},{"title":"David Barrett, JD, on Future Trends for Genomic Medicines","url":{"current":"barrett-future-trends-genomic-medicines","_type":"slug"},"thumbnail":{"_type":"mainImage","alt":"David Barrett, JD, the chief executive officer of ASGCT","caption":"David Barrett, JD","asset":{"_ref":"image-73c396ac9ec1c25274e5148785e53cc12aad6577-755x426-png","_type":"reference"}},"published":"2024-11-22T12:00:05.483Z"},{"title":"Evaluating Allogeneic CAR-T P-BCMA-ALLO1 in R/R Multiple Myeloma","url":{"current":"evaluating-allogeneic-car-t-p-bcma-allo1-multiple-myeloma","_type":"slug"},"thumbnail":{"_type":"mainImage","alt":"Bhagirathbhai R. 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The FDA’sIND clearance allows for the geographic expansion of the phase 1b trial, which has previously been ","_key":"a44ba534f14b0"},{"text":"cleared in Italy and France","_key":"a44ba534f14b1","_type":"span","marks":["bf68ffd82333"]},{"text":", to the United States.","_key":"a44ba534f14b2","_type":"span","marks":[]},{"_type":"span","marks":["superscript"],"text":"1,2","_key":"b7e1e9b319dc"},{"_type":"span","marks":[],"text":" Notably, the expansion of the trial to the US is supported by funding from The Dion Foundation for Children with Rare Diseases. ATA-200, which delivers a functional copy of the disease-targeted human ","_key":"107dee127ce4"},{"marks":["em"],"text":"SGCG","_key":"3be97082fcd6","_type":"span"},{"marks":[],"text":" gene, was tolerated and demonstrated the ability to correct symptoms and disease biomarkers in mice models. It will be administered intravenously in the trial.","_key":"2737330ee903","_type":"span"},{"text":"3","_key":"6f70c7e9e05c","_type":"span","marks":["superscript"]}],"_type":"block","style":"normal","_key":"b61f24123382"},{"markDefs":[],"children":[{"_type":"span","marks":[],"text":"“This IND clearance in an important step to bring ATA-200 to US children suffering from this highly debilitating LGMD-2C/R5 disease” Sophie Olivier, MD, the chief medical officer of Atamyo, said in a statement.","_key":"fe4cc87afe5e0"},{"marks":["superscript"],"text":"1","_key":"53db804e4e64","_type":"span"}],"_type":"block","style":"normal","_key":"4f1bacaa23f2"},{"_key":"740abbc23b34","markDefs":[{"_type":"link","href":"https://www.cgtlive.com/view/atamyo-therapeutics-hits-milestones-limb-girdle-muscular-dystrophy-programs","_key":"3652cc16ed87"}],"children":[{"_type":"span","marks":[],"text":"Atamyo originally ","_key":"ba0aea8ea0e70"},{"_type":"span","marks":["3652cc16ed87"],"text":"submitted the IND to the FDA","_key":"ba0aea8ea0e71"},{"_type":"span","marks":[],"text":" in September 2024.","_key":"ba0aea8ea0e72"},{"_type":"span","marks":["superscript"],"text":"4","_key":"79dafabf2806"},{"_key":"72d14647a8e9","_type":"span","marks":[],"text":" The company noted alongside the news of the IND submission to the FDA that the agency had granted rare pediatric disease designation to ATA-200. Atamyo additionally statedat the time that it anticipates that dosing of patients in the trial for ATA-200 will begin before the end of 2024."}],"_type":"block","style":"normal"},{"markDefs":[],"children":[{"marks":[],"text":"“We are proud to be the first treatment for LGMD-2C/R5 to enter into clinical development in the US and we are committed to opening the first US center before year-end” Stéphane Degove, Atamyo’s Chief Executive Officer, added to the statement.","_key":"8f1d5254679b0","_type":"span"},{"marks":["superscript"],"text":"1","_key":"36210722bbc1","_type":"span"}],"_type":"block","style":"normal","_key":"1cae330d9072"},{"markDefs":[{"_type":"link","href":"https://www.cgtlive.com/view/atamyo-therapeutics-submits-cta-europe-limb-girdle-muscular-dystrophy-type-2c-r5-gene-therapy","_key":"5b2b26781a98"}],"children":[{"_type":"span","marks":[],"text":"The clinical trial application (CTA) leading to the trial’s clearance in France and Italy ","_key":"69df838c51a80"},{"_type":"span","marks":["5b2b26781a98"],"text":"was originally submitted","_key":"69df838c51a81"},{"marks":[],"text":" in September 2023.","_key":"69df838c51a82","_type":"span"},{"_type":"span","marks":["superscript"],"text":"3","_key":"6aa0279f763d"},{"_type":"span","marks":[],"text":" In addition to announcing the CTA submission, Atamyo also noted that it has received $8.6 million in nondilutive financing from France 2030, a program operated by Bpifrance, for ATA-200's development.","_key":"1b8e1c64f083"}],"_type":"block","style":"normal","_key":"8cbc9cad2c52"},{"style":"normal","_key":"43da752759f0","markDefs":[],"children":[{"_type":"span","marks":[],"text":"“ATA-200 incorporates a new promoter that enhances the liver and cardiac safety of gene therapy,” Isabelle Richard, PhD, the cofounder and chief scientific officer of Atamyo, upon whose research ATA-200's design was based, said in a September 2023 statement.","_key":"4c984da8a1910"},{"text":"3","_key":"33afd14d3209","_type":"span","marks":["superscript"]},{"text":" “This first-in-class experimental treatment represents a new hope for the patients”.","_key":"693d5edb31c9","_type":"span","marks":[]}],"_type":"block"},{"style":"normal","_key":"0086d240afe1","markDefs":[],"children":[{"_type":"span","marks":[],"text":"In addition to ATA-200, Atamyo is also developing ATA-100, an AAV vector-based gene therapy that is administered as a single dose and is intended to deliver a functional copy of the human ","_key":"743abb2d36b20"},{"marks":["em"],"text":"FKRP","_key":"5ec4378c4906","_type":"span"},{"_type":"span","marks":[],"text":" gene in order to treat fukutin-related protein limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9).","_key":"c817e50ec0bb"},{"_type":"span","marks":["superscript"],"text":"5-6","_key":"a21cfb1056d1"},{"_type":"span","marks":[],"text":" ATA-100 is currently being evaluated in a multicenter phase 1/2 clinical trial (EudraCT 2021-004276-33, NCT05224505) in Denmark, France, and the United Kingdom under CTAs that were cleared in each of those countries between 2021 and 2022.","_key":"089eb743b377"},{"text":"7-9","_key":"35b9bb61847c","_type":"span","marks":["superscript"]},{"_type":"span","marks":[],"text":" A later IND clearance from the FDA in 2023 allows expansion of the trial to the United States.","_key":"c1193cdd972c"},{"_type":"span","marks":["superscript"],"text":"10","_key":"b58f2f85fe47"},{"text":" Recently, in September 2024, Atamyo announced that the dose escalation portion of the phase 1b trial for ATA-200 had finished recruiting patients.","_key":"58cab6202c47","_type":"span","marks":[]},{"_key":"aa69960474f4","_type":"span","marks":["superscript"],"text":"4"}],"_type":"block"},{"children":[{"marks":[],"text":"“We are thrilled to have completed the recruitment of the dose escalation phase of our phase 1b study of ATA-100,” Olivier said in a statement at the time.","_key":"f0006a6902530","_type":"span"},{"_type":"span","marks":["superscript"],"text":"4","_key":"580b86606930"},{"_type":"span","marks":[],"text":" “Data of the dose escalation will serve at selecting the dose to carry over in the pivotal phase of the study. We look forward to presenting the preliminary results of the first dose cohort at the forthcoming World Muscle Society (WMS) Congress and at American Society of Gene \u0026 Cell Therapy’s Breakthroughs in Muscular Dystrophy Conference, held November 19 to 20, 2024 in Chicago.”","_key":"32437f379820"}],"_type":"block","style":"normal","_key":"5fbf322a32c6","markDefs":[]},{"_key":"01c53092d4f7","markDefs":[],"children":[{"_type":"span","marks":[],"text":"REFERENCES\n1. IND for ATA-200, a gene therapy for the treatment of limb-girdle muscular dystrophy type 2C/R5 (LGMD2C/R5), cleared to proceed by FDA. News release. Atamyo Therapeutics. November 12, 2024. Accessed November 19, 2024. https://atamyo.com/press-releases/ind-for-ata-200-a-gene-therapy-for-the-treatment-of-limb-girdle-muscular-dystrophy-type-2c-r5-lgmd2c-r5-cleared-to-proceed-by-fda/\n2. Atamyo Therapeutics obtains regulatory authorization in Europe to initiate a clinical trial for ATA-200, its gene therapy to treat limb-girdle muscular dystrophy type 2C/R5. News release. News release. Atamyo Therapeutics. March 26, 2024. Accessed November 19, 2024. https://atamyo.com/press-releases/atamyo-therapeutics-obtains-regulatory-authorization-in-europe-to-initiate-a-clinical-trial-for-ata-200-its-gene-therapy-to-treat-limb-girdle-muscular-dystrophy-type-2c-r5/\n3. ATA-200, AtamyoTherapeutics’ gene therapy to treat limb-girdle muscular dystrophy type 2C/R5, reaches key milestones with the filing of a clinical trial application in Europe and a non-dilutive financing from France 2030 program. News release. Atamyo Therapeutics. September 19, 2023. Accessed November 19, 2024. https://atamyo.com/press-releases/https-atamyo-com-wp-content-uploads-pr-sept-19-2023-ata-200-reaches-key-milestones-with-cta-in-europe-and-france2030-financing-pdf/\n4. Atamyo Therapeutics observes LGMD awareness day with updates on key milestones in its clinical development of gene therapies for patients suffering from limb-girdle muscular dystrophies. News releases. September 30, 2024. Accessed November 19, 2024. https://atamyo.com/press-releases/atamyo-therapeutics-observes-lgmd-awareness-day-with-updates-on-key-milestones-in-its-clinical-development-of-gene-therapies-for-patients-suffering-from-limb-girdle-muscular-dystrophies/\n5. IND for ATA-100, a gene therapy for the treatment of limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9), cleared to proceed by FDA. News release. Atamyo Therapeutics. September 5, 2023. Accessed November 19, 2024. https://atamyo.com/press-releases/2023/ind-for-ata-100-a-gene-therapy-for-the-treatment-of-limb-girdle-muscular-dystrophy-type-2i-r9-lgmd2i-r9-cleared-to-proceed-by-fda/\n6. Atamyo Therapeutics announces first patient dosed with ATA-100 gene therapy in LGMD-R9 clinical trial. News Release. Atamyo Therapeutics. September 26, 2022. Accessed November 19, 2024. https://atamyo.com/press-releases/atamyo-therapeutics-announces-first-patient-dosed-with-ata-100-gene-therapy-in-lgmd-r9-clinical-trial/\n7. Atamyo Therapeutics obtains first regulatory authorization in Europe to initiate a clinical trial for ATA-100, its gene therapy to treat limb-girdle muscular dystrophy type 2I/R9. News release. Atamyo Therapeutics. December 6, 2021. Accessed November 19, 2024. https://atamyo.com/press-releases/2021/atamyo-therapeutics-obtains-first-regulatory-authorization-in-europe-to-initiate-a-clinical-trial-for-ata-100-its-gene-therapy-to-treat-limb-girdle-muscular-dystrophy-type-2i-r9/\n8. Atamyo Therapeutics reaches significant regulatory and financial milestones for ATA-100, its gene therapy to treat limb-girdle muscular dystrophy type 2I/R9. News Release. Atamyo Therapeutics. February 24, 2022. Accessed November 19, 2024. https://atamyo.com/press-releases/atamyo-therapeutics-reaches-significant-regulatory-and-financial-milestones-for-ata-100-its-gene-therapy-to-treat-limb-girdle-muscular-dystrophy-type-2i-r9/\n9. Atamyo Therapeutics announces significant milestones for ATA-100 and ATA-200, its gene therapy programs to treat limb-girdle muscular dystrophy 2I/R9 and 2C/R5. News release. Atamyo Therapeutics. May16, 2022. Accessed November 19, 2024. https://atamyo.com/press-releases/atamyo-therapeutics-announces-significant-milestones-for-ata-100-and-ata-200-its-gene-therapy-programs-to-treat-limb-girdle-muscular-dystrophy-2i-r9-and-2c-r5/\n10. IND for ATA-100, a gene therapy for the treatment of limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9), cleared to proceed by FDA. News release. Atamyo Therapeutics. September 5, 2023. Accessed November 19, 2024. https://atamyo.com/press-releases/2023/ind-for-ata-100-a-gene-therapy-for-the-treatment-of-limb-girdle-muscular-dystrophy-type-2i-r9-lgmd2i-r9-cleared-to-proceed-by-fda/","_key":"7a21f0b565a40"}],"_type":"block","style":"h6"},{"_key":"1448b4f785d6","markDefs":[],"children":[{"text":"","_key":"429c1aab68820","_type":"span","marks":[]}],"_type":"block","style":"normal"}],"_createdAt":"2024-11-19T10:08:39Z","_rev":"tJBCVnDTgUimCQOCgAzW7I","authors":[{"displayName":"Noah Stansfield","url":"noah-stansfield"}],"documentGroupMapping":null,"ExcludeFromPubMedXML":false,"display_summary":true,"thumbnail":{"caption":"Sophie Olivier, MD","asset":{"_ref":"image-0ad104c0cc1fd9a47e3e9a1b13dafff2d2418ed5-300x300-jpg","_type":"reference"},"_type":"mainImage","alt":"Sophie Olivier, MD, the chief medical officer of Atamyo"},"published":"2024-11-19T12:00:18.703Z","summary":"In addition to the IND clearance, the FDA also granted ATA-200 orphan drug designation.","targeting":{"content_placement":["topic/neurology","news"],"document_url":["atamyo-therapeutics-gene-therapy-ata-200-cleared-trial-lgmd-type-2c-r5"],"document_group":null,"rootDocumentGroup":[],"issue_url":"","publication_url":""},"relatedArticles":[{"title":"Exploring CAR-T for Autoimmune Disease","url":{"_type":"slug","current":"exploring-car-t-autoimmune-disease"},"thumbnail":{"_type":"mainImage","alt":"Georg Schett, MD, vice president research and chair of internal medicine at the University of Erlangen – Nuremberg","caption":"Georg Schett, MD","asset":{"_ref":"image-6808278d4a529c2b98af3c3297a83a2440fa06a2-300x300-png","_type":"reference"}},"published":"2024-11-25T12:00:57.366Z"},{"title":"Alzheimer Disease Awareness Month 2024: Looking Back at a Year of Progress in Cell and Gene Therapy","url":{"current":"alzheimer-disease-awareness-month-2024-looking-back-year-progress-cell-gene-therapy","_type":"slug"},"thumbnail":{"asset":{"_ref":"image-bcf8b7384dbe616cc22cb250d8946d9b6758e997-1200x674-jpg","_type":"reference"},"_type":"mainImage"},"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"},{"title":"David Barrett, JD, on Future Trends for Genomic Medicines","url":{"current":"barrett-future-trends-genomic-medicines","_type":"slug"},"thumbnail":{"_type":"mainImage","alt":"David Barrett, JD, the chief executive officer of ASGCT","caption":"David Barrett, JD","asset":{"_ref":"image-73c396ac9ec1c25274e5148785e53cc12aad6577-755x426-png","_type":"reference"}},"published":"2024-11-22T12:00:05.483Z"},{"title":"Evaluating Allogeneic CAR-T P-BCMA-ALLO1 in R/R Multiple Myeloma","url":{"current":"evaluating-allogeneic-car-t-p-bcma-allo1-multiple-myeloma","_type":"slug"},"thumbnail":{"alt":"Bhagirathbhai R. 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