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/_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fajmc%2F7e20ff65b7abea493e84dace957fb80991ce7bb1-6048x4024.jpg%3Ffit%3Dcrop%26auto%3Dformat&w=1200&q=75 1200w, /_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fajmc%2F7e20ff65b7abea493e84dace957fb80991ce7bb1-6048x4024.jpg%3Ffit%3Dcrop%26auto%3Dformat&w=1920&q=75 1920w, /_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fajmc%2F7e20ff65b7abea493e84dace957fb80991ce7bb1-6048x4024.jpg%3Ffit%3Dcrop%26auto%3Dformat&w=2048&q=75 2048w, /_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fajmc%2F7e20ff65b7abea493e84dace957fb80991ce7bb1-6048x4024.jpg%3Ffit%3Dcrop%26auto%3Dformat&w=3840&q=75 3840w" src="/_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fajmc%2F7e20ff65b7abea493e84dace957fb80991ce7bb1-6048x4024.jpg%3Ffit%3Dcrop%26auto%3Dformat&w=3840&q=75" decoding="async" data-nimg="fill" style="position:absolute;top:0;left:0;bottom:0;right:0;box-sizing:border-box;padding:0;border:none;margin:auto;display:block;width:0;height:0;min-width:100%;max-width:100%;min-height:100%;max-height:100%" class="object-contain" loading="lazy"/></noscript></span></a></div><div class="border-t-gray-100 border-t-2 flex-auto py-2 "><span class="text-sm text-gray-500 py-2">November 23rd 2024</span><div class="py-3"><a class="lg:text-[20px] text-[19px] font-[500]" href="/view/viltolarsen-slows-dmd-progression-in-small-study">Viltolarsen Slows DMD Progression in Small Study</a></div><div class="pb-2"><div><span class="text-md "><span class="mr-1 italic">By </span><a class="mr-1 text-sky-800 hover:text-primary" href="/authors/hayden-e-klein"><i>Hayden E. Klein</i></a></span></div></div><a href="/view/viltolarsen-slows-dmd-progression-in-small-study"><a href="/view/viltolarsen-slows-dmd-progression-in-small-study"><p class="mt-1 text-gray-800 text-[13px] line-clamp-6 text-hidden">Further research is needed to determine how once-weekly viltolarsen can slow disease progression and preserve motor function in boys and young men with Duchenne muscular dystrophy (DMD).</p></a></a></div></div><div class="flex-wrap w-[60%] flex "><a class="flex w-[50%] flex-col sm:pl-[35px] pl-[20px] pb-[35px] " href="/view/sarepta-halts-development-testing-of-dmd-therapy-srp-5051"><div class="w-full lg:h-[134px] h-[100px] relative bg-default-logo-background "><span style="box-sizing:border-box;display:block;overflow:hidden;width:initial;height:initial;background:none;opacity:1;border:0;margin:0;padding:0;position:absolute;top:0;left:0;bottom:0;right:0"><img alt="" src="data:image/gif;base64,R0lGODlhAQABAIAAAAAAAP///yH5BAEAAAAALAAAAAABAAEAAAIBRAA7" decoding="async" data-nimg="fill" class="lg:object-cover object-contain " 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 alt="" sizes="100vw" srcSet="/_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fajmc%2Fa7019eb0cbb8b85d5d49a47769ca1af1a24407d5-5472x3648.jpg%3Ffit%3Dcrop%26auto%3Dformat&w=640&q=75 640w, /_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fajmc%2Fa7019eb0cbb8b85d5d49a47769ca1af1a24407d5-5472x3648.jpg%3Ffit%3Dcrop%26auto%3Dformat&w=750&q=75 750w, /_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fajmc%2Fa7019eb0cbb8b85d5d49a47769ca1af1a24407d5-5472x3648.jpg%3Ffit%3Dcrop%26auto%3Dformat&w=828&q=75 828w, /_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fajmc%2Fa7019eb0cbb8b85d5d49a47769ca1af1a24407d5-5472x3648.jpg%3Ffit%3Dcrop%26auto%3Dformat&w=1080&q=75 1080w, /_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fajmc%2Fa7019eb0cbb8b85d5d49a47769ca1af1a24407d5-5472x3648.jpg%3Ffit%3Dcrop%26auto%3Dformat&w=1200&q=75 1200w, /_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fajmc%2Fa7019eb0cbb8b85d5d49a47769ca1af1a24407d5-5472x3648.jpg%3Ffit%3Dcrop%26auto%3Dformat&w=1920&q=75 1920w, /_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fajmc%2Fa7019eb0cbb8b85d5d49a47769ca1af1a24407d5-5472x3648.jpg%3Ffit%3Dcrop%26auto%3Dformat&w=2048&q=75 2048w, /_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fajmc%2Fa7019eb0cbb8b85d5d49a47769ca1af1a24407d5-5472x3648.jpg%3Ffit%3Dcrop%26auto%3Dformat&w=3840&q=75 3840w" src="/_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fajmc%2Fa7019eb0cbb8b85d5d49a47769ca1af1a24407d5-5472x3648.jpg%3Ffit%3Dcrop%26auto%3Dformat&w=3840&q=75" decoding="async" data-nimg="fill" style="position:absolute;top:0;left:0;bottom:0;right:0;box-sizing:border-box;padding:0;border:none;margin:auto;display:block;width:0;height:0;min-width:100%;max-width:100%;min-height:100%;max-height:100%" class="lg:object-cover object-contain " loading="lazy"/></noscript></span></div><div class="border-t-gray-100 border-t-2 pt-2 pb-2 flex-auto "><a href="/view/sarepta-halts-development-testing-of-dmd-therapy-srp-5051"><span class="text-sm text-gray-500">November 14th 2024</span></a><div><a class="text-[14px]" href="/view/sarepta-halts-development-testing-of-dmd-therapy-srp-5051"><p class="line-clamp-4 text-hidden font-[500]">Sarepta Halts Development, Testing of DMD Therapy SRP-5051</p></a></div><div class="py-2"><div><span class="text-md "><span class="mr-1 italic">By </span><a class="mr-1 text-sky-800 hover:text-primary" href="/authors/hayden-e-klein"><i>Hayden E. Klein</i></a></span></div></div></div></a><a class="flex w-[50%] flex-col sm:pl-[35px] pl-[20px] pb-[35px] " href="/view/fda-approves-gene-therapy-to-treat-aadc-deficiency"><div class="w-full lg:h-[134px] h-[100px] relative bg-default-logo-background "><span style="box-sizing:border-box;display:block;overflow:hidden;width:initial;height:initial;background:none;opacity:1;border:0;margin:0;padding:0;position:absolute;top:0;left:0;bottom:0;right:0"><img alt="" src="data:image/gif;base64,R0lGODlhAQABAIAAAAAAAP///yH5BAEAAAAALAAAAAABAAEAAAIBRAA7" decoding="async" data-nimg="fill" class="lg:object-cover object-contain " 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 alt="" sizes="100vw" 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/_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fajmc%2F2eda2b798654c7967a80cb8bda8ab07ab614a58b-2880x1618.png%2FScreen%2520Shot%25202023-10-23%2520at%252012.01.31%2520PM.png%3Ffit%3Dcrop%26auto%3Dformat&w=3840&q=75 3840w" src="/_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fajmc%2F2eda2b798654c7967a80cb8bda8ab07ab614a58b-2880x1618.png%2FScreen%2520Shot%25202023-10-23%2520at%252012.01.31%2520PM.png%3Ffit%3Dcrop%26auto%3Dformat&w=3840&q=75" decoding="async" data-nimg="fill" style="position:absolute;top:0;left:0;bottom:0;right:0;box-sizing:border-box;padding:0;border:none;margin:auto;display:block;width:0;height:0;min-width:100%;max-width:100%;min-height:100%;max-height:100%" class="object-cover" loading="lazy"/></noscript></span></div><div class="jsx-1637274924 content-wrap"><span class="jsx-1637274924 py-3 text-sm text-gray-500 text-left"><i class="jsx-1637274924">October 23rd 2023</i></span><div class="jsx-1637274924 w-full h-fit mb-4"><p class="jsx-1637274924 text-[18px] font-bold text-[#000000] text-left line-clamp-3 text-hidden">Dr Landon Marshall Discusses Gene Therapy Forecasting Benefits, Future Advancements</p></div></div></div></a><a target="" style="scroll-snap-align:center;text-decoration:none" class="jsx-286612495 sm:w-[280px] xs:w-[240px] w-[65%] h-auto flex flex-col space-y-3 flex-none select-none no-underline" href="/view/payers-providers-still-navigating-uncharted-waters-for-gene-therapies"><div class="jsx-286612495 w-full h-full flex flex-col overflow-hidden relative bg-white text-center"><div class="jsx-286612495 relative w-full shadow-md shadow-grey-800 aspect-video"><span style="box-sizing:border-box;display:block;overflow:hidden;width:initial;height:initial;background:none;opacity:1;border:0;margin:0;padding:0;position:absolute;top:0;left:0;bottom:0;right:0"><img alt="" src="data:image/gif;base64,R0lGODlhAQABAIAAAAAAAP///yH5BAEAAAAALAAAAAABAAEAAAIBRAA7" decoding="async" data-nimg="fill" class="object-cover" 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 alt="" sizes="100vw" srcSet="/_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fajmc%2Fc39d430a8717d06cbba2f663a66cdc84200bebff-1280x719.png%2FBooth-Killpack.png%3Ffit%3Dcrop%26auto%3Dformat&w=640&q=75 640w, /_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fajmc%2Fc39d430a8717d06cbba2f663a66cdc84200bebff-1280x719.png%2FBooth-Killpack.png%3Ffit%3Dcrop%26auto%3Dformat&w=750&q=75 750w, /_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fajmc%2Fc39d430a8717d06cbba2f663a66cdc84200bebff-1280x719.png%2FBooth-Killpack.png%3Ffit%3Dcrop%26auto%3Dformat&w=828&q=75 828w, 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src="/_next/image?url=https%3A%2F%2Fcdn.sanity.io%2Fimages%2F0vv8moc6%2Fajmc%2Fc39d430a8717d06cbba2f663a66cdc84200bebff-1280x719.png%2FBooth-Killpack.png%3Ffit%3Dcrop%26auto%3Dformat&w=3840&q=75" decoding="async" data-nimg="fill" style="position:absolute;top:0;left:0;bottom:0;right:0;box-sizing:border-box;padding:0;border:none;margin:auto;display:block;width:0;height:0;min-width:100%;max-width:100%;min-height:100%;max-height:100%" class="object-cover" loading="lazy"/></noscript></span></div><div class="jsx-286612495 content-wrap"><span class="jsx-286612495 py-3 text-sm text-gray-500 text-left"><i class="jsx-286612495">April 10th 2023</i></span><div class="jsx-286612495 w-full h-fit mb-4"><p class="jsx-286612495 text-[18px] font-bold text-[#000000] text-left line-clamp-3 text-hidden">Payers, Providers Still Navigating Uncharted Waters for Gene Therapies</p></div></div></div></a></div></div></div><div class="flex items-center space-x-4 border-0 border-b border-secondary select-none py-1"><h2 class="text-3xl text-primary">More News</h2></div><div class="flex flex-wrap w-full pb-4"><div class="jsx-ad50481d5ee26850 w-full h-full"><div><div><div class="text-[8px] text-center text-gray-500 hidden">Advertisement</div><div id="div-gpt-ad-infeed-1"></div></div></div><div class="jsx-ad50481d5ee26850 flex flex-col sm:flex-row justify-between my-4 "><a class="jsx-ad50481d5ee26850" href="/view/a-year-of-dmd-gene-therapy-trial-failures"><img src="https://cdn.sanity.io/images/0vv8moc6/ajmc/f17e24452d6c5da3f5c3add1df744b5a59621ffe-6000x4000.jpg?fit=crop&auto=format" alt="Gene therapy research | Image credit: RFBSIP – stock.adobe.com" width="288" class="jsx-ad50481d5ee26850 w-full xs:w-[288px] "/></a><div class="jsx-ad50481d5ee26850 article-detail flex flex-col gap-[0.2rem] w-full sm:w-[46%] md:w-[65%]"><span class="jsx-ad50481d5ee26850 font-bold article-publish-date block italic text-sm text-gray-500 mt-[1rem] sm:mt-0">Published: October 29th 2024 | Updated: October 30th 2024</span><p class="jsx-ad50481d5ee26850 article-title font-bold text-[1rem]"><a class="jsx-ad50481d5ee26850" href="/view/a-year-of-dmd-gene-therapy-trial-failures">A Year of DMD Gene Therapy Trial Failures</a></p><div class="jsx-ad50481d5ee26850 authors flex-row wrap gap-[0.2rem]"><a class="jsx-ad50481d5ee26850 text-[#000] text-sm italic" href="/authors/hayden-e-klein">Hayden E. Klein</a></div><div class="jsx-ad50481d5ee26850 article-summary"><a class="jsx-ad50481d5ee26850" href="/view/a-year-of-dmd-gene-therapy-trial-failures"><div class="jsx-ad50481d5ee26850 text-sm text-gray-500 py-1">The outcomes of the EMBARK and CIFFREO trials in boys aged 4 to 7 have raised concerns in the Duchenne muscular dystrophy (DMD) community and prompted layoffs at Pfizer.</div></a></div></div></div><div style="border-bottom:1px solid #CCCCCC" class="jsx-ad50481d5ee26850"></div></div><div class="jsx-ad50481d5ee26850 w-full h-full"><div class="jsx-ad50481d5ee26850 flex flex-col sm:flex-row justify-between my-4 "><a class="jsx-ad50481d5ee26850" href="/view/different-cost-effectiveness-models-of-car-t-yield-similar-results"><img src="https://cdn.sanity.io/images/0vv8moc6/ajmc/0500ff75db50ce96c1716d94f373d42fc8148c20-7280x4080.jpg?fit=crop&auto=format" alt="Varying models for CAR-T therapy are similarly cost effective, report finds | image credit: inthasone - stock.adobe.com" width="288" class="jsx-ad50481d5ee26850 w-full xs:w-[288px] "/></a><div class="jsx-ad50481d5ee26850 article-detail flex flex-col gap-[0.2rem] w-full sm:w-[46%] md:w-[65%]"><span class="jsx-ad50481d5ee26850 font-bold article-publish-date block italic text-sm text-gray-500 mt-[1rem] sm:mt-0">October 22nd 2024</span><p class="jsx-ad50481d5ee26850 article-title font-bold text-[1rem]"><a class="jsx-ad50481d5ee26850" href="/view/different-cost-effectiveness-models-of-car-t-yield-similar-results">Different Cost-Effectiveness Models of CAR T Yield Similar Results </a></p><div class="jsx-ad50481d5ee26850 authors flex-row wrap gap-[0.2rem]"><a class="jsx-ad50481d5ee26850 text-[#000] text-sm italic" href="/authors/jared-kaltwasser">Jared Kaltwasser</a></div><div class="jsx-ad50481d5ee26850 article-summary"><a class="jsx-ad50481d5ee26850" href="/view/different-cost-effectiveness-models-of-car-t-yield-similar-results"><div class="jsx-ad50481d5ee26850 text-sm text-gray-500 py-1">Investigators said the unique features of chimeric antigen receptor (CAR) T-cell therapy may warrant novel approaches to cost-effectiveness analysis.</div></a></div></div></div><div style="border-bottom:1px solid #CCCCCC" class="jsx-ad50481d5ee26850"></div></div><div class="jsx-ad50481d5ee26850 w-full h-full"><div class="jsx-ad50481d5ee26850 flex md:hidden justify-center items-center"></div><div class="jsx-ad50481d5ee26850 flex flex-col sm:flex-row justify-between my-4 "><a class="jsx-ad50481d5ee26850" href="/view/cns-directed-aav-gene-therapy-immune-dynamics-and-clinical-implications"><img src="https://cdn.sanity.io/images/0vv8moc6/ajmc/64a1f45a5ebb7f0f665f1e955a9016e4250c9519-1200x724.jpg?fit=crop&auto=format" alt="Illustration of gene therapy | Image Credit: © RFBSIP - stock.adobe.com" width="288" class="jsx-ad50481d5ee26850 w-full xs:w-[288px] "/></a><div class="jsx-ad50481d5ee26850 article-detail flex flex-col gap-[0.2rem] w-full sm:w-[46%] md:w-[65%]"><span class="jsx-ad50481d5ee26850 font-bold article-publish-date block italic text-sm text-gray-500 mt-[1rem] sm:mt-0">October 17th 2024</span><p class="jsx-ad50481d5ee26850 article-title font-bold text-[1rem]"><a class="jsx-ad50481d5ee26850" href="/view/cns-directed-aav-gene-therapy-immune-dynamics-and-clinical-implications">CNS-Directed AAV Gene Therapy: Immune Dynamics and Clinical Implications</a></p><div class="jsx-ad50481d5ee26850 authors flex-row wrap gap-[0.2rem]"><a class="jsx-ad50481d5ee26850 text-[#000] text-sm italic" href="/authors/kimberly-rath-pharmd">Kimberly Rath, PharmD</a></div><div class="jsx-ad50481d5ee26850 article-summary"><a class="jsx-ad50481d5ee26850" href="/view/cns-directed-aav-gene-therapy-immune-dynamics-and-clinical-implications"><div class="jsx-ad50481d5ee26850 text-sm text-gray-500 py-1">A new study reviews how various routes of adeno-associated virus (AAV) gene therapy administration influence local and peripheral immune responses, with a focus on those observed in clinical trials targeting the central nervous system (CNS).</div></a></div></div></div><div style="border-bottom:1px solid #CCCCCC" class="jsx-ad50481d5ee26850"></div></div><div class="jsx-ad50481d5ee26850 w-full h-full"><div class="jsx-ad50481d5ee26850 flex flex-col sm:flex-row justify-between my-4 "><a class="jsx-ad50481d5ee26850" href="/view/voretigene-neparvovec-helps-patients-with-rpe65-related-retinopathy-regain-bilateral-function"><img src="https://cdn.sanity.io/images/0vv8moc6/ajmc/58ebe9965752c640b53e33522ef233409ac2c4cf-1200x629.jpg?fit=crop&auto=format" alt="eye computer | Image Credit: © eevi - stock.adobe.com" width="288" class="jsx-ad50481d5ee26850 w-full xs:w-[288px] "/></a><div class="jsx-ad50481d5ee26850 article-detail flex flex-col gap-[0.2rem] w-full sm:w-[46%] md:w-[65%]"><span class="jsx-ad50481d5ee26850 font-bold article-publish-date block italic text-sm text-gray-500 mt-[1rem] sm:mt-0">October 9th 2024</span><p class="jsx-ad50481d5ee26850 article-title font-bold text-[1rem]"><a class="jsx-ad50481d5ee26850" href="/view/voretigene-neparvovec-helps-patients-with-rpe65-related-retinopathy-regain-bilateral-function">Voretigene Neparvovec Helps Patients With RPE65-Related Retinopathy Regain Bilateral Function</a></p><div class="jsx-ad50481d5ee26850 authors flex-row wrap gap-[0.2rem]"><a class="jsx-ad50481d5ee26850 text-[#000] text-sm italic" href="/authors/karen-jacobson-sive">Karen Jacobson-Sive</a></div><div class="jsx-ad50481d5ee26850 article-summary"><a class="jsx-ad50481d5ee26850" href="/view/voretigene-neparvovec-helps-patients-with-rpe65-related-retinopathy-regain-bilateral-function"><div class="jsx-ad50481d5ee26850 text-sm text-gray-500 py-1">Three patients with RPE65-related retinopathy showed improved function in both eyes following gene augmentation therapy treatment with voretigene neparvovec.</div></a></div></div></div><div style="border-bottom:1px solid #CCCCCC" class="jsx-ad50481d5ee26850"></div></div><div class="jsx-ad50481d5ee26850 w-full h-full"><div class="jsx-ad50481d5ee26850 flex flex-col sm:flex-row justify-between my-4 "><a class="jsx-ad50481d5ee26850" href="/view/protein-s-role-in-insulin-signaling-could-have-implications-for-gene-therapy"><img src="https://cdn.sanity.io/images/0vv8moc6/ajmc/01d6f0461a162bcac5760581ec29ecf04ac1bf76-1200x800.jpg?fit=crop&auto=format" alt="Molecular model of insulin molecule | Image Credit: © Dr_Microbe - stock.adobe.com" width="288" class="jsx-ad50481d5ee26850 w-full xs:w-[288px] "/></a><div class="jsx-ad50481d5ee26850 article-detail flex flex-col gap-[0.2rem] w-full sm:w-[46%] md:w-[65%]"><span class="jsx-ad50481d5ee26850 font-bold article-publish-date block italic text-sm text-gray-500 mt-[1rem] sm:mt-0">October 4th 2024</span><p class="jsx-ad50481d5ee26850 article-title font-bold text-[1rem]"><a class="jsx-ad50481d5ee26850" href="/view/protein-s-role-in-insulin-signaling-could-have-implications-for-gene-therapy">Protein's Role in Insulin Signaling Could Have Implications for Gene Therapy</a></p><div class="jsx-ad50481d5ee26850 authors flex-row wrap gap-[0.2rem]"><a class="jsx-ad50481d5ee26850 text-[#000] text-sm italic" href="/authors/jared-kaltwasser">Jared Kaltwasser</a></div><div class="jsx-ad50481d5ee26850 article-summary"><a class="jsx-ad50481d5ee26850" href="/view/protein-s-role-in-insulin-signaling-could-have-implications-for-gene-therapy"><div class="jsx-ad50481d5ee26850 text-sm text-gray-500 py-1">Insulin resistance plays a role in many diseases. The new research provides insights into why insulin resistance appears to be associated with neurodegenerative disorders.</div></a></div></div></div><div style="border-bottom:1px solid #CCCCCC" class="jsx-ad50481d5ee26850"></div></div><div class="jsx-ad50481d5ee26850 w-full h-full"><div class="jsx-ad50481d5ee26850 flex flex-col sm:flex-row justify-between my-4 "><a class="jsx-ad50481d5ee26850" href="/view/meta-study-analyzes-immunosuppressive-protocols-for-aav-gene-therapy-for-monogenic-disorders"><img src="https://cdn.sanity.io/images/0vv8moc6/ajmc/df17be9ab7803f252c60bc1d6eea8586c27dcc08-3898x2598.jpg?fit=crop&auto=format" alt="Gene editing | Image Credit: © Degimages - stock.adobe.com" width="288" class="jsx-ad50481d5ee26850 w-full xs:w-[288px] "/></a><div class="jsx-ad50481d5ee26850 article-detail flex flex-col gap-[0.2rem] w-full sm:w-[46%] md:w-[65%]"><span class="jsx-ad50481d5ee26850 font-bold article-publish-date block italic text-sm text-gray-500 mt-[1rem] sm:mt-0">September 18th 2024</span><p class="jsx-ad50481d5ee26850 article-title font-bold text-[1rem]"><a class="jsx-ad50481d5ee26850" href="/view/meta-study-analyzes-immunosuppressive-protocols-for-aav-gene-therapy-for-monogenic-disorders">Meta-Study Analyzes Immunosuppressive Protocols for AAV Gene Therapy for Monogenic Disorders </a></p><div class="jsx-ad50481d5ee26850 authors flex-row wrap gap-[0.2rem]"><a class="jsx-ad50481d5ee26850 text-[#000] text-sm italic" href="/authors/karen-jacobson-sive">Karen Jacobson-Sive</a></div><div class="jsx-ad50481d5ee26850 article-summary"><a class="jsx-ad50481d5ee26850" href="/view/meta-study-analyzes-immunosuppressive-protocols-for-aav-gene-therapy-for-monogenic-disorders"><div class="jsx-ad50481d5ee26850 text-sm text-gray-500 py-1">Immunosuppressants used in adeno-associated virus (AAV) gene therapy for monogenic disorders present an adverse effect profile that is consistent with high-dose steroid use in other conditions, according to a meta-study that analyzed immunosuppressive protocols.</div></a></div></div></div><div style="border-bottom:1px solid #CCCCCC" class="jsx-ad50481d5ee26850"></div></div><div class="jsx-ad50481d5ee26850 w-full h-full"><div><div><div class="text-[8px] text-center text-gray-500 hidden">Advertisement</div><div id="div-gpt-ad-infeed-7"></div></div></div><div class="jsx-ad50481d5ee26850 flex flex-col sm:flex-row justify-between my-4 "><a class="jsx-ad50481d5ee26850" href="/view/emerging-technology-engineers-oncogenic-mutation-into-cell-lines"><img src="https://cdn.sanity.io/images/0vv8moc6/ajmc/ea97471e4b0613f5b7be5bf9898c5082213545f3-5472x3072.jpg?fit=crop&auto=format" alt="Gene editing | image credit: 4K_Heaven - stock.adobe.com" width="288" class="jsx-ad50481d5ee26850 w-full xs:w-[288px] "/></a><div class="jsx-ad50481d5ee26850 article-detail flex flex-col gap-[0.2rem] w-full sm:w-[46%] md:w-[65%]"><span class="jsx-ad50481d5ee26850 font-bold article-publish-date block italic text-sm text-gray-500 mt-[1rem] sm:mt-0">September 11th 2024</span><p class="jsx-ad50481d5ee26850 article-title font-bold text-[1rem]"><a class="jsx-ad50481d5ee26850" href="/view/emerging-technology-engineers-oncogenic-mutation-into-cell-lines">Emerging Technology Engineers Oncogenic Mutation Into Cell Lines </a></p><div class="jsx-ad50481d5ee26850 authors flex-row wrap gap-[0.2rem]"><a class="jsx-ad50481d5ee26850 text-[#000] text-sm italic" href="/authors/gwen-mergian">Gwen Mergian</a></div><div class="jsx-ad50481d5ee26850 article-summary"><a class="jsx-ad50481d5ee26850" href="/view/emerging-technology-engineers-oncogenic-mutation-into-cell-lines"><div class="jsx-ad50481d5ee26850 text-sm text-gray-500 py-1">A new proof-of-concept method reliably engineered a hotspot mutation of SF3B1, a gene-splicing gene, into diverse cancer cell lines, outperforming other contemporary editing approaches. </div></a></div></div></div><div style="border-bottom:1px solid #CCCCCC" class="jsx-ad50481d5ee26850"></div></div><div class="jsx-ad50481d5ee26850 w-full h-full"><div class="jsx-ad50481d5ee26850 flex flex-col sm:flex-row justify-between my-4 "><a class="jsx-ad50481d5ee26850" href="/view/review-car-t-cells-could-transform-pediatric-sle-therapy"><img src="https://cdn.sanity.io/images/0vv8moc6/ajmc/86002120662bc086c89a9494427508ac7a4ceea2-1200x724.jpg?fit=crop&auto=format" alt="Genetic analysis | Image Credit: © angellodeco - stock.adobe.com" width="288" class="jsx-ad50481d5ee26850 w-full xs:w-[288px] "/></a><div class="jsx-ad50481d5ee26850 article-detail flex flex-col gap-[0.2rem] w-full sm:w-[46%] md:w-[65%]"><span class="jsx-ad50481d5ee26850 font-bold article-publish-date block italic text-sm text-gray-500 mt-[1rem] sm:mt-0">September 4th 2024</span><p class="jsx-ad50481d5ee26850 article-title font-bold text-[1rem]"><a class="jsx-ad50481d5ee26850" href="/view/review-car-t-cells-could-transform-pediatric-sle-therapy">Review: CAR T Cells Could Transform Pediatric SLE Therapy</a></p><div class="jsx-ad50481d5ee26850 authors flex-row wrap gap-[0.2rem]"><a class="jsx-ad50481d5ee26850 text-[#000] text-sm italic" href="/authors/jared-kaltwasser">Jared Kaltwasser</a></div><div class="jsx-ad50481d5ee26850 article-summary"><a class="jsx-ad50481d5ee26850" href="/view/review-car-t-cells-could-transform-pediatric-sle-therapy"><div class="jsx-ad50481d5ee26850 text-sm text-gray-500 py-1">Early data suggest chimeric antigen receptor (CAR) T-cell therapy can achieve meaningful long-term results.</div></a></div></div></div><div style="border-bottom:1px solid #CCCCCC" class="jsx-ad50481d5ee26850"></div></div><div class="jsx-ad50481d5ee26850 w-full h-full"><div class="jsx-ad50481d5ee26850 flex md:hidden justify-center items-center"></div><div class="jsx-ad50481d5ee26850 flex flex-col sm:flex-row justify-between my-4 "><a class="jsx-ad50481d5ee26850" href="/view/neurological-complications-in-car-t-cell-therapy-a-case-of-parkinsonism"><img src="https://cdn.sanity.io/images/0vv8moc6/ajmc/b1f0d37eaf7b6b59492c384b92052c2f1b8dee6a-6144x3072.jpg?fit=crop&auto=format" alt="CAR T-cell therapy has revolutionized cancer treatment | image credit: Serhii - stock.adobe.com" width="288" class="jsx-ad50481d5ee26850 w-full xs:w-[288px] "/></a><div class="jsx-ad50481d5ee26850 article-detail flex flex-col gap-[0.2rem] w-full sm:w-[46%] md:w-[65%]"><span class="jsx-ad50481d5ee26850 font-bold article-publish-date block italic text-sm text-gray-500 mt-[1rem] sm:mt-0">September 4th 2024</span><p class="jsx-ad50481d5ee26850 article-title font-bold text-[1rem]"><a class="jsx-ad50481d5ee26850" href="/view/neurological-complications-in-car-t-cell-therapy-a-case-of-parkinsonism">Neurological Complications in CAR T-Cell Therapy: A Case of Parkinsonism </a></p><div class="jsx-ad50481d5ee26850 authors flex-row wrap gap-[0.2rem]"><a class="jsx-ad50481d5ee26850 text-[#000] text-sm italic" href="/authors/kimberly-rath-pharmd">Kimberly Rath, PharmD</a></div><div class="jsx-ad50481d5ee26850 article-summary"><a class="jsx-ad50481d5ee26850" href="/view/neurological-complications-in-car-t-cell-therapy-a-case-of-parkinsonism"><div class="jsx-ad50481d5ee26850 text-sm text-gray-500 py-1">A case report highlights a rare instance of parkinsonism following CAR T-cell therapy for multiple myeloma. </div></a></div></div></div><div style="border-bottom:1px solid #CCCCCC" class="jsx-ad50481d5ee26850"></div></div><div class="jsx-ad50481d5ee26850 w-full h-full"><div class="jsx-ad50481d5ee26850 flex flex-col sm:flex-row justify-between my-4 "><a class="jsx-ad50481d5ee26850" href="/view/looking-to-car-t-cell-therapy-in-refractory-pediatric-sle"><img src="https://cdn.sanity.io/images/0vv8moc6/ajmc/4243be11cacd5398be186f3048e776d01e5aa2d5-1124x717.jpg?fit=crop&auto=format" alt="The safety profile of CAR T for patients with SLE is more favorable compared with that for the oncology population, likely due to a smaller target amount of T cells and thus a lower underlying risk of cytokine release syndrome. | Image credit: AIGen - stock.adobe.com" width="288" class="jsx-ad50481d5ee26850 w-full xs:w-[288px] "/></a><div class="jsx-ad50481d5ee26850 article-detail flex flex-col gap-[0.2rem] w-full sm:w-[46%] md:w-[65%]"><span class="jsx-ad50481d5ee26850 font-bold article-publish-date block italic text-sm text-gray-500 mt-[1rem] sm:mt-0">August 28th 2024</span><p class="jsx-ad50481d5ee26850 article-title font-bold text-[1rem]"><a class="jsx-ad50481d5ee26850" href="/view/looking-to-car-t-cell-therapy-in-refractory-pediatric-sle">Looking to CAR T-Cell Therapy in Refractory Pediatric SLE</a></p><div class="jsx-ad50481d5ee26850 authors flex-row wrap gap-[0.2rem]"><a class="jsx-ad50481d5ee26850 text-[#000] text-sm italic" href="/authors/gayle-turim-dickstein">Gayle Turim Dickstein</a></div><div class="jsx-ad50481d5ee26850 article-summary"><a class="jsx-ad50481d5ee26850" href="/view/looking-to-car-t-cell-therapy-in-refractory-pediatric-sle"><div class="jsx-ad50481d5ee26850 text-sm text-gray-500 py-1">Several years of preliminary research results suggest chimeric antigen receptor (CAR) T-cell therapy could benefit pediatric patients with refractory systemic lupus erythematosus (SLE).</div></a></div></div></div><div style="border-bottom:1px solid #CCCCCC" class="jsx-ad50481d5ee26850"></div></div><div class="jsx-ad50481d5ee26850 w-full h-full"><div class="jsx-ad50481d5ee26850 flex flex-col sm:flex-row justify-between my-4 "><a class="jsx-ad50481d5ee26850" href="/view/an-updated-snapshot-of-gene-therapy-clinical-trials-worldwide"><img src="https://cdn.sanity.io/images/0vv8moc6/ajmc/e453d2b550779778cb1700cdcf01fc4997e11c99-1200x738.jpg?fit=crop&auto=format" alt="Gene therapy trials | Image Credit: ArtemisDiana-stock.adobe.com" width="288" class="jsx-ad50481d5ee26850 w-full xs:w-[288px] "/></a><div class="jsx-ad50481d5ee26850 article-detail flex flex-col gap-[0.2rem] w-full sm:w-[46%] md:w-[65%]"><span class="jsx-ad50481d5ee26850 font-bold article-publish-date block italic text-sm text-gray-500 mt-[1rem] sm:mt-0">August 23rd 2024</span><p class="jsx-ad50481d5ee26850 article-title font-bold text-[1rem]"><a class="jsx-ad50481d5ee26850" href="/view/an-updated-snapshot-of-gene-therapy-clinical-trials-worldwide">An Updated Snapshot of Gene Therapy Clinical Trials Worldwide</a></p><div class="jsx-ad50481d5ee26850 authors flex-row wrap gap-[0.2rem]"><a class="jsx-ad50481d5ee26850 text-[#000] text-sm italic" href="/authors/gayle-turim-dickstein">Gayle Turim Dickstein</a></div><div class="jsx-ad50481d5ee26850 article-summary"><a class="jsx-ad50481d5ee26850" href="/view/an-updated-snapshot-of-gene-therapy-clinical-trials-worldwide"><div class="jsx-ad50481d5ee26850 text-sm text-gray-500 py-1">The gene therapy landscape has changed dramatically in the past 5 years, and investigators wanted to capture and analyze the details of current and upcoming trials, along with their potential impact on patients.</div></a></div></div></div><div style="border-bottom:1px solid #CCCCCC" class="jsx-ad50481d5ee26850"></div></div><div class="jsx-ad50481d5ee26850 w-full h-full"><div class="jsx-ad50481d5ee26850 flex flex-col sm:flex-row justify-between my-4 "><a class="jsx-ad50481d5ee26850" href="/view/novel-car-e-platform-enhances-car-t-cell-functionality-in-cancer-treatment"><img src="https://cdn.sanity.io/images/0vv8moc6/ajmc/70fb2572859a305b82403a2b2a90815e69440fa4-1200x738.jpg?fit=crop&auto=format" alt="CAR T interaction | Image Credit: Alpha Tauri 3D-stock.adobe.com" width="288" class="jsx-ad50481d5ee26850 w-full xs:w-[288px] "/></a><div class="jsx-ad50481d5ee26850 article-detail flex flex-col gap-[0.2rem] w-full sm:w-[46%] md:w-[65%]"><span class="jsx-ad50481d5ee26850 font-bold article-publish-date block italic text-sm text-gray-500 mt-[1rem] sm:mt-0">August 16th 2024</span><p class="jsx-ad50481d5ee26850 article-title font-bold text-[1rem]"><a class="jsx-ad50481d5ee26850" href="/view/novel-car-e-platform-enhances-car-t-cell-functionality-in-cancer-treatment">Novel CAR-E Platform Enhances CAR T-Cell Functionality in Cancer Treatment</a></p><div class="jsx-ad50481d5ee26850 authors flex-row wrap gap-[0.2rem]"><a class="jsx-ad50481d5ee26850 text-[#000] text-sm italic" href="/authors/kimberly-rath-pharmd">Kimberly Rath, PharmD</a></div><div class="jsx-ad50481d5ee26850 article-summary"><a class="jsx-ad50481d5ee26850" href="/view/novel-car-e-platform-enhances-car-t-cell-functionality-in-cancer-treatment"><div class="jsx-ad50481d5ee26850 text-sm text-gray-500 py-1">Researchers introduce a chimeric antigen receptor–enhancer (CAR-E) therapeutic platform that significantly extends the effectiveness and memory of CAR T cells, potentially preventing relapse in patients who have cancer.</div></a></div></div></div><div style="border-bottom:1px solid #CCCCCC" class="jsx-ad50481d5ee26850"></div></div><div class="jsx-ad50481d5ee26850 w-full h-full"><div><div><div class="text-[8px] text-center text-gray-500 hidden">Advertisement</div><div id="div-gpt-ad-infeed-13"></div></div></div><div class="jsx-ad50481d5ee26850 flex flex-col sm:flex-row justify-between my-4 "><a class="jsx-ad50481d5ee26850" href="/view/high-costs-of-sickle-cell-therapies-highlight-paradox-of-drug-development"><img src="https://cdn.sanity.io/images/0vv8moc6/ajmc/222da18b351cb067cd438953b4e1201622e19986-1200x738.jpg?fit=crop&auto=format" alt="expensive medicine | Image Credit: jadamprostore-stock.adobe.com" width="288" class="jsx-ad50481d5ee26850 w-full xs:w-[288px] "/></a><div class="jsx-ad50481d5ee26850 article-detail flex flex-col gap-[0.2rem] w-full sm:w-[46%] md:w-[65%]"><span class="jsx-ad50481d5ee26850 font-bold article-publish-date block italic text-sm text-gray-500 mt-[1rem] sm:mt-0">August 9th 2024</span><p class="jsx-ad50481d5ee26850 article-title font-bold text-[1rem]"><a class="jsx-ad50481d5ee26850" href="/view/high-costs-of-sickle-cell-therapies-highlight-paradox-of-drug-development">High Costs of Sickle Cell Therapies Highlight Paradox of Drug Development</a></p><div class="jsx-ad50481d5ee26850 authors flex-row wrap gap-[0.2rem]"><a class="jsx-ad50481d5ee26850 text-[#000] text-sm italic" href="/authors/jared-kaltwasser">Jared Kaltwasser</a></div><div class="jsx-ad50481d5ee26850 article-summary"><a class="jsx-ad50481d5ee26850" href="/view/high-costs-of-sickle-cell-therapies-highlight-paradox-of-drug-development"><div class="jsx-ad50481d5ee26850 text-sm text-gray-500 py-1">Experts argue for new mechanisms to fund research in oft-ignored rare diseases.</div></a></div></div></div><div style="border-bottom:1px solid #CCCCCC" class="jsx-ad50481d5ee26850"></div></div><div class="jsx-ad50481d5ee26850 w-full h-full"><div class="jsx-ad50481d5ee26850 flex flex-col sm:flex-row justify-between my-4 "><a class="jsx-ad50481d5ee26850" href="/view/crispr-cas9-gene-therapy-holds-promise-to-treat-epidermolysis-bullosa"><img src="https://cdn.sanity.io/images/0vv8moc6/ajmc/b4db9eee75fd88b300f5a6df4aa74dc6ea845477-1200x738.jpg?fit=crop&auto=format" alt="epidermolysis bullosa | Image Credit: Yurii Kibalnik-stock.adobe.com" width="288" class="jsx-ad50481d5ee26850 w-full xs:w-[288px] "/></a><div class="jsx-ad50481d5ee26850 article-detail flex flex-col gap-[0.2rem] w-full sm:w-[46%] md:w-[65%]"><span class="jsx-ad50481d5ee26850 font-bold article-publish-date block italic text-sm text-gray-500 mt-[1rem] sm:mt-0">August 2nd 2024</span><p class="jsx-ad50481d5ee26850 article-title font-bold text-[1rem]"><a class="jsx-ad50481d5ee26850" href="/view/crispr-cas9-gene-therapy-holds-promise-to-treat-epidermolysis-bullosa">CRISPR/Cas9 Gene Therapy Holds Promise to Treat Epidermolysis Bullosa </a></p><div class="jsx-ad50481d5ee26850 authors flex-row wrap gap-[0.2rem]"><a class="jsx-ad50481d5ee26850 text-[#000] text-sm italic" href="/authors/karen-jacobson-sive">Karen Jacobson-Sive</a></div><div class="jsx-ad50481d5ee26850 article-summary"><a class="jsx-ad50481d5ee26850" href="/view/crispr-cas9-gene-therapy-holds-promise-to-treat-epidermolysis-bullosa"><div class="jsx-ad50481d5ee26850 text-sm text-gray-500 py-1">An exon-skipping technique using dual single-guide RNA/Cas9 ribonucleoproteins targeted at 3 novel COL7A1 exons with pathogenic heterozygous mutations achieved exon deletion rates of up to 95%. </div></a></div></div></div><div style="border-bottom:1px solid #CCCCCC" class="jsx-ad50481d5ee26850"></div></div><div class="jsx-ad50481d5ee26850 w-full h-full"><div class="jsx-ad50481d5ee26850 flex flex-col sm:flex-row justify-between my-4 "><a class="jsx-ad50481d5ee26850" href="/view/progress-in-precise-gene-expression-control-with-crispr-cas12f"><img src="https://cdn.sanity.io/images/0vv8moc6/ajmc/df17be9ab7803f252c60bc1d6eea8586c27dcc08-3898x2598.jpg?fit=crop&auto=format" alt="Artist rendering of a gene being edited | Image credit: Degimages - stock.adobe.com" width="288" class="jsx-ad50481d5ee26850 w-full xs:w-[288px] "/></a><div class="jsx-ad50481d5ee26850 article-detail flex flex-col gap-[0.2rem] w-full sm:w-[46%] md:w-[65%]"><span class="jsx-ad50481d5ee26850 font-bold article-publish-date block italic text-sm text-gray-500 mt-[1rem] sm:mt-0">July 27th 2024</span><p class="jsx-ad50481d5ee26850 article-title font-bold text-[1rem]"><a class="jsx-ad50481d5ee26850" href="/view/progress-in-precise-gene-expression-control-with-crispr-cas12f">Progress in Precise Gene Expression Control With CRISPR-Cas12f</a></p><div class="jsx-ad50481d5ee26850 authors flex-row wrap gap-[0.2rem]"><a class="jsx-ad50481d5ee26850 text-[#000] text-sm italic" href="/authors/kimberly-rath-pharmd">Kimberly Rath, PharmD</a></div><div class="jsx-ad50481d5ee26850 article-summary"><a class="jsx-ad50481d5ee26850" href="/view/progress-in-precise-gene-expression-control-with-crispr-cas12f"><div class="jsx-ad50481d5ee26850 text-sm text-gray-500 py-1">An innovative CRISPR-Cas12f-based activator system may hold significant potential in the future of gene therapy and biological research, suggest researchers of a new study.</div></a></div></div></div><div style="border-bottom:1px solid #CCCCCC" class="jsx-ad50481d5ee26850"></div></div><div class="jsx-ad50481d5ee26850 w-full h-full"><div class="jsx-ad50481d5ee26850 flex flex-col sm:flex-row justify-between my-4 "><a class="jsx-ad50481d5ee26850" href="/view/hemophilia-a-gene-therapy-superior-to-standard-of-care-in-phase-3-trial"><img src="https://cdn.sanity.io/images/0vv8moc6/ajmc/217c874543101839aacf463159b863d6e797d4cf-1200x800.jpg?fit=crop&auto=format" alt="Red blood drop with double helix | Image credit: Елена Бутусова-stock.adobe.com" width="288" class="jsx-ad50481d5ee26850 w-full xs:w-[288px] "/></a><div class="jsx-ad50481d5ee26850 article-detail flex flex-col gap-[0.2rem] w-full sm:w-[46%] md:w-[65%]"><span class="jsx-ad50481d5ee26850 font-bold article-publish-date block italic text-sm text-gray-500 mt-[1rem] sm:mt-0">July 26th 2024</span><p class="jsx-ad50481d5ee26850 article-title font-bold text-[1rem]"><a class="jsx-ad50481d5ee26850" href="/view/hemophilia-a-gene-therapy-superior-to-standard-of-care-in-phase-3-trial">Hemophilia A Gene Therapy Superior to Standard of Care in Phase 3 Trial</a></p><div class="jsx-ad50481d5ee26850 authors flex-row wrap gap-[0.2rem]"><a class="jsx-ad50481d5ee26850 text-[#000] text-sm italic" href="/authors/laura-joszt">Laura Joszt, MA</a></div><div class="jsx-ad50481d5ee26850 article-summary"><a class="jsx-ad50481d5ee26850" href="/view/hemophilia-a-gene-therapy-superior-to-standard-of-care-in-phase-3-trial"><div class="jsx-ad50481d5ee26850 text-sm text-gray-500 py-1">The gene therapy giroctocogene fitelparvovec demonstrated superiority compared with the standard of care, routine prophylaxis, in hemophilia A treatment.</div></a></div></div></div><div style="border-bottom:1px solid #CCCCCC" class="jsx-ad50481d5ee26850"></div></div><div class="jsx-ad50481d5ee26850 w-full h-full"><div class="jsx-ad50481d5ee26850 flex md:hidden justify-center items-center"></div><div class="jsx-ad50481d5ee26850 flex flex-col sm:flex-row justify-between my-4 "><a class="jsx-ad50481d5ee26850" href="/view/key-considerations-for-optimizing-b-vec-in-dystrophic-epidermolysis-bullosa"><img src="https://cdn.sanity.io/images/0vv8moc6/ajmc/dc87888d5428fdd4da3f31d55d53741ee29ecddd-1200x728.jpg?fit=crop&auto=format" alt="symptoms of epidermolysis bullosa | Image Credit: ангелина ковальчук-stock.adobe.com" width="288" class="jsx-ad50481d5ee26850 w-full xs:w-[288px] "/></a><div class="jsx-ad50481d5ee26850 article-detail flex flex-col gap-[0.2rem] w-full sm:w-[46%] md:w-[65%]"><span class="jsx-ad50481d5ee26850 font-bold article-publish-date block italic text-sm text-gray-500 mt-[1rem] sm:mt-0">July 22nd 2024</span><p class="jsx-ad50481d5ee26850 article-title font-bold text-[1rem]"><a class="jsx-ad50481d5ee26850" href="/view/key-considerations-for-optimizing-b-vec-in-dystrophic-epidermolysis-bullosa">Key Considerations for Optimizing B-VEC in Dystrophic Epidermolysis Bullosa</a></p><div class="jsx-ad50481d5ee26850 authors flex-row wrap gap-[0.2rem]"><a class="jsx-ad50481d5ee26850 text-[#000] text-sm italic" href="/authors/ajmc-contributor">AJMC Contributor</a></div><div class="jsx-ad50481d5ee26850 article-summary"><a class="jsx-ad50481d5ee26850" href="/view/key-considerations-for-optimizing-b-vec-in-dystrophic-epidermolysis-bullosa"><div class="jsx-ad50481d5ee26850 text-sm text-gray-500 py-1">Beremagene geperpavec-svdt (B-VEC) entered the market in 2023 as the first approved corrective treatment for dystrophic epidermolysis bullosa (DEB), a rare genetic disease affecting the skin and nails that is caused by mutations in the COL7A1 gene. </div></a></div></div></div><div style="border-bottom:1px solid #CCCCCC" class="jsx-ad50481d5ee26850"></div></div><div class="jsx-ad50481d5ee26850 w-full h-full"><div class="jsx-ad50481d5ee26850 flex flex-col sm:flex-row justify-between my-4 "><a class="jsx-ad50481d5ee26850" href="/view/understanding-coverage-financing-and-future-trends-of-gene-therapy-in-employer-health-plans"><img src="https://cdn.sanity.io/images/0vv8moc6/ajmc/1e602bba35858bd62230432132517d1d42061e3d-1280x800.jpg?fit=crop&auto=format" alt="Medicine doctor hold icon health and electronic medical record on interface. Digital healthcare and network connection on hologram virtual screen, insurance. medical technology and network concept - ipopba - stock.adobe.com.jpeg" width="288" class="jsx-ad50481d5ee26850 w-full xs:w-[288px] "/></a><div class="jsx-ad50481d5ee26850 article-detail flex flex-col gap-[0.2rem] w-full sm:w-[46%] md:w-[65%]"><span class="jsx-ad50481d5ee26850 font-bold article-publish-date block italic text-sm text-gray-500 mt-[1rem] sm:mt-0">July 21st 2024</span><p class="jsx-ad50481d5ee26850 article-title font-bold text-[1rem]"><a class="jsx-ad50481d5ee26850" href="/view/understanding-coverage-financing-and-future-trends-of-gene-therapy-in-employer-health-plans">Understanding Coverage, Financing, and Future Trends of Gene Therapy in Employer Health Plans</a></p><div class="jsx-ad50481d5ee26850 authors flex-row wrap gap-[0.2rem]"><a class="jsx-ad50481d5ee26850 text-[#000] text-sm italic" href="/authors/giuliana-grossi">Giuliana Grossi</a></div><div class="jsx-ad50481d5ee26850 article-summary"><a class="jsx-ad50481d5ee26850" href="/view/understanding-coverage-financing-and-future-trends-of-gene-therapy-in-employer-health-plans"><div class="jsx-ad50481d5ee26850 text-sm text-gray-500 py-1">The Greater Philadelphia Business Coalition on Health webinar series provided an in-depth framework for the advantages of including cell and gene therapy coverage in employer health plans, as well as the challenges brought by manufacturing complexities and the need to address accessibility to treatment.</div></a></div></div></div><div style="border-bottom:1px solid #CCCCCC" class="jsx-ad50481d5ee26850"></div></div><div class="jsx-ad50481d5ee26850 w-full h-full"><div><div><div class="text-[8px] text-center text-gray-500 hidden">Advertisement</div><div id="div-gpt-ad-infeed-19"></div></div></div><div class="jsx-ad50481d5ee26850 flex flex-col sm:flex-row justify-between my-4 "><a class="jsx-ad50481d5ee26850" href="/view/lexeo-s-shares-positive-interim-data-for-fa-cardiomyopathy-gene-therapy"><img src="https://cdn.sanity.io/images/0vv8moc6/ajmc/d20c253c8b12f9440a8a5120f98b413d5fce97b1-1200x738.jpg?fit=crop&auto=format" alt="cardiac genetics | Image Credit: bluebay2014-stock.adobe.com" width="288" class="jsx-ad50481d5ee26850 w-full xs:w-[288px] "/></a><div class="jsx-ad50481d5ee26850 article-detail flex flex-col gap-[0.2rem] w-full sm:w-[46%] md:w-[65%]"><span class="jsx-ad50481d5ee26850 font-bold article-publish-date block italic text-sm text-gray-500 mt-[1rem] sm:mt-0">July 16th 2024</span><p class="jsx-ad50481d5ee26850 article-title font-bold text-[1rem]"><a class="jsx-ad50481d5ee26850" href="/view/lexeo-s-shares-positive-interim-data-for-fa-cardiomyopathy-gene-therapy">Lexeo Shares Positive Interim Data for FA Cardiomyopathy Gene Therapy</a></p><div class="jsx-ad50481d5ee26850 authors flex-row wrap gap-[0.2rem]"><a class="jsx-ad50481d5ee26850 text-[#000] text-sm italic" href="/authors/jared-kaltwasser">Jared Kaltwasser</a></div><div class="jsx-ad50481d5ee26850 article-summary"><a class="jsx-ad50481d5ee26850" href="/view/lexeo-s-shares-positive-interim-data-for-fa-cardiomyopathy-gene-therapy"><div class="jsx-ad50481d5ee26850 text-sm text-gray-500 py-1">The therapy led to increased frataxin levels and decreased left ventricular mass in patients with Friedreich ataxia (FA) cardiomyopathy, the authors said. </div></a></div></div></div><div style="border-bottom:1px solid #CCCCCC" class="jsx-ad50481d5ee26850"></div></div><div class="jsx-ad50481d5ee26850 w-full h-full"><div class="jsx-ad50481d5ee26850 flex flex-col sm:flex-row justify-between my-4 "><a class="jsx-ad50481d5ee26850" href="/view/gpr176-may-be-potential-gene-therapy-target-in-ovarian-cancer"><img src="https://cdn.sanity.io/images/0vv8moc6/ajmc/97665e972696ce982819d10600e6d512624efe9a-1200x728.jpg?fit=crop&auto=format" alt="Female reproductive system | Image Credit: mi_viri-stock.adobe.com" width="288" class="jsx-ad50481d5ee26850 w-full xs:w-[288px] "/></a><div class="jsx-ad50481d5ee26850 article-detail flex flex-col gap-[0.2rem] w-full sm:w-[46%] md:w-[65%]"><span class="jsx-ad50481d5ee26850 font-bold article-publish-date block italic text-sm text-gray-500 mt-[1rem] sm:mt-0">July 15th 2024</span><p class="jsx-ad50481d5ee26850 article-title font-bold text-[1rem]"><a class="jsx-ad50481d5ee26850" href="/view/gpr176-may-be-potential-gene-therapy-target-in-ovarian-cancer">GPR176 May Be Potential Gene Therapy Target in Ovarian Cancer</a></p><div class="jsx-ad50481d5ee26850 authors flex-row wrap gap-[0.2rem]"><a class="jsx-ad50481d5ee26850 text-[#000] text-sm italic" href="/authors/ajmc-contributor">AJMC Contributor</a></div><div class="jsx-ad50481d5ee26850 article-summary"><a class="jsx-ad50481d5ee26850" href="/view/gpr176-may-be-potential-gene-therapy-target-in-ovarian-cancer"><div class="jsx-ad50481d5ee26850 text-sm text-gray-500 py-1">The biomarker, G-protein-coupled receptor 176 (GPR176), may be a driver in the progression of ovarian cancer and potential target for gene therapy, suggested researchers of a new study. </div></a></div></div></div><div style="border-bottom:1px solid #CCCCCC" class="jsx-ad50481d5ee26850"></div></div><div class="jsx-ad50481d5ee26850 w-full h-full"><div class="jsx-ad50481d5ee26850 flex flex-col sm:flex-row justify-between my-4 "><a class="jsx-ad50481d5ee26850" href="/view/five-children-two-hearing-improved-ears-each-successful-dfnb9-gene-therapy"><img src="https://cdn.sanity.io/images/0vv8moc6/ajmc/d32530e4c78aa913166b6bcb0261ff05354e7486-1200x728.jpg?fit=crop&auto=format" alt="Hearing test for children | Image Credit: Microgen-stock.adobe.com" width="288" class="jsx-ad50481d5ee26850 w-full xs:w-[288px] "/></a><div class="jsx-ad50481d5ee26850 article-detail flex flex-col gap-[0.2rem] w-full sm:w-[46%] md:w-[65%]"><span class="jsx-ad50481d5ee26850 font-bold article-publish-date block italic text-sm text-gray-500 mt-[1rem] sm:mt-0">July 8th 2024</span><p class="jsx-ad50481d5ee26850 article-title font-bold text-[1rem]"><a class="jsx-ad50481d5ee26850" href="/view/five-children-two-hearing-improved-ears-each-successful-dfnb9-gene-therapy">Five Children, 2 Hearing-Improved Ears Each: Successful DFNB9 Gene Therapy</a></p><div class="jsx-ad50481d5ee26850 authors flex-row wrap gap-[0.2rem]"><a class="jsx-ad50481d5ee26850 text-[#000] text-sm italic" href="/authors/gayle-turim-dickstein">Gayle Turim Dickstein</a></div><div class="jsx-ad50481d5ee26850 article-summary"><a class="jsx-ad50481d5ee26850" href="/view/five-children-two-hearing-improved-ears-each-successful-dfnb9-gene-therapy"><div class="jsx-ad50481d5ee26850 text-sm text-gray-500 py-1">The interim results of a single-arm trial in children with autosomal recessive deafness 9 (DFNB9) show the benefits of binaural administration of gene therapy: better hearing with no serious adverse effects. </div></a></div></div></div><div style="border-bottom:1px solid #CCCCCC" class="jsx-ad50481d5ee26850"></div></div><div class="jsx-ad50481d5ee26850 w-full h-full"><div class="jsx-ad50481d5ee26850 flex flex-col sm:flex-row justify-between my-4 "><a class="jsx-ad50481d5ee26850" href="/view/aav-therapy-a-successful-one-patient-spg50-gene-therapy-trial"><img src="https://cdn.sanity.io/images/0vv8moc6/ajmc/793336e1256fe4a7af9fd07850899011c60e1a5c-1200x728.jpg?fit=crop&auto=format" alt="DNA research | Image Credit: vladimircaribb-stock.adobe.com" width="288" class="jsx-ad50481d5ee26850 w-full xs:w-[288px] "/></a><div class="jsx-ad50481d5ee26850 article-detail flex flex-col gap-[0.2rem] w-full sm:w-[46%] md:w-[65%]"><span class="jsx-ad50481d5ee26850 font-bold article-publish-date block italic text-sm text-gray-500 mt-[1rem] sm:mt-0">July 1st 2024</span><p class="jsx-ad50481d5ee26850 article-title font-bold text-[1rem]"><a class="jsx-ad50481d5ee26850" href="/view/aav-therapy-a-successful-one-patient-spg50-gene-therapy-trial">AAV Therapy: A Successful 1-Patient SPG50 Gene Therapy Trial</a></p><div class="jsx-ad50481d5ee26850 authors flex-row wrap gap-[0.2rem]"><a class="jsx-ad50481d5ee26850 text-[#000] text-sm italic" href="/authors/gayle-turim-dickstein">Gayle Turim Dickstein</a></div><div class="jsx-ad50481d5ee26850 article-summary"><a class="jsx-ad50481d5ee26850" href="/view/aav-therapy-a-successful-one-patient-spg50-gene-therapy-trial"><div class="jsx-ad50481d5ee26850 text-sm text-gray-500 py-1">An adeno-associated virus (AAV) gene therapy for hereditary spastic paraplegia type 50 (SPG50)—funded by a young patient’s family—gives hope not only to a 4-year-old, but to researchers working on similar projects for other rare diseases.</div></a></div></div></div><div style="border-bottom:1px solid #CCCCCC" class="jsx-ad50481d5ee26850"></div></div><div class="jsx-ad50481d5ee26850 w-full h-full"><div class="jsx-ad50481d5ee26850 flex flex-col sm:flex-row justify-between my-4 "><a class="jsx-ad50481d5ee26850" href="/view/stem-cell-based-gene-editing-strategy-shows-promise-in-dmd"><img src="https://cdn.sanity.io/images/0vv8moc6/ajmc/df17be9ab7803f252c60bc1d6eea8586c27dcc08-3898x2598.jpg?fit=crop&auto=format" alt="Gene editing shows promise in Duchenne muscular dystrophy. | Image credit: Degimages - stock.adobe.com" width="288" class="jsx-ad50481d5ee26850 w-full xs:w-[288px] "/></a><div class="jsx-ad50481d5ee26850 article-detail flex flex-col gap-[0.2rem] w-full sm:w-[46%] md:w-[65%]"><span class="jsx-ad50481d5ee26850 font-bold article-publish-date block italic text-sm text-gray-500 mt-[1rem] sm:mt-0">June 29th 2024</span><p class="jsx-ad50481d5ee26850 article-title font-bold text-[1rem]"><a class="jsx-ad50481d5ee26850" href="/view/stem-cell-based-gene-editing-strategy-shows-promise-in-dmd">Stem Cell–Based Gene Editing Strategy Shows Promise in DMD</a></p><div class="jsx-ad50481d5ee26850 authors flex-row wrap gap-[0.2rem]"><a class="jsx-ad50481d5ee26850 text-[#000] text-sm italic" href="/authors/jared-kaltwasser">Jared Kaltwasser</a></div><div class="jsx-ad50481d5ee26850 article-summary"><a class="jsx-ad50481d5ee26850" href="/view/stem-cell-based-gene-editing-strategy-shows-promise-in-dmd"><div class="jsx-ad50481d5ee26850 text-sm text-gray-500 py-1">Early tests show stem cells can be used to spark expression of a miniature version of the dystrophin protein. </div></a></div></div></div><div style="border-bottom:1px solid #CCCCCC" class="jsx-ad50481d5ee26850"></div></div><div class="jsx-ad50481d5ee26850 w-full h-full"><div class="jsx-ad50481d5ee26850 flex flex-col sm:flex-row justify-between my-4 "><a class="jsx-ad50481d5ee26850" href="/view/fda-grants-2-approvals-to-delandistrogene-moxeparvovec-for-dmd"><img src="https://cdn.sanity.io/images/0vv8moc6/ajmc/34c5e95e8f78398a57d78153d1c62046bb968351-1200x628.jpg?fit=crop&auto=format" alt="Blue stamp of FDA approval | Image Credit: argus-stock.adobe.com" width="288" class="jsx-ad50481d5ee26850 w-full xs:w-[288px] "/></a><div class="jsx-ad50481d5ee26850 article-detail flex flex-col gap-[0.2rem] w-full sm:w-[46%] md:w-[65%]"><span class="jsx-ad50481d5ee26850 font-bold article-publish-date block italic text-sm text-gray-500 mt-[1rem] sm:mt-0">June 20th 2024</span><p class="jsx-ad50481d5ee26850 article-title font-bold text-[1rem]"><a class="jsx-ad50481d5ee26850" href="/view/fda-grants-2-approvals-to-delandistrogene-moxeparvovec-for-dmd">FDA Grants 2 Approvals to Delandistrogene Moxeparvovec for DMD</a></p><div class="jsx-ad50481d5ee26850 authors flex-row wrap gap-[0.2rem]"><a class="jsx-ad50481d5ee26850 text-[#000] text-sm italic" href="/authors/maggie-l-shaw">Maggie L. Shaw</a></div><div class="jsx-ad50481d5ee26850 article-summary"><a class="jsx-ad50481d5ee26850" href="/view/fda-grants-2-approvals-to-delandistrogene-moxeparvovec-for-dmd"><div class="jsx-ad50481d5ee26850 text-sm text-gray-500 py-1">Accelerated approval was originally granted in June 2023 for patients aged 4 to 5 years, indicating there was an unmet clinical need for a potentially life-saving treatment for the rare genetic muscle disorder.</div></a></div></div></div><div style="border-bottom:1px solid #CCCCCC" class="jsx-ad50481d5ee26850"></div></div><div class="jsx-ad50481d5ee26850 w-full h-full"><div><div><div class="text-[8px] text-center text-gray-500 hidden">Advertisement</div><div id="div-gpt-ad-infeed-25"></div></div></div><div class="jsx-ad50481d5ee26850 flex md:hidden justify-center items-center"></div><div class="jsx-ad50481d5ee26850 flex flex-col sm:flex-row justify-between my-4 "><a class="jsx-ad50481d5ee26850" href="/view/rett-syndrome-gene-therapy-granted-rmat-designation"><img src="https://cdn.sanity.io/images/0vv8moc6/ajmc/5a79382241da4877610a308e516bbd5b919a82b7-1280x720.jpg?fit=crop&auto=format" alt="Molecular engineering of DNA for Gene therapy | Image Credit: © immimagery - stock.adobe.com" width="288" class="jsx-ad50481d5ee26850 w-full xs:w-[288px] "/></a><div class="jsx-ad50481d5ee26850 article-detail flex flex-col gap-[0.2rem] w-full sm:w-[46%] md:w-[65%]"><span class="jsx-ad50481d5ee26850 font-bold article-publish-date block italic text-sm text-gray-500 mt-[1rem] sm:mt-0">June 12th 2024</span><p class="jsx-ad50481d5ee26850 article-title font-bold text-[1rem]"><a class="jsx-ad50481d5ee26850" href="/view/rett-syndrome-gene-therapy-granted-rmat-designation">Rett Syndrome Gene Therapy Granted RMAT Designation</a></p><div class="jsx-ad50481d5ee26850 authors flex-row wrap gap-[0.2rem]"><a class="jsx-ad50481d5ee26850 text-[#000] text-sm italic" href="/authors/victoria-johnson">Victoria Johnson</a></div><div class="jsx-ad50481d5ee26850 article-summary"><a class="jsx-ad50481d5ee26850" href="/view/rett-syndrome-gene-therapy-granted-rmat-designation"><div class="jsx-ad50481d5ee26850 text-sm text-gray-500 py-1">The FDA granted the Regenerative Medicine Advanced Therapy (RMAT) designation after reviewing the phase 1 safety and efficacy data.</div></a></div></div></div><div style="border-bottom:1px solid #CCCCCC" class="jsx-ad50481d5ee26850"></div></div><div class="jsx-ad50481d5ee26850 w-full h-full"><div class="jsx-ad50481d5ee26850 flex flex-col sm:flex-row justify-between my-4 "><a class="jsx-ad50481d5ee26850" href="/view/advances-in-colon-cancer-delivering-dual-mrna-with-lysate-based-nanoparticles"><img src="https://cdn.sanity.io/images/0vv8moc6/ajmc/f9707d6ef810913dca040b92861c37327ef0f9d7-1200x628.jpg?fit=crop&auto=format" alt="mRNA | Image Credit: ArtemisDiana-stock.adobe.com" width="288" class="jsx-ad50481d5ee26850 w-full xs:w-[288px] "/></a><div class="jsx-ad50481d5ee26850 article-detail flex flex-col gap-[0.2rem] w-full sm:w-[46%] md:w-[65%]"><span class="jsx-ad50481d5ee26850 font-bold article-publish-date block italic text-sm text-gray-500 mt-[1rem] sm:mt-0">June 11th 2024</span><p class="jsx-ad50481d5ee26850 article-title font-bold text-[1rem]"><a class="jsx-ad50481d5ee26850" href="/view/advances-in-colon-cancer-delivering-dual-mrna-with-lysate-based-nanoparticles">Advances in Colon Cancer: Delivering Dual-mRNA With Lysate-Based Nanoparticles</a></p><div class="jsx-ad50481d5ee26850 authors flex-row wrap gap-[0.2rem]"><a class="jsx-ad50481d5ee26850 text-[#000] text-sm italic" href="/authors/gayle-turim-dickstein">Gayle Turim Dickstein</a></div><div class="jsx-ad50481d5ee26850 article-summary"><a class="jsx-ad50481d5ee26850" href="/view/advances-in-colon-cancer-delivering-dual-mrna-with-lysate-based-nanoparticles"><div class="jsx-ad50481d5ee26850 text-sm text-gray-500 py-1">Building on prior research, a team tackled mouse-model colon cancer with a novel nonviral nanovector to carry dual–messenger RNA (mRNA) immunogene therapy.</div></a></div></div></div><div style="border-bottom:1px solid #CCCCCC" class="jsx-ad50481d5ee26850"></div></div><div class="jsx-ad50481d5ee26850 w-full h-full"><div class="jsx-ad50481d5ee26850 flex flex-col sm:flex-row justify-between my-4 "><a class="jsx-ad50481d5ee26850" href="/view/proof-of-concept-study-highlights-potential-of-gene-based-therapy-for-timothy-disease"><img src="https://cdn.sanity.io/images/0vv8moc6/ajmc/6cdd1ab5f025491ef555c6f3c9f111601845853b-1200x724.jpg?fit=crop&auto=format" alt="Genetic and gene manipulation concept | Image Credit: © ipopba - stock.adobe.com" width="288" class="jsx-ad50481d5ee26850 w-full xs:w-[288px] "/></a><div class="jsx-ad50481d5ee26850 article-detail flex flex-col gap-[0.2rem] w-full sm:w-[46%] md:w-[65%]"><span class="jsx-ad50481d5ee26850 font-bold article-publish-date block italic text-sm text-gray-500 mt-[1rem] sm:mt-0">June 2nd 2024</span><p class="jsx-ad50481d5ee26850 article-title font-bold text-[1rem]"><a class="jsx-ad50481d5ee26850" href="/view/proof-of-concept-study-highlights-potential-of-gene-based-therapy-for-timothy-disease">Proof-of-Concept Study Highlights Potential of Gene-Based Therapy for Timothy Disease</a></p><div class="jsx-ad50481d5ee26850 authors flex-row wrap gap-[0.2rem]"><a class="jsx-ad50481d5ee26850 text-[#000] text-sm italic" href="/authors/ajmc-contributor">AJMC Contributor</a></div><div class="jsx-ad50481d5ee26850 article-summary"><a class="jsx-ad50481d5ee26850" href="/view/proof-of-concept-study-highlights-potential-of-gene-based-therapy-for-timothy-disease"><div class="jsx-ad50481d5ee26850 text-sm text-gray-500 py-1">The proof-of-concept study showed that antisense oligonucleotides developed by the researchers successfully restored cellular development and brain cell function in patients with the disease characterized by multiorgan dysfunction.</div></a></div></div></div><div style="border-bottom:1px solid #CCCCCC" class="jsx-ad50481d5ee26850"></div></div><div class="jsx-ad50481d5ee26850 w-full h-full"><div class="jsx-ad50481d5ee26850 flex flex-col sm:flex-row justify-between my-4 "><a class="jsx-ad50481d5ee26850" href="/view/gene-therapy-offers-promise-in-treating-inborn-errors-of-immunity"><img src="https://cdn.sanity.io/images/0vv8moc6/ajmc/a4049ec21ae36544bfa87b9df4b04bb0f18a1d38-1200x724.jpg?fit=crop&auto=format" alt="CRISPR DNA Editor | Image Credit: NathanDeverycom-stock.adobe.com" width="288" class="jsx-ad50481d5ee26850 w-full xs:w-[288px] "/></a><div class="jsx-ad50481d5ee26850 article-detail flex flex-col gap-[0.2rem] w-full sm:w-[46%] md:w-[65%]"><span class="jsx-ad50481d5ee26850 font-bold article-publish-date block italic text-sm text-gray-500 mt-[1rem] sm:mt-0">May 20th 2024</span><p class="jsx-ad50481d5ee26850 article-title font-bold text-[1rem]"><a class="jsx-ad50481d5ee26850" href="/view/gene-therapy-offers-promise-in-treating-inborn-errors-of-immunity">Gene Therapy Offers Promise in Treating Inborn Errors of Immunity</a></p><div class="jsx-ad50481d5ee26850 authors flex-row wrap gap-[0.2rem]"><a class="jsx-ad50481d5ee26850 text-[#000] text-sm italic" href="/authors/jared-kaltwasser">Jared Kaltwasser</a></div><div class="jsx-ad50481d5ee26850 article-summary"><a class="jsx-ad50481d5ee26850" href="/view/gene-therapy-offers-promise-in-treating-inborn-errors-of-immunity"><div class="jsx-ad50481d5ee26850 text-sm text-gray-500 py-1">A new report says early evidence of using gene therapy to treat monogenic diseases offers reason for optimism.</div></a></div></div></div><div style="border-bottom:1px solid #CCCCCC" class="jsx-ad50481d5ee26850"></div></div><div class="jsx-ad50481d5ee26850 w-full h-full"><div class="jsx-ad50481d5ee26850 flex flex-col sm:flex-row justify-between my-4 "><a class="jsx-ad50481d5ee26850" href="/view/valoctocogene-roxaparvovec-dramatically-reduces-bleeding-in-hemophilia-a"><img src="https://cdn.sanity.io/images/0vv8moc6/ajmc/f8510957779b2631356ec6e55fe7d580e5d694f6-1200x724.jpg?fit=crop&auto=format" alt="Hemophilia definition | Image Credit: VitezslavVylicil-stock.adobe.com" width="288" class="jsx-ad50481d5ee26850 w-full xs:w-[288px] "/></a><div class="jsx-ad50481d5ee26850 article-detail flex flex-col gap-[0.2rem] w-full sm:w-[46%] md:w-[65%]"><span class="jsx-ad50481d5ee26850 font-bold article-publish-date block italic text-sm text-gray-500 mt-[1rem] sm:mt-0">May 15th 2024</span><p class="jsx-ad50481d5ee26850 article-title font-bold text-[1rem]"><a class="jsx-ad50481d5ee26850" href="/view/valoctocogene-roxaparvovec-dramatically-reduces-bleeding-in-hemophilia-a">Valoctocogene Roxaparvovec Dramatically Reduces Bleeding in Hemophilia A</a></p><div class="jsx-ad50481d5ee26850 authors flex-row wrap gap-[0.2rem]"><a class="jsx-ad50481d5ee26850 text-[#000] text-sm italic" href="/authors/kimberly-rath-pharmd">Kimberly Rath, PharmD</a></div><div class="jsx-ad50481d5ee26850 article-summary"><a class="jsx-ad50481d5ee26850" href="/view/valoctocogene-roxaparvovec-dramatically-reduces-bleeding-in-hemophilia-a"><div class="jsx-ad50481d5ee26850 text-sm text-gray-500 py-1">New study findings demonstrate that valoctocogene roxaparvovec, a gene therapy for severe hemophilia A, significantly reduces bleeding episodes and dependency on factor VIII infusions over a 3-year period, while maintaining a favorable safety profile. </div></a></div></div></div><div style="border-bottom:1px solid #CCCCCC" class="jsx-ad50481d5ee26850"></div></div><div class="jsx-ad50481d5ee26850 w-full h-full"><div class="jsx-ad50481d5ee26850 flex flex-col sm:flex-row justify-between my-4 "><a class="jsx-ad50481d5ee26850" href="/view/new-strategy-shows-promise-for-cd7-positive-leukemia-lymphoma"><img src="https://cdn.sanity.io/images/0vv8moc6/ajmc/04663c88d2d04992175ced53fda8bb885d2d2ee8-1200x724.jpg?fit=crop&auto=format" alt="3D rendering of a CAR T cell | Image Credit: Naeblys-stock.adobe.com" width="288" class="jsx-ad50481d5ee26850 w-full xs:w-[288px] "/></a><div class="jsx-ad50481d5ee26850 article-detail flex flex-col gap-[0.2rem] w-full sm:w-[46%] md:w-[65%]"><span class="jsx-ad50481d5ee26850 font-bold article-publish-date block italic text-sm text-gray-500 mt-[1rem] sm:mt-0">May 9th 2024</span><p class="jsx-ad50481d5ee26850 article-title font-bold text-[1rem]"><a class="jsx-ad50481d5ee26850" href="/view/new-strategy-shows-promise-for-cd7-positive-leukemia-lymphoma">New Strategy Shows Promise for CD7-Positive Leukemia/Lymphoma</a></p><div class="jsx-ad50481d5ee26850 authors flex-row wrap gap-[0.2rem]"><a class="jsx-ad50481d5ee26850 text-[#000] text-sm italic" href="/authors/kimberly-rath-pharmd">Kimberly Rath, PharmD</a></div><div class="jsx-ad50481d5ee26850 article-summary"><a class="jsx-ad50481d5ee26850" href="/view/new-strategy-shows-promise-for-cd7-positive-leukemia-lymphoma"><div class="jsx-ad50481d5ee26850 text-sm text-gray-500 py-1">This study introduces a novel "all-in-one" strategy combining CD7 chimeric antigen receptor T-cell therapy with haploidentical hematopoietic stem-cell transplantation, showing significant potential to improve survival and reduce toxicities in patients with relapsed or refractory CD7-positive hematologic cancers. </div></a></div></div></div><div style="border-bottom:1px solid #CCCCCC" class="jsx-ad50481d5ee26850"></div></div></div><div class="w-full text-center flex justify-center pb-24"><a class="px-4 py-2 border-y border-r bg-primary text-white" href="/compendium/gene-therapy?page=1">1</a><a class="px-4 py-2 border-y border-r " href="/compendium/gene-therapy?page=2">2</a><a class="px-4 py-2 border-y border-r " href="/compendium/gene-therapy?page=3">3</a><a class="px-4 py-2 border-y border-r" 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And oftentimes, [we're] trying to find a way of replacing the full length of the dystrophin gene, as opposed to micro-dystrophin or mini-dystrophin—so, finding alternative methods of having perhaps viral vectors that could replace parts of it, and then they'll assemble back together intracellularly again, or finding other ways of having gene therapy delivered without viral vectors that don't activate the immune system.","_key":"910e88bf611f","_type":"span","marks":[]}],"_type":"block","style":"normal","_key":"240e55f05547"},{"children":[{"_type":"span","marks":[],"text":"","_key":"be13c8b5b82e"}],"_type":"block","style":"normal","_key":"09a23ae955e1","markDefs":[]},{"markDefs":[],"children":[{"text":"I think those will be areas for future research that are really, really promising, as well as other combinations of therapies—not just the steroids, but nonsteroidal things at the cellular level, working on regenerating muscle for the patients that have already had some of that muscle [loss] before they receive their gene therapy. 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That's the question that remains unanswered that we're trying to navigate.","_key":"0b54aa36fc301","_type":"span"}],"_type":"block","style":"normal","_key":"23ae4d39b98c"},{"children":[{"text":"","_key":"ddc3846352950","_type":"span","marks":[]}],"_type":"block","style":"normal","_key":"4f8829f40ea5","markDefs":[]},{"_key":"b6271759605a","markDefs":[],"children":[{"_type":"span","marks":[],"text":"Then tied to that is: What if they are and what if they aren't? Should it wear off over time, and you have to go back to the standard of care today, what does that mean for that treatment that was administered? What does that mean for the payer? What does that mean for the patient? What does that mean for the manufacturer? And those are questions that are unanswered today. Those are the things that ongoing discussions are going to have to take place. Some kind of agreement should be decided upon between the parties involved. And, as of yet, we don't have that. But those are the questions that are out there that we need to be talking about and looking to answer as more time comes on and more information is available with this real-world evidence.","_key":"fdd13bb357d80"}],"_type":"block","style":"normal"},{"markDefs":[],"children":[{"_key":"6a7796dd97d30","_type":"span","marks":[],"text":""}],"_type":"block","style":"normal","_key":"253cde94c522"},{"_type":"block","style":"normal","_key":"e66a93617b76","markDefs":[],"children":[{"_key":"1139718793450","_type":"span","marks":["strong"],"text":"Theoretically, should payers find these therapies attractive despite the high price tag because they lead to avoidance of other costs?"}]},{"style":"normal","_key":"d11384354255","markDefs":[],"children":[{"_type":"span","marks":["strong"],"text":"Booth: ","_key":"72886accbf5e0"},{"text":"Yeah, because we're taking into consideration [emergency department] visits, hospitalization, home care. 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