American Society of Gene & Cell Therapy

📰 Check out the latest issue of ASGCT Insider, the newsletter from ASGCT's Communications Committee! In this issue, read about the FDA approval of #Encelto, the first and only treatment for MacTel type 2, as well as a collection of journal articles and clinical development news. Thank you to this month's contributors: Jon Brudvig, PhD; Christopher Leidli, MS, MBA; Tsai-Yu Lin, PhD; Florie Borel, PhD; and Benjamin McLeod

🔜 #ASGCT2025 in #NOLA is just a few weeks away and we can't wait! Thanks for the shout out, New Orleans & Company! What else will you discover in New Orleans this May? View the full video + make sure you register to join us. #celltherapy #genetherapy #annualmeeting https://lnkd.in/gKwcfrWm

🩸 March is #HemophiliaAwarenessMonth! Hemophilia is a genetic disease that prevents blood from clotting properly, leading to prolonged internal and external bleeding. Recent #CGT approvals and ongoing research are opening new possibilities for better treatment options and improved quality of life. Learn more about how #genetherapy is changing the trajectory of hemophilia care: https://lnkd.in/gj5UWGW2

𝗝𝗼𝗶𝗻 𝘂𝘀 𝗳𝗼𝗿 𝗔𝗦𝗚𝗖𝗧'𝘀 𝗠𝗼𝗹𝗲𝗰𝘂𝗹𝗮𝗿 𝗧𝗵𝗲𝗿𝗮𝗽𝘆 𝗣𝗿𝗲𝘀𝗲𝗻𝘁𝘀: 𝟮𝟱𝘁𝗵 𝗔𝗻𝗻𝗶𝘃𝗲𝗿𝘀𝗮𝗿𝘆 𝗦𝗽𝗲𝗰𝗶𝗮𝗹 𝗜𝘀𝘀𝘂𝗲 𝗹𝗶𝘃𝗲 𝘄𝗲𝗯𝗶𝗻𝗮𝗿! We are excited to invite you to a special webinar celebrating the 25th anniversary of 𝘔𝘰𝘭𝘦𝘤𝘶𝘭𝘢𝘳 𝘛𝘩𝘦𝘳𝘢𝘱𝘺, ASGCT's flagship scientific journal. Since its launch on January 1, 2000, Molecular Therapy has grown to become the premier destination for groundbreaking research in cell and gene therapy, achieving an impact factor of over 10 in 2020. 📅 Wednesday, April 23rd  🕚 11:00 a.m. CT 𝗝𝗼𝗶𝗻 𝗘𝗱𝗶𝘁𝗼𝗿-𝗶𝗻-𝗖𝗵𝗶𝗲𝗳 𝗗𝗿. 𝗝𝗼𝘀𝗲𝗽𝗵 𝗖. 𝗚𝗹𝗼𝗿𝗶𝗼𝘀𝗼 𝗜𝗜𝗜 𝗮𝗻𝗱 𝗱𝗶𝘀𝘁𝗶𝗻𝗴𝘂𝗶𝘀𝗵𝗲𝗱 𝗽𝗮𝗻𝗲𝗹𝗶𝘀𝘁𝘀 𝗳𝗼𝗿 𝘁𝗵𝗲𝘀𝗲 𝗹𝗶𝘃𝗲, 𝗶𝗻𝘀𝗶𝗴𝗵𝘁𝗳𝘂𝗹 𝗽𝗿𝗲𝘀𝗲𝗻𝘁𝗮𝘁𝗶𝗼𝗻𝘀: • Dr. Terry Flotte on "Gene Therapy Then and Now: A Look Back at Changes in the Field Over the Past 25 Years" • Dr. R. Jude Samulski on "AAV Vector Development, Back to the Future" • Dr. Nour Shobaki on "From Concept to Cure: The Evolution of CAR-T Cell Therapy" Don't miss this opportunity to hear live from leaders who have shaped the CGT field over the past quarter-century! https://lnkd.in/eiy85pPU #GeneTherapy #CellTherapy #ASGCT #MolecularTherapy

What we're reading: MIT engineers turn skin cells directly into neurons for cell therapy, written by Anne Trafton | MIT News "A new, highly efficient process for performing this conversion could make it easier to develop therapies for spinal cord injuries or diseases like ALS." Read more here: https://lnkd.in/eJq4h6cj

📢 Have you voted yet? ASGCT Election voting is open through April 11 for all current Members and Transitional Members! https://lnkd.in/gyb5Qk23

❓ Did you know? There are different types of #amyloidosis, each caused by abnormal protein buildup in the body. One type is known as ATTR amyloidosis which can be passed on genetically or be caused by age related factors. • Hereditary amyloidosis: the TTR gene variant is passed along from the parents • Wild-type ATTR amyloidosis: caused by age-related factors #Genetherapy for ATTR amyloidosis aims to limit production of amyloid deposits or amyloid buildup in the body by limiting the production of the TTR protein. Gene therapies are being explored with a few approaches to treat ATTR: • Gene Editing: Prevents TTR protein production by removing the TTR gene from DNA. This one-time, in vivo treatment is delivered directly into the body using a viral vector. Scientists modify these vectors to ensure only beneficial genetic material reaches target cells. • Gene Silencers: An FDA-approved treatment uses gene silencers like siRNA and ASOs to block TTR protein production, preventing harmful amyloid deposits. This helps manage nerve damage (polyneuropathy) in hereditary ATTR amyloidosis (hATTR-PN). Learn more and help raise awareness this Amyloidosis Awareness Month! #PatientEducation https://lnkd.in/gQkgGiPh

We're grateful to Rep. Jake Auchincloss’ office for the thoughtful conversation about robust #NIH research funding and its critical role in developing breakthrough #CGTs. Your support for the research ecosystem helps ensure America remains at the forefront of biomedical innovation. #ASGCTadvocacy 

With a record number of abstracts submitted for #ASGCT2025, this Annual Meeting promises to be another exceptional opportunity for attendees to learn from leading researchers across the cell and gene therapy community. #ASGCT2025 abstracts will be released online on April 28, 2025 (4:30 p.m. ET). ✨ Join us May 13-17 in #NewOrleans or virtually for the premier #CGT event of the year! Register here: https://lnkd.in/d6busdKn #celltherapy #genetherapy #NOLA

We appreciate Rep. Richard Neal’s office for the opportunity to discuss how sustained NIH research funding strengthens the innovation pipeline for cell and gene therapies. Your support helps ensure greater patient access and global competitiveness. #ASGCTadvocacy