American Society of Gene & Cell Therapy

Thank you, ASGCT Members, for all of your support this year! Our offices will be closed Dec. 23-Jan. 1 for the holiday break. Email support will be available in emergency circumstances, but the great majority of inquiries will be immediately addressed when we're back in the office on Thursday, Jan. 2.   While we're out of office, here are a few ways you can be prepared for the new year at ASGCT: - Renew your ASGCT membership to make sure you're maintaining benefits next year - Register for #ASGCT2025 early for the best rates - Submit an abstract for the opportunity to present it in front of 8,000+ colleagues at the 28th Annual Meeting We'll see you in 2025!

BREAKING: FDA approved #Tryngolza (also known as #Olezarsen) an investigational, first-in-class, RNA-targeted medicine for the treatment of adults with familial chylomicronemia syndrome (FCS). FCS is a rare genetic disease that prevents the body from breaking down fats, which results in extremely high triglyceride levels. Read our statement from ASGCT President Paula Cannon on our website. https://lnkd.in/gMAxBnps #rarediseases

Yesterday, #Ryoncil became the first FDA-approved mesenchymal stromal #celltherapy indicated to treat steroid-refractory acute graft-versus-host disease (SR-aGVHD) in patients who are two months and older. SR-aGVHD is a potentially life-threatening complication that can occur after a patient receives an allogeneic #stemcell transplant. Read FDA's statement on the approval to learn more about Ryoncil. https://lnkd.in/eH63UdNn

Paul Edmonds lived with #HIV for decades before he became one of a few people worldwide to be cured (City of Hope). His story reminds us why research and patient voices are so critical. Hear his story and the cutting-edge science ASGCT members are working on. Read this blog post from ASGCT President Paula Cannon, PhD, and watch our recent Congressional briefing on #genetherapy + #celltherapy research for HIV. https://lnkd.in/gwsCcbZN #ASGCTadvocacy 

The December issue of The Patient Press is OUT NOW! This month, we're introducing two brand-new #PatientEducation units on #genetherapy for genetic hearing loss and for ATTR amyloidosis. You can also read about upcoming events, like next month's ASGCT + FDA AAV workshop, the Empowering Patients Summit in March, and patient advocate opportunities at #ASGCT2025! Check it out + share with your network! https://lnkd.in/gH__TBWS

💡 Happening today at 1 p.m. CT: Focused Ultrasound: Cutting-Edge Applications in Cell and Gene Therapies Join ASGCT & the Focused Ultrasound Foundation to discover how this groundbreaking technology is revolutionizing therapeutic delivery. Learn from leading experts about latest developments and real-world applications. Expand your research horizons and connect with innovators in the field. Check out the comments on this post to see the event in your time zone and register to view the event live or on-demand!

📰 The ASGCT Communications Committee created a newsletter! Let us know what you think of the inaugural issue + what else you'd like to read in the future. #scicomm #genetherapy #celltherapy

We can't wait to welcome you to New Orleans for #ASGCT2025! Register NOW to lock in your spot May 13-17 and explore the lastest breakthroughs in CGT. If you're an Associate Member and a first and presenting abstract author you may be eligible for FREE registration! Have #genetherapy or #celltherapy research to share? We're accepting abstracts through Jan. 31 and we'd love to see what you're working on. Submit your work for the opportunity to win an award, be published in #MolecularTherapy, and present in New Orleans for 8,000 of the field's professionals. https://lnkd.in/eKiSKA4

Have you subscribed to The Patient Press? The second issue of this new #PatientEducation-focused newsletter is coming out next week; sign up to get it straight to your inbox! Upcoming highlights in the December issue include: --New units on Genetic #HearingLoss + #TransthyretinAmyloidosis (ATTR) --World AIDS Day --Plain language summaries from #ASGCTBreakthroughs24 Read + subscribe on our website. https://lnkd.in/gcGRa-tt #ASGCTadvocacy

ASGCT thanks Centers for Medicare & Medicaid Services for finalizing important proposals in the proposed 2025 Hospital Outpatient Prospective Payment System rule. In the draft rule, CMS proposed to exclude qualifying cell and gene therapies (CGTs) from C-APC packaging for one-year. ASGCT supported CMS’ proposal to exclude CGTs from C-APC packaging methodology, and suggested the change be made indefinitely. Therefore, we were pleased to see CMS accept our recommendation and extended indefinitely the changes to excluded CGTs from C-APC packaging methodology. We look forward to continued collaboration with CMS to ensure patient access to these safe and efficacious therapies. Read the comments on our website. #ASGCTAdvoacy #PatientAccess https://lnkd.in/g6EBabbi