American Society of Gene + Cell Therapy
@asgct.bsky.social
Advancing knowledge, awareness, and education leading to the discovery and clinical application of genetic and cellular therapies to alleviate human disease.
🌎 Join us on Tuesday, Dec. 16, for the next installment of our Around the World webinar series! Guilherme Baldo, Ph.D., of Universidade Federal do Rio Grande do Sul, moderates a deep dive into South American CGT frontiers. Attend at no cost: https://bit.ly/489b0mT
December 12, 2025 at 7:26 PM
🌎 Join us on Tuesday, Dec. 16, for the next installment of our Around the World webinar series! Guilherme Baldo, Ph.D., of Universidade Federal do Rio Grande do Sul, moderates a deep dive into South American CGT frontiers. Attend at no cost: https://bit.ly/489b0mT
FDA approves an expanded indication for Breyanzi, the first CAR T-cell therapy for relapsed/refractory marginal zone lymphoma. The CD19-directed therapy offers a one-time treatment for patients. Read more: https://bit.ly/48JGyzS
Bristol Myers Squibb’s Application for Breyanzi Accepted for Priority Review by U.S. Food and Drug Administration (FDA) in Fifth Cancer Type for Relapsed or Refractory Margi...
Breyanzi has the potential to be the first and only CAR T cell therapy approved for MZL, addressing a high unmet need Application based on results from MZL cohort of TRANSCEND FL presented at ICML in ...
bit.ly
December 11, 2025 at 9:33 PM
FDA approves an expanded indication for Breyanzi, the first CAR T-cell therapy for relapsed/refractory marginal zone lymphoma. The CD19-directed therapy offers a one-time treatment for patients. Read more: https://bit.ly/48JGyzS
"Multiple sessions established that precise genomic modification in various tissues is transitioning from an experimental concept to a clinical reality." – Kiran Musunuru, MD, Ph.D.
Read more + access recordings from the live event: https://bit.ly/3YmtziR
Read more + access recordings from the live event: https://bit.ly/3YmtziR
Celebrating Breakthrough Moments: Highlights from ASGCT Breakthroughs…
ASGCT’s 2025 Breakthroughs in In Vivo Gene Editing Conference delivered precisely what its name promised: breakthrough moments that are reshaping our…
www.asgct.org
December 11, 2025 at 5:14 PM
"Multiple sessions established that precise genomic modification in various tissues is transitioning from an experimental concept to a clinical reality." – Kiran Musunuru, MD, Ph.D.
Read more + access recordings from the live event: https://bit.ly/3YmtziR
Read more + access recordings from the live event: https://bit.ly/3YmtziR
The @fda.gov has approved Waskyra, the first gene therapy for Wiskott-Aldrich syndrome and a major milestone for nonprofit-led development. Clinical trials showed sharp drops in severe infections and bleeding. ASGCT supports solutions to help these therapies reach patients. https://bit.ly/48RoGDj
In a first, FDA approves a gene therapy from a nonprofit
The FDA on Tuesday approved Waskyra for a rare immune disease called Wiskott-Aldrich syndrome, for which patients have little options besides a bone marrow transplant.
endpoints.news
December 10, 2025 at 10:57 PM
The @fda.gov has approved Waskyra, the first gene therapy for Wiskott-Aldrich syndrome and a major milestone for nonprofit-led development. Clinical trials showed sharp drops in severe infections and bleeding. ASGCT supports solutions to help these therapies reach patients. https://bit.ly/48RoGDj
Your research deserves the spotlight! 🌟 The ASGCT Annual Meeting offers a crucial opportunity to showcase your research to a prestigious global audience, boosting the visibility of your work and helping you forge career-shaping connections. Learn more: https://bit.ly/3KmVlbP
December 10, 2025 at 6:43 PM
Your research deserves the spotlight! 🌟 The ASGCT Annual Meeting offers a crucial opportunity to showcase your research to a prestigious global audience, boosting the visibility of your work and helping you forge career-shaping connections. Learn more: https://bit.ly/3KmVlbP
We recently hosted a Congressional briefing on #GeneTherapy approaches for #HIV with experts from @usc.edu, @ohsunews.bsky.social, and patient advocates. Read our recap to learn how decades of NIH-funded research is bringing us closer to a cure: https://bit.ly/4oJbLt4 #ASGCTAdvocacy
ASGCT Briefs Congress on Gene Therapies to Treat HIV
Today, more than 1.2 million Americans live with HIV. Gene and cell therapy approaches hold the potential to deliver one-time therapies that could free…
www.asgct.org
December 9, 2025 at 5:23 PM
We recently hosted a Congressional briefing on #GeneTherapy approaches for #HIV with experts from @usc.edu, @ohsunews.bsky.social, and patient advocates. Read our recap to learn how decades of NIH-funded research is bringing us closer to a cure: https://bit.ly/4oJbLt4 #ASGCTAdvocacy
Huge thanks to @coons.senate.gov for helping spotlight how gene therapy is shifting HIV treatment from lifelong management toward durable, potentially curative outcomes. We appreciate your commitment to advancing biomedical research. #ASGCTadvocacy
December 5, 2025 at 7:21 PM
Huge thanks to @coons.senate.gov for helping spotlight how gene therapy is shifting HIV treatment from lifelong management toward durable, potentially curative outcomes. We appreciate your commitment to advancing biomedical research. #ASGCTadvocacy
📢 Registration for the ASGCT 2026 Annual Meeting is officially open! Secure your spot to join thousands of cell and gene therapy experts in Boston, May 11-15, 2026. Register today: https://annualmeeting.asgct.org/register-attend/rates-registration
December 3, 2025 at 7:18 PM
📢 Registration for the ASGCT 2026 Annual Meeting is officially open! Secure your spot to join thousands of cell and gene therapy experts in Boston, May 11-15, 2026. Register today: https://annualmeeting.asgct.org/register-attend/rates-registration
Join us this Thursday for a FREE webinar exploring the evolution of aptamers, from their initial discovery to their current role as precise, versatile tools surpassing antibodies in diagnostic and therapeutic applications. Register: https://bit.ly/3XLi7Np
Molecular Therapy Presents: Innovations in Aptamer Technology
Thursday, December 4 at 1 p.m. CT
www.asgct.org
December 1, 2025 at 3:05 PM
Join us this Thursday for a FREE webinar exploring the evolution of aptamers, from their initial discovery to their current role as precise, versatile tools surpassing antibodies in diagnostic and therapeutic applications. Register: https://bit.ly/3XLi7Np
FDA approves Itvisma, expanding access to gene therapy for spinal muscular atrophy. The one-time intrathecal treatment is approved for patients two years and older – the first gene replacement therapy available for this broader SMA population. Read more: https://bit.ly/3Kn9mpM
FDA Approves Gene Therapy for Treatment of Spinal Muscular Atrophy
FDA Approves Gene Therapy for Treatment of Spinal Muscular Atrophy
www.fda.gov
November 28, 2025 at 7:30 PM
FDA approves Itvisma, expanding access to gene therapy for spinal muscular atrophy. The one-time intrathecal treatment is approved for patients two years and older – the first gene replacement therapy available for this broader SMA population. Read more: https://bit.ly/3Kn9mpM
ASGCT is pleased to see two CGT products recognized with the Commissioner’s National Priority Voucher (CNVP). We continue to advocate for safe and effective regulatory frameworks. Read a summary of our recent comment letters on other FDA initiatives: https://bit.ly/43Tm5qP
ASGCT Comments on Key FDA Initiatives Supporting Cell and Gene…
ASGCT recently submitted three comment letters to the US Food and Drug Administration (FDA) focused on advancing regulatory flexibility and modernization…
www.asgct.org
November 26, 2025 at 4:38 PM
ASGCT is pleased to see two CGT products recognized with the Commissioner’s National Priority Voucher (CNVP). We continue to advocate for safe and effective regulatory frameworks. Read a summary of our recent comment letters on other FDA initiatives: https://bit.ly/43Tm5qP
Last week, the FDA approved Plozasiran as an adjunct to diet to reduce triglycerides in adults with familial chylomicronemia syndrome (FCS). This RNAi therapy represents a new treatment option for patients with FCS, a severe and rare genetic disease. Read more: https://bit.ly/47XTSlc
FDA approves drug to reduce triglycerides in adults with familial chyl
FDA approves drug to reduce triglycerides in adults with familial chylomicronemia syndrome
www.fda.gov
November 25, 2025 at 2:45 PM
Last week, the FDA approved Plozasiran as an adjunct to diet to reduce triglycerides in adults with familial chylomicronemia syndrome (FCS). This RNAi therapy represents a new treatment option for patients with FCS, a severe and rare genetic disease. Read more: https://bit.ly/47XTSlc
Master CGT fundamentals in our new FREE online course! Learn the essentials – from CRISPR to clinical trial design and more – on your own schedule. Visit the ASGCT Learning Center to enroll today: https://bit.ly/44bFKCj
November 24, 2025 at 6:43 PM
Master CGT fundamentals in our new FREE online course! Learn the essentials – from CRISPR to clinical trial design and more – on your own schedule. Visit the ASGCT Learning Center to enroll today: https://bit.ly/44bFKCj
FDA announces first nine recipients of Commissioner's National Priority Voucher program – including DB-OTO, a gene therapy for genetic hearing loss! The program aims to cut review times from 10-12 months to 1-2 months. More in The Advocate: https://bit.ly/4pr8Bek #ASGCTadvocacy
The Advocate: November 2025
Senate HELP Committee highlights CGT breakthroughs like sickle cell treatments as examples of U.S. biotech leadership – while raising concerns about…
bit.ly
November 21, 2025 at 3:05 PM
FDA announces first nine recipients of Commissioner's National Priority Voucher program – including DB-OTO, a gene therapy for genetic hearing loss! The program aims to cut review times from 10-12 months to 1-2 months. More in The Advocate: https://bit.ly/4pr8Bek #ASGCTadvocacy
Thanks to all who kicked off Breakthroughs in Targeted In Vivo Gene Editing with us today – what an incredible day of cutting-edge science and insights in this exciting field!
November 21, 2025 at 2:20 AM
Thanks to all who kicked off Breakthroughs in Targeted In Vivo Gene Editing with us today – what an incredible day of cutting-edge science and insights in this exciting field!
As cell and gene therapies revolutionize modern medicine, South America is emerging as a dynamic player on the global stage. Join this FREE webinar exploring South American technological frontiers: https://bit.ly/489b0mT
Around the World: Technological Frontiers in South American Gene and…
As gene and cell therapies revolutionize modern medicine, South America is emerging as a dynamic player on the global stage—leveraging scientific…
bit.ly
November 20, 2025 at 3:15 PM
As cell and gene therapies revolutionize modern medicine, South America is emerging as a dynamic player on the global stage. Join this FREE webinar exploring South American technological frontiers: https://bit.ly/489b0mT
"Our moment is now. It's now that we have the very first tools to start asking, what happens if we deliver gene therapy to the whole brain? Can we get this done?"
Watch our latest Community Quick Take with Dr. Sonia Vallabh on gene editing for prion disease: https://bit.ly/3X4YPCp
Watch our latest Community Quick Take with Dr. Sonia Vallabh on gene editing for prion disease: https://bit.ly/3X4YPCp
November 19, 2025 at 9:25 PM
"Our moment is now. It's now that we have the very first tools to start asking, what happens if we deliver gene therapy to the whole brain? Can we get this done?"
Watch our latest Community Quick Take with Dr. Sonia Vallabh on gene editing for prion disease: https://bit.ly/3X4YPCp
Watch our latest Community Quick Take with Dr. Sonia Vallabh on gene editing for prion disease: https://bit.ly/3X4YPCp
Secure your livestream access and unlock one year of on-demand recordings! 🔓 Don't miss out on this must-attend gene editing event: asgct.org/breakthroughs
November 19, 2025 at 6:40 PM
Secure your livestream access and unlock one year of on-demand recordings! 🔓 Don't miss out on this must-attend gene editing event: asgct.org/breakthroughs
Recent news reports highlighting commercial efforts to normalize heritable human genome editing (HHGE) underscore the urgent need for sustained vigilance, ethical clarity, and enforceable global governance. Read more: https://bit.ly/4r8iIGt
Global Scientific Consortium Reaffirms Position on Human Heritable…
Recent news reports highlighting commercial efforts to normalize heritable human genome editing (HHGE) underscore the urgent need for sustained vigilance,…
www.asgct.org
November 18, 2025 at 10:52 PM
Recent news reports highlighting commercial efforts to normalize heritable human genome editing (HHGE) underscore the urgent need for sustained vigilance, ethical clarity, and enforceable global governance. Read more: https://bit.ly/4r8iIGt
Can't travel to San Diego? Join our targeted gene editing event via livestream and get one year of on-demand access! 📱 Register: asgct.org/breakthroughs
November 17, 2025 at 5:05 PM
Can't travel to San Diego? Join our targeted gene editing event via livestream and get one year of on-demand access! 📱 Register: asgct.org/breakthroughs
We’d love to see you next week at Breakthroughs in Targeted In Vivo Gene Editing – in San Diego or online! You’ll hear new research from CGT leaders, including these featured speakers and many more: asgct.org/breakthroughs
November 13, 2025 at 4:07 PM
We’d love to see you next week at Breakthroughs in Targeted In Vivo Gene Editing – in San Diego or online! You’ll hear new research from CGT leaders, including these featured speakers and many more: asgct.org/breakthroughs
Check out the Q3 Landscape Report for the latest in the therapeutics pipeline, clinical targets, developer progress, and more! 🧬 Read the full report: https://www.asgct.org/news-publications/landscape-report
November 12, 2025 at 7:06 PM
Check out the Q3 Landscape Report for the latest in the therapeutics pipeline, clinical targets, developer progress, and more! 🧬 Read the full report: https://www.asgct.org/news-publications/landscape-report
The latest episode of the Molecular Therapy podcast is out now! This in-depth conversation focuses on an article recently published in @moltherapy.bsky.social titled “Efficient in vivo generation of CAR T cells using a retargeted fourth-generation lentiviral vector.”
🎙️Listen: https://bit.ly/43o18UG
🎙️Listen: https://bit.ly/43o18UG
Generating CAR T cells in vivo with lentiviral vectors with… | ASGCT
Editor-in-Chief of Molecular Therapy, Dr. Joseph Glorioso, is joined by Rachael Nimmo, PhD, and Kyriacos Mitrophanous, PhD, to discuss “Efficient in vivo…
bit.ly
November 11, 2025 at 8:16 PM
The latest episode of the Molecular Therapy podcast is out now! This in-depth conversation focuses on an article recently published in @moltherapy.bsky.social titled “Efficient in vivo generation of CAR T cells using a retargeted fourth-generation lentiviral vector.”
🎙️Listen: https://bit.ly/43o18UG
🎙️Listen: https://bit.ly/43o18UG
The #ASGCT2026 abstract submission window is officially OPEN! Present your research live in Boston, get published in @moltherapy.bsky.social, and network with global cell & gene therapy experts. Don’t miss this opportunity: https://bit.ly/3LVBtg0
Submission Information
Lean how and when to submit your abstracts, how abstracts are reviewed and selected, and how you'll be notified if your abstract is accepted.
annualmeeting.asgct.org
November 11, 2025 at 4:50 PM
The #ASGCT2026 abstract submission window is officially OPEN! Present your research live in Boston, get published in @moltherapy.bsky.social, and network with global cell & gene therapy experts. Don’t miss this opportunity: https://bit.ly/3LVBtg0
The Empowering Patients Summit is back! 🎉 Join us March 11 + 12 for this free virtual event to gain the latest insights and be inspired by powerful stories from the patient community. https://bit.ly/4hGwXOh
Empowering Patients 2026: A Cell and Gene Therapies Summit
This free, virtual Summit will bring together patients, caregivers, advocates, researchers, and industry leaders for two dynamic half-days of learning the…
www.asgct.org
November 7, 2025 at 7:45 PM
The Empowering Patients Summit is back! 🎉 Join us March 11 + 12 for this free virtual event to gain the latest insights and be inspired by powerful stories from the patient community. https://bit.ly/4hGwXOh