American Society of Gene & Cell Therapy

12/24/2025

Happy holidays from all of us at ASGCT! We wish our entire community a joyful and restful season. The ASGCT office will be closed Dec. 24 – Jan. 5. We look forward to a bright and productive 2026.

12/22/2025

The U.S. Food and Drug Administration has approved Waskyra, the first gene therapy for Wiskott-Aldrich syndrome and the first-ever gene therapy to be brought through regulatory approval by a nonprofit organization, demonstrating a vital new pathway for ultra-rare disease treatments.

Read more from ASGCT President Terry Flotte, MD, on the clinical impact and the importance of nonprofit research:

This approval represents a significant milestone as the first gene therapy developed and brought through regulatory approval by a nonprofit organization.

12/22/2025

The entire CGT community is invited to join us in Boston, May 11-15, 2026, for the ASGCT 2026 Annual Meeting! ✔️ Check registration off your list today + save with our early bird discount.

12/18/2025

The December issue of The Advocate is live! 🏛️ Don't miss our latest deep dive into CGT advocacy, including key policy news and regulatory milestones from the past month:

ASGCT convened Congressional leaders & patient advocates to discuss next-generation HIV gene therapy approaches – moving beyond lifelong treatment toward…

12/17/2025

🎙️ The Issue is back! Listen to the latest episode as Dr. Jim Burns, CEO of Ensoma, discusses the company's novel technological approach to treating both rare diseases and solid tumor oncology.

Host Emily Walsh Martin welcomes Dr. Jim Burns, CEO of Ensoma, to discuss the company's novel technological approach to treating both rare diseases and…

12/16/2025

The latest edition of The Patient Press is out now! Catch up on CGT news for patients, caregivers, advocates, and the rare disease community:

Check out key policy updates, including the Rare Pediatric Disease PRV reauthorization, plus new resources on AAV immune responses and Epidermolysis…

12/15/2025

Start the new year strong with our new Foundations of CGT online course – self-paced, interactive, and free! Check out the ASGCT Learning Center: https://www.asgct.org/education-career-resources/asgct-learning-center

12/12/2025

🌎 Join us on Tuesday, Dec. 16, for the next installment of our Around the World webinar series! Guilherme Baldo, Ph.D., of UFRGS - Universidade Federal do Rio Grande do Sul, moderates a deep dive into South American CGT frontiers with speakers:

Osvaldo L. Podhajcer, Ph.D., Fundación Instituto Leloir
Maroun Khoury, Ph.D., Universidad de los Andes
Hilda Petrs Silva, Ph.D., UFRJ - Universidade Federal do Rio de Janeiro

💻 Reserve your spot (free!) today: https://www.asgct.org/events-meetings/around-the-world-technological-frontiers-in-south-american-gene-and-cell-therapy

12/11/2025

"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

ASGCT’s 2025 Breakthroughs in In Vivo Gene Editing Conference delivered precisely what its name promised: breakthrough moments that are reshaping our…

12/10/2025

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

12/09/2025

ASGCT recently hosted a successful Congressional briefing on next-generation cell and gene therapy approaches for HIV. We were honored to have Senator Chris C***s (D-DE) open the session, highlighting the critical need for sustained federal investment in innovative HIV research.

The briefing featured a powerhouse panel, including:

➡️ Dr. Paula Cannon (ASGCT Past President + University of Southern California): Insights on HIV biology and GTx strategies.
➡️ Dr. Jonah Sacha (Oregon Health & Science University (OHSU)): Lessons from patients cured through bone marrow transplants.
➡️ Paul Edmonds & Michael Louella: Essential perspectives from a cured patient and a community advocate.

These promising clinical approaches rely on robust research funding and science-informed policy to ensure breakthroughs become accessible treatments.

Read our full briefing summary for key takeaways:

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…

12/05/2025

Huge thanks to U.S. Senator Chris C***s 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.