American Society of Gene + Cell Therapy

Advancing CGTs in 2025: ASGCT's Policy and Advocacy Highlights ASGCT reflects on a transformative year for the cell and gene therapy (CGT) field - one marked by exciting scientific achievements, expanded patient…

As January comes to an end, we’re reflecting on a major year for #ASGCTAdvocacy! Read our 2025 recap: https://www.asgct.org/news-publications/asgct-news/advancing-cgts-in-2025-asgcts-policy-and-advocacy-highlights

MORE TIME TO SUBMIT! ⏳ We’ve extended the #ASGCT2026 abstract deadline to Feb. 3 at 8 p.m. ET. Submit now: https://annualmeeting.asgct.org/abstracts/submission-information

Book your hotel for #ASGCT2026 through our official block today! Stay in the heart of Boston's biotech hub with maximum flexibility and our best rate guarantee.

Take advantage of our early registration discount before it’s gone: https://annualmeeting.asgct.org/register-attend/travel-accommodation

Rare Pediatric Disease Priority Review Voucher (PRV) - Sign On | ASGCT ASGCT is proud to lead nonprofits, academic medical centers, and patient advocacy groups in urging Congress to pass the Mikaela Naylon Give Kids a Chance…

ASGCT is proud to lead nonprofits, academic medical centers, and patient advocacy groups in urging Congress to pass the Mikaela Naylon Give Kids a Chance Act and reauthorize the Rare Pediatric Disease Priority Review Voucher Program: https://bit.ly/4sZ2alh

Deadline: January 30 ⏱️ Don't miss out on the opportunity to present your groundbreaking work #ASGCT2026! Submit your abstract: https://annualmeeting.asgct.org/abstracts/submission-information

Gene Editing Gene editing aims to be a one-time therapy that directly edits pieces of DNA within the cell.

Ever wonder how gene editing actually works? 🤔 From CRISPR to base editing, the science is moving fast—and we’re here to help you make sense of it all. https://patienteducation.asgct.org/understanding-cell-gene-therapy/types-of-cell-gene-therapy/gene-editing

⏱️ It's time to finalize your #ASGCT2026 abstract. Hit that submit button today! https://annualmeeting.asgct.org/abstracts/submission-information

The Advocate: January 2026 ASGCT launches CGTxchange to rescue shelved therapies, urges Senate action on PRV reauthorization, and provides critical updates on federal funding and…

ASGCT launches CGTxchange to rescue shelved therapies, urges Senate action on PRV reauthorization, and provides critical updates on federal funding and FDA manufacturing programs.

Read more in the latest edition of The Advocate: https://bit.ly/45ItIkK

Don't miss out on the opportunity to present your groundbreaking work at this essential gathering of cell and gene therapy professionals. Submit your abstract today! https://annualmeeting.asgct.org/abstracts/submission-information

Join ASGCT President Dr. Terry Flotte at the Empowering Patients Summit! He kicks off two virtual half-days of expert-led sessions on March 11 at 10 a.m. ET. Reserve your spot (free!): asgct.org/empowering

#EmpoweringASGCT

American Society of Gene & Cell Therapy and Orphan Therapeutics… First-of-its-kind venture to facilitate and catalyze development of deprioritized cell and gene therapies, supported by AI-enabled platform

We’re proud to announce a new partnership with Orphan Therapeutics Accelerator to launch CGTxchange – a first-of-its-kind, jointly owned marketplace for deprioritized CGTs, accelerating development for ultra-rare diseases. #ASGCT #OTXL

Learn more: https://bit.ly/3LuypaS

Curious about cell and gene therapy? Dr. Phil Tai will guide you through Module 1 in our new Foundations of CGT online course – self-paced, convenient, and free! Check it out: asgct.org/learning

Funded by an independent educational grant from Pfizer, Inc.

Entrepreneurship, Fundraising, and the Future of Cell & Gene… | ASGCT Dr. Paulk offers perceptive insights into the complex world of biotech entrepreneurship, covering essential fundraising strategies from Seed and Series…

Dr. Niki Paulk, CEO and Founder of Siren Biotechnology joins the podcast for a timely conversation on turning scientific promise into commercial success. 🎧 Listen now: https://bit.ly/49oQeAm

ASGCT Committees As a rapidly growing community, ASGCT offers a unique opportunity for members at any career stage to contribute meaningfully to the cell and gene therapy…

Looking to expand your professional network and leadership experience in the CGT field? Apply to serve on an ASGCT committee! Our committees drive the programs and initiatives that advance the field.

Submit your application by January 15: https://www.asgct.org/membership/asgct-committees

“We’re in such an exciting moment, and how to marry the potential of all of this with getting the details right for each application is the tension of our moment.”

Hear Dr. Sonia Vallabh's powerful personal journey to develop a treatment for prion disease: https://bit.ly/3X4YPCp

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.

FDA Approves Waskyra, First Gene Therapy for Wiskott-Aldrich Syndrome This approval represents a significant milestone as the first gene therapy developed and brought through regulatory approval by a nonprofit organization.

The @fda.gov has approved Waskyra, the first gene therapy for #WiskottAldrichSyndrome and the first-ever gene therapy to be brought through regulatory approval by a nonprofit organization. Read more from ASGCT President Terry Flotte, MD: https://bit.ly/3NcZzn7

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.

Unlocking New Therapeutic Potential: Ensoma’s Platform for… | ASGCT 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…

🎙️ 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. https://bit.ly/4j1uRtg

The Patient Press: December 2025 Check out key policy updates, including the Rare Pediatric Disease PRV reauthorization, plus new resources on AAV immune responses and Epidermolysis…

The latest edition of The Patient Press is out now! Catch up on CGT news for patients, caregivers, advocates, and the rare disease community: https://patienteducation.asgct.org/the-patient-press-newsletter/the-patient-press-december-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

🌎 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

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 ...

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

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…

"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

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.

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

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…

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

📢 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

Molecular Therapy Presents: Innovations in Aptamer Technology Thursday, December 4 at 1 p.m. CT

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