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.

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

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.

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.

🎙️ 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 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

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

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

We recently hosted a Congressional briefing on #GeneTherapy approaches for #HIV with experts from @usc.edu, @ohsunews.bsky.social#GeneTherapy#HIVand 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

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

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

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

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

"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

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

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

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

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

📢 AVAILABLE NOW: Breakthroughs in Targeted In Vivo Gene Editing abstracts! View and download the collection of original research on our website, and don't miss your chance to dive deeper Nov. 20-21 in San Diego and virtually. https://bit.ly/47R1ndv

October is #HealthLiteracyMonth! 🧠 Simple, trusted info on cell and gene therapies is just a click away: patienteducation.asgct.org

Sending a heartfelt thank you to @nordrare.bsky.social for hosting us at last week’s inspiring Rare Diseases and Orphan Products Breakthrough Summit! 🦓💙 We were grateful for the opportunity to celebrate the success stories that remind us what’s possible when innovation meets hope.

The October edition of The Patient Press is here! Save the date for the 2026 Empowering Patients Summit, watch a clip of our Patient Education resources on @goodmorningamerica.bsky.social, and more: https://bit.ly/3Jl866c

Big news! We’ve relaunched our ASGCT websites with a fresh look and enhanced functionality to make accessing critical CGT resources easier than ever. 🌐 asgct.org 🗓️ annualmeeting.asgct.org 💙 patienteducation.asgct.org

ICYMI: Leaders from across the #CGT ecosystem gathered in Washington, DC last month for ASGCT’s 2025 Policy Summit, a forum that brought regulators, patient advocates, industry, + others together to discuss challenges + opportunities shaping the field. https://bit.ly/43vpSdy

⏰ Early registration for ASGCT's Breakthroughs in Targeted In Vivo Gene Editing ends Oct. 20! Secure the lowest rates for this Nov. 20-21 conference in San Diego + virtually. www.asgct.org/breakthroughs

Learn what happens to #NIH grants and #FDA user fees during a government #shutdown. Our latest blog examines operational impacts on #CGT research and development. https://bit.ly/46WvP4n #ASGCTadvocacy

📢 AVAILABLE NOW: Advancing CGTs for Cancer abstracts! Explore the latest research from leading scientists + clinicians working to advance #CGT for cancer. Download 40+ pieces of original research on our website and register to join us in Philly! https://bit.ly/4nVlxIm

Key #PS25 takeaway from CMC session: Harmonization efforts are especially critical for bespoke “n of 1” therapies, ensuring patients can access treatment without unnecessary delays. #ASGCTAdvocacy

That’s a wrap on #PS25! Thank you to our speakers, attendees, + sponsors for an outstanding event! #ASGCTAdvocacy We want to hear your feedback! Let us know how we did by filling out this short evaluation: https://bit.ly/46mRYd0

Welcome back to Day 2 of #PS25! This morning, we're kicking off with experts from @LifeEditTx, @Dyno_Tx, and @darkhorsecagt who will tell us how #AI is transforming #CGT discovery, trial design, and regulation. #ASGCTadvocacy

Carolyn Hutter of @genome_gov and Allison Bradbury at @nationwidekids dive into @NIH’s role to advance genomic medicine and patient-focused research. #ASGCTadvocacy #PS25

Live at #PS25: Melissa Majerol of CMMI digs into the CGT Access Model and how the federal value-based arrangement pilot is helping drive broader patient access. #ASGCTadvocacy