The BIO-ASGCT CGT Science Series is back! The latest session dove into advanced analytical approaches for rAAV characterization, including genome/capsid analysis and standardization efforts. Check out The Advocate for more details: https://bit.ly/3OCZLxd
#ASGCTAdvocacy
Congress nears completion of FY26 appropriations with modest #NIH increase (~$415M) and stable ARPA-H funding at $1.5B. Critically, F&A cost reimbursement rates preserved for research institutions. Read more in The Advocate: https://bit.ly/3OyPsK1
#ASGCTAdvocacy #ResearchFunding
As January comes to an end, we’re reflecting on a major year for #ASGCTAdvocacy! Read our 2025 recap: www.asgct.org/news-publications/asgct-...
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
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
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
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
Dr. Snehal Naik
Participants network at the 2025 ASGCT Policy Summit.
Panelists discuss critical CGT challenges at the 2025 ASGCT Policy Summit.
Panelists discuss critical CGT challenges at the 2025 ASGCT Policy Summit.
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
What does it take to onboard newly approved CGTs in hospitals? And where does LTFU come in as both a regulatory and patient experience issue? Speakers discussing real-world bottlenecks to clinical care at #PS25. #ASGCTadvocacy
Michelle McClure (@ACMG) and Dylan Simon (@EveryLifeOrg) are talking all things newborn screening at #PS25: where the system stands today, efforts to update and strengthen the system, and why patient advocates are at the heart of it all. #ASGCTadvocacy
#PS25 update: Experts are discussing EU HTA scientific advice for CGTs. Takeaway - early engagement with HTA bodies is critical for access strategies. #ASGCTadvocacy
Dr. Mimi Lee presents @ARPA_H’s multi-ecosystem collaboration for precision medicine. Exciting examples of cross-sector innovation in #raredisease #CGT development! #ASGCTAdvocacy #PS25
We’re live! Kicking off the 2025 Policy Summit with opening remarks from Dr. Snehal Naik, ASGCT Regulatory Affairs Committee Chair. Stay tuned for critical policy discussions shaping the #CGT landscape. #ASGCTadvocacy #PS25
✨ The Policy Summit (#PS25) STARTS TOMORROW in Washington, D.C.!
Dr. Snehal Naik, Chair of ASGCT's Regulatory Affairs Committee, shares why this event is important to the #CGT field. REGISTER NOW: https://bit.ly/3IyraNR
#ASGCTadvocacy
💡 "I strongly believe we are in a moment," says @arpa-h.bsky.social's Dr. Mimi Lee, who is speaking at the 8th Annual Policy Summit NEXT WEEK in Washington, D.C.!
Hear why she's excited to attend this event and make sure to register: https://bit.ly/3KaYScC #ASGCTadvocacy #rarediseases
ASGCT appreciates Senate appropriators for their bipartisan leadership in advancing #CGTs. The $400M NIH increase and strong CGT support in the FY26 Labor-HHS bill reflect a shared commitment to transforming patient care through innovative science. #ASGCTadvocacy
⚡ BREAKING: New #NewbornScreening session added to Policy Summit!
Hear how ASGCT advances gene therapy access for all patients.
🎧 Podcast: newbornscreeningspotlight.podbean.com/e/american-society-of-ge...
📍 Register: https://bit.ly/4o4es9k
#ASGCTadvocacy #PolicySummit
At the 2025 Policy Summit, "you will be armed with new insights about how to ensure you are starting with the end in mind from even the earliest stages of discovery and development," says ASGCT Member Emily Walsh Martin.
💡 Why attend the ASGCT Policy Summit in two weeks?
"You will be armed with new insights about how to ensure you are starting with the end in mind from even the earliest stages of discovery and development," says Dr. Emily Walsh Martin. #ASGCTadvocacy https://bit.ly/4niZ8Vc
💡 ON THE BLOG: Last week, ASGCT President Dr. Terry Flotte + Past President Dr. Paula Cannon participated in @fda.gov's first #celltherapy + #genetherapy roundtable.
Watch them deliver remarks on the field's beginnings, recent news on Baby KJ, + more! #ASGCTadvocacy www.asgct.org/publications...
THANK YOU for making #ASGCT2025 a success! Access sessions on demand until July 18. We can't wait to see you May 12-16, 2026 in #Boston! #ASGCT2025 #CGT #ASGCTadvocacy
Navigating global regulatory requirements for #CGT development? Join our "Global Convergence: CMC Harmonization" symposium at #ASGCT2025, featuring experts from @Takeda and @BristolMyerSquibb who will share real-world strategies! May 16, 1:30-3:15 pm #ASGCTadvocacy
What happens when promising CGT programs are deprioritized? Join our #ASGCT2025 fireside chat featuring Dr. Donald Kohn (@UCLA), Rachel McMinn (@NeurogeneInc), and other experts as they explore challenges and potential pathways forward for these therapies. #ASGCTadvocacy
Congratulations to Rami Major, PhD, ASGCT's ‘25-26 Congressional Policy Fellow!
Congratulations to Rami Major, PhD, ASGCT's ‘25-26 Congressional Policy Fellow! She'll head to Capitol Hill starting in Sept. to bring science-based policy advice to legislators - hosted in partnership with @aaas.org. #ASGCTadvocacy
Calling all patient advocacy organizations, academic institutions, + other nonprofits: Sign onto a group letter to support NIH research funding through March 4.
📰 The February issue of The Patient Press is out now! 📰
Calling all patient advocacy organizations, academic institutions, + other nonprofits: Sign onto a group letter to support NIH research funding through March 4. #PatientEducation #ASGCTAdvocacy www.asgct.org/publications...
We recently caught up with our inaugural Congressional Policy Fellow @abankston.bsky.social to see what she's been up to in Washington, D.C.
Learn what she's working on and APPLY through Feb. 24 to serve in this paid role next year. #ASGCTadvocacy www.asgct.org/publications...
From March 12-13, join us for this free, virtual event to inform and educate those who can benefit from #celltherapy and #genetherapy treatments.
From March 12-13 at Empowering Patients 2025, we'll explore:
✔️The Value of Patients + Advocates
✔️Why Are Clinical Trials for #RareDiseases Different?
✔️Developing #GeneTherapies as Platform Technologies
Register for FREE! #ASGCTAdvocacy #PatientEducation www.asgct.org/events/empow...
