American Society of Gene & Cell Therapy

WATCH: As the number of approved #genetherapy products increases, so too do questions about their long-term safety, durability, and accessibility. The Working Group on Pediatric #GeneTherapy and Medical Ethics (#PGTME) at NYU Langone Health recently hosted its fifth annual Lunchtime Lecture Series, featuring clinicians, advocates, regulators and other stakeholders in the pediatric #CGT space. Check out the recap and watch all the lectures about ongoing and emerging issues in the space on our blog! #PatientEducation https://lnkd.in/ezvgSTQp

Empowering Patients 2025: A Cell and Gene Therapies Summit is a free, virtual event open to the public. Taking place on March 12-13 from 9 a.m.-12:30 p.m. ET, experts from across the CGT field will join ASGCT live to provide timely education and engaging discussions on cell and gene therapies. Two such sessions are featured below! Complexity of Study Design in Cell and Gene Therapy March 12, 2025 | 10:25 - 11:25 AM ET Dr. Raymond Wang (Children's Hospital Orange County) and Dr. Heather Lau (Ultragenyx) will lead an expert discussion on the critical decisions that shape clinical trials, including participant eligibility, dosing protocols, and control group implementation. Sharon King will moderate. When Innovation Stumbles + Pathways Forward March 12, 2025 | 11:30 AM - 12:25 PM ET Dr. Terry Flotte (University of Massachusetts) and Dr. Cara O'Neill (Cure Sanfilippo Foundation) will examine the state of commercially pre-viable therapies, offering both clinical and patient advocacy perspectives on addressing development challenges. Register today at www.asgct.org/ep2025!

The American Society of Gene & Cell Therapy (#ASGCT) has extended the abstract submission deadline for the 28th Annual Meeting to 8 p.m. ET/5 p.m. PT on Tuesday, February 4. The abstract deadline extension is open to all potential abstract authors to share cutting-edge research and foster scientific collaboration among academic, industry, and government researchers—the long-standing cornerstones of ASGCT’s Annual Meeting. Learn more and submit an abstract for presentation at the ASGCT 28th Annual Meeting at https://lnkd.in/dkPVZQdB.

❓ Did you know that most therapies circulating in the bloodstream can't reach the brain because of the blood-brain barrier? #FocusedUltrasound has the potential to transform treatments for #braindisorders, bringing hope to patients and families. Focused Ultrasound Foundation Dr. Agessandro Abrahao (Sunnybrook) discusses the new technology in our latest Community Quick Take! Check out more resources on our website + let us know what you think. #PatientEducation https://lnkd.in/gpR-JMJj

🔬 You have a powerful role to play in developing treatments for your disease. Get started by learning: - What information you can share about your disorder that can influence researchers - The steps for getting the green light to repurpose approved drugs for your condition - The phases of the drug development process in which patient participation is most important Together with FDA and Critical Path Institute (C-Path), the National Organization for Rare Disorders created a FREE training course to help the #RareCommunity better understand #raredisease drug development and share real stories from experts who have been in YOUR shoes! https://lnkd.in/eqhw3Zk3

We recently caught up with our inaugural Congressional Policy Fellow Adriana Bankston to see what she's been up to in Washington, D.C. over the past several months. "During my time here, I delved into several policy issues through legislation, letters, and events and meetings within my policy portfolio. . . In addition to performing the day to day duties of a Congressional staffer, one exciting opportunity has been the ability to contribute to policy projects on issues that I am very passionate about." Learn more about Dr. Bankston's work on Capitol Hill + apply to serve in this paid role next year. https://lnkd.in/gsnbmJpH

📈 The ASGCT-Citeline Q4 report is live! Q4 delivered approvals across three modalities: #Aucatzyl for acute lymphocytic leukemia, #RegeneCyte for blood disorders, and #Tryngolza for lipoprotein lipase deficiency. #CART #celltherapy #stemcelltherapy Read + download the full report. https://lnkd.in/gR3z_HAK

Last summer, 17-month-old Giulia received a #genetherapy for #Canavan disease. The AAV9 vector gene therapy is based on research led by ASGCT Past President Guangping Gao, PhD; and Associate Member Dominic J. Gessler, M.D., Ph.D., assistant professor of neurological surgery at UMass Chan Medical School. “You have to remember everything you do, day and night, is for a cause,” said Dr. Gao. “This cause is this: can we develop therapy to save those babies and change their quality of life?” https://lnkd.in/gYCqcV8R

We're live for day two of Class Considerations on Immunogenicity for AAV Gene Therapy Products: Assessing Current Practice and New Data! Check out the updated program and register for free at www.asgct.org/2025aav. #AAV #CGT #ASGCT

We hope that you found the first day of ASGCT’s workshop, Class Considerations on Immunogenicity for AAV Gene Therapy Products: Assessing Current Practice and New Data, to be insightful and informative. Join us tomorrow, Thursday, January 23 to continue the dialog. Visit ASGCT.org/2025aav for an updated program and schedule of events.