Human Gene and Cell Therapy Center

29/07/2025

🧬 NOW OPEN: Registration for Breakthroughs in Targeted In Vivo Gene Editing!

On Nov. 20-21, dive into cutting-edge solutions for targeted delivery of technology in vivo in San Diego + virtually. https://bit.ly/4lIckCG

13/02/2025

https://annualmeeting.asgct.org/register/patient-advocates?fbclid=IwY2xjawIajJZleHRuA2FlbQIxMAABHSsCujZyVHrNHG04ATpJioW1JhlkWr-16ts69euwze-_vZHFfzphBdi3Lw_aem_j2meWRwsiBmsPRN3-M9-YA

ASGCT recognizes the critical role patient advocacy organizations and foundations play in developing treatments and helping those with a disease or disorder navigate the complex journey of  gene and cell therapies.  ASGCT offers a limited number of complimentary registrations to support the abilit...

22/01/2025

Starting TOMORROW: ASGCT and FDA are hosting a free scientific workshop exploring immunogenicity challenges across AAV gene therapies!

Join leading researchers and regulators for discussions on immune responses, antibody testing methodologies, and redosing strategies in AAV development. Seven FDA OTP representatives will share regulatory perspectives alongside experts from UMass Chan Medical School, University of Florida, Sarepta Therapeutics, and other institutions.

January 22-23, 2025
9:00 AM - 1:00 PM ET
Virtual | Free
Register: www.asgct.org/2025aav

21/12/2024

Olezarsen@is the first approved treatment in the U.S. for FCS, which prevents the body from breaking down fats. The treatment is an@antisense oligonucleotide (A*O).