Philadelphia-Much progress has been made in the development of gene therapies for retinal dystrophies during the last decade. Albert Maguire, MD
CGTLive®’s Weekly Rewind – September 20, 2024
Review top news and interview highlights from the week ending September 20, 2024.
Continuing the Marathon of Muscular Dystrophy Research
Jeffrey Chamberlain, PhD, McCaw Endowed Chair of Muscular Dystrophy at University of Washington, shared his outlook on the trajectory of research in the field.
Poseida Therapeutics Garners FDA RMAT Designation for Allogeneic CAR-T P-BCMA-ALLO1 in R/R Multiple Myeloma
The product is currently being evaluated in a phase 1/1b clinical trial.
Rocket Pharmaceuticals Maxes Out Recruitment in Pivotal Trial for Danon Disease Gene Therapy RP-A501
The single-arm trial enrolled 12 male patients in total across the United States and European Union.
Around the Helix: Cell and Gene Therapy Company Updates – September 18, 2024
Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
Ongoing Research Seeks to Address Challenges With Muscular Dystrophy Gene Therapy
Jeffrey Chamberlain, PhD, McCaw Endowed Chair of Muscular Dystrophy at University of Washington, highlighted studies presented at MDA's 2024 conference.