Review top news and interview highlights from the week ending August 30, 2024.
CGTLive®’s Weekly Rewind
Welcome to CGTLive®’s Weekly Rewind! We’ve compiled 5 highlights from this week’s coverage of advances in gene and cell therapies, including FDA actions, notable research, and interviews with experts across the field.
Among more than 100 patients with hemophilia A, no FVIII-specific responses showed associations with the assessed safety or efficacy parameters.
The research scientist at Seattle Children’s Research Institute discussed findings from mouse research he presented at ASGCT’s 2024 conference.
In light of the IND clearance, Galpagos intends to bring its phase 1/2 ATALANTA-1 clinical trial (NCT06561425), which is currently ongoing in Europe, to the United States.
The NMPA’s decision was made with reference to results from the phase 2 CARTIFAN-1 clinical trial (NCT03758417), which took place at multiple sites in China.
Mark Walters, MD, a professor in residence for pediatrics at the Sickle Cell Center of Excellence at the University of California, discussed gene therapy’s ongoing transformation of the treatment landscape.
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Jeffrey Chamberlain, PhD, on the Import of Early Intervention in DMD
July 10th 2025The McCaw Endowed Chair of Muscular Dystrophy at University of Washington, discussed how comprehensive care for DMD patients involves early diagnosis, steroid treatment, consideration of mutation-specific therapies, and more.