CGTLive®’s Weekly Rewind – August 30, 2024

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.

1. Immunogenicity Outcomes for Val-Rox Reported From Phase 3 GENEr8-1 Study

Among more than 100 patients with hemophilia A, no FVIII-specific responses showed associations with the assessed safety or efficacy parameters.

2. Chun-Yu Chen, PhD, on Addressing Hemophilia A With CRISPR/Cas9 mRNA LNP Gene Editing

The research scientist at Seattle Children’s Research Institute discussed findings from mouse research he presented at ASGCT’s 2024 conference.

3. Galapagos Cleared by FDA to Bring Trial for CAR-T Therapy GLPG5101 in R/R NHL to US

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.

4. Legend Biotech’s CAR-T Carvykti Approved for R/R MM Indication in China

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.

5. The Future of Sickle Cell Disease’s Landscape of Care

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.