CGTLive®’s Weekly Rewind – July 12, 2024
Review top news and interview highlights from the week ending July 12, 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.
1. High-Dose Gene Therapy Slows Huntington Disease Progression
Treated patients had a –0.2 mean change in cUHDRS compared with a –1.0 change in external control data.
2. Paul Y. Song, MD, on Taking a Holistic Approach to Treating Parkinson Disease
The chairman and chief executive officer of NKGen discussed the potential of SNK01, the company’s autologous natural killer cell therapy, in treating PD.
3. SPEARHEAD-1 Supports Potential TCR-T Approval for Synovial Sarcoma
The Prescription Drug User Fee Act (PDUFA) target action date is August 4, 2024.
4. Leigh Ramos-Platt, MD, on Looking Forward to Gene Therapy’s Growth
The clinical professor of neurology and pediatrics at Keck School of Medicine of USC also discussed current strategies with gene therapy administration.
5. Nkarta Launches Trial for CAR-NK Therapy NKX019 in Lupus and Gains Clearance for Separate Trial in Other Autoimmune Diseases
Ntrust-2 will assess NKX019 in systemic sclerosis, idiopathic inflammatory myopathy, and ANCA-associated vasculitis.
News and Expert Insights in Hemophilia Gene Therapy for Bleeding Disorders Awareness Month 2025
March 9th 2025In observance of Bleeding Disorders Awareness Month, held annually in March, catch up on some of the latest news and expert insights in gene therapy for hemophilia, which is among the most common of these disorders.
