CGTLive®’s Weekly Rewind – July 12, 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. 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.