CGTLive®’s Weekly Rewind – July 5, 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. Navigating a Novel Therapy Frontier: Gene Therapy's Stunted Impact on Leukodystrophies

CGTLive takes a look at the path gene therapies have blazed in leukodystrophies, the challenges they’ve faced, and the road they’ve paved.

2. Mark Walters, MD, on the Future of Sickle Cell Disease’s Landscape of Care

The 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.

3. Wave Life Sciences' Antisense Oligonucleotide WVE-003 Demonstrates Ability to Lower Mutant Huntingtin in Phase 1b/2a Trial

The placebo-controlled multidose portion of the SELECT-HD trial included 23 patients with Huntington Disease in total.

4. Deborah Phippard, PhD, on the Challenges of Evaluating Gene Therapy for Parkinson Disease

The chief scientific officer of Precision for Medicine discussed unique difficulties of designing clinical trials for PD gene therapy candidates.

5. Lyell’s ROR1-Targeted CAR-T LYL797 Attains 60% Clinical Benefit Rate at High Dose Level in Phase 1 Solid Tumor Trial

Notably, 1 patient treated in the trial died of respiratory failure, but it is not clear whether the death was related to LYL797.