CGTLive®’s Weekly Rewind – July 19, 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. CBER To Conduct Interactive CGT Site Tours to Improve Communication, Share Best Practices

The FDA announced its START Pilot Program in October 2023 with a similar goal.

2. David Dimmock, MBBS, on AI-Guided ASO Development for Ultra-Rare Diseases

The chief medical officer at Creyon Bio overviewed the development of an allele-selective TNPO2 protein for a single patient with a de novo pathology.

3. Gene Therapy Improves Some Disease Biomarkers of Friedreich Ataxia Cardiomyopathy

Despite the improvements, Lexeo was unable to discern a benefit on cardiovascular fitness and Peak VO2 from LX2006 with current measurements.

4. Subhash Tripathi, PhD, on Generating In Vivo CARs With A2-CAR-CISC EngTreg Cells

The senior researcher at Seattle Children’s discussed the development and validation of A2-CAR-CISC EngTreg cells.

5. Dopaminergic Progenitor Cell Therapy Shows Some Symptom Improvement in Parkinson Disease

Data from the high-dose cohort of a phase 1/2 clinical trial in South Korea will be shared in Fall 2024.