CGTLive®’s Weekly Rewind – March 15, 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. FDA Finalizes Sets of Guidelines for Both CAR-T Products and Genome Editing Products

According to Federal Register notices, the guidance documents are updated versions of drafts originally published in March 2022.

2. Jeffrey Chamberlain, PhD, on MDA Legacy Award: From Grad Work to Gene Therapy Approval

The McCaw Endowed Chair of Muscular Dystrophy at University of Washington discussed his research career with muscular dystrophies.

3. CK0803 Treg Therapy Seems Safe for ALS, Trial Set to Treat Second Cohort

The positive DSMB recommendation came after no participants were found to experience any serious adverse events from the therapy.

4. Arshad Khanani, MD, on Comparing Wet AMD Gene Therapy 4D-150 to Aflibercept in a Randomized Clinical Trial

The assistant professor in the department of pediatrics at the University of Florida College of Medicine discussed also discussed the latest results announced from the phase 2 portion of the PRISM study.

5. Gene Editing Investigations Mature With heart-1 Trial for Familial Hypercholesterolemia

Verve Therapeutics plans to expand evaluations into the US after an IND clearance was delayed by a clinical hold.