CGTLive®’s Weekly Rewind – June 13, 2025

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. Sarepta Garners Platform Technology Designation for Limb-Girdle Muscular Dystrophy Gene Therapy Vector

The platform technology designation is a new designation established by the FDA.

2. Shahzad Raza, MD, on Future Plans for CAR-T NXC-201 in R/R Light Chain Amyloidosis

The hematologist/oncologist at the Cleveland Clinic discussed next steps after early promising results were presented at ASCO’s 2025 meeting.

3. Nuevocor Snags IND Clearance for Gene Therapy Trial in LMNA DCM

In light of the IND clearance, the company intends to go forward with plans for a first-in-human phase 1/2 ascending-dose clinical trial.

4. Deevyashali Parekh, MBBS, on Evaluating the Prevalence of Second Primary Malignancies After CAR-T

The internal medicine resident at SUNY Upstate Medical University Hospital discussed the implications of findings from a deidentified database of EMR data.

5. Study Suggests β-Thalassemia Gene Therapy May Help Improve Growth in Pediatric Patients

The study included 3 boys and 6 girls who were treated with gene therapy for TDT at 5 to 16 years of age.