CGTLive®’s Weekly Rewind – April 25, 2025

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. Cartesian Therapeutics’ mRNA CAR-T Descartes-08 Shows Efficacy and Safety in Myasthenia Gravis in Phase 2 Study

During the 12-month study, Descartes-08 was deemed well-tolerated.

2. Linda Marbán, PhD, on Evaluating Capricor’s Cardiomyopathy Cell Therapy Deramiocel for DMD

The CEO of Capricor Therapeutics discussed clinical data presented at MDA’s 2025 meeting.

3. UniQure's Huntington Disease Gene Therapy AMT-130 Snags FDA Breakthrough Therapy Designation

Notably, AMT-130 has previously been granted regenerative medicine advanced therapy, orphan drug, and fast track designations by the agency.

4. Questions of Access: Gene Therapy’s Next Chapter in β-Thalassemia

Tami John, MD and Shalini Shenoy, MD, MBBS, offer insight into the clinical experience of patients treated with Casgevy and Zynteglo thus far.

5. BrainChild Bio's Pediatric Brain Tumor CAR-T BCB-276 Reels in Breakthrough Therapy Designation

The FDA’s decision was supported by data indicating overall survival benefit for patients with brain tumors from the phase 1 BrainChild-03 clinical trial.