CGTLive®’s Weekly Rewind – March 21, 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. FDA Approves Alnylam’s Vutrisiran for the Treatment of ATTR Amyloidosis With Cardiomyopathy

The product is marketed under the name Amvuttra.

2. Michael Flanagan, PhD, on Del-Zota for Duchenne Muscular Dystrophy

The chief scientific officer at Avidity Biosciences discussed the function and mechanism of the investigational antisense treatment, which is in development for DMD amenable to exon 44 skipping.

3. Patient Dies of Acute Liver Failure After Treatment With Sarepta’s DMD Gene Therapy Elevidys

Acute liver injury is known to be a possible adverse event associated with AAV vector-based gene therapies such as Elevidys.

4. REGENXBIO’s DMD Gene Therapy RGX-202 Generates Microdystrophin Expression in Additional Patients Treated in Phase 1/2 Trial

One of the patients, who was aged 3 years at the time of treatment, achieved 122.3% microdystrophin expression compared to control.

5. SpliceBio Doses First Patient in Trial for Stargardt Gene Therapy SB-007

The ASTRA and accompanying POLARIS studies are actively enrolling patients.