CGTLive®’s Weekly Rewind – May 30, 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. Patient Dies After Treatment With Rocket Pharmaceuticals’ Danon Disease Gene Therapy RP-A501 in Phase 2 Trial

The patient’s death occurred after an acute systemic infection and was associated with an unexpected SAE of clinical complications related to a capillary leak syndrome.

2. ImmunoLogic, Episode 4: "Inflammatory Storms and Gentle Balances: Navigating Toxicities in Immunotherapies" With Caroline Diorio, MD, FRCPC, FAAP

In Episode 4 of ImmunoLogic, Caroline Diorio, MD, FRCPC, FAAP, discussed her research on adverse events associated with cell therapy.

3. ALS Awareness Month 2025: A Review of the Cell and Gene Therapy Pipeline

In observance of ALS Awareness Month, held annually in May, we took a look at the clinical pipeline of advanced therapeutics currently in development for the neurodegenerative disease.

4. Helping to Bring Preclinical Gene Therapy Research to First-In-Human Trials

Roger Hajjar, MD, the director of the Mass General Brigham Gene and Cell Therapy Institute, also discussed the Institute’s presentations at ASGCT’s 2025 conference.

5. Ocugen’s Gene Therapy OCU410ST Nets Rare Pediatric Disease Designation for ABCA4-Associated Retinopathies

Alongside the announcement, Ocugen noted that it intends to initiate a phase 2/3 confirmatory clinical trial for OCU410ST in the coming weeks.