CGTLive®’s Weekly Rewind – May 16, 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. Spur Therapeutics’ Gene Therapy SBT101 Generally Well-Tolerated in Phase 1 Trial for Adrenoleukodystrophy

The new data were presented in a poster at ASGCT’s 2025 meeting.

2. Nicholas Giovannone, PhD, on Using Bispecific Antibodies to Redose AAV Gene Therapy

The senior principal scientist at Regeneron discussed an advanced approach to enabling gene therapy redosing.

3. REGENXBIO’s BLA for MPSII Gene Therapy RGX-121 Accepted for Priority Review by the FDA

The PDUFA action date for the BLA has been set as November 9, 2025.

4. Rebecca Ahrens-Nicklas, MD, PhD, and Kiran Musunuru, MD, PhD, MPH, ML, MRA, on Treating CPS1 Deficiency With Personalized Gene Editing

The physician-scientists at CHOP discussed an n-of-1 clinical trial for a CRISPR gene-editing strategy.

5. Aneal Khan MD, MSc, FCCMG, FRCPC, on Evaluating Gene Therapy for Fabry Disease

The president of MAGIC Clinic and CEO of Discovery DNA discussed the implications of gene therapy data presented at the 2025 WORLDSymposium.