CGTLive®’s Weekly Rewind – August 29, 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 Bumps Back PDUFA for REGENXBIO's MPS II Gene Therapy RGX-121

The new Prescription Drug User Fee Act (PDUFA) goal date is set for February 8, 2026.

2. Annaiz Grimm, BS, on Using EngTregs to Treat Autoimmune Disease

The research scientist at Seattle Children's Research Institute discussed a potential alternative to standard of care immunosuppressive therapy.

3. FDA Approves Precigen’s Recurrent Respiratory Papillomatosis Gene Therapy Papzimeos

Notably the FDA granted Papzimeos a full approval, without a requirement for a confirmatory trial.

4. Prerna Mewawalla, MD, on Unmet Needs Following Early-Line CAR-T for R/R Multiple Myeloma

The medical director of Apheresis and a hematologist-oncologist in the Division of Hematology and Cellular Therapy at Allegheny Health Network spoke about unmet needs in patients who receive early-line CAR-T for R/R MM.

5. Ultragenyx Puts Rolling BLA for Glycogen Storage Disease Gene Therapy DTX401 in Front of FDA

Ultragenyx anticipates that the full BLA will have been submitted in the fourth quarter of this year.