CGTLive®’s Weekly Rewind – February 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 Accepts BLA for Ultragenyx’s MPSIIIA Gene Therapy UX111 With Priority Review

The PDUFA action date for the BLA has been set at August 18, 2025, and no advisory committee meeting is currently planned.

2. Nirav Shah, MD, MSHP, on Evaluating Zamto-Cel in R/R DLBCL

The associate professor of medicine at Medical College of Wisconsin discussed efficacy data presented at the 2025 Tandem Meetings.

3. BioRestorative Therapies’ MSC Therapy BRTX-100 Fast Tracked for Chronic Lumbar Disc Disease

BRTX-100 is currently being evaluated in a prospective, randomized, and controlled phase 2 clinical trial.

4. Bhagirathbhai R. Dholaria, MD, on Evaluating P-BCMA-ALLO1 in R/R Myeloma

The associate professor of medicine in malignant hematology & stem cell transplantation at Vanderbilt University Medical Center discussed results from a phase 1 clinical trial.

5. Pfizer Jettisons FDA-Approved Hemophilia B Gene Therapy Beqvez

The company made the decision based on a lack of interest from the patient and clinician communities.