CGTLive®’s Weekly Rewind – February 21, 2025
Review top news and interview highlights from the week ending February 21, 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. 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.
Newsletter
Stay at the forefront of cutting-edge science with CGT—your direct line to expert insights, breakthrough data, and real-time coverage of the latest advancements in cell and gene therapy.
Jeffrey Chamberlain, PhD, on the Import of Early Intervention in DMD
July 10th 2025The McCaw Endowed Chair of Muscular Dystrophy at University of Washington, discussed how comprehensive care for DMD patients involves early diagnosis, steroid treatment, consideration of mutation-specific therapies, and more.
