CGTLive’s Weekly Rewind – August 4, 2023

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 Issues CRL for Mesoblast's Allogeneic Stem Cell Therapy for SR-Graft-Versus-Host-Disease

Four years after submitting its initial BLA for remestemcel-L for steroid-refractory acute graft versus host disease, Mesoblast has encountered another hurdle toward possible approval.

2. J. Andrew Livingston, MD, and Brian Van Tine, MD, PhD, on Sarcoma Awareness Month 2023

The clinicians from MD Anderson Cancer Center and Washington University – St. Louis shared messages on sarcoma awareness.

3. BLA Submission Gives Libmeldy a Shot at US Review for Metachromatic Leukodystrophy

The gene-edited cell therapy OTL-200 was approved in Europe in 2020.

4. Maria Pia Morelli, MD, PhD, on Promising Safety of Sleeping Beauty TCR-T Therapy in Solid Tumors

The assistant professor at MD Anderson Cancer Center discussed progress of the phase 1/2 trial being conducted at the center.

5. Trial for Wilson Disease Gene Therapy UX701 Starts Dosing Second Cohort

The initiation of dosing the second cohort in Ultragenyx’s study was recommended by the Data Safety Monitoring Board after a review of safety results from the trial’s first cohort.