CGTLive®’s Weekly Rewind – August 14, 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. Alzheimer Disease Gene Therapy Provides Restoration of Entorhinal Cortex FDG-PET Activity in Phase 1 Trial

AAV2-BDNF gene therapy shows promise in preventing neuronal loss and cognitive decline in early Alzheimer disease, according to the trial results.

2. Kathleen A. Dorritie, MD, on the ‘Explosion of New Therapies’ in Hematologic Malignancies

The hematologist/oncologist at the University of Pittsburgh Medical Center Hillman Cancer Center in Pennsylvania discussed updates to NCCN guidelines and research to pay attention to in hematologic malignancies.

3. FDA Updates Labeling for bluebird's Active Cerebral Adrenoleukodystrophy Gene Therapy Eli-Cel to Reflect Risk of Hematologic Malignancy

The move was based on updated safety data from an FDA probe into the risk of hematologic malignancies in patients treated with eli-cel.

4. Galapagos Snags FDA RMAT Designation for R/R Mantle Cell Lymphoma CAR T-Cell Therapy

The RMAT designation was granted based on data from the phase 1/2 ATALANTA-1 clinical trial, which includes a subset of patients with r/r MCL.

5. EMA's CHMP Gives Ocugen the Green Light to Submit MAA for OCU410ST Based on GARDian3 Trial

Notably, the first patient was dosed in the GARDian3 in July 2025.