GeneTherapyLive’s Weekly Rewind – February 4, 2022

Welcome to GeneTherapyLive’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. A Snapshot of Ophthalmologic Cell and Gene Therapies in Development

Cell and gene therapies are currently being evaluated as possible avenues of treatment for multiple inherited retinal diseases. We’re spotlighting promising therapies currently in development.

2. Challenges in Managing Hemophilia: Steven W. Pipe, MD

The director of the Pediatric Hemophilia and Coagulation Disorders Program at CS Mott Children’s Hospital discussed challenges of currently available strategies for managing hemophilia A and B, including adherence to factor replacement therapy.

3. First Patient Dosed in Wet AMD Gene Therapy Trial

The first patient has been dosed in the phase 1/2 trial (NCT05197270) of the gene therapy 4D-150 (4D Molecular Therapeutics) for the potential treatment of wet age-related macular degeneration (wet AMD).

4. Cilta-Cel in the Multiple Myeloma Treatment Landscape: Thomas G. Martin, MD

The clinical professor of medicine, Helen Diller Family Comprehensive Cancer Center, UCSF, discussed data from both the CARTITUDE-1 and CARTITUDE-2 studies. He also touched oncilta-cel's place in the multiple myeloma treatment landscape.

5. Bispecific TCR Cell Therapy Approved for Unresectable or Metastatic Uveal Melanoma

The FDA has approved Immunocore's tebentafusp-tebn (Kimmtrak) for the treatment of HLA-A*02:01–positive unresectable or metastatic uveal melanoma.