CGTLive’s Weekly Rewind – December 8, 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 Approves bluebird bio's Lovo-Cel Gene Therapy for Sickle Cell Disease

The decision was supported by efficacy data from 36 patients from the ongoing phase 1/2 HGB-206 trial (NCT02140554) and 2 patients in the phase 3 HGB-210 trial (NCT04293185).

2. FDA Approves Exa-cel, Vertex and CRISPR Therapeutics’ Gene Therapy, for Sickle Cell

The FDA’s decision was based on data from the CLIMB-121 and CLIMB-131 clinical trials, and the decision makes it the first CRISPR-based gene therapy to be approved in the US.

3. Sukumar Nagendran, MD, on Addressing Unmet Needs in Rett Syndrome With Gene Therapy

The president and head of R&D at Taysha Gene Therapies discussed the early clinical evaluation of TSHA-102.

4. Adaptimmune Submits Afami-Cel First Engineered TCR-T Therapy BLA for Synovial Sarcoma

The cell therapy yielded a median overall survival of around 17 months compared with a historical overall survival of less than 12 months.

5. Benitec Biopharma’s Oculopharyngeal Muscular Dystrophy Gene Therapy Trial Doses First Patient

Benetic anticipates an announcement of interim results sometime around the midpoint of 2024.