CGTLive’s Weekly Rewind – September 30, 2022

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. Sarepta’s DMD Gene Therapy Goes In Front of FDA With BLA Submission

SRP-9001 has shown safety and efficacy across multiple studies compared to controls, with additional efficacy data expected in 2024 from the EMBARK trial.

2. Marty Geidlin, PhD, on Advantages of Allogeneic NK Cell Therapy Approaches

The senior vice president of Technical Operations at Senti Biosciences discussed the company’s NK cell logic-gating platform and allogeneic approach.

3. Vertex Continues Gene Therapy Momentum in Blood Disorders With Exa-Cel Submission

Bluebird bio’s ZYNTEGLO was the first gene therapy to be approved in the space in August 2022.

4. Working With the Community to Improve Patient Access to Trials, Therapies

Rob Richards, administrative director, cell therapy and transplant, University of Pennsylvania, discussed improving reach of gene therapy trials and therapies.

5. KIR-CAR T-Cell Therapy Recognized for Mesothelioma Treatment

Verismo plans to initiate a phase 1, first-in-human trial of SynKIR-110 in the first quarter of 2023.