CGTLive®’s Weekly Rewind – February 28, 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. AskBio Garners RMAT Designation for Parkinson Disease Gene Therapy AB-1005

The FDA’s decision was based on a review of findings from a phase 1b clinical trial.

2. David Barrett, JD, on ASGCT’s Q4 2024 Landscape Report

The chief executive officer of ASGCT broke down the highlights of the organization’s final report for 2024.

3. Regeneron’s Gene Therapy DB-OTO Improves Hearing in Children With Otoferlin-Related Hearing Loss

Out of 11 treated children who had at least 1 posttreatment assessment, 10 showed improved hearing at various decibel hearing levels.

4. Reena Sharma, MD, on Future Plans for Evaluating FLT201

The adult metabolic consultant at Salford Royal Hospital discussed areas of interest for future study for the Gaucher disease gene therapy product.

5. Patient With Frontotemporal Dementia Treated With NKGen’s Autologous NK Cell Therapy Troculeucel in Compassionate Use Context

The company intends to pursue a full IND application for troculeucel in FTD.