CGTLive®’s Weekly Rewind – November 15, 2024

CGTLive®’s Weekly Rewind

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 Obe-Cel for Adults With R/R B-Cell Precursor Acute Lymphoblastic Leukemia

The CAR-T therapy was approved based on the pivotal phase 1b/2 FELIX clinical trial (NCT04404660), which showed good rates of overall complete remission and median duration of remission.

2. Anelloviruses, a Potential Alternative to AAV for Gene Therapy

Chris Wright, MD, PhD, the chief medical officer and head of translational research at Ring Therapeutics, discussed research presented at ASGCT 2024.

3. FDA Approves PTC Therapeutics’ Gene Therapy Kebilidi for AADC Deficiency

The gene therapy has been approved in the UK and European Union since 2022 under the brand name Upstaza.

4. FDA Accepts Abeona Therapeutics' BLA for Epidermolysis Bullosa Gene Therapy Pz-Cel

The agency has set the PDUFA action date for the BLA for April 29, 2025.

5. Allogene’s CAR-T ALLO-316 Demonstrates Ability to Produce Responses in Renal Cell Carcinoma, but Safety Data Includes 3 On-Study Deaths

The safety data set for the trial included 39 patients who were treated with ALLO-316.