CGTLive’s Weekly Rewind – August 26, 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. Dystrophic Epidermolysis Bullosa Gene Therapy BLA Accepted With Priority Review

The BLA was supported by data from 2 intrapatient, placebo-controlled clinical trials.

2. Laurent Poirot, PhD, on the FDA’s IND Clearance for UCART20x22

The senior vice president of immunology at Cellectis discussed the company’s investigational dual-targeted CAR-T therapy for B-cell malignancies.

3. Resurrecting ALS Mesenchymal Cell Therapy for Regulatory Approval

Merit Cudkowicz, MD, discussed corrected data from a phase 3 trial of NurOwn that BrainStorm believes supports a BLA submission.

4. Alexis Thompson, MD, MPH, on Qualifying Treatment Centers for Beti-Cel in Thalassemia

The primary investigator and chief of hematology at Children’s Hospital of Philadelphia discussed requirements for administering beti-cel.

5. GPC3 CAR-T Leads to Long Term Complete Response in Hepatocellular Carcinoma

A case study showed that the patient achieved a complete response in 12 months after treatment with CARsgen Therapeutics’ cell therapy.