CGTLive’s Weekly Rewind – May 27, 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. Gene Therapy for Knee Cartilage Lesions Receives RMAT Designation

NeoCart was originally developed by the company Histogenics.

2. EtranaDez Accepted for Priority Review in Hemophilia B

A final analysis of the pivotal HOPE-B study was presented at ASGCT 2022.

3. Developing Safer Viral Vectors for Gene Therapy

The chief executive officer of Ring Therapeutics, Tuyen Ong, MD, discussed the potential of anellovirus vectors for use in gene therapy.

4. Ami J. Shah, MD, on Reaching Normalized Hemoglobin With Gene Therapy in PKD

The clinical professor of pediatrics at Stanford Medicine discussed positive interim findings from a phase 1 study of RP-L301.

5. Gene Therapy for AADC Deficiency Receives Positive CHMP Opinion

The positive opinion was based on findings from studies conducted in Taiwan.