CGTLive’s Weekly Rewind – May 27, 2022

Article

Review top news and interview highlights from the week ending 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.

Recent Videos
Barry J Byrne, MD, PhD, the chief medical advisor of MDA and a physician-scientist at the University of Florida
Sarah Larson, MD, the medical director of the Immune Effector Cell Therapy Program in the Division of Hematology/Oncology at David Geffen School of Medicine at University of California, Los Angeles (UCLA)
Manali Kamdar, MD, the associate professor of medicine–hematology and clinical director of lymphoma services at the University of Colorado
Manali Kamdar, MD, the associate professor of medicine–hematology and clinical director of lymphoma services at the University of Colorado
Ben Samelson-Jones, MD, PhD, assistant professor pediatric hematology, Perelman School of Medicine, University of Pennsylvania and Associate Director, Clinical In Vivo Gene Therapy, Children’s Hospital of Philadelphia
Manali Kamdar, MD, the associate professor of medicine–hematology and clinical director of lymphoma services at the University of Colorado
Steven W. Pipe, MD, a professor of pediatric hematology/oncology at CS Mott Children’s Hospital
Haydar Frangoul, MD, the medical director of pediatric hematology/oncology at Sarah Cannon Research Institute and Pediatric Transplant and Cellular Therapy Program at TriStar Centennial
Bhagirathbhai R. Dholaria, MD, an associate professor of medicine in malignant hematology & stem cell transplantation at Vanderbilt University Medical Center
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