GeneTherapyLive’s Weekly Rewind – November 26, 2021

Welcome to GeneTherapyLive’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 Accepts Beti-Cel BLA for β-thalassemia

The FDA has accepted bluebird bio’s Biologics License Application for betibeglogene autotemcel (beti-cel) for the potential treatment of β-thalassemia. The gene therapy, which will receive priority review, has a PDUFA date of May 20, 2022.

2. Overcoming Challenges With CAR T Therapies in Solid Tumors: Julian Molina, MD, PhD

The hematologist/oncologist from Mayo Clinic discussed the role of HLA loss in a variety of cancers. He also outlined the challenges of CAR T-cell therapies in treating solid tumors and how Tmod technology may overcome these.

3. Gene-Edited, Autologous HSP Cells Correct Metabolic Activity in Hurler Syndrome

Autologous hematopoietic stem and progenitor cells transduced ex vivo with an IDUA–encoding lentiviral vector yielded extensive metabolic correction in peripheral tissues and the central nervous system in patients with Hurler syndrome (mucopolysaccharidosis type 1, Hurler variant) according to data from a recent phase 1/2 study (NCT03488394).

4. Targeting the Putamen in AADC Deficiency: Paul Wuh-Liang Hwu, MD, PhD

The professor from National Taiwan University Hospital discussed the benefits of delivering gene therapy directly to the putamen in AADC deficiency. Hwu is an investigator on the trials of PTC-AADC, an investigational gene therapy in this indication.

5. Hemophilia A Gene Therapy Shows Sustained Factor VIII Expression

SPK-8011, an investigational gene therapy for hemophilia A, was well-tolerated and yielded sustained Factor VIII expression in treated men, according to data from a phase 1/2 study (NCT03003533) and its long-term follow-up study (NCT03432520).