Review top news and interview highlights from the week ending 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.
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.
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.
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).
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.
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).