CGTLive’s Weekly Rewind – December 1, 2023
Review top news and interview highlights from the week ending December 1, 2023.
CGTLive’s Weekly Rewind – December 1, 2023
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. FDA to Investigate Risk of T-Cell Malignancy After CAR T-Cell Therapy
The FDA has received reports of multiple T-cell malignancies in patients after approved CAR-T treatment.
2. Marcela Maus, MD, PhD, on New Approaches to CAR T-Cell Therapy for Solid Tumors
The Associate Professor of Medicine at Harvard Medical School discussed upcoming research and strategies to combat present hurdles in the treatment field.
3. Second Gene Therapy for Epidermolysis Bullosa up for Priority Review
Pz-cel has a PDUFA date of May 25, 2024.
4. Fernanda Mesquita, PhD, on Using LAMA2-Exosomes to Improve Cardiomyocyte Survival in Stroke
The research associate at the The Texas Heart Institute discussed preclinical research she presented at AHA’s 2023 Scientific Sessions on MSC-derived exosomes.
5. Autolus Submits Obe-Cel BLA for Adult R/R B-ALL
The company also plans to submit an MAA to the EMA in the first half of 2024.
FDA Announces Probe Into bluebird's Elivaldogene Autotemcel for Hematologic Malignancies
November 27th 2024Approved as Skysona, the therapy has been reported to be related to cases of hematologic malignancies, including life-threatening instances of myelodysplastic syndrome and acute myeloid leukemia.
First Patient Dosed in RIDGE-1 Trial for Tenaya’s ARV Cardiomyopathy Gene Therapy TN-401
November 26th 2024The patient’s dosing took place at the University of California, San Francisco, although the multicenter study is expected to eventually dose patients at other locations in the United States, United Kingdom, and Europe.
