CGTLive’s Weekly Rewind – February 3, 2023
Review top news and interview highlights from the week ending February 3, 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 Blocks 4D’s Gene Therapy Program for Fabry, Clears IND for Diabetic Macular Edema
The clinical hold comes a few weeks after the company announced it was stopping enrollment in the phase 1/2 clinical trial.
2. Raj Mehra, PhD, and Krishna Subramanian, PhD, on Striving For Disease-Modifying Therapies in Parkinson Disease
Mehra and Subramanian discussed preclinical research with the investigational gene therapy SLS-004.
3. Giant Axonal Neuropathy Gene Therapy Needs Double-blind Trial Before BLA Submission
The dosing of additional patients in a double-blind, placebo-controlled design will help support a BLA submission for TSHA-120.
4. Gerhard Ehninger, MD, on Managing Toxicities With Switchable CARs in AML
The professor at University Hospital Dresden discussed the positive safety profile of Unicar-T-CD123.
5. Remestemcel-L Cell Therapy Makes Comeback With Resubmitted BLA for Pediatric GvHD
Mesoblast is resubmitting its BLA with new CMC, survival, and mechanism of action data on the therapy and phase 3 trial.
Evaluating Allogeneic CAR-T P-BCMA-ALLO1 in R/R Multiple Myeloma
November 21st 2024Bhagirathbhai R. Dholaria, MD, an associate professor of medicine in malignant hematology & stem cell transplantation at Vanderbilt University Medical Center, discussed interim data from the phase 1/1b clinical trial evaluating Poseida's CAR-T.
World Pancreatic Cancer Day 2024: Looking Back at Progress in Cell and Gene Therapy
November 21st 2024In observance of World Pancreatic Cancer Day, held on the third Thursday of November each year, we took a look back at the past year's news in cell and gene therapy for pancreatic cancer indications.
