CGTLive’s Weekly Rewind – August 12, 2022
Review top news and interview highlights from the week ending August 12, 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. Zolgensma Gene Therapy Linked to 2 Deaths in SMA Patients, Novartis Reports
The 2 deaths, due to acute liver failure, occurred in patients treated in Kazakhstan and Russia.
2. Ide-Cel Shows Benefit in Earlier Use in R/R Multiple Myeloma
A recent comparison of health-related quality of life scores also favored ide-cel over belantamab mafodotin treatment.
3. UniQure Pauses High Dose of AMT-130 Huntington Disease Gene Therapy for Severe Adverse Events
No such adverse events have occurred in the lower-dose group.
4. First Patient Dosed in Sickle Cell Gene Therapy Clinical Trial From Graphite Bio
Nula-cel is intended to directly correct the mutation that causes sickle cell disease.
5. Frontotemporal Dementia Gene Therapy Trial Initiates Dosing
PBFT02, developed by Passage Bio, is delivered via intra cisterna magna administration to the brain.
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.
