CGTLive’s Weekly Rewind – June 3, 2022
Review top news and interview highlights from the week ending June 3, 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. ICER Finds Beti-Cel Superior to Standard-of-Care for β-Thalassemia
Beti-cel was rated a B+ for lingering questions about durability and unknown risks.
2. Val-Rox BLA for Hemophilia A Delayed Again
BioMarin now expects to file in September 2022 instead of June.
3. FDA Approves Tisa-Cel Expanded Indication in R/R Follicular Lymphoma
The approval was based on data from the phase 2 ELARA clinical trial, in which a complete response of more than 65% was observed.
4. Expediting the Development of Patient-Specific iSPC Cell Therapies
Stefan Braam, PhD, chief executive officer of Ncardia and Cellistic discussed the companies’ work in the cell therapy space.
5. Liso-Cel Yields High Response Rates and Positive PROs in LBCL
Treated patients had significant improvements in fatigue and lymphoma symptoms.
News and Expert Insights in Hemophilia Gene Therapy for Bleeding Disorders Awareness Month 2025
March 9th 2025In observance of Bleeding Disorders Awareness Month, held annually in March, catch up on some of the latest news and expert insights in gene therapy for hemophilia, which is among the most common of these disorders.
