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.
Exploring New Pathways in Muscular Dystrophy Research: From Fibrosis to Function
April 30th 2025Matthew Alexander, PhD, a neuromuscular expert, discussed the evolving landscape of targeted therapies in muscular dystrophies, from fibrosis and glycosylation to combination strategies with gene therapy.
