CGTLive’s Weekly Rewind – 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.