CGTLive’s Weekly Rewind – October 14, 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. Val-Rox up for Review in Hemophilia A 2 Years After Initial BLA Submission

The FDA has accepted BioMarin’s resubmitted biologics license application (BLA) for valoctocogeneroxaparvovec (val-rox) for review in treating severe hemophilia A.

2. Patients With Fabry Disease Withdrawn From ERT After Isaralgagene Civaparvovec Treatment

All 5 patients on ERT at study start have since been able to withdraw after gene therapy treatment.

3. Roberto Giugliani, MD, PhD, on Advantages of Gene Therapy in MPS Type 2

The professor of genetics at Federal University of Rio Grande do Sul discussed how RGX-121 has the potential to change the treatment landscape of MPSII.

4. Multiple Sclerosis Cell Therapy Demonstrates Promising Safety and Efficacy in Phase 2 Trial

Nearly 20% of treated patients achieved at least 25% improvement in timed 25-foot walk speed and/or 9-Hole Peg test at 28 weeks.

5. Nick Boyle, PhD, on Advantages of In Vivo CAR Therapies

The chief executive officer of Abintus Bio discussed the company’s in vivo approach and how it could help expand patient access to cell therapies.