Review top news and interview highlights from the week ending 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.
The FDA has accepted BioMarin’s resubmitted biologics license application (BLA) for valoctocogeneroxaparvovec (val-rox) for review in treating severe hemophilia A.
All 5 patients on ERT at study start have since been able to withdraw after gene therapy treatment.
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.
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.
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.