Findings in the Area of Gene Therapy Reported from BioMarin Pharmaceutical Inc. (Clinical Immunogenicity Outcomes From Gener8-1, a Phase 3 Study of Valoctocogene Roxaparvovec, an Aav5-vectored Gene Therapy for Hemophilia A).

Researchers from BioMarin Pharmaceutical Inc. have conducted a phase 3 study on valoctocogene roxaparvovec, a gene therapy for the treatment of hemophilia A. The study found that no inhibitors were detected following the administration of the therapy, and immune responses were primarily directed towards the AAV5 capsid. FVIII-specific cellular immune responses were less frequent and showed no association with safety or efficacy parameters. This research provides valuable insights into the immunogenicity outcomes of gene therapy for hemophilia A. [Extracted from the article]