Study to Evaluate the Efficacy and Safety of Valoctocogene Roxaparvovec, With Prophylactic Steroids in Hemophilia A

Clinical Publications

• Evaluation of one-stage and chromogenic assays for the laboratory measurement of factor VIII activity following valoctocogene roxaparvovec infusion
• Blood biodistribution and vector shedding of valoctocogene roxaparvovec in people with severe hemophilia A
• Valoctocogene roxaparvovec gene therapy provides durable haemostatic control for up to 7 years for haemophilia A
• Activity of transgene-produced B-domain-deleted factor VIII in human plasma following AAV5 gene therapy
• Clinical immunogenicity outcomes from GENEr8-1, a phase 3 study of valoctocogene roxaparvovec, an AAV5-vectored gene therapy for hemophilia A
• Efficacy, safety, and quality of life 4 years after valoctocogene roxaparvovec gene transfer for severe hemophilia A in the phase 3 GENEr8-1 trial
• Three-year outcomes of valoctocogene roxaparvovec gene therapy for hemophilia A
• Comparative Effectiveness of Valoctocogene Roxaparvovec and Prophylactic Factor VIII Replacement in Severe Hemophilia A
• Long-term safety and efficacy outcomes of valoctocogene roxaparvovec gene transfer up to 6 years post-treatment
• Health-related quality of life following valoctocogene roxaparvovec gene therapy for severe hemophilia A in the phase 3 trial GENEr8-1
• Safety and efficacy of valoctocogene roxaparvovec with prophylactic glucocorticoids: 1-year results from the phase 3b, single-arm, open-label GENEr8-3 study
• Two-Year Outcomes of Valoctocogene Roxaparvovec Therapy for Hemophilia A
• Outcomes and management of invasive procedures in participants with hemophilia A post gene therapy: a post hoc analysis of the GENEr8-1 phase III trial
• Interindividual variability in transgene mRNA and protein production following adeno-associated virus gene therapy for hemophilia A
• Valoctocogene Roxaparvovec Gene Therapy for Hemophilia A
• Persistence of haemostatic response following gene therapy with valoctocogene roxaparvovec in severe haemophilia A
• Early Phase Clinical Immunogenicity of Valoctocogene Roxaparvovec, an AAV5-Mediated Gene Therapy for Hemophilia A
• Matching-adjusted indirect comparison of bleeding outcomes in severe haemophilia A: Comparing valoctocogene roxaparvovec gene therapy, emicizumab prophylaxis, and FVIII replacement prophylaxis

Preclinical Publications

• Molecular analysis of AAV5-hFVIII-SQ vector-genome-processing kinetics in transduced mouse and nonhuman primate livers
• Induction of ER Stress by an AAV5 BDD FVIII Construct Is Dependent on the Strength of the Hepatic-Specific Promoter
• Prednisolone Does Not Regulate Factor VIII Expression in Mice Receiving AAV5-hFVIII-SQ: Valoctocogene Roxaparvovec
• The Impact of Pre-existing Immunity on the Non-clinical Pharmacodynamics of AAV5-Based Gene Therapy
• Young mice administered adult doses of AAV5-hFVIII-SQ achieve therapeutic factor VIII expression into adulthood
• Lack of germline transmission in male mice following a single intravenous administration of AAV5-hFVIII-SQ gene therapy

Protocol

• FDA Approval Documents
• Clinical Trial Protocol
• Statistical Analysis Plan