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Advancing our understanding of gene therapy research, one publication at a time

Science | March 25, 2022

Dr. Wing Yen Wong is the Group Vice President and Head of Global Medical Affairs leading medical aspects of BioMarin’s development of medicines for optimal patient outcomes with a proven track record of drug development spanning clinical research to global medical affairs activities to support approved and pipeline therapeutic agents.

BioMarin Publishes Data from Phase 3 Hemophilia A Trial in the New England Journal of Medicine

Forty years ago, the idea of gene therapy seemed like science fiction. Deliver a genetic blueprint to human cells using modified viruses or other molecular tools? It’s not hard to imagine the Sisyphean journey required to move this technology from skeptics’ territory to clinical research. Yet science has brought us to an exciting point at which gene therapy research is progressing rapidly with a wealth of ongoing studies teaching us more by the day about how to target genetic diseases and manufacture gene therapies.

We’re excited to be part of that journey. At BioMarin, among our many ongoing studies is a phase 3 trial for severe hemophilia A—one of the largest phase 3 studies of any gene therapy, and the longest duration of clinical experience for any gene therapy in hemophilia A. additional data from that study was published in the New England Journal of Medicine (NEJM), marking the third article detailing this investigational therapy that has been published by the Journal. This publication provides one-year data from the phase 3 clinical study, which is further supported by the learnings we presented last month at the European Association for Haemophilia and Allied Disorders (EAHAD). The article is also referenced in an editorial published in the same issue of the Journal putting gene therapy research into the context of existing management options.

“We are proud to be pioneers in the study of gene therapy for severe hemophilia A and to share individual patient data that facilitates a more complete understanding of the full data set of this potentially transformative medicine,” said Hank Fuchs, M.D., President of Worldwide Research and Development at BioMarin. “Valoctocogene roxaparvovec has been studied longer than any other gene therapy for hemophilia A, and year after year, we continue to increase our knowledge of how this investigational therapy may potentially benefit the lives of people with hemophilia A. We are grateful to the study participants and investigators for their essential role in this development program, which includes GENEr8-1, the largest gene therapy study in hemophilia A.” It is important to note that research into valoctocogene roxaparvovec is ongoing; it has not been determined to be safe or effective or approved for use outside of a clinical trial.

In addition to the findings shared in NEJM, BioMarin is conducting multiple concurrent studies with valoctocogene roxaparvovec, including an analysis of human liver biopsies and a study to evaluate the impact of a prophylactic corticosteroid regimen on FVIII expression following administration of valoctocogene roxaparvovec in severe hemophilia A.

Each of these studies helps answer specific questions about BioMarin programs and about gene therapy as a whole. BioMarin’s ongoing investment in gene therapy research aims to help advance the entire field of gene therapy to meet the urgent needs of patients. The publication of this research marks an exciting step in that process, and we’re looking forward to learning and sharing more.

Dr. Wing Yen Wong is the Group Vice President and Head of Global Medical Affairs leading medical aspects of BioMarin’s development of medicines for optimal patient outcomes with a proven track record of drug development spanning clinical research to global medical affairs activities to support approved and pipeline therapeutic agents.

Dr. Wong is also an accomplished physician in the treatment and management of patients with hemophilia and hemoglobinopathies with over 20 years of experience in pediatric hematology/oncology with over 50 peer-reviewed clinical and scientific publications.