BioMarin Pharmaceutical Inc. recently updated research results regarding an investigational gene therapy treatment for severe hemophilia A, presenting its findings at the 59th American Society of Hematology (ASH) Annual Meeting and Exposition in Atlanta.
The California-based company, a provider of gene therapy solutions for addressing unmet serious medical needs, delivered an oral presentation of its revised phase 1/2 study data examining the potential of valoctocogene roxaparvovec (formerly BMN 270), after 1.5 years, including information gleaned through mid-November, according to a company press release.
Primary investigator John Pasi, a professor of haemostasis and thrombosis from Barts and the London School of Medicine and Dentistry, presented the information, updating earlier reports from July and October.
"With 1.5 years of data on valoctocogene roxaparvovec, our understanding of this novel gene therapy is considerable, and we are looking forward to drawing on that knowledge in the Phase 3 GENEr8 studies, which we expect will begin enrolling patients this month," Dr. Hank Fuchs, BioMarin’s president for worldwide research and development, said in the release.
BioMarin simultaneously published the newer findings in the New England Journal of Medicine. Its research study centered on six patients with severe hemophilia A.
"We're pleased that this work is being presented in one of the most highly regarded peer reviewed publications and at a pace that is faster than other in vitro gene therapies,” Fuchs said in the release. “This most current data presented at ASH combined with the one-year data published in NEJM will contribute to an increase in understanding within the scientific community about gene therapy in general and specifically about the exciting new developments in a potential one-time treatment for severe hemophilia A."