BioMarin Pharmaceutical Publishes Follow-Up Data for Hemophilia A Gene Therapy Phase I/II Study

January 7, 2020

BioMarin Pharmaceutical Publishes Follow-Up Data for Hemophilia A Gene Therapy Phase I/II Study

BioMarin announced the publication in the New England Journal of Medicine of a peer-reviewed article covering up to three years of follow-up data from an ongoing phase II/II study evaluating valoctocogene roxaparvovec, an investigational AAV-based gene therapy for severe hemophilia A. The results demonstrate a "sustained, clinically relevant benefit" to patients from a single infusion of valoctocogene roxaparvovec. With our extensive temperature-controlled storage, packaging, and courier capabilities, Yourway is well equipped to support clinical trials for gene therapies and other advanced therapies.

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