Data Show Potential of Roctavian Gene Therapy for Hemophilia A

Data Show Potential of Roctavian Gene Therapy for Hemophilia A

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Data Show Potential of Roctavian Gene Therapy for Hemophilia A

Gene constructs making up Roctavian, an investigational gene therapy for hemophilia A, did not permanently integrate into the genome of non-human primates or cause any signs of tumors or malignancy in the animals.  These are key findings from preclinical studies that BioMarin Pharmaceutical, the therapy’s developer, presented at the 24th American Society of Gene and Cell Therapy (ASGCT) Annual Meeting, held online, May 11–14. “BioMarin is committed to furthering our scientific understanding of gene therapy…

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