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BioMarin, Pioneer in Phenylketonuria (PKU) and Gene Therapy, Doses First Participant in Global PHEARLESS Phase 1/2 Study of BMN 307 Gene Therapy
BioMarin Builds Upon 15+ Year Commitment to PKU Community with Potential 3rd Therapy in PKU Franchise Company Leverages Gene Therapy Manufacturing Expertise
About this update from Biomarin Pharmaceutical Inc.
[{"type":"text","content":"BioMarin Builds Upon 15+ Year Commitment to PKU Community with Potential 3rd Therapy in PKU Franchise\n Company Leverages Gene Therapy Manufacturing Expertise Using Commercial-Ready Process\n\n\nSAN RAFAEL, Calif., Sept. 24, 2020 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) announced today that it has dosed the first participant in the global PHEARLESS Phase 1/2 study with BMN 307, an investigational gene therapy for the treatment of individuals with PKU. BMN 307 is an AAV5-phenylalanine hydroxylase (PAH) gene therapy designed to normalize blood phenylalanine (Phe) concentration levels in patients with PKU by inserting a correct copy of the PAH gene into liver cells. BMN 307 will be evaluated to determine safety and whether a single dose of treatment can restore natural Phe metabolism, normalize plasma Phe levels, and enable a normal diet in patients with PKU. \n\n \n \n \n \n \n \n\n \nBioMarin will conduct this study with material manufactured with a commercial-ready process to facilitate rapid clinical development and potentially support approval. BMN 307 represents a potential third PKU treatment option in BioMarin's PKU franchise and a second gene therapy development program. \n\"More than 70 years ago, the first child was treated for PKU in the United Kingdom at Birmingham Women's and Children's Hospital. Today, we continue to make strides in PKU treatment through the clinical study of a gene therapy for PKU,\" said Tarekegn G. Hiwot at University Hospitals Birmingham NHS Foundation Trust and principal investigator for the PHEARLESS study. \"There is a tremendous unmet need for PKU patients. As a treating physician, it is important to me to be involved in clinical research to evaluate innovative therapies that have the potential to change the treatment paradigm in PKU for good.\"\n\"BioMarin has been committed to the PKU community for more than 15 years and remains dedicated to the research and development of innovative therapies to advance the standard of care for people with PKU,\" said Hank Fuchs, M.D., President, Worldwide Research and Development at BioMarin. \"Building upon our experience of delivering two approved PKU therapies to the PKU community, BMN 307 gene therapy combines BioMarin's leadership in the development of PKU therapies with our expertise in gene therapy development and manufacturing.\...