Posted by Michael Wonder on 04 Oct 2020
BioMarin receives FDA fast track designation for investigational gene therapy, BMN 307
2 October 2020 - Second investigational gene therapy in clinic, potential third therapy in PKU franchise.
BioMarin Pharmaceutical announced today that the U.S. FDA has granted fast track designation to BMN 307, an investigational gene therapy for the treatment of individuals with phenylketonuria.
Last week, BioMarin announced that it had dosed the first participant in the global Phearless Phase 1/2 study with BMN 307, an AAV5-phenylalanine hydroxylase (PAH) gene therapy designed to normalize blood phenylalanine concentration levels in patients with phenylketonuria by inserting a correct copy of the PAH gene into liver cells.
