BioMarin announces gene therapy candidate for PKU

BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) said in its 1Q18 earnings report that it plans to submit an IND next year for a gene therapy candidate to treat phenylketonuria (PKU).

The candidate will be an adeno-associated virus (AAV) vector containing the DNA sequence that codes for the phenylalanine hydroxylase enzyme, which is deficient in PKU. BioMarin said the candidate led to sustained normal levels of the amino acid phenylalanine without inducing hypophenylalanemia in preclinical models...