BioMarin readies study of gene therapy for PKU

BioMarin Pharmaceutical (BMRN -0.5%) has submitted an application to UK regulators seeking signoff to launch a Phase 1/2 clinical trial evaluating a single administration of BMN 307 in patients with phenylketonuria (PKU), a rare inborn error of metabolism that causes the amino acid phenylalanine to build up in the body causing severe health problems.

The company expects to start enrollment in Q1 2020 in the UK and is actively preparing regulatory submissions in other countries.

BMN 307 is an AAV5 phenylalanine hydroxylase gene therapy.

PKU-related tickers: Synlogic (SYBX -1.9%), Rubius Therapeutics (RUBY -0.5%), Homology Medicines (FIXX -0.9%), Retrophin (RTRX -0.2%), Codexis (CDXS -1%),

https://seekingalpha.com/news/3502066-biomarin-readies-study-gene-therapy-pku