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RUBY - BioMarin readies study of gene therapy for PKU
Rubius Therapeutics Inc.
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.
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