FDA accepts Homology’s IND for Phase 1/2 study of PKU gene therapy


Homology Medicines (NASDAQ:FIXX) received FDA clearance to commence its Phase 1/2 study of HMI-102 in adults with phenylketonuria (PKU), a rare genetic disease affecting metabolism.

Individuals with PKU lack the phenylalanine hydroxylase (PAH) enzyme that metabolizes phenylalanine. If left untreated, phenylalanine levels rise significantly, putting patients at risk of severe neurological impairment. HMI-102 is a one-time gene therapy that delivers a functional copy of the PAH gene to liver cells, enabling production of the enzyme.

“A potentially curative gene therapy that provides the functional gene and relieves the burden of PKU would be a major advance for patients and their families,” Arthur Tzianabos, Homology’s president and CEO, said in a statement.

Homology notes that this Phase 1/2 trial would represent the first PKU gene therapy to enter the clinic. The company anticipates initial data will be available by the end of 2019.

Abby Hardy