14 May 2019 - Company provides details of pheNIX, the first gene therapy trial for PKU, and remains on track to report initial clinical results from the Phase 1/2 study in 2019.

Homology Medicines announced today that the U.S. FDA has granted fast track designation for HMI-102, Homology’s one-time gene therapy development candidate for the treatment of adults with phenylketonuria (PKU). PKU is an inborn error of metabolism primarily caused by mutations in the PAH gene that lead to phenylalanine hydroxylase (PAH) deficiency resulting in the inability to breakdown phenylalanine (Phe), an essential amino acid found in natural protein.

In April 2019, Homology announced that FDA cleared the Investigational New Drug (IND) application for HMI-102 for the treatment of PKU. Previously, the FDA and the European Medicines Agency (EMA) granted orphan drug designation for HMI-102 in the United States and European Union for the use of human hematopoietic stem cell-derived adeno-associated virus AAVHSC15 to treat PAH deficiency, the primary cause of PKU. The Company is currently working with treatment centers to begin the Phase 1/2 pheNIX trial for adult patients with PKU, and expects to report initial clinical data in 2019.

Read Homology Medicines press release