Homology Medicines is focused on advancing our gene therapy and nuclease-free gene editing technology forward as one-time treatments and potential cures for patients with rare diseases.
Clinical trials are an important part of the drug development process, and vital to treatments being reviewed and approved before potentially becoming available to patients.
Homology is conducting the pheNIX clinical trial with investigational gene therapy HMI-102 in adults with phenylketonuria (PKU). Gene therapy refers to a technique that uses genes to treat and potentially cure certain diseases.
PKU is caused by mutations in the PAH gene responsible for breaking down phenylalanine (Phe) as it enters the body through diet. As a result of the body’s inability to properly break down Phe, it accumulates to toxic levels in the blood, causing the symptoms experienced by PKU patients. HMI-102 is an investigational gene therapy that contains a functioning PAH gene to potentially override the effects of the disease-causing mutation. HMI-102 is designed as a one-time treatment.
HMI-102 is not approved by the U.S. Food and Drug Administration (FDA) or any other health authority outside of the U.S.