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Clinical Trials

Clinical Trials

Homology Medicines is focused on advancing our genetic medicines platform 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.

What are the stages of drug development?
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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 designed as a one-time treatment that contains a functioning PAH gene to potentially override the effects of the disease-causing mutation.

How Could HMI-102 Work in the Body?

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To learn more about pheNIX, download our fact sheet (English or Spanish), visit, search for “HMI-102” on or talk to your physician.

HMI-102 is not approved by the U.S. Food and Drug Administration (FDA) or any other health authority outside of the U.S.

pheEDBACK Natural History Study

We are also conducting pheEDBACK, a one-year natural history study for adults with PKU due to PAH deficiency. To learn more, visit and search the study ID HMI-100-102 or the identifier NCT04768348.