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.
pheNIX Gene Therapy Clinical Trial for PKU
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.
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 clinicaltrials.gov and search the study ID HMI-100-102 or the identifier NCT04768348.
juMPStart Gene Therapy Clinical Trial for Hunter Syndrome
Homology is conducting the juMPStart clinical trial with investigational gene therapy HMI-203 in adults with Hunter syndrome.
Hunter syndrome is caused is caused by mutations in the IDS gene, which results in the inability to produce the key functional enzyme idurnate-2-sulfatase, or I2S. This leads to toxic lysosomal accumulation of glycosaminoglycans (GAGs), which causes progressive debilitation. HMI-203 is an investigational gene therapy that contains a functioning IDS gene to potentially override the effects of the disease-causing mutation. HMI-203 is designed as a one-time treatment.
To learn more about juMPStart, download our fact sheet.