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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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pheEDIT Gene Editing Clinical Trial for PKU 

We are conducting the pheEDIT clinical trial with investigational gene editing product HMI-103 in adults with PKU. Gene editing is designed to make permanent correction to the genome where there is a missing or mutated gene. 

HMI-103 is designed as a one-time, in vivo product candidate incorporating nuclease-free gene editing for PKU. Once safety and efficacy are established in adults, we plan to initiate trials in pediatric patients. 

 

How Could HMI-103 Work in the Body?

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To learn more about pheEDIT, download our fact sheet.

pheEDBACK Natural History Study

Aditionally, we are 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.

HMI-203 is designed as a one-time, in vivo treatment that delivers functioning IDS genes to the peripheral organs and the central nervous system. 

 

How Could HMI-203 Work in the Body?

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To learn more about juMPStart, download our fact sheet.

HMI-103 and HMI-203 are not approved by the U.S. Food and Drug Administration (FDA) or any other health authority outside of the U.S.