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Therapeutic Focus

Therapeutic Focus

Homology Medicines’ genetic medicines platform has the potential to treat and cure a wide range of genetic diseases through gene editing or gene therapy.

We are initially working to cure monogenic diseases, or diseases that are caused by a defect in a single gene. We are prioritizing diseases with significant unmet medical need and validated regulatory pathways to bring new treatments forward as rapidly as possible.

For each target, we are deploying either a gene therapy or gene editing approach based on disease biology. In both approaches, we are using our proprietary human hematopoietic stem cell-derived adeno-associated virus vectors (AAVHSCs) to deliver the treatment to patients.

Gene editing involves the insertion of a corrected gene directly into the genome. With gene editing, a person’s DNA is permanently corrected. This approach can potentially be curative including in rapidly dividing cells (e.g., hematopoietic CD34+ cells and pediatric liver cells).

Gene therapy involves the transfer of corrected genes to specific cells in the body. With gene therapy, the genes do not integrate into the genome. This approach can be potentially curative in slow- or non-dividing cells (e.g., adult liver or central nervous system).

Homology’s Genetic Medicines Pipeline

*Homology retains U.S. rights to in vivo programs; Novartis has ex-U.S. rights and worldwide rights to ex vivo programs.


Adult Phenylketonuria (PKU)
Our most advanced product candidate is a gene therapy treatment (HMI-102) for phenylketonuria, or PKU. PKU is a rare inborn error of metabolism caused by a mutation in the PAH gene. PKU results in a loss of function of the enzyme phenylalanine hydroxylase, which is responsible for the metabolism of phenylalanine (Phe), an amino acid obtained exclusively from the diet. If left untreated, toxic levels of Phe can accumulate in the blood and result in progressive and severe neurological impairment. Currently, there are no treatment options for PKU that target the underlying genetic cause of the disease. It has been estimated that PKU affects nearly 15,000 people in the U.S. with a worldwide prevalence of 50,000 people.

We are in IND-enabling studies with HMI-102, a gene therapy candidate designed to address the genetic cause of the disease by delivering functional copies of the PAH gene to adult patients via our proprietary AAVHSCs. HMI-102 was granted Orphan Drug Designation by the U.S. Food and Drug Administration.