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).