We are at a point in time where it is possible to translate genetic discoveries into cures.
The field of human genetics is discovering disease-causing mutations at an unprecedented rate. These genetic mutations drive many rare, debilitating and fatal disorders. While some treatments are available to address symptoms, or slow the progression of genetic diseases, treatments that directly address the causative genes are needed to develop cures for patients.
Two advanced approaches to curing genetic diseases are gene editing and gene therapy. Homology Medicines' technology offers the option to pursue both approaches and can provide important advantages over current methods. Our proprietary technology is based on the discovery of a novel class of human-derived adeno-associated virus (AAV) vectors that can be used to achieve precise and efficient in vivo and ex vivo gene editing, as well as gene therapy.
Our AAV technology is based on the pioneering research of Saswati Chatterjee, Ph.D., Professor of Virology at the Beckman Research Institute at the City of Hope in California. Dr. Chatterjee and her team led the first AAV vector-mediated gene transfer studies into human hematopoietic stem cells (HSCs) and subsequently identified and isolated a series of naturally-occurring AAVHSCs from human CD34+ cells. These vectors have many unique properties, including high efficiency and precise gene editing, plus the ability to deliver to the central nervous system (CNS), which can be bolstered by technology we licensed from the California Institute of Technology.
Our gene editing platform, AMEnDR™ (AAV-Mediated Editing by Direct Homologous Recombination), harnesses the naturally occurring process of homologous recombination to correct gene mutations. Homologous recombination is a natural biological mechanism used by cells to ensure highly precise DNA repair.
Homology Medicines' approach to gene editing begins with designing homology sequence arms that are highly specific to a region of the human genome and results in a permanent therapeutic correction in the DNA when delivered to cells. The delivery of Homology Medicines' gene editing therapy is accomplished using our proprietary AAVHSC vectors. These AAVHSC vectors travel to the cell's nucleus and facilitate homologous recombination directed repair. Our scientists can engineer this combination of homology arms with AAVHSC vectors to treat a range of disease-causing mutations through:
Gene editing provides the potential to not only treat, but cure genetic diseases with a one-time administration.
Learn more about our advanced technology:
Homology Medicines' gene editing platform offers unique potential advantages:
- Single platform that enables in vivo gene editing
- Facilitates highly precise gene editing in an ex vivo approach
- Leverages the natural gene repair process of homologous recombination and does not require a nuclease for gene cutting
- Precise on-target editing with potential to detect any off-target sites
- Editing efficiencies that can achieve the requisite therapeutic threshold
Our gene therapy approach utilizes our proprietary AAVHSC vectors to deliver a functional gene to a cell where there is a missing or mutated gene. Once delivered, the functional gene may lead to therapeutic protein expression. In non-dividing cells, gene therapy could cure a genetic disease. Our industry-leading number of unique vectors have demonstrated significant systemic biodistribution to multiple tissue types in preclinical studies, including liver, muscle (skeletal and cardiac), ocular and CNS. This enables the selection of the optimal therapeutic vector based on the tissue involved in the disease.