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’ proprietary technology platform offers the option to pursue both approaches and can provide important advantages over current methods.
Our technology platform is based on the pioneering research of one of our founders, 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 adeno-associated virus (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.
Our proprietary AAVHSC vectors have many unique properties that can enable the development of potentially one-time curative treatments:
Homology’s novel AAVHSCs are packaged with either a gene editing or gene therapy construct. Our gene editing construct includes lengthy guide sequences, or homology arms, which are designed to enable the specific alignment to the desired genomic location and then, through the natural process of homologous recombination, correction of the diseased gene in the genome by replacement with a whole functional copy. Our gene therapy construct includes a functional copy of the gene and a promotor sequence that is designed to enable the gene to be turned on in the cell and ultimately transcribed to express a therapeutic protein without integrating into the genome.