Homology is leveraging its genetic medicines platform to develop one-time gene therapy and gene editing treatments caused by variants in a single gene. In addition, the company is leveraging its gene therapy approach to develop one-time GTx-mAb treatments for continuous antibody production.
Its platform is based on the discovery of a family of adeno-associated viruses, which were found inside human stem cells (AAVHSCs). They are capable of delivering genetic material throughout the body, including across the blood-brain-barrier to the central nervous system.
In developing a genetic medicine, the company begins by choosing the AAVHSC that reaches the area(s) of the body needed to address the specific disease we are targeting. It then designs the product candidate to precisely and efficiently deliver genetic medicines following a one-time I.V. infusion (in vivo) using either a gene therapy, nuclease-free gene editing, or GTx-mAb modality.
Its platform is based on the discovery of a family of adeno-associated viruses, which were found inside human stem cells (AAVHSCs). They are capable of delivering genetic material throughout the body, including across the blood-brain-barrier to the central nervous system.
In developing a genetic medicine, the company begins by choosing the AAVHSC that reaches the area(s) of the body needed to address the specific disease we are targeting. It then designs the product candidate to precisely and efficiently deliver genetic medicines following a one-time I.V. infusion (in vivo) using either a gene therapy, nuclease-free gene editing, or GTx-mAb modality.