AAV-gene therapy

Affinia Therapeutics is a gene therapy company developing adeno-associated virus (AAV) gene therapies for rare and prevalent diseases, starting with diseases of the heart and nervous system. Its proprietary ART (Affinia Rationally-designed Therapies) platform uses AI and structural modeling to rationally design novel AAV capsids and therapeutic payloads. Its first program, AFTX-201, targets BAG3-associated dilated cardiomyopathy (DCM).

AI and structural modelingcapsid engineeringgene editinggene replacementgene therapy manufacturingknockdownpayload designvectorized antibody

BioViva USA Inc is a research and development company working on medical technologies and treatments aimed at addressing aging-associated disorders and enhancing human health through genetic interventions. It develops gene therapy platforms, including AAV and CMV gene therapy, designed to help cells behave more youthfully and target the biological processes of aging. BioViva states it does not provide gene therapy to patients.

CMV gene therapygene therapy developmentresearch and development

Kinea Bio is a Seattle-based biotechnology company founded in 2020 by scientific leaders in the neuromuscular disease space. It is developing next-generation genetic medicine for neuromuscular diseases, including Duchenne Muscular Dystrophy (DMD), dysferlinopathy, and DM1, using AAV-gene therapy approaches such as its Split-Intein Dual-AAV approach and SIMPLI-GT™ technology.

gene therapynon-viral gene therapy deliverySIMPLI-GT™ technologySplit-Intein Dual-AAV approach