AAVAA Therapeutics

AAVAA Therapeutics is a preclinical-stage biotechnology company focused on developing adeno-associated virus (AAV)-mediated gene therapies and gene editing solutions for skeletal and bone-related diseases. Co-founded by world-renowned gene therapy pioneer Dr. Guangping Gao and bone disease expert Dr. Jae-Hyuck Shim, the company leverages bone-directed recombinant AAV (rAAV) vectors and CRISPR-Cas9 technology to target genetic and chronic bone disorders. Its pipeline includes therapeutic programs for rare skeletal diseases such as Fibrodysplasia Ossificans Progressiva (FOP) and Osteogenesis Imperfecta (OI), as well as prevalent conditions like osteoporosis, osteoarthritis, and rheumatoid arthritis. By utilizing tissue-specific promoters and advanced gene silencing/editing techniques, AAVAA Therapeutics aims to deliver curative, single-injection treatments that address the root genetic causes of debilitating skeletal conditions.