Principal Investigators: Abramson
Genascence is a clinical-stage biotechnology company developing life-changing gene therapy products for musculoskeletal diseases. GNSC-001 is a genetic medicine—a recombinant adeno-associated virus vector carrying a coding sequence for interleukin-1 receptor antagonist (IL-1Ra), a potent inhibitor of IL-1 signaling. GNSC-001 is administered as a one-time intra-articular injection, which is a straightforward procedure performed in a physician’s office. GNSC-001 is designed to offer long-term, sustained inhibition of IL-1 following a single injection.
GNSC-001 is currently being developed for osteoarthritis of the knee. In pre-clinical studies, a single treatment with GNSC-001 lasted beyond one year and was able to improve both symptoms and joint structure in a large animal model.