Genascence

Genascence Corporation is a clinical-stage biotechnology company founded in 2017, focusing on the development of gene therapies for prevalent musculoskeletal diseases. The company was established by a team with extensive experience in gene therapy, including CEO Thomas W. Chalberg, Ph.D., Annahita Keravala, Ph.D., Christopher H. Evans, Ph.D., Paul Robbins, Ph.D., and Steven Ghivizzani, Ph.D. Chalberg, a scientist and serial entrepreneur, has a background in molecular biology and has founded other successful biotech companies like SightGlass Vision and Avalanche Biotechnologies. His academic credentials include a Ph.D. in genetics from Stanford University and an MBA from UC Berkeley, reflecting a career aimed at translating genetic knowledge into medical treatments.

The company's core business revolves around developing life-changing medicines for common conditions that affect millions, such as osteoarthritis (OA). Its technology is licensed from prominent U.S. research institutions, including the Mayo Clinic, the University of Florida, and NYU Langone Health. Genascence's business model is centered on the research, development, and eventual commercialization of its gene therapy products. The company's primary client base will be patients suffering from musculoskeletal diseases, a market with significant unmet needs as current treatments primarily manage symptoms without slowing disease progression. Genascence has secured funding through a seed round in 2019 and a Series A round in 2022, raising a total of $10.5 million from investors like Pacira BioSciences, DeepWork Capital, and the University of Florida. It also received an $11.6 million award from the California Institute for Regenerative Medicine (CIRM).

Genascence's lead product candidate is GNSC-001, a gene therapy for osteoarthritis of the knee. This treatment involves a single, intra-articular injection designed to provide long-term relief. GNSC-001 uses a recombinant adeno-associated vector (AAV) to deliver a gene that produces Interleukin-1 receptor antagonist (IL-1Ra), a naturally occurring protein that blocks the inflammatory actions of Interleukin-1 (IL-1). IL-1 is a key molecule in the progression of OA, causing inflammation, pain, and cartilage destruction. By enabling joint cells to continuously produce IL-1Ra, GNSC-001 aims to provide sustained inhibition of inflammation and potentially slow or reverse joint degeneration. The company is conducting the DONATELLO Phase 1b clinical trial to evaluate the safety and efficacy of GNSC-001, with initial data expected in late 2024. The FDA has granted Fast Track designation for GNSC-001, highlighting the urgent need for new OA treatments.

Keywords: gene therapy, musculoskeletal diseases, osteoarthritis, clinical-stage, biotechnology, AAV vector, IL-1Ra, joint disease, intra-articular injection, biopharmaceuticals