Grant Award Details
To develop a small molecule treatment for osteoarthritis injected intra-articularly to enhance MSC chondrogenic differentiation in vivo. This award is running in parallel with the team's PC1 award. This CLIN1 award funds the GMP manufacturing of the drug and the conduct of the IND-enabling toxicology studies to complete the IND package and file an IND with the FDA for a Phase 1 trial.
Grant Application Details
- Development of a Chondrogenic Drug Candidate Targeting Resident Mesenchymal Stem Cells for the Treatment of Osteoarthritis
Therapeutic Candidate or Device
The therapeutic candidate is a drug-like small molecule that promotes cartilage resident mesenchymal stem cell differentiation into chondrocytes.
Osteoarthritis and cartilage injury
The therapeutic candidate, through intra-articular administration, promotes cartilage resident mesenchymal stem cell differentiation into chondrocytes. The newly formed chondrocytes replace the dead chondrocytes, synthesize and secret extracellular matrix proteins, which leads to the repair of damaged cartilage in OA patients or following traumatic injury.
Unmet Medical Need
Current therapeutic options for OA are limited to pain or symptom-modifying drugs and joint replacement surgery; no disease-modifying drugs are approved for clinical use. The therapeutic candidate, if successful, will be the first-in-class regenerative medicine for OA and cartilage injury.
IND filing and initiating Phase 1 clinical trial
Major Proposed Activities
- IND document preparation and filing
- GLP toxicology and safety profiling of the therapeutic candidate
- Non-GLP determination of maximum tolerated doses upon local administration
Osteoarthritis (OA) is the most prevalent musculoskeletal disease and globally the 4th leading cause of Years Lost to Disease (YLD). The annual economic impact of arthritis in the U.S. is estimated at over $100 billion. No disease-modifying OA drugs are approved for clinical use. Clearly the development of a new disease-modifying therapeutic would have a significant impact on the well-being of Californians and reduce the negative economic impact on the state.