Agonist monoclonal antibody therapeutic activating GPR49 for regenerative medicine

Funding Type: 
Early Translational III
Grant Number: 
ICOC Funds Committed: 
Public Abstract: 

Advancement in our understanding of stem cell biology over the last decade has provided a tremendous opportunity to pursue regenerative medicines for diseases with unmet medical needs. We plan to accelerate the promise of regenerative medicine by leveraging our experience in conventional antibody drug development to modulate a critical stem cell pathway involved in intestinal tissue regeneration. Crohn's disease and ulcerative colitis, collectively known as inflammatory bowel disease (IBD), affect as many as 1.4 million Americans. IBD is characterized by a chronic relapsing course of diarrhea, abdominal pain, weight loss and rectal bleeding. Direct treatment costs for IBD in US were estimated at $2 billion annually in 1999 while indirect costs may be much higher. Patients struggle with debilitating symptoms for much of their adult life. While several treatment option exist, as many as 60% of patients do not respond to primary therapy or show signs of acute relapse and/or disease progression, indicating a considerable unmet need for more effective therapies. Our promising discovery work has led to a development program to achieve proof of concept for an antibody treatment to drive intestinal renewal in these diseases.

Statement of Benefit to California: 

This project will benefit the citizens of California by leveraging experience in conventional drug therapies to realize the potential of regenerative medicine. This will benefit citizens in providing better treatment options for diseases with unmet medical needs. In addition, this project will provide the state with the benefit of decreased financial burden on the health care system from savings due to more effective treatments. Successful completion of the project will also support the approach of utilizing conventional drug development in regenerative medicine. This will benefit the robust California biotechnology industry by validating this strategy which could lead to additional drugs in this category or in other indications in the future. Finally, this project will benefit the state of California through job creation. Progression of our drug candidate will correlate with increased employment at our company and support contract research organizations.