Columbia University

Technology Ventures

Engineered Deubiquitinases (enDUBs)

Engineered Deubiquitinases (enDUBs): Many diseases are caused by deficits in the stability, expression, or proper targeting of one or more proteins. Post-translational stabilization of such proteins is a potentially powerful therapeutic strategy that has not yet been achieved. We have developed a proprietary platform technology¾ engineered deubiquitinases (enDUBs)¾ to selectively tune the ubiquitin status of target proteins to restore expression and function to treat diseases. As a first indication for this technology, we have targeted cystic fibrosis (CF), a devastating monogenic rare disease caused by loss-of-function mutations in a chloride channel (CFTR), that results in lung damage and premature death. CF represents a huge unmet clinical need with a multi-billion-dollar market potential. Many CF mutations result in reduced trafficking of CFTR to the surface of epithelial cells lining the lungs. In proof-of-concept studies we have shown that enDUBs correct this basic deficit with greater efficiency than the FDA-approved pharmacological corrector, lumacaftor, currently on the market. Excitingly, enDUB and lumacaftor together have a synergistic impact leading to a super rescue of CFTR surface trafficking and function. We are seeking investment partners to move this early-stage technology through pre-clinical studies with the aim of filing an IND for CF therapy within two years.

To learn more about this opportunity, contact:

Joan José Martínez
Technology Licensing Officer
joan.martinez@columbia.edu