Collaborative led by Dr. Hansen has named a Phase I winner of The National Institutes of Health Common Fund’s Targeting RNA in Disease with Novel Technologies (TRDNT) Challenge.

Some inherited diseases begin with a single genetic change that tells a cell to stop making a protein too soon. These “nonsense mutations” account for roughly one in ten genetic defects behind inherited disease.

The University of California, San Francisco-Harvard COUPLET Collaborative has been named a Phase I winner of The National Institutes of Health Common Fund’s Targeting RNA in Disease with Novel Technologies (TRDNT) Challenge.

Led by Dr. Scott Hansen, chief of the Department of Surgery Division of Plastic and Reconstructive Surgery, alongside Dr. Jason Pomerantz, Dr. Shailesh Agarwal, and Research Fellow Shaan Sekhon, the team is developing a precision RNA platform designed to restore production of full-length proteins from a patient’s own RNA.

Epidermolysis bullosa is the first target, with cystic fibrosis to follow. For people with cystic fibrosis caused by these mutations, the approach could help address a gap left by treatments that have transformed care for many other patients.

Congratulations to the COUPLET team!