Drug for rare genetic disorder receives FDA approval with help of licensing by Michele Gunness

Michele Gunness, Ph.D.

UPDATE: On June 30, 2020, the FDA approved the use of triheptanoin to treat pediatric and adult patients with molecularly confirmed long-chain fatty acid oxidation disorders (LC-FAOD). See the Ultragenyx press release.

Published in Research News

Rare pediatric disease dataset license by Michele Gunness receives Deal of Distinction Award

Michele Gunness, Ph.D., has been recognized for her role in a creative technology licensing deal for a rare pediatric disease dataset. The deal resulted in Fast Track Designation and Rare Pediatric Disease Designation by the FDA for the treatment of long-chain fatty acid oxidation disorders.

Unlike traditional licensing models, the intellectual property in this deal was a limited data set from 32 subjects with a fatty acid oxidation disorder. There are approximately 2,500 patients in the U.S. with these disorders.

OHSU School of Medicine researchers Melanie B. Gillingham, Ph.D., and Cary O. Harding, M.D., conducted the largest investigator-initiated clinical trial in fatty acid oxidation disorders and the only double-blind randomized trial in the field. The FDA-funded trial was conducted at OHSU and the University of Pittsburgh.

The deal negotiated by Gunness among OHSU, the University of Pittsburgh, and Ultragenyx Pharmaceutical brings together the assets and talents of academic medical centers and a biomedical company.

The 2019 Deal of Distinction Award for the Industry-University-Government Interface Sector was given to Gunness, senior technology development manager at OHSU Technology Transfer, by the Licensing Executives Society at the LES Annual Meeting Award Luncheon, Oct. 21.