Date: 2016-05-04
Type of information: Collaboration agreement
Compound: AT342
Company: Audentes Therapeutics (USA - CA) University of Pennsylvania (US - PA)
Therapeutic area: Rare diseases - Genetic diseases - Hepatic diseases - Liver diseases
Type agreement: collaboration - research
Action mechanism:
- gene therapy. AT342, is an AAV vector containing a functional version of the UGT1A1 gene. Preclinical data in murine models of the disease demonstrate AAV-UGT1A1 significantly reduces bilirubin levels, even at UGT1A1 liver expression levels of just five to eight percent of normal.
Disease: Crigler-Najjar Syndrome
Details:
- • On May 4, 2016, Audentes Therapeutics announced a collaboration with the Orphan Disease Center at the Perelman School of Medicine at the University of Pennsylvania ("Penn") to develop AT342, an AAV gene therapy for Crigler-Najjar Syndrome, a rare, inherited, metabolic liver disease. In addition, Audentes has expanded its existing partnership with RegenXBio to include an exclusive worldwide license for the treatment of Crigler-Najjar using RegenXBio's NAV® AAV8 vector. This program was initiated at Penn under the leadership of James M. Wilson, M.D., Ph.D., a pioneer in human gene therapy and the director of the Gene Therapy Program and the Orphan Disease Center at Penn.
- Audentes plans to file an IND for AT342 in 2016 and expects preliminary data from a Phase 1/2 clinical trial to be available in 2017.
Financial terms:
Latest news:
Is general: Yes
