Date: 2016-05-04
Type of information: R&D agreement
Compound: AT342
Company: Audentes Therapeutics (USA - CA) Perelman School of Medicine at the University of Pennsylvania (USA - PA)
Therapeutic area: Rare diseases - Genetic diseases - Hepatic diseases - Liver diseases
Type agreement: R&D
Action mechanism: gene therapy.
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 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.
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