Date: 2016-05-04
Type of information: Licensing agreement
Compound: NAV rAAV8 and rAAV9 vectors
Company: Audentes Therapeutics (USA - CA) Regenx Biosciences (USA - MD)
Therapeutic area: Rare diseases - Genetic diseases
Type agreement: licensing development commercialisation
Action mechanism: gene therapy.
Disease: X-Linked Myotubular Myopathy (XLMTM), Pompe disease
Details: * On July 30, 2013, REGENX Biosciences and Audentes Therapeutics announced that they have entered into an agreement for the development and commercialization of products to treat X-Linked Myotubular Myopathy (XLMTM) and Pompe disease using NAV vectors. Under the terms of the Agreement, REGENX granted Audentes an exclusive worldwide license, with rights to sublicense, to REGENX’s NAV rAAV8 and rAAV9 vectors for treatment of XLMTM and Pompe disease in humans.
Financial terms: In return for these rights, REGENX receives an up-front payment, certain milestone fees and royalties on net sales of products incorporating NAV rAAV8 and rAAV9.
Latest news: * 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.
