Genable, Spark Therapeutics deal
Spark granted Genable exclusive, worldwide rights to patents covering manufacturing of adeno-associated virus (AAV) vectors to treat rhodopsin-linked retinitis pigmentosa (RP). Spark will be the exclusive manufacturer for Genable's lead product, GT038, which is in preclinical testing to treat RP. Spark will provide development advice in the development of GT038. Spark will receive an upfront payment, payments for the manufacture and supply of GT038 and is eligible to receive milestones and royalties. GT038 has Orphan Drug designation in the U.S. to treat RP and in the EU to treat rhodopsin-linked RP. The product is an AAV vector encoding an RNAi targeting rhodopsin (Rho; Opn2) in combination with an AAV vector containing a Rho gene. Genable expects to start Phase I testing in 2016. The partners declined to disclose financial details. ...