Spark Therapeutics of Philadelphia and Genable Technologies, a privately held Dublin, Ireland-based biopharmaceutical company, are collaborating on Genable's lead therapeutic GT038, used to treat rhodopsin-linked autosomal dominant retinitis pigmentosa (RHO adRP). Genable will license certain adeno-associated virus (AAV) vector manufacturing patents from Spark. Spark will be the exclusive manufacturer of the product and provide development advice and experience to Genable to help in the ongoing development of GT038. Spark will receive milestone payments and royalties on future sales of GT038.
Jason Loveridge, CEO of Genable Technologies, said, "We have chosen Spark as our partner to advise, lend their experience and manufacture GT038 based on their broad expertise in gene therapy.”
RHO adRP is an inherited retinal dystrophy that leads to blindness in most cases. There currently is no approved pharmacologic treatment for adRP, which affects an estimated 30,000 patients worldwide. GT038 utilizes AAV vectors with an established safety and efficacy profile to deliver RNA interference (RNAi) molecules to suppress the expression of faulty and normal copies of RHO and restore normal gene expression. GT038 has been granted Orphan Drug Designation in both the U.S. and Europe.