Genable Technologies, Spark Therapeutics to advance novel therapy for rare form of retinitis pigmentosa

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Spark Therapeutics and Genable Technologies announced today that they have entered into a collaboration agreement for Genable's lead therapeutic to treat rhodopsin-linked autosomal dominant retinitis pigmentosa (RHO adRP), GT038. Under the terms of the collaboration, Genable will license certain adeno-associated virus (AAV) vector manufacturing patents from Spark. The parties have entered into a broad agreement in which Spark will be the exclusive manufacturer of the product and provide development advice and expertise to Genable to help in the ongoing development of GT038. Spark will receive milestone payments and royalties on future sales of GT038, as well as near-term revenue from the manufacture and supply of product.

"We are excited to apply our deep expertise in AAV clinical development and manufacturing to augment Genable's great work, and expand the number of debilitating diseases of the eye that can be addressed through gene therapy," said Jeffrey D. Marrazzo, Spark Therapeutics co-founder, president and CEO.

Dr. Jason Loveridge, CEO of Genable Technologies commented, "We have chosen Spark as our partner to advise, lend their experience and manufacture GT038 based on their broad expertise in gene therapy.  We see them as a world-class organization, and we are excited to be advancing our novel therapy GT038 into the clinic."

"The collaboration with Spark provides an exciting opportunity to greatly expedite development of Genable's novel therapy targeted towards RHO-adRP," said Professor Jane Farrar, founder and director, Genable Technologies; professor, Trinity College (Dublin).

GT038 is a potential treatment for rhodopsin (RHO)-linked autosomal dominant retinitis pigmentosa (adRP), an inherited retinal dystrophy that leads to blindness in most cases. There is currently 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.

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