News Feature | March 26, 2014

Spark And Genable Tech Collaborate On Rare Gene Therapy

By Estel Grace Masangkay

Spark Therapeutics and Genable Technologies announced that they have signed a collaboration agreement to develop GT038, Genable’s lead therapeutic drug for the treatment of rhodopsin-linked autosomal dominant retinitis pigmentosa (RHO adRP).

The partnership will allow Genable to license specific adeno-associated virus (AAV) vector manufacturing patents from Spark. Spark will gain exclusive manufacturing rights of the product and will receive milestone payments and royalty on future sales of GT038. The company is also eligible to receive near term revenue from the manufacture and supply of GT038, for which it will provide development advice and expertise.

Jeffrey Marrazzo, Spark Therapeutics co-founder, president and CEO, said, “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.”

GT038 is a potential drug for the treatment of rhodopsin (RHO)-linked autosomal dominant retinitis pigmentosa (adRP), which affects approximately 30,000 patients around the world. The disease is an inherited retinal dystrophy that often leads to blindness. No approved pharmacologic treatment for adRP currently exists. GT038 has an established safety and efficacy profile for the utilization of AAV vectors to deliver RNA interference (RNAi) molecules. This suppresses the expression of faulty and normal copies of RHO, and restores normal gene expression. Professor Jane Farrar, founder and director of Genable Technologies, and a professor at Trinity College in Dublin, said, “The collaboration with Spark provides an exciting opportunity to greatly expedite development of Genable's novel therapy targeted towards RHO-adRP.”

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 drug has been granted Orphan Drug Designation status in both the EU and U.S.