Retinitis pigmentosa is the most common inherited eye disease. Mutations in the gene for rhodopsin, the pigment for photoreceptor cells in the retina responsible for the perception of light, account for 15 percent of retinitis pigmentosa cases. CHOP spinout Spark Therapeutics, which has a late stage retinitis pigmentosa gene therapy treatment under development, is collaborating with Ireland-based biopharma company Genable Technologies, according to a company statement.
The deal will focus on Genable’s lead therapeutic to treat rhodopsin-linked autosomal dominant retinitis pigmentosa, called GT038. Under the deal terms, Genable will license certain manufacturing patents from Spark. Philadelphia-based Spark will be the exclusive manufacturer of the product and provide development advice and expertise to Genable to help in its development of GT038. In exchange, Spark received an initial payment and will receive subsequent milestone payments and royalties on future sales of GT038, and near-term revenue from the manufacture and supply of the product, according to the statement.
In response to emailed questions a spokeswoman for Spark Therapeutics said: “Generally, the company recognizes that there are a significant number of severe genetic diseases without effective treatments in therapeutic areas where it has significant expertise (for example, inherited blindness, hematologic disorders, neurodegenerative diseases), and if Spark can play a role in expanding the number of products that get to market through partnerships, the team will certainly entertain those possibilities.”
Rhodopsin-linked autosomal dominant retinitis pigmentosa affects roughly 30,000 people worldwide and there are currently no drugs to treat it. Genable’s retinitis pigmentosa gene therapy is designed to suppresses the expression of faulty and normal copies of the rhodopsin gene and restores normal gene expression.
Its treatment has been given Orphan Drug Designation in both the U.S. and Europe which extends the exclusivity for the treatment, if it’s approved, and gives the company certain tax credits. Genable has the rights to market its treatment globally, if it succeeds in getting the treatment approved.
Spark Therapeutics has a few gene therapies under development. One is for patients with inherited Leber’s congenital amaurosis and retinitis pigmentosa. Another is for Hemophilia B in Phase 1/2 development. Earlier this year CEO Jeff Marrazzo said the company expects to have data from a Phase 3 open-label, randomized control trial for its treatment for blindness caused by mutations of the RPE65 gene available in the first half of next year.
