Biotech company Spark Therapeutics, Irish firm to partner on blindness drug

Spark Therapeutics L.L.C., a Philadelphia company developing gene-based medicines for debilitating diseases, has signed a collaborative partnership with a gene-therapy firm in Ireland to develop a product to treat a rare form of blindness, the companies announced Tuesday.

Spark, a biotechnology company spun out of research at Children's Hospital of Philadelphia, said Dublin-based Genable Technologies Ltd. will license certain patents from Spark, which will be the exclusive manufacturer and provide development expertise for a potential treatment for blindness caused by inherited retinal dystrophy.

Spark will receive milestone payments and royalties on future sales, as well as revenue from the manufacturing and supply of the product. Financial terms were not disclosed.

Children's Hospital has committed to investing up to $50 million in Spark, which seeks to be the nation's first commercial provider of gene therapy.

"The scientific community has identified about 207 different genes that are involved with various forms of retinal degeneration that cause blindness," said Jeffrey D. Marrazzo, Spark's cofounder and CEO.

Genable's therapeutic strategy "knocks down" or suppresses the "over-expression of one gene" causing a disease, while at the same time "replacing something else that is missing in the patient," Marrazzo said in an interview.

Spark is developing two potential products to treat inherited retinal disease. One is in a late-stage, or Phase 3, clinical trial, "which is likely to be the first approved gene therapy in the United States," Marrazzo said.

A second drug candidate for retinal disease will begin clinical trials shortly. "We haven't announced the specifics of that program yet," he said.

Initial results on its lead drug candidate in late-stage testing have been promising, Spark said: Once-blind patients could recognize faces and were moved out of Braille classrooms. They were no longer considered legally blind, and some were able to drive a car.

"In that earlier trial, five of the 12 participants were school-age," Marrazzo said. "All five went from Braille to sighted classrooms" after one injection in one eye with the treatment.

Spark also is overseeing an early-stage clinical trial to treat hemophilia B.

The company's founding team includes scientists who led the movement to develop gene therapy as a new treatment paradigm. Gene therapy is the concept of treating disease by replacing or correcting faulty genes.

Many scientists supporting Spark are still working at Children's Hospital.

"Our goal is to grow to about 50 people or so by the end of the year," Marrazzo said.