Spark Therapeutics Purchases the Irish Company Genable

March 07, 2016

Spark Therapeutics, the Philadelphia-based, gene-therapy company responsible for developing a treatment targeting retinal diseases caused by mutations in the RPE65  gene, has purchased Genable, an Irish bio-pharmaceutical company developing  a gene therapy for the treatment of autosomal dominant retinitis pigmentosa (adRP). The Foundation Fighting Blindness was an early investor in Genable’s treatment, known as RhoNova™, and it is hoped that this transaction will expedite the development of the therapy.

“We are delighted that this transaction with Spark Therapeutics will ensure the expeditious clinical development of RhoNova™ for the treatment of autosomal dominant, rhodopsin-linked retinitis pigmentosa, a leading cause of inherited blindness,” says Annette Clancy, chair of the board of directors of Genable. “The unanimous recommendation of the board was that Spark Therapeutics, world leaders in AAV gene therapies, has the relevant resources and expertise to maximize the chance of success for RhoNova™ and deliver its benefit for patients. We wish them every success.”

Bill Schmidt, CEO of the Foundation, added, “Genable, with early FFB support, has done extensive work in the area of gene therapy for people with adRP. As we well know, this type of research, specifically moving it from the lab to clinical trials, is a very expensive venture. We believe Spark’s purchase of Genable will allow the research progress on RhoNova™ to continue and ultimately, we hope, move into delivery to patients.”

The advance of RhNova™ is a just one of many examples of FFB’s strategy to fund promising research at its early stages, as a means of de-risking it for later investment.