Genable Receives $6.8 Million Investment to Develop Gene Therapy for Dominant RP

November 14, 2011
Image of a row of test tubes in a laboratoryThanks to success in preclinical studies funded in part by the Foundation Fighting Blindness, the Irish biopharmaceutical company Genable Technologies has received a €5 million (equivalent to about $6.8 million) venture capital investment to advance its gene therapy for autosomal dominant retinitis pigmentosa (adRP) into a clinical trial.

The company plans to launch the clinical study in 2013. In 2010, the European Medicines Agency, a regulatory organization similar to the FDA, gave the treatment an orphan designation, which provides Genable with marketing and development incentives to facilitate its advancement.

“We are very excited to see Genable’s treatment attracting significant venture capital investment. It is important for us to fund emerging therapies that can attract outside investment to move them into and through clinical trials,” says Dr. Stephen Rose, chief research officer, Foundation Fighting Blindness. Dr. Rose also serves on Genable’s board of directors in a non-voting, observational role.

Known as GT038, Genable’s gene therapy is for people with adRP caused by mutations in the gene rhodopsin. The treatment works by silencing, or “knocking down,” the defective copies of the rhodposin gene and, at the same time, delivering normally functioning copies of the gene to retinal cells. A manmade virus known as an adeno-associated virus, or AAV, is used to deliver the treatment to retinal cells.

Researchers have identified mutations in 23 genes that can cause adRP. Defects in rhodopsin are the most common cause of adRP, accounting for about 25 percent of all adRP cases.

“The rhodopsin gene is an important target for treatment, because it can affect so many members of so many families,” says Dr. Rose. “As a cause of dominant disease, the gene can get passed down from generation to generation. Genable’s therapy provides real hope that we can stop blindness from being part of these families’ legacies.”