Partnership Aims at Treatment for Dominant RP

FFB is partnering with the Irish biopharmaceutical company Genable to develop an innovative gene therapy for people affected with autosomal dominant forms of retinitis pigmentosa or adRP.

Genable has reported encouraging results from an early, preclinical study of their treatment. The company aims to begin a clinical trial of the therapy in 2010.

The emerging treatment employs small interfering RNA (siRNA) to silence the defective genes that cause progressive vision loss in dominant RP. In addition to shutting down the disease-causing gene, a new, healthy gene is delivered to the retina.

A key advantage of the treatment is that it shows potential to work regardless of the genetic variation that is causing vision loss. Approximately 18 genes have been linked to dominant RP.

The approach uses an adeno-associated virus (AAV) to deliver the therapeutic gene to the affected retina. AAVs are also being used in other retinal disease studies including the landmark clinical trial at The Children’s Hospital of Philadelphia, Moorfields Eye Hospital, and the Universities of Pennsylvania and Florida that resulted in vision-restoring outcomes for patients with Leber congenital amaurosis, a severe form of RP that manifests early in life.

“We are excited about the partnership, because it is a promising technology and an approach that may help many people with different forms of dominant RP,” says Mauricio Vargas-Cortes, Ph.D., Chief Drug Development Officer, NNRI. “Furthermore, there haven’t been many other options for people with dominant RP.”

The partnership is being formed through FFB’s clinical support organization, the National Neurovision Research Institute (NNRI). Fighting Blindness Ireland is also a partner in the agreement.

NNRI is providing funding for hiring senior scientists to perform additional preclinical research. NNRI will also obtain an equity stake in Genable, as well as an observational (non-voting) seat on Genable’s Board of Directors.