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Owings Mills, MD - September 24, 2008 - The Foundation Fighting Blindness (FFB) has announced an agreement with Genable, an Irish biopharmaceutical company, to develop therapies to treat retinal degenerative diseases. 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.
Genable’s emerging treatment employs RNA interference to silence defective genes that cause progressive vision loss in dominant forms of retinitis pigmentosa (RP). A key advantage of the treatment is that in principle it has the potential to work regardless of the specific mutation in a given gene that is causing vision loss. Multiple disease-causing mutations within the same gene represent a significant problem associated with the development of treatments for dominantly inherited RP. Genable’s technologies address this particular therapeutic problem.
The approach utilizes an adeno-associated virus vector (AAV) to deliver the therapeutic to the affected retina. AAVs are also being used in other types of gene therapy including the landmark clinical studies recently reported by researchers in the Children’s Hospital of Philadelphia, Moorfields Eye Hospital, and the Universities of Pennsylvania and Florida that resulted in positive outcomes for patients with Leber congenital amaurosis, an early-onset form of RP that causes severe vision loss.
“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, Chief Drug Development Officer, NNRI. “Furthermore, there haven’t been many other options for people with dominant RP.”
NNRI is providing funding to expedite additional preclinical development. NNRI will have an observational (non-voting) seat on Genable’s Board of Directors.
About the Foundation Fighting Blindness and National Neurovision Research Institute
The Foundation Fighting Blindness is a nonprofit organization headquartered in Owings Mills, Maryland. The urgent mission of FFB is to drive the research that will provide preventions, treatments and cures for people affected by retinitis pigmentosa, macular degeneration, Usher syndrome, and the entire spectrum of retinal degenerative diseases. |
