Fighting Blindness Annual Report

This year the Foundation continued its long-standing strategy of funding the most promising retinal science studies across the spectrum of these diseases. We also made several major investments in ongoing research through collaborations with industry and clinical partners.

The Foundation currently funds 75 projects at prominent laboratories and research clinical centers around the world. Listed below are a few of the significant developments and investments made by the Foundation during the past year. Following these summaries is a list of clinical trials currently underway which were made possible, in part, due to FFB support. Taken together, this growing number of clinical trials and the increasing number of FFB–industry collaborations demonstrate the ways in which FFB is helping to move treatments closer to the marketplace and to the patients who will benefit from them.

FDA Advisory Committee Recommends Approval of RPE65
Gene Therapy

The most exciting news of the year was the unanimous vote by the U.S. Food and Drug Administration’s Advisory Committee on Cellular, Tissue, and Gene Therapies to recommend FDA approval of voretigene neparvovec. The emerging treatment, developed by Spark Therapeutics and to be marketed as Luxturna™, is for people with vision loss from Leber congenital amaurosis and retinitis pigmentosa caused by RPE65 gene mutations. The treatment delivers functional copies of the RPE65 gene directly into the retina thereby compensating for the nonfunctional, mutated copies. FFB invested $10 million in research over two decades that made clinical trials for the gene therapy possible. At press time, the ultimate FDA decision on the treatment was expected by January 2018.

SparingVision Formed to Advance Sight-Saving Protein

The development of a treatment with the potential to save vision for people with retinitis pigmentosa by slowing the progression of the disease is getting a major boost thanks to the formation of the French biotech company SparingVision, to move it into a clinical trial and out to the international marketplace. The Foundation Fighting Blindness Clinical Research Institute (FFB-CRI) made an $8.2 million equity investment in the new company.

A spinoff of the Institut de la Vision, SparingVision was established to clinically develop and commercialize a protein known as rod-derived cone viability factor (RdCVF). The emerging, vision-saving therapy performed well in several FFB-CRI-funded lab studies at the Institut de la Vision in Paris. SparingVision’s goal is to launch a clinical trial in 2019.

Foundation Investing in Drug to Slow Many Forms of
Retinitis Pigmentosa

Sometimes, fighting blindness means helping people save the vision they have, or at least slowing disease progression enough so they can maintain useful vision for all of their lives.

That’s the idea behind a promising, emerging drug for retinitis pigmentosa (RP) known as N-acetylcysteine-amide (NACA).

The Foundation Fighting Blindness Clinical Research Institute announced an investment of up to $7.5 million to advance the potential therapy into and through a Phase 2 clinical trial.