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
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
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
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.
In several animal models, including previous FFB-funded lab
studies of rodent models at Johns Hopkins University, NACA
slowed retinal degeneration.
Nacuity Pharmaceuticals, Inc., a start-up company in Fort Worth,
Texas, owns the rights to NACA for ophthalmology and will be
Foundation Research Funding Commitments
and Investment Highlights