I’m pleased to welcome you to the 2017 Foundation Fighting
Blindness Annual Report. While I have the privilege of opening this
report, it takes a team effort to achieve FFB’s mission. I encourage
you, our most loyal donors, to take a few minutes to read the full
report. In doing so you will see how your generosity has helped
fuel a revolution in inherited retinal disease science — one that puts
us solidly into an era of clinical trials and on the cusp of providing
treatments for some retinal degenerations!
FFB provides the all-important seed money to support the work of the world’s best retinal disease scientists. We propel their
best ideas forward so they can be studied, attract larger funding investments from private and public sources, and, in a few
cases, earn FDA approval!
This strategy has yielded strong dividends. One of the most significant was this past October’s recommendation by the U.S.
Food and Drug Administration Advisory Committee on Cellular, Tissue, and Gene Therapies that Spark Therapeutics’ gene
therapy for the treatment of RPE65 gene mutations be approved. This anticipated therapy will not only be life-changing
for people with LCA caused by the RPE65 mutation, it will help move additional gene-based ocular and other treatments
forward. See pages 3—5 for other highlights of the year and for a list of ongoing clinical trials that have their roots in FFB
funding — 24 to date and across the spectrum of inherited retinal diseases.
What’s behind this amazing progress? We take your donated dollars and, through a review process involving the field’s
leading researchers and clinicians, deploy them worldwide, leveraging your donation for additional investments. Give us
$100 or $1,000 or $25,000, and we will carefully invest that money in research that we believe will not only improve the
field’s understanding of how to treat retinal diseases but will also attract the larger investments required to bring those
treatments to market.
Our current priority is moving more and more therapies that show promise in the lab to clinical trials to show safety and
efficacy. We need your continued help to do so.
In closing I want to reiterate that fulfilling our mission to end blindness caused by inherited retinal disease takes a team.
I want to thank the score of FFB researchers and clinicians who are working hard every day to find cures. I want to thank
those experts who sit on FFB’s Research Oversight Committee and our Scientific Advisory Board for continually lending
their expertise to the effort. I want to recognize the patients who courageously participate in clinical trials. Thanks as well to
our hardworking staff. And, finally, I want to again thank our donors. Without you, none of the treatment progress FFB has
helped create over the last four decades would have been possible.
in this issue
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