Benjamin Yerxa, PhD, CEO, Foundation Fighting Blindness
How Will FFB Steer and Sustain
the Field’s New Pace of Discovery?
IT’S BEEN A THRILLING couple of months for
the retinal disease research field. In October Spark
Therapeutics made a strong case before the U.S. Food
and Drug Administration’s Cellular, Tissue, and Gene
Therapies Advisory Committee that its testing of
voretigene neparvovec, a gene therapy for mutations of
the RPE65 gene, demonstrated the therapy’s safety and
effectiveness. The data presented led to a unanimous
recommendation that the treatment be approved.
Then in December, voretigene neparvovec, now being
marketed as LUXTURNA™, received full FDA approval.
This approval of the first gene therapy for the eye or any
inherited disease was a historic milestone for the field
and for our community. (See more about its significance
in the page 1 story in this newsletter issue.) Without a
doubt, the field is at a new pace of play, one that holds
exciting promise for the immediate future.
In the 47 years since the Foundation Fighting Blindness,
then called the RP Foundation, was created, the
discoveries and accumulated knowledge achieved are
truly remarkable. When the Foundation opened its
doors in 1971, very little was known about inherited
retinal diseases, and almost no research on cures was
being done. In the now almost five decades since, the
field has identified about 250 of the mutated genes that
cause inherited vision loss. We have delivered a few
treatments to the marketplace, and numerous others are
being tested now in 25 clinical trials underway or about
to begin. It’s fair to say that while progress hasn’t always
been fast, that pace has now quickened considerably.
The question before us is how best to steer and sustain
this new pace of discovery.
One of the most critical results of the FDA approval
of LUXTURNA, beyond that people with mutations
to the RPE65 gene will enjoy the gift of sight due to
the treatment, is the very real momentum it provides
for other gene therapies now in the pipeline. Simply
put, momentum will beget more momentum. That will
be especially true in the area of industry investments in
emerging gene therapies. But, we also need to remember
that advances in science are often non-linear. That’s
the fascinating thing about science and the reason for
the need for sustained and innovative investment in
research; you never know which study is going to unlock
the mystery that will deliver the next cure.
How will FFB work to sustain the increased pace of
progress? We’ll do a few things:
• Work to increase our already successful public/
private partnerships with the biotech and
• Focus on the most promising treatments across
multiple technologies including gene, cell, and drug
• Search for gene-independent, possible pan-retinal
• Find financial, academic, and corporate partners;
• Invest early to attract industry and mainstream
venture investment (funding that’s critical to close
still-significant research funding gaps);
• Attract the best and brightest science minds to
retinal disease research through FFB’s early career
awards programs and by investing in the early
research that researchers often have a difficult time
attracting support for; and
• Continue to partner with our dedicated volunteer
leaders and generous donors to fulfill our shared
dream of a world without vision loss due to
inherited retinal disease.
In closing, I wholeheartedly salute all the members
of the Foundation Fighting Blindness team — our
governance and scientific advisory leaders, our research
and industry partners, and, most importantly, field
volunteers and donors. The last few years have been
exciting, but not as exciting as the next few will be.