programs and the option to license discovery programs for three
additional indications at the time of clinical candidate selection.
Stem Cells for RP Move Into Human Studies
In 2015, two companies, ReNeuron (U.K.) and jCyte (California),
received FDA authorization to launch clinical trials of their stem-cell therapies for retinitis pigmentosa (RP).
In 2016, both companies reported they were treating patients
without any serious adverse events. While ReNeuron did not
disclose how many participants had received its cell injections,
jCyte reported they had treated nearly two dozen people. Both
clinical trials, which are initially focused on safety, are the first-ever stem-cell trials for RP.
Gene Therapy Leads to Sustained Vision Benefits
In 2016, researchers at the University of Oxford (U.K.) reported
that the vision benefits of its gene therapy for people with
choroideremia had been sustained for the first patients treated
in its clinical trial, which launched in 2013. Gene therapy
clinical trials for people with choroideremia are now underway
(or being launched) at Bascom Palmer Eye Institute in Miami,
the University of Alberta, Children’s Hospital of Philadelphia,
the University of Pennsylvania, and Massachusetts Eye and Ear
In 2016, several other clinical trials for potential treatments
are advancing impressively, including gene therapy studies
for achromatopsia, Usher syndrome, Stargardt disease, and
FFB’s Clinical Research Institute Advances Human Research
In 2016, the Foundation’s Clinical Research Institute continued
to boost the global retinal research field with patient natural
history studies such as ProgSTAR (Stargardt disease); its patient
registry, MyRetina Tracker.org; and a consortium of clinical
experts ready to quickly and effectively launch more clinical trials
for vision-saving therapies. In 2017, the consortium will launch
a natural history study for people with Usher syndrome and RP
caused by mutations in the gene USH2A.
The Foundation Fighting Blindness Clinical Research Institute
(FFB-CRI), Bpifrance (BPI), and the Fondation Voir et Entendre
(FVE) announced the formation of SparingVision, a company
headquartered in France, to advance a promising, emerging
therapy for the treatment of blinding retinal disease retinitis
pigmentosa (RP) into a clinical trial and, ultimately, out to
the international marketplace. A total of € 15. 5 million in
tranche funding has been announced, with FFB-CRI and BPI
each investing € 7 million and FVE providing € 1. 5 million.
SparingVision’s goal is to launch a clinical trial for RdCVF in 2019.
FDA Clinical Trial Endpoints Meeting
FFB-CRI — along with the FDA, National Eye Institute (NEI), and
the Association for Research in Vision and Ophthalmology (ARVO)
— hosted a one-day public workshop to review clinical trial design
considerations and outcome measures for potential treatments
for inherited retinal diseases. As a result of the meeting, the
FDA validated an endpoint measurement tool known as EZ Area,
which efficiently and effectively measures viable photoreceptors
in a patient’s retina. EZ Area, developed by researchers funded by
FFB-CRI, will greatly help drug developers and pharmaceutical
companies design and launch human studies for treatments for
people with retinitis pigmentosa.
Therapy Review Meetings
FFB-CRI organized and hosted a dozen therapy review meetings
that brought together the world’s leading drug development
experts to evaluate the status of emerging stem-cell, gene, and
pharmaceutical treatments for inherited retinal diseases. The
goal of these meetings was to determine what preclinical studies
companies and researchers needed to do to move potential
therapies into human studies. The meetings also helped FFB-CRI determine which companies and treatments warranted
Track Your Vision. Drive the Research.