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Study Suggests Vitamin A May Benefit Children with RP

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An FFB-funded study at Massachusetts Eye and Ear Infirmary (MEEI) suggests that vitamin A palmitate supplementation may slow the decline of cone function by nearly 50 percent in children with retinitis pigmentosa (RP). Cones are the photoreceptors that normally provide daytime vision, and the ability to read, recognize faces, and perceive colors. Cone function was evaluated in the study by a full-field electroretinogram or ERG, which measures the retina’s electrical response to light.
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Vision Improvements Reported in Early Stem Cell Trial for Wet AMD

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Two patients with advanced wet age-related macular degeneration (AMD) in a Phase I clinical trial demonstrated improved visual acuity sustained for one year after a sheet of retinal pigment epithelial (RPE) cells derived from embryonic stem cells was transplanted under their retinas. Each patient had one eye treated. Vision improvement for one patient was 29 letters or about 6 lines on an eye chart. The other had a gain of 21 letters or about 4 lines.
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Best Disease Gene Therapy Advances Toward Clinical Trial

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BEST1-Untreated cBest patho MCT1-E66

Untreated retina

BEST1-treated MCT1-E66

Retina treated with gene therapy

Using gene therapy, FFB-funded researchers at the University of Pennsylvania School of Veterinary Medicine (Penn Vet) and Perelman School of Medicine have reversed the disease process in a canine model of Best disease, an inherited form of macular degeneration that can lead to severe vision loss in humans. The therapeutic effect of the treatment has been sustained for as long as five years. Results of the study led by Karina Guziewicz, PhD, and Artur Cideciyan, PhD, were published online in the journal Proceedings of the National Academy of Sciences (PNAS).
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Choroideremia Gene Therapy Moves into Phase 3 Human Study

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Nightstar Therapeutics, a retinal-disease, gene-therapy development company in the UK, is advancing its emerging gene therapy for choroideremia into a Phase 3 clinical trial known as STAR. The study will enroll approximately 140 patients at 18 clinical sites in the US, Europe, Canada, and South America.
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Natural History Study Launches for LCA Caused by Specific Mutation in CEP290

Massachusetts Eye and Ear (MEE) is participating in a natural history study for people with Leber congenital amaurosis (LCA) type 10 caused by a mutation referred to as “c.2991+1655A>G” in intron 26 of the CEP290 gene.
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FFB-CRI Investing $7.5 Million in Emerging Therapy for USH2A

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The Foundation Fighting Blindness Clinical Research Institute (FFB-CRI) has entered into a partnership with ProQR to develop a retinal therapy for people with Usher syndrome type 2A (USH2A) caused by mutations in exon 13 of the USH2A gene. FFB-CRI will be investing up to $7.5 million in milestone-based funding to advance the treatment, known as QR-421a, toward a Phase 1/2 clinical trial during 2018. ProQR plans to issue the initial data report for the clinical study in 2019.
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AGTC Launches XLRP Gene Therapy Clinical Trial at Five Sites in U.S.

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Applied Genetic Technologies Corporation (AGTC) is now recruiting for its Phase 1/2 gene therapy clinical trial for males with X-linked retinitis pigmentosa (XLRP) caused by mutations in the gene RPGR. Approximately 15 patients will be enrolled in the study, which is primarily evaluating safety. Three doses of the gene therapy will be tested. The trial is taking place at five sites in the U.S.
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Ophthotech Launching Human Study of Emerging Therapy for Stargardt Disease

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Ophthotech, a biopharmaceutical company developing therapies for eye diseases, has enrolled the first patient in its Phase 2b clinical trial of Zimura® for people with Stargardt disease caused by mutations in the gene ABCA4. The 120-participant study will be taking place at more than 30 sites. Data and knowledge gleaned from ProgStar, a natural history study for people with Stargardt disease funded by the Foundation Fighting Blindness, was used in the design of the clinical trial.
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Clinical Trial to Launch for System Combining Optogenetics and Eyewear

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gensightThe French biotech GenSight Biologics has received regulatory authorization in the UK to launch the PIONEER Phase 1/2 clinical trial for its GS030 system — a light-sensing gene therapy (optogenetics) coupled with eyewear, which enhances visual stimulation. The system is designed to restore vision for people who are blind from retinitis pigmentosa (RP) and potentially other retinal conditions such as: Usher syndrome, Stargardt disease, and dry age-related macular degeneration.

The GS030 will be evaluated in 18 people with RP who can see no better than counting fingers. Additional trial details are available at the clinical trials Web site hosted by the National Institutes of Health.
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Top Retinal Research Advances for 2017

To view and listen to Ben Shaberman’s presentation of “Top Retinal Research Advances for 2017,” with full slides and audio, click here. The text to the presentation is as follows:

This is Ben Shaberman, director of science communications, at the Foundation Fighting Blindness (FFB), and I’m pleased to present a quick overview of some of the exciting research advances for inherited retinal diseases made during 2017. It has been an exciting year with several promising therapies moving into and through clinical trials.
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