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More than two decades ago, the Foundation Fighting Blindness (FFB) began funding RPE65 gene therapy research that led in late 2017 to LUXTURNA™, the first FDA-approved gene therapy for the eye or an inherited condition. Ultimately, the Foundation provided more than $10 million in funding for the groundbreaking effort.
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Ophthotech is a biopharmaceutical company committed to developing therapeutics and gene therapy solutions to treat retinal diseases. The company is aggressively pursuing therapies for orphan conditions like retinitis pigmentosa (RP), Stargardt disease, and Leber congenital amaurosis (LCA), as well as common indications such as wet and dry age-related macular degeneration (AMD).
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The Foundation Fighting Blindness has announced funding for seven new research projects to advance the development of treatments and cures for retinal degenerative diseases. Each project will receive a total of $300,000 over a three-year period.
The grants were selected through FFB’s annual call for research proposals from individual investigators. Seventy scientists submitted requests for funding. Applications were reviewed by FFB’s Scientific Advisory Board, which is comprised of the world’s leading retinal experts.
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Hosted by the Foundation Fighting Blindness and Casey Eye Institute at Oregon Health & Science University, the Innovation Summit for Retinal Cell and Gene Therapy has emerged as one of the most essential events for researchers and companies developing treatments and cures for retinal degenerative diseases.
In its fifth year, the Innovation Summit featured 40 presentations from industry experts from around the world. More than 250 people were in attendance. The event was held on April 27, the day before the annual meeting of the Association for Research in Vision and Ophthalmology (ARVO) in Honolulu.
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Horama, a French biotech developing gene therapies for rare eye diseases, was established in 2014 as a spin-off of INSERM, France’s public scientific and technology institute. Today, the company has three gene-therapy development programs underway for rare inherited retinal diseases, targeting people with mutations in the genes PDE6B, RPE65, and RLBP1.
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Untreated retina
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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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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The 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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Ashley and Cole Carper traveled from Little Rock, AR, to tell their family’s story at the FDA hearing.
Spark Therapeutics has taken a major step closer to gaining marketing approval for its vision-restoring gene therapy for people with RPE65 mutations causing Leber congenital amaurosis (LCA) and retinitis pigmentosa. At the conclusion of a public hearing on October 12, 2017, an advisory committee comprised of FDA-selected experts voted unanimously – 16 to 0 – to recommend approval. The FDA is due to make a final decision on marketing approval for the treatment, known as voretigene neparvovec, by January 12, 2018.
The event held at FDA headquarters included the presentation of trial results from Spark representatives, as well as compelling testimony from patients, family members, and industry stakeholders.
Twenty-four-year-old Katelyn Corey told hearing attendees that before receiving the treatment, her constant adaptation to dwindling vision didn’t leave time for much else in her life. But her circumstances changed dramatically in December 2013, after she received the RPE65 gene therapy in Spark’s Phase III clinical trial.
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Applied Genetics Technology Corporation (AGTC) reported that its gene therapy for X-linked retinoschisis (XLRS) has performed encouragingly in a Phase I/II, safety-oriented clinical trial taking place at seven sites in the U.S.
XLRS is an inherited disease that leads to significant vision loss due to splitting of the layers of the retina. The condition affects about 35,000 males in the U.S. and Europe. XLRS is caused by mutations in the gene retinoschisin. AGTC’s gene therapy uses a human-engineered virus — and adeno-associated virus or AAV — to deliver normal copies of retinoschisin to the patient’s retina.
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As the Foundation's Senior Director of Communi-cations, Ben writes science and research articles for the Foundation’s website, newsletters and Eye on the Cure blog. Read More...
Steve is highly respected for his expertise and tireless commitment to finding treatments and cures for vision-robbing retinal diseases. Read More...