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Archive for the Research Category

Acucela Enrolling Patients in Phase 3 Trial for Stargardt Disease Treatment

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The Seattle biotech Acucela is now enrolling participants in its Phase 3 clinical trial for emixustat hydrochloride, an emerging oral drug for slowing vision loss in people with Stargardt disease, an inherited form of a macular degeneration. The progressive retinal condition affects 30,000 people in the US.
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Ophthotech to Boost Development of Gene Therapy for Best Disease

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Ophthotech Corporation, a biopharmaceutical company focused on the development of therapies for orphan retinal diseases and age-related macular degeneration, has announced an evolving, commercial partnership with the University of Pennsylvania and University of Florida to develop a gene therapy for Best disease caused by BEST1 mutations.
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Foundation Invests $2.5 Million in Search for Elusive Retinal Disease Genes and Mutations

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Since the identification in 1989 of the first gene associated with an inherited retinal disease (IRD) – that gene was RHO, which when mutated, is a frequent cause of retinitis pigmentosa (RP) – genetic researchers, many funded by the Foundation, have identified approximately 270 genes linked to IRDs. In most cases, defects in a single gene can cause a retinal disease and vision loss.

The cumulative breakthroughs in IRD gene discovery over the past three decades are indeed impressive. It means that, today, about 65-70 percent of IRD patients will have their mutated gene identified when getting tested. However, it also means that the gene mutations for about one-third of patients are still not identified.
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Recording Available for Achromatopsia Teleconference Hosted by AGTC, Foundation, and Achroma Corp

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Achromatopsia is a challenging inherited retinal disease causing extreme light sensitivity, as well as impaired visual acuity and color perception. Approximately 75 percent of cases are caused by mutations in the genes CNGA3 or CNGB3.

Applied Genetic Technologies Corporation (AGTC), Achroma Corp, and the Foundation Fighting Blindness hosted a one-hour teleconference on the condition. The call highlighted the difficulties patients have in getting a diagnosis for achromatopsia, the importance of genetic testing, and gene therapy clinical trials underway. Presenters included Dr. Christine Kay, a clinical trial investigator with Vitreo Retinal Associates (Gainesville, Florida), and Bridget Vissari, president of Achroma Corp, which is expediting cures for achromatopsia.
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Apellis Launches Phase 3 Clinical Trial Program for Advanced Dry AMD Treatment

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The biopharmaceutical company Apellis has treated the first patient in its Phase 3 clinical trial program for APL-2, a compound designed to slow the progression of advanced dry age-related macular degeneration (AMD) also known as geographic atrophy (GA).
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FFB Congratulates RPE65 Gene Therapy Researchers for Champalimaud Award

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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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Vision Improvements Reported in ProQR’s Clinical Trial for LCA10 Treatment

ProQR, a biotech company in the Netherlands, has reported vision improvements for patients in a Phase 1/2 clinical trial for QR-110, a therapy for people with Leber congenital amaurosis 10 (LCA10), which is caused by the p.Cys998X mutation in the CEP290 gene. The mutation is estimated to affect about 2,000 people in the Western world.

The company reported that 60 percent of subjects in the trial demonstrated improvements in visual acuity and their ability to navigate a mobility course. The treatment was also safe for patients.
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Ophthotech is Advancing an Impressive Portfolio of Cutting-Edge Therapies for Retinal Diseases

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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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FFB Provides Four Career Development Awards to Up-and-Coming Clinical Researchers

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“The first time I examined a person’s retina as a junior resident, something clicked. All the things I found interesting came together — surgery, patient care, genetics, regenerative medicine, and cell biology,” says Mandeep Singh, MD, PhD, assistant professor in ophthalmology, Wilmer Eye Institute, Johns Hopkins Medicine. “I knew retina was what I wanted to focus on for my career.”

Dr. Singh is one of four new recipients of career development awards (CDAs) from the Foundation Fighting Blindness. Each recipient will receive a total of $375,000 over five years to help build an independent research program in addition to their clinical practices. FFB currently funds 12 CDAs for up-and-coming clinicians to advance their experience and expertise in research for retinal degenerative diseases. Since its inception, FFB has given CDAs to more than 100 clinical investigators.
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FFB Funding More than $2 Million in New Research

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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