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Apellis Launches Phase 3 Clinical Trial Program for Advanced Dry AMD Treatment

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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Forty High-Impact Retinal-Research Efforts Highlighted at FFB-Casey Innovation Summit

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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Call to Action: Ask Congress to Support $1 Billion in Eye Research

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An important bill has been introduced to the US House of Representatives that has the potential to greatly increase research funding for all blinding eye conditions, including retinal diseases such as retinitis pigmentosa, Leber congenital amaurosis, Usher syndrome, and age-related macular degeneration.
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Retinal Regeneration: Releasing Your Inner Salamander

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For someone with a retinal disease such as retinitis pigmentosa or macular degeneration, their vision loss is caused by photoreceptor degeneration. Photoreceptors are the retinal cells that capture light and convert it into electrical signals, which are sent back to the brain where they are used to create the images we see.
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