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

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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.As a result of the encouraging interim results, ProQR has concluded the Phase 1/2 trial and plans to move the treatment into a Phase 2/3 clinical trial. Ten people were treated in the Phase 1/2 study.Results from the interim analysis were presented on September 5, 2018, at the Retinal Degeneration 2018 meeting in Killarney,Ireland, by principal investigatorArtur Cideciyan, PhD, research professor of ophthalmology at theScheie Eye Institute,University of Pennsylvan…

September is Healthy Aging Month

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Today, people are living longer than ever before so it’s important to be proactive and take responsibility for your health as you age. Like any other organ in your body, your eyes do not stay the same as you get older. Vision changes are normal with age but vision loss and blindness is not.  Older adults are at higher risk for certain eye diseases and conditions, including age-related macular degeneration, cataract, diabetic retinopathy, glaucoma, dry eye, and low vision.  To continue to enjoy healthy vision as you advance in years, it’s important to have a comprehensive dilated eye examination with an ophthalmologist on a regular basis. Here are some other tips to maintain healthy vision now and as you age: Eat a healthy, balanced diet. Fruits and vegetables can help keep your eyes healthy. Visit our website for healthy eye recipes, click here Eye Cook.Maintain a healthy weight. Being overweight increases your risk for diabetes. By exercising regularly, you can help prevent vision…

Ophthotech is Advancing an Impressive Portfolio of Cutting-Edge Therapies for Retinal Diseases

Audio version: 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). Most impressive, the company is taking on a multi-track strategy that includes retinal gene-therapy development, including delivery of over-sized genes and design of a two-step process of gene knockdown and replacement for autosomal dominant conditions. While these are the scientific challenges that keep most retinal researchers awake at night, Ophthotech thrives on them and sees them as opportunities to meet significant unmet medical needs for people with retinal diseases. Kourous Rezaei, MD, the company’s chief medical officer, says his team’s clinical and drug-development expertise, as well…

FFB Funding More than $2 Million in New Research

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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. “Many of the funded research projects are cross-cutting, meaning they have the potential to benefit a broad range of people, independent of the mutated genes causing their retinal diseases,” says Stephen Rose, PhD, FFB’s chief scientific officer. “Also, some projects address a critical gap in our understanding and modeling of disease, and have potential to move the field forward in a significant way.” Here are summaries of the seven new projects: Gene Therapy to Pres…

Eye Safety Tips for the Solar Eclipse

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Here’s what you need to know about the upcoming solar eclipse and how to safely view the eclipse without losing vision from solar retinopathy! A solar eclipse occurs when the moon passes between the Sun and the Earth, and the Moon casts its shadow, known as the umbra, on Earth. Partial or total eclipses occur somewhere on Earth about every 18 months as you can see here in in this photo showing solar eclipses since 2001. Photo credit: Wikipedia On August 21, the entire United States will experience at least a partial eclipse, but only those in what is known as the path of totality will witness a total solar eclipse. The eclipse will occur quite quickly, and even those within the path of totality will see, at most, 2 minutes and 40 seconds of the total solar eclipse. So how can you safely view the solar eclipse? Well, if you are IN the path of totality you will first witness a partial eclipse, and then the short time period of the total eclipse, and then the partial eclipse once ag…

A Second Vision

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Kristin McDonald For the last few years Kristin McDonald, a former actress and television spokeswoman, has been applying her make-up without the aid of her eyes due to retinitis pigmentosa, a condition that reduces a person’s peripheral vision until all that is left is a pinpoint of sight. Today, she is in a first stage study that is offering her and others hope that the injection of stem cells might be the mechanism that could slow, and maybe even halt the effects of this horrible disease. Discovery Eye Foundation helped support many of the preliminary translational studies necessary to bring the clinical trial to the FDA and get this exciting, novel approach to the patients. I am Tom Sullivan, Ambassador of Vision for the Discovery Eye Foundation, and I was rocked when my phone rang recently and I heard the sound of my friend Kristin crying. “What’s the matter?” I asked with real concern. Her tears were quickly replaced by laughter, joyous laughter. “It might be working,” she s…

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

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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. Spark Therapeutics, Applied Genetic Technologies Corporation (AGTC), REGENXBIO, and Nightstar Therapeutics sponsored the event. Summit co-hosts were Casey’s Mark Pennesi, MD, PhD, and Trevor McGill, PhD, and Steven Rose, PhD, FFB’s chief scientific officer. While the ARVO meeting is the world’s largest eye research conference with about 12,000 attendees, the Innovation Summit provi…