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Market/Novel Tech

Spark Therapeutics announces additional efficacy outcomes from first randomised US trial in gene therapy

Spark Therapeutics (NASDAQ:ONCE), a gene therapy company based in Philadelphia, USA, has announced announced that the U.S. Food and Drug Administration’s (FDA) Cellular, Tissue and Gene Therapies Advisory Committee has unanimously recommended (16-0) approval of “LUXTURNA” (voretigene neparvovec), an investigational, “potential one-time gene therapy, for the treatment of patients with vision loss due to confirmed […]

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Complement C3 inhibitor (APL-2) meets primary end-point in Phase II clinical trial to treat geographic atrophy

A complement 3 (C3) inhibitor (APL-2) has shown significant results in the experimental treatment of geographic atrophy.   In the Phase II multi-center, randomized, single-masked, sham-controlled study, 246 patients were enrolled across 40 clinical sites in the USA, Australia and New Zealand. Patients received 5 mg APL-2/100 µL, administered via intravitreal injection, either monthly or every […]

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National Eye Institute (NEI) announces winner of research competition to develop a 3-D retina organoid

The National Eye Institute of the US National Institutes of Health (NIH) have announced a winner of their ambitious competition for researchers to build a working model of the human retina from stem cells. The initiative, termed the “3-D Retina Organoid Challenge” (see www.nei.nih.gov/3droc) seeks to “clarify the mechanisms of retinal disease, stimulate new technologies […]

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October 16th, 2017: “EURETINA-Brief”© Issue No. 163

by Dr. Gearóid Tuohy   Dear EURETINA Members,   A very warm welcome to the October 16th, 2017 edition of EURETINA’s web-based digital magazine, “EURETINA Brief”. EURETINA are delighted to continue our delivery of up-to-date summary briefs on a range of topics of interest to retinal clinicians, specialists and researchers across Europe. This resource is […]

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Whole exome sequencing (WES) continues to yield significant efficiencies in clinical identification of inherited retinal degenerations.

Research results reported by investigators based at the Department of Genetics & Microbiology, University of Barcelona, Spain, have shown that whole exome sequencing (WES) continues to provide efficient returns in the identification of the genetic diagnosis for a range of inherited retinal disorders. The Spanish research group worked on a cohort of 33 families with […]

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Findings from an international genomics consortium almost double the number of associated genetic loci and variants for AMD

Research conducted by the International AMD Genomics Consortium (IAMDGC) has recently reported an almost doubling in the number of genetic risk loci associated with age-related macular degeneration (AMD). In a significant publication in the journal Nature Genetics, the research group has reported on an analysis of >12 million gene variants, “including 163,714 directly genotyped, mostly […]

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