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New gene therapy for inherited blindness could cost patients up to $1m per treatment
A new gene therapy for the treatment of Leber’s congenital amaurosis (LCA) is predicted to be priced at somewhere between $650,000 and $1,000,000 per patient per treatment, sparking considerable debate in the gene therapy market. According to Wall St. analysts, the new gene therapy for LCA may earn up to $200M in revenues in its […]Read full story
New geographic atrophy drug for AMD fails to meet primary endpoint in age-related macular degeneration
A new antibody drug (lampalizumab) targeting geographic atrophy in age related macular degeneration has missed its primary endpoint in two pivotal Phase III studies comprising >1,800 patients. The drug, sponsored by Genentech (Roche), was designed to target a key molecular component in the complement pathway, understood to be a critical pathway leading to the accumulation […]Read full story
Spanish research suggests gene editing may provide a potential treatment for Usher’s syndrome.
A Spanish research team, based at the Instituto de Investigación Sanitaria La Fe (IIS La Fe), Valencia, have published data showing preliminary proof-of-principle for the use of a new gene editing technology aimed at correcting gene mutations that cause Usher’s syndrome. The technology, known as “CRISPR”, was used to correct mutations in the USH2A gene […]Read full story
November 17th, 2017: “EURETINA-Brief”© Issue No. 165
by Dr. Gearóid Tuohy Dear EURETINA Members, A very warm welcome to the November 17th, 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 […]Read full story
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 […]Read full story
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 […]Read full story