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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 seeks to “clarify the mechanisms of retinal disease, stimulate new technologies […]

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