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Spark Therapeutics files marketing authorization application at EMA for the treatment of Leber’s congenital amaurosis (LCA)

Spark Therapeutics (NASDAQ:ONCE), a gene therapy company based in Philadelphia, USA, has announced the submission of a Marketing Authorization Application (MAA) to the European Medicines Agency for Investigational “LUXTURNA” (voretigene neparvovec). The gene therapy treatment, designed for patients with vision loss due to Leber’s congenital amurosis, has similarly undergone regulatory submission in the United States. […]

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Phase II results from encapsulated cell therapy technology show benefit in the treatment of patients with macular telangiectasia 2

Results from a Phase II multicentre randomised trial, which had enrolled 67 patients, with macular telangiectasia 2, has reported meeting its primary endpoint, defined as a change in the ellipsoid zone from baseline to month 24, measured by Spectral Domain Optical Coherence Tomography (SD-OCT). According to the sponsor of the study, an increase in the […]

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Reprogramming of Müller glia cells in mammalian retina promotes regeneration of neuronal cells following injury

A US study, published in the journal Nature (Aug 3rd, 2017), has shown that a regenerative gene from zebrafish is capable of driving the generation of neuronal cells in the mammalian retina. Damaged retina in zebrafish results in cells within the eye regenrating new cells to permit the fish to maintain vision, in contrast to […]

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