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GenSight to launch trial of gene therapy, visual stimulation device combo in UK

The UK’s Medicines and Healthcare Regulatory Agency has approved GenSight Biologics‘ (EPA:SIGHT) application to combine gene therapy and a wearable optronic visual stimulation device in patients with retinitis pigmentosa. The first-in-man Phase I/II trial is designed to study the safety and tolerability of GenSight’s GS030 combination therapy in three groups of three patients each. Get the full story at our sister site, Drug Delivery Business News. The post GenSight to launch trial of gene therapy, visual stimulation device combo in UK appeared first on MassDevice.
Source: Mass Device - Category: Medical Devices Authors: Tags: Clinical Trials Optical/Ophthalmic Pharmaceuticals Regulatory/Compliance Wall Street Beat gensightbiologics Source Type: news

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This study recapitulated the pathogenesis of RPGR using patient-specific organoids and achieved targeted gene therapy of RPGR mutations in a dish as proof-of-concept evidence. Graphical abstract Teaser Jin and colleagues demonstrate that patient-specific iPSC-derived 3D retinae can recapitulate disease progress of retinitis pigmentosa through presenting defects in photoreceptor morphology, gene profile, and electrophysiology, as well as the defective ciliogenesis in iPSCs, iPSC-RPE, and 3D retinae. CRISPR/Cas9-mediated gene correction can rescue not only photoreceptor structure and electrophysiological property but also observed ciliopathy.
Source: Stem Cell Reports - Category: Stem Cells Source Type: research
GenSight Biologics, a biopharmaceutical company based in France, is developing gene therapies for rare diseases that cause blindness. The company has recently received approval in the UK for its Phase I/II PIONEER trial, which will test a treatment c...
Source: Medgadget - Category: Medical Devices Authors: Tags: Exclusive Genetics Ophthalmology Source Type: blogs
Authors: Nanda A, Salvetti AP, Martinez-Fernandez de la Camara C, MacLaren RE Abstract Inherited retinal diseases are thought to be the leading cause of sight loss in the working age population. Mutations found in the RPGR and CHM genes cause retinitis pigmentosa (RP) and choroideremia, respectively. In the first instance, an X-linked family history of visual field loss commonly raises the suspicion of one of these two genes. In choroideremia, the classic description of a white fundal reflex secondary to the widespread chorioretinal degeneration was made over a hundred years ago in Caucasians. But, it is not so obv...
Source: Ophthalmic Genetics - Category: Opthalmology Tags: Ophthalmic Genet Source Type: research
Authors: Chuang K, Fields MA, Del Priore LV Abstract The advent of gene editing has introduced the ability to make changes to the genome of cells, thus allowing for correction of genetic mutations in patients with monogenic diseases. Retinal diseases are particularly suitable for the application of this new technology because many retinal diseases, such as Stargardt disease, retinitis pigmentosa (RP), and Leber congenital amaurosis (LCA), are monogenic. Moreover, gene delivery techniques such as the use of adeno-associated virus (AAV) vectors have been optimized for intraocular use, and phase III trials are well un...
Source: The Yale Journal of Biology and Medicine - Category: Universities & Medical Training Tags: Yale J Biol Med Source Type: research
Authors: Yusuf IH, Shanks ME, Clouston P, MacLaren RE Abstract FLVCR1 (feline leukemia virus subgroup c receptor 1) is a transmembrane protein involved in the trafficking of intracellular heme. Homozygous variants in FLVCR1 have been described in association with a clinical syndrome of posterior column ataxia with retinitis pigmentosa (PCARP). Here, we describe a patient with non-syndromic retinitis pigmentosa homozygous for a splice-site variant in FLVCR1 (c.1092 + 5G>A) without evidence of posterior column ataxia or cerebellar degeneration. We suggest an association between intronic splice-site varia...
Source: Ophthalmic Genetics - Category: Opthalmology Tags: Ophthalmic Genet Source Type: research
Human Gene Therapy Dec 2017, Vol. 28, No. 12: 1180-1188.
Source: Human Gene Therapy - Category: Genetics & Stem Cells Authors: Source Type: research
Human Gene Therapy Dec 2017, Vol. 28, No. 12: 1189-1201.
Source: Human Gene Therapy - Category: Genetics & Stem Cells Authors: Source Type: research
Condition:   Non-syndromic Retinitis Pigmentosa Intervention:   Combination Product: Gene therapy: GS030-DP AND Medical device: GS030-MD Sponsor:   GenSight Biologics Not yet recruiting
Source: ClinicalTrials.gov - Category: Research Source Type: clinical trials
More News: Clinical Trials | Eyes | Gene Therapy | Genetics | Medical Devices | Opthalmology | Retinitis Pigmentosa | Study