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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Publication date: March 2018Source: Progress in Retinal and Eye Research, Volume 63Author(s): Marina França Dias, Kwangsic Joo, Jessica A. Kemp, Silvia Ligório Fialho, Armando da Silva Cunha, Se Joon Woo, Young Jik KwonAbstractRetinitis Pigmentosa (RP) is a hereditary retinopathy that affects about 2.5 million people worldwide. It is characterized with progressive loss of rods and cones and causes severe visual dysfunction and eventual blindness in bilateral eyes. In addition to more than 3000 genetic mutations from about 70 genes, a wide genetic overlap with other types of retinal dystrophies has been report...
Source: Progress in Retinal and Eye Research - Category: Opthalmology Source Type: research
Precision medicine seeks to treat disease with molecular specificity. Advances in genome sequence analysis, gene delivery, and genome surgery have allowed clinician-scientists to treat genetic conditions at the level of their pathology. As a result, progress in treating retinal disease using genetic tools has advanced tremendously over the past several decades. Breakthroughs in gene delivery vectors, both viral and nonviral, have allowed the delivery of genetic payloads in preclinical models of retinal disorders and have paved the way for numerous successful clinical trials. Moreover, the adaptation of CRISPR-Cas systems f...
Source: Journal of Clinical Investigation - Category: Biomedical Science Authors: Source Type: research
X-linked forms of retinitis pigmentosa (XLRP) are relatively severe blinding disorders, resulting from progressive photoreceptor dysfunction primarily caused by mutations in RPGR or RP2 gene.This technology is poised to advance RPGR or RP2 gene therapy to clinical stage using AAV8 or AAV9 vector carrying human full-length RPGR or RP2-coding sequence.   The investigators have performed a wide dose range study over 18-months and found it to preserve rod and/or cone function as evidenced by ERG and/or OCT, optomotor tests. Morphologically, the treatment preserved rod and cone viability, and corrected mistrafficking of co...
Source: NIH OTT Licensing Opportunities - Category: Research Authors: Source Type: research
(Michigan State University) A Michigan State University veterinary ophthalmologist has modified a gene therapy that reverses blindness in dogs that have a certain form of a disease known as progressive retinal atrophy, or PRA, and is now looking to advance the treatment for human use. Simon Petersen-Jones in the College of Veterinary Medicine has received a five-year, $8.2 million grant from the National Institutes of Health to further the therapy for people who have a type of retinitis pigmentosa.
Source: EurekAlert! - Medicine and Health - Category: International Medicine & Public Health Source Type: news
The objective here is a set of tests that (a) match up to the expected outcome based on human trials of mitochondrially targeted antioxidants, and (b) that anyone can run without the need to involve a physician, as that always adds significant time and expense. These tests are focused on the cardiovascular system, particularly measures influenced by vascular stiffness, and some consideration given to parameters relevant to oxidative stress and the development of atherosclerosis. A standard blood test, with inflammatory markers. An oxidized LDL cholesterol assessment. Resting heart rate and blood pressure. Heart r...
Source: Fight Aging! - Category: Research Authors: Tags: Newsletters Source Type: blogs
Authors: Huang XF Abstract Retinitis pigmentosa (RP) encompasses a heterogeneous group of inherited retinal disorders characterized by progressive photoreceptor and/or retinal pigment epithelial (RPE) degenerations with a prevalence approximately 1 in 4000 in the general population. Over 70 causative genes have been defined in RP families, and a number of animal models have been identified so far. However there have been no widely recognized treatments able to recover or reverse the degenerating retina, to prevent the disease deterioration, ultimately to restore the remaining vision. Therapeutics advancements have ...
Source: Advances in Experimental Medicine and Biology - Category: Research Tags: Adv Exp Med Biol Source Type: research
Authors: Koch SF, Tsang SH Abstract Retinal gene therapy has yet to achieve sustained rescue after disease onset - perhaps because transduction efficiency is insufficient ("too little") and/or the disease is too advanced ("too late") in humans. To test the latter hypothesis, we used a mouse model for retinitis pigmentosa (RP) that allowed us to restore the mutant gene in all diseased rod photoreceptor cells, thereby generating optimally treated retinas. We then treated mice at an advanced disease stage and analyzed the rescue. We showed stable, sustained rescue of photoreceptor structure an...
Source: Advances in Experimental Medicine and Biology - Category: Research Tags: Adv Exp Med Biol Source Type: research
Many of the methods by which aging can be modestly slowed in laboratory species are characterized by increased cellular housekeeping: more repair, more clearance of broken molecular machinery, more removal of metabolic waste. The extended life span produced by calorie restriction appears to depend on this increase: it doesn't happen in mice in which housekeeping processes are disabled. Most of the work on cellular housekeeping in aging is focused on autophagy, responsible for removing protein aggregates and cellular structures. The proteasome is a part of a separate system of housekeeping that deals with broken or otherwis...
Source: Fight Aging! - Category: Research Authors: Tags: Medicine, Biotech, Research Source Type: blogs
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
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