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

Related Links:

Purpose of review Retinitis pigmentosa is a group of genetically diverse inherited blinding disorders for which there are no treatments. Owing to recent advances in imaging technology, DNA sequencing, gene therapy, and stem cell biology, clinical trials have multiplied and the landscape is rapidly changing. This review provides a relevant and timely update of current trends and future directions for the diagnosis and management of this disease. Recent findings This review will highlight the use of retinal imaging to measure progression of disease, next-generation sequencing for genetic diagnosis, the use of electronic...
Source: Current Opinion in Pediatrics - Category: Pediatrics Tags: GENETICS: Edited by Nathaniel H. Robin Source Type: research
GenSight Biologics (EPA:SIGHT) said today that the first person was treated in a first-in-man trial evaluating GenSight’s gene therapy candidate, GS030-DP, in combination with a wearable optronic visual stimulation device, GS030-MD. Scientists are evaluating the drug-device combination in 18 people with retinitis pigmentosa. Get the full story at our sister site, Drug Delivery Business News. The post GenSight Biologics launches trial for gene therapy, eye device combo appeared first on MassDevice.
Source: Mass Device - Category: Medical Devices Authors: Tags: Clinical Trials Drug-Device Combinations Optical/Ophthalmic Pharmaceuticals Wall Street Beat gensightbiologics Source Type: news
Authors: Miraldi Utz V, Coussa RG, Antaki F, Traboulsi EI Abstract Significant discoveries in the etiology and pathogenesis of inherited retinal diseases (IRDs) have been made in the last few decades. Of the large number genes that cause IRDs, bi-allelic mutations in RPE65 lead to Leber Congenital Amaurosis type 2 (LCA 2), and can also result in phenotypes described as severe early childhood onset retinal dystrophy (SECORD) and Retinitis pigmentosa 20 (RP20). Following the publication of the successful Phase-III clinical trials of gene augmentation surgery for RPE65-related IRDs with voretigene neparvovec, the FDA ...
Source: Ophthalmic Genetics - Category: Opthalmology Tags: Ophthalmic Genet Source Type: research
Hug shirts, smellphones, virtual tastes, bionic eyes and hearing aids doing translations – just a few keywords showing how technology will take human perception to a higher level in the future. Innovative healthcare solutions will go way beyond improving our senses when we experience problems, they will augment our capabilities and open new horizons for humanity. Let’s jump into the pool of details. How humans perceive the sensory cacophony called the world Car. Flower. Smartphone. Leaf. Shadow. Ponytail. Red Sweater. Monitor. Water. Coffee. Beeps. Sidney Bechet tunes. Bicycle. Laugh. Light breeze. Holiday mem...
Source: The Medical Futurist - Category: Information Technology Authors: Tags: Biotechnology Cyborgization Health Sensors & Trackers Medical Professionals Patients body augmentation future Healthcare hearing human human perception Medicine sense sensing smell taste touch vision Source Type: blogs
(University of Miami Miller School of Medicine) A Puerto Rican patient with X-linked retinitis pigmentosa (XLRP) is hoping to save his vision after an innovative gene therapy procedure at Bascom Palmer Eye Institute at the University of Miami Miller School of Medicine. On August 23, Julio Adorno Nieves, 23, became the first US patient to be given new genes for his inherited blinding condition in a worldwide Nightstar Therapeutics clinical trial.
Source: EurekAlert! - Medicine and Health - Category: International Medicine & Public Health Source Type: news
AbstractRetinal degeneration diseases, such as age-related macular degeneration and retinitis pigmentosa, affect millions of people worldwide and are major causes of irreversible blindness. Effective treatments for retinal degeneration, including drug therapy, gene augmentation or transplantation approaches, have been widely investigated. Nevertheless, more research should be dedicated to therapeutic methods to improve future clinical treatments. Recently, with the rapid development of genome-editing technology, gene therapy has become a potentially effective treatment for retinal degeneration diseases. A clustered regular...
Source: Human Genetics - Category: Genetics & Stem Cells Source Type: research
Scientists funded by NIH preserve vision in dogs with a disease similar to retinitis pigmentosa in humans.
Source: National Institutes of Health (NIH) News Releases - Category: American Health Source Type: news
This article will explore an overview of the clinical trials of gene supplementation therapy for retinal diseases that are underway or planned for the near future. PMID: 30040511 [PubMed - as supplied by publisher]
Source: Ophthalmic Genetics - Category: Opthalmology Tags: Ophthalmic Genet Source Type: research
Publication date: Available online 24 July 2018Source: Journal of BiotechnologyAuthor(s): Diogo B. Bitoque, Gabriela A. SilvaAbstractGene therapy has the potential for treating retinal diseases, and we have been developing delivery vehicles and expression vectors for this purpose. In this short communication, we describe the generation of tools for both in vitro studies of the disease mechanism and for in vivo testing of therapeutic approaches. We have cloned the PDE6β gene and also recreated the same mutation present in the rd10 mouse using an optimized plasmid vector. To allow visual detection, we have also generate...
Source: Journal of Biotechnology - Category: Biotechnology Source Type: research
ConclusionsHere we report the first genetically confirmed CLN3 disease in Chinese, with a novel pathogenetic mechanism relating gene and protein, and highlights the potential ethnic differences in the mutation spectrum. We wish to establish the importance of clinical awareness and laboratory diagnosis of CLN3 disease, especially in the promising age of gene therapy.
Source: Clinica Chimica Acta - Category: Laboratory Medicine Source Type: research
More News: Clinical Trials | Eyes | Gene Therapy | Genetics | Medical Devices | Opthalmology | Retinitis Pigmentosa | Study