Spark to charge $850,000 per patient for blindness gene therapy

(Reuters) - Spark Therapeutics Inc said on Wednesday it will charge $850,000 per patient for its breakthrough gene therapy to treat a rare form of blindness, a lower than expected price that the company said reflects patient and insurer concerns about access and cost.
Source: Reuters: Health - Category: Consumer Health News Tags: healthNews Source Type: news

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(University of Pennsylvania) Gene therapies have had success in treating blindness but can't save areas of the retina where cells have already died. In a new effort, University of Pennsylvania scientists John Wolfe, also of CHOP, and William Beltran, along with David Gamm of the University of Wisconsin-Madison, will attempt to develop a stem-cell-based approach that restores vision.
Source: EurekAlert! - Medicine and Health - Category: International Medicine & Public Health Source Type: news
AbstractAberrant growth of blood vessels in the choroid layer of the eye, termed choroidal neovascularization (CNV), is the pathological hallmark of exudative age-related macular degeneration (AMD), causing irreversible blindness among the elderly. Co-localization of proangiogenic factors and hypoxia inducible factors (HIF) in neovascular membranes from AMD eyes suggests the role of hypoxia in pathogenesis of CNV. In order to utilize hypoxic conditions in RPE for therapeutic purposes, we developed an optimized hypoxia regulated, RPE cell-specific gene therapy to inhibit choroidal neovascularization. An adeno-associated vir...
Source: Journal of Molecular Medicine - Category: Molecular Biology Source Type: research
Presentations in a botanical garden. Workshops in an actual work shop. Disco in a museum. The 2018 Thrival Festival eschewed tradition and challenged attendees to ponder: Are we asking the right questions when it comes to humanity + technology + art? The annual Thrival Festival held in Pittsburgh PA is truly unique. It combines art, technology, philosophy, music, and yes, even healthcare, into an event that is part science fair and part theatre. Instead of holding the event in a traditional auditorium or hotel, the organizers chose the beautiful Phipps Conservatory and Botanical Gardens as the setting for this year’s...
Source: EMR and HIPAA - Category: Information Technology Authors: Tags: Connected Health Digital Health Healthcare HealthCare IT Healthcare IT Conferences John Battelle Laura Montoya Rasu Shrestha Thrival Source Type: blogs
Yet another gene therapy spinoff from Nationwide Children's Hospital has been acquired for $100 million. Celenex is developing a treatment for Batten Disease, a rare, fatal neurological condition that causes blindness and progressive loss of speech and movement. Children who inherit it typically don't live past age 12. The startup was acquired by Amicus Therapeutics Inc., a Cranbury, New Jersey-base d biotechnology company that focuses on rare metabolic disorders. Columbus-based Rev1 Ventures,…
Source: bizjournals.com Health Care:Pharmaceuticals headlines - Category: Pharmaceuticals Authors: Source Type: news
The European Medicines Agency advisory panel voted Friday to recommend approval of Luxturna, a one-time gene therapy developed by Philadelphia-based Spark Therapeutics for an inherited eye disorder that leads to blindness if untreated. Luxturna received Food and Drug Administration approval in late 2017. The European Medicines Agency typically follows the removal of its advisory panels. If the treatment is approved in Europe, it would become the first gene therapy for a genetic disease approved…
Source: bizjournals.com Health Care:Biotechnology headlines - Category: Biotechnology Authors: Source Type: news
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
In this study, we found that TXNIP deficiency induces accelerated senescent phenotypes of mouse embryonic fibroblast (MEF) cells under high glucose condition and that the induction of cellular ROS or AKT activation is critical for cellular senescence. Our results also revealed that TXNIP inhibits AKT activity by a direct interaction, which is upregulated by high glucose and H2O2 treatment. In addition, TXNIP knockout mice exhibited an increase in glucose uptake and aging-associated phenotypes including a decrease in energy metabolism and induction of cellular senescence and aging-associated gene expression. We propose that...
Source: Fight Aging! - Category: Research Authors: Tags: Newsletters Source Type: blogs
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
Seven scientists in the United States and Britain who have come up with a revolutionary gene therapy cure for a rare genetic form of childhood blindness won a 1 million euro ($1.15 million)prize on Tuesday, Portugal's Champalimaud Foundation said.
Source: Reuters: Health - Category: Consumer Health News Tags: healthNews Source Type: news
(University of Pennsylvania School of Medicine) Three Penn Medicine ophthalmology innovators received the 2018 Ant ó nio Champalimaud Vision Award for their revolutionary work leading to the first successful gene therapy to cure an inherited cause of childhood blindness.
Source: EurekAlert! - Medicine and Health - Category: International Medicine & Public Health Source Type: news
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