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Posts tagged optogenetics

Clinical Trial to Launch for System Combining Optogenetics and Eyewear

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gensightThe French biotech GenSight Biologics has received regulatory authorization in the UK to launch the PIONEER Phase 1/2 clinical trial for its GS030 system — a light-sensing gene therapy (optogenetics) coupled with eyewear, which enhances visual stimulation. The system is designed to restore vision for people who are blind from retinitis pigmentosa (RP) and potentially other retinal conditions such as: Usher syndrome, Stargardt disease, and dry age-related macular degeneration.

The GS030 will be evaluated in 18 people with RP who can see no better than counting fingers. Additional trial details are available at the clinical trials Web site hosted by the National Institutes of Health.
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Scientists Receive $25 Million to Develop a Vision-Restoring System that Connects to the Brain

Labels in English are (clockwise): Intelligent neuromorphic camera/LED; Matrix for optical stimulation of neurons/electronic; Implant for data processing/external; Antenna for energy transfer and wireless communication; External module for visual information transfer

Labels in English are (clockwise from upper left): Intelligent neuromorphic camera/LED; Matrix for optical stimulation of neurons/electronic; Implant for data processing/external; Antenna for energy transfer and wireless communication; External module for visual information transfer

While researchers around the world are developing numerous drugs and biological therapies for ocular diseases and injuries, an international scientific team coordinated in France is developing an innovative approach to bring back eyesight to the blind that bypasses the eye entirely. Their high-tech, vision-restoring system interfaces with the visual cortex, the back of the brain where visual input is processed to create the images we see.

The CorticalSight Consortium, led by Jose-Alain Sahel, MD, at the Institut de la Vision and the University of Pittsburgh, and Serge Picaud, PhD, at the Institut de la Vision, was recently awarded a contract of up to $24.9 million by the Defense Advanced Research Projects Agency (DARPA) for development of their system, which combines optogenetics and advanced image-capturing and -processing technologies. A major advantage of the approach is it is designed to provide vision restoration for people regardless of their eye disease or condition.
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FFB-Funded Scientists Report on Nine Promising Translational Research Efforts

Translational research — moving promising science out of laboratories and into clinical trials — is essential to getting vision-saving, retinal-disease treatments out to the millions who need them. With that said, translational research is also costly and high risk and requires extensive clinical development and regulatory knowledge.

The Foundation Fighting Blindness has taken the translational challenge head on by investing more than $75 million in therapy-development projects with strong clinical-trial potential through its Translational Research Acceleration Program (TRAP), which includes Gund-Harrington Scholar Awards.
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Optogenetic Therapy Takes First Step Forward in Clinical Trial

Retrosense logoRetroSense Therapeutics has reported that three participants have received injections of its potential optogenetic therapy, known as RST-01, in a Phase I/II clinical trial. The patients were given the lowest dose of RST-01, and no adverse ocular events were observed. Furthermore, the treatment showed some biological activity, though RetroSense did not provide details about what that activity was or what it meant.

More information on safety and efficacy will likely be reported about the RetroSense trial after more trial participants have been observed over a longer period of time, and after discussions with the U.S. Food and Drug Administration. Continue Reading…

For Rare Disease Day – The Many Benefits of Genetic Research

Rare Disease Day logoIt’s apropos that Rare Disease Day 2016 will be held on the rarest day on the calendar—Leap Day, February 29.

However, collectively, rare diseases are not uncommon. About 30 million Americans, nearly 10 percent of our population, are affected by one of 7,000 rare diseases. They’re an important public health issue, making it incumbent upon us to work hard to eradicate them.
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Treatments for Retinal Diseases are Leading to Therapies for the Brain

An image of the brainThe retina—the thin, fragile layer of tissue lining the back of the eye—gives us the invaluable gift of vision. It works like film or digital sensors in a camera by converting the light that enters our eye into electrical signals. Those signals are sent over the optic nerve to the back of the brain, where they are used to create the images we see.
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The Top Research Advancements of 2014: How Fast Can We Go?

lab photoAs we approach 2015, it’s inspiring to look back on 2014 and recount the numerous advancements we’ve made in developing vision-saving treatments and cures. When I joined the Foundation nearly a decade ago, virtually nothing was in a human study. We were curing lots of blind mice, and clinical trials seemed elusive. But, today, more than a dozen promising therapies are being evaluated in people, and at least a dozen more clinical trials are expected to begin in the next few years.
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How Evolution is Leading to Gene Therapies for More Retinal Diseases

Dr. John FlanneryAt first blush, gene therapy for retinal diseases seems so simple: Inject a tiny drop of liquid containing good copies of a gene to replace the bad, and you’re home free. Vision is saved, and, in some cases, it’s even restored.

But the reality is: Developing gene therapies that are safe, effective and long-lasting is very challenging in our world of genetically diverse retinal degenerations. Scientists have to design a delivery system that gets the genes to the right types of cells across the entire retina, but without affecting other cells.
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ARVO Update on Optogenetics: New Light for People with Advanced Vision Loss

Keirnan Willett.One of the promising emerging approaches to restoring vision in people with advanced retinal diseases is a form of therapy known as optogenetics. In essence, this type of treatment restores light sensitivity — and, potentially, meaningful vision — to people who have very little, if any, vision remaining. An important benefit of optogenetics is that it should work regardless of the underlying genetic defect causing the disease. While it is too early to speculate how well optogenetics will ultimately work, we’re hoping it ends up retiring a lot of white canes. Continue Reading…

Optogenetics Workshop Sheds Light on Opportunities for Restoring Vision

Dr. Richard Masland, Massachusetts Eye and Ear Infirmary

Dr. Richard Masland, Massachusetts Eye and Ear Infirmary

Last Friday, the Foundation hosted an outstanding science workshop in Boston on the development of optogenetic therapies for restoring vision in people with advanced retinal diseases. We convened an impressive cadre of the world’s top retinal scientists — including experts from Japan, France, Italy and the United States, as well as local researchers from Harvard and MIT — to discuss available optogenetic technologies and advancing them into and through clinical trials.

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