In 2018, the PRO RETINA workgroup will once again organize a seminar for Choroideremia patients and people interested in the disease. The objective of this workgroup is to provide an update on the research and new options regarding the diagnosis and treatment. Initial studies of a gene therapy have already been done worldwide. Gene therapy has already been applied in Germany. We will also report on vision aids and counselling options. This is an excellent opportunity to exchange experiences in dealing with the disease.
MeiraGTx, a London and New York based gene therapy company, announced today the European Medicines Agency (EMA) has granted Priority Medicines (PRIME) designation to MeiraGTx’s gene therapy product candidate A002 for the treatment of patients with achromatopsia (ACHM) due to mutations in the CNGB3 gene. Achromatopsia is a chronically debilitating inherited eye disorder that severely limits a person’s sight. Patients are legally blind from birth with very poor visual acuity as well as extreme light sensitivity, involuntary eye movement, and complete color blindness.
ViaOpta Hello Application
ViaOpta Hello is a multi-tool application that can assist blind or visually impaired people by using cognitive services and Microsoft latest image analysis technology to identify people, items, and scenes.
Foundation Fighting Blindness will provide up to $7.5 million in funding to develop ProQR’s candidate QR-421a for Usher syndrome type 2A 12
Foundation Fighting Blindness and ProQR have entered into a partnership to develop QR-421a for Usher syndrome type 2A, targeting mutations in exon 13 of the causative USH2A gene. Foundation Fighting Blindness will provide milestone-based co-funding of up to $7.5 million to ProQR to advance the program into the clinic. QR-421a received orphan drug designation from the FDA.
The Food and Drug Administration (FDA) in the United States approved the first gene therapy to treat an inherited disease on Tuesday December 19th 2017. Although a gene therapy was approved for an ultra-rare disease in Europe a number of years ago and the FDA approved two gene therapies already this year, one for a form of leukaemia and one for a form of lymphoma, this is the first time such a therapy has been approved for an inherited retinal dystrophy (IRD) and marks a new era of hope for our community as a whole.
Since 1941, the Edward S. Harkness Eye Institute at the Columbia University Department of Ophthalmology has offered an ophthalmology basic science course. While the focus of the course is resident training, it has also proven useful for the visual sciences community as a whole. Coursework examines the fundamentals of vision and emphasizes how these basic principles relate to patient care.
On World Sight Day, eye care organisations around the world take note of new data that shows that global eye care efforts are at serious risk of being overwhelmed—unless we act now.
New data published in the Lancet Global Health shows that decades of declining “avoidable blindness”—those with uncorrected refractive errors and cataract—is plateauing, and is projected to increase between 2015 and 2050.
Novartis has reported that RTH258 (brolucizumab) 6 mg met the primary and key secondary endpoints in two Phase III studies, HAWK and HARRIER. RTH258 3 mg, evaluated in HAWK, also met these endpoints. These pivotal studies enrolled more than 1,800 patients with neovascular age-related macular degeneration (nAMD) across 400 centres worldwide.
NEI 3-D Retina Organoid Challenge spurs next-generation models to study blinding diseases and test therapies
A proposal to create a living model of the human retina, the light-sensitive tissue at the back of the eye, won $90,000 in the National Eye Institute (NEI) 3-D Retina Organoid Challenge (3-D ROC). The NEI 3-D ROC is an initiative that seeks to design human retinas from stem cells. Erin Lavik, Sc.D., at the University of Maryland, Baltimore County, led the awarded team. The NEI is part of the National Institutes of Health.