Four-year study will follow patients at 20 clinical sites worldwide
The first patient has been enrolled in a four-year natural history study of people with mutations in the USH2A gene which are the leading cause of the combined vision and hearing loss associated with Usher syndrome type 2A and vision loss from autosomal recessive retinitis pigmentosa. There are currently no treatments for the vision loss associated with these inherited retinal diseases.
A Symposium in honor of Dr. Gustavo Aguirre, recipient of the 2017 Proctor Medal, Association for Research in Vision and Ophthalmology (ARVO) will take place on Thursday November 16, 2017; 1:00pm—6:00pm at the School of Veterinary Medicine, University of Pennsylvania Please visit http://TRRaTConference.website2.me
Attendance is free but registration is essential to secure a place.
MeiraGTx, a New York and London based gene therapy company, have announced the first patient in its clinical study for X-Linked Retinitis Pigmentosa (XLRP) was treated at Moorfields Eye Hospital in London. This clinical study is the first-in-man Phase I/II dose escalation study of AAV2/5-hRKp.RPGR, MeiraGTx’s AAV-mediated gene therapy for XLRP caused by mutations in the RP GTPase regulator gene (RPGR).
Retina International will host its annual EURetina Symposium from 9am to 10.30am on Thursday September 7th in room 124 of the Barcelona Convention Centre.
The symposium will feature presentations on the European Reference Network for Rare Eye Disease (ERN-EYE), results from a recent study on the Economic Burden of Blindness in Europe, Patient Reported Outcome Measures for the retina and an introduction to the Retina International toolkit on genetic testing.
Potential for first gene therapy for a genetic disease to be approved in both the U.S. and EU
4D Molecular Therapeutics (4DMT), a leader in adeno-associated virus (AAV) gene therapy vector discovery and product development, and the Foundation Fighting Blindness (FFB), the world’s largest non-governmental source of research funding for inherited retinal degenerations (IRD) and dry age-related macular degeneration (AMD), today announced a partnership to develop intravitreal gene therapeutics for patients with these blinding conditions using 4DMT-proprietary AAV vectors.
A massive step forward for the Retina Community as The FDA grants a Priority Review with Prescription Drug User Fee Act (PDUFA) date of Jan. 12, 2018
LUXTURNATM (voretigene neparvovec) unveiled as proposed trade name.
Data show that 65 percent of participants demonstrated the maximal improvement in the clinical trial’s primary endpoint, bilateral multi-luminance mobility testing (MLMT), at year one
Bpifrance and Foundation Fighting Blindness have launched SparingVision to develop vision-saving treatment for Inherited Retinal Diseases. The newly established French company, SparingVision has won the 2017 i-Lab Grand Prize recognizing new and innovative companies. The i-Lab competition is organized by the French Ministry of Higher Education, Research and Innovation and Bpifrance (BPI).