On Wednesday January 3rd 2018, Spark Therapeutics INC announced a series of payment models designed to help the process of patient access to its new gene therapy LUXTURNA in the United States, which is the only country where the once in a lifetime therapy is currently approved. The company also announced the cost of the treatment.
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.
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
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.