Contact Information Available for logged-in reporters only Newswise Orlando, Fla. In two separate studies, vision scientists have developed healthy genes to prevent blinding diseases that stem from genetic defects. The research is being presented at the 2014 Annual Meeting of the Association for Research in Vision and Ophthalmology (ARVO) this week in Orlando, Fla. In a clinical trial to treat choroideremia, a rare disease that causes progressive and irreversible blindness, scientists developed a virus that can replace the missing gene (that causes the disease) in the cells at the back of the eye. Six months after the virus was injected into patients, findings showed that some patients experienced improved vision. Abstract Title: Improved visual function in patients with choroideremia undergoing subretinal gene therapy Presentation Start/End Time: Sunday, May 4, 3:15 3:30pm Location: S 320AB Session Number: 147 In a separate study, researchers developed a gene therapy to stop the progression of a form of retinitis pigmentosa, an inherited disease transmitted from mothers to sons. Two years after the therapy was used to treat dogs at an early stage of the disease, the treatment remained effective. Further use of the technique in dogs with mid and late stages of the disease … Continue reading →

By RTT News, May 05, 2014, 08:34:00 AM EDT (RTTNews.com) - Regeneron Pharmaceuticals, Inc. ( REGN ) and Avalanche Biotechnologies, Inc. formed a broad collaboration to discover, develop and commercialize novel gene therapy products to treat ophthalmologic diseases. The collaboration includes novel gene therapy vectors and proprietary molecules, discovered jointly by Avalanche and Regeneron, and developed using the Avalanche Ocular BioFactory, an adeno-associated virus (AAV)-based, proprietary, next-generation platform for the discovery and development of gene therapy vectors for ophthalmology. As part of the agreement, Avalanche would receive an upfront cash payment, contingent payments of up to $640 million upon achievement of certain development and regulatory milestones, plus a royalty on worldwide net sales of collaboration products. The collaboration comprises up to eight distinct therapeutic targets, and Regeneron will have exclusive worldwide rights for each product it moves forward in clinical development. Further, Avalanche has the option to share in development costs and profits for products directed toward two collaboration therapeutic targets selected by Avalanche. Pursuant to the agreement, Regeneron has a time-limited right of first negotiation for certain rights to AVA-101, Avalanche's gene therapy product targeting vascular endothelial growth factor currently under development for the treatment of wet age-related macular … Continue reading →