First Human Test of Optogenetics Could Restore Sight to the Blind

Before our brains build a visual image of our world, a chain of cells in our eyes must convert light into electrical signals that are processed in the brain. Photoreceptor cells in the retina represent the first link in this chain, and they are reactive to photons, or the fundamental particle of visible light. Retinitis pigmentosa causes these cells to…

SOCS3 in retinal neurons and glial cells suppresses VEGF signaling to prevent pathological neovascular growth

Neurons and glial cells in the retina contribute to neovascularization, or the formation of abnormal new blood vessels, in proliferative retinopathy, a condition that can lead to vision loss or blindness. We identified a mechanism by which suppressor of cytokine signaling 3 (SOCS3) in neurons and glial cells prevents neovascularization. We found that Socs3 expression…

Repair of Rhodopsin mRNA by Spliceosome-Mediated RNA Trans-Splicing: A New Approach for Autosomal Dominant Retinitis Pigmentosa

The promising clinical results obtained for ocular gene therapy in recent years have paved the way for gene supplementation to treat recessively inherited forms of retinal degeneration.  We used spliceosome-mediated RNA trans-splicing as a strategy for repairing the transcript of the rhodopsin gene, the gene most frequently mutated in autosomal dominant retinitis pigmentosa (RP).  Retinitis…

Avalanche Biotechnologies and Annapurna Therapeutics Announce Proposed Merger

Avalanche Biotechnologies, Inc. (“Avalanche”) (AAVL) and Annapurna Therapeutics SAS (“Annapurna”), a privately held biopharmaceutical company, today announced that they have entered into a definitive agreement providing for the acquisition of all outstanding shares of Annapurna by Avalanche in exchange for approximately 17.6 million newly issued shares of Avalanche common stock.  Upon completion of the proposed…

Scientists Successfully Apply Gene Therapy against Retinitis Pigmentosa

A collaboration between scientists in the UK and the USA has shown that gene therapy can give life-long protection to the light-sensitive photoreceptor cells responsible for colour vision in a mouse model of the most common inherited eye disorder.

  These findings are significant because they open up a new line of research to prevent nerve…

Retinal function, structure changes in proliferative diabetic retinopathy revealed

Patients with proliferative diabetic retinopathy (PDR) exhibit marked visual dysfunction and structural changes in both the inner and outer retinal layers, research findings indicate. Retinal function was evaluated using a number of tests, including contrast sensitivity, frequency doubling perimetry (FDP) and photostress testing. And semiautomatically segmented spectral-domain optical coherence tomography scans were used to quantify…

New drug candidate is promising therapeutic option for angiogenic retinal diseases

A research team led by scientists at Beth Israel Deaconess Medical Center (BIDMC) and the University of New Mexico School of Medicine has identified a small molecule that treats animal models of aged macular degeneration (AMD) and retinopathy of prematurity (ROP) by preventing the overgrowth of blood vessels that are characteristic of these two retinal…

Gene Therapy Staves Off Blindness from Retinitis Pigmentosa in Canine Model

Gene therapy preserved vision in a study involving dogs with naturally occurring, late-stage retinitis pigmentosa, according to research funded by the National Eye Institute (NEI), part of the National Institutes of Health. The findings contribute to the groundwork needed to move gene therapy forward into clinical trials for people with the blinding eye disorder, for…

Aerie Pharmaceuticals Reports Positive Rhopressa™ Phase 3 Efficacy Results

Aerie Pharmaceuticals, a clinical-stage pharmaceutical company focused on the discovery, development and commercialization of first-in-class glaucoma therapies, today reported the successful results of its second Phase 3 trial for Rhopressa™, a novel once-daily, triple-action eye drop being tested for its ability to lower intraocular pressure (IOP) in patients with glaucoma or ocular hypertension. Read the entire…