Posters describe positive preclinical results of RetinoStat in wet age-related macular degeneration and the potential application of the LentiVector system in corneal endothelial disorders
Oxford BioMedica and its collaborators at Johns Hopkins University School of Medicine, Baltimore, Maryland, USA, presented three posters at the Association for Research in Vision and Ophthalmology (Arvo) Annual Meeting, held from 30 April to 4 May 2006 in Fort Lauderdale, Florida, USA.
These posters describe positive preclinical results of RetinoStat in wet age-related macular degeneration and the potential application of the LentiVector system in corneal endothelial disorders.
RetinoStat is a gene-based medicine based on Oxford BioMedica's proprietary LentiVector system, delivering two anti-angiogenic genes that halt the aberrant growth of blood vessels that leads to vision loss in wet age-related macular degeneration (AMD), a leading cause of adult blindness in developed countries.
The preclinical data presented at Arvo confirm that RetinoStat provides statistically significant efficacy in an industry-standard in vivo model of wet AMD.
In addition, by precisely engineering gene switches in the product, the company has achieved highly specific gene expression in the target cells of the retina.
This substantially enhances the potential safety and efficacy of RetinoStat.
The new data also confirm that the optimised configuration of RetinoStat, with two anti-angiogenic genes (endostatin and angiostatin), has efficacy superior to configurations based on single genes Oxford BioMedica's collaborators at Johns Hopkins University also showed data from an in vitro study demonstrating that the company's LentiVector system can be used to deliver therapeutic genes safely and effectively to human corneal endothelial cells.
Hence, the LentiVector system may have application in the development of gene-based therapies for the treatment of corneal endothelial disorders such as Fuchs' dystrophy.
This inherited disease affects about 1% of the population, and leads to gradual loss of vision as the inner layer of the cornea degenerates.
Today, the only real cure for Fuchs' dystrophy is a cornea transplant.
Oxford BioMedica and Johns Hopkins University, with support from the Foundation Fighting Blindness and its translational-oriented subsidiary, the National Neurovision Research Institute (NNRI), are conducting further preclinical studies with RetinoStat and commencing manufacturing scale-up and non-clinical studies to support the start of clinical trials.
The company is on track with its objective to start clinical trials of RetinoStat in wet AMD in 2007.
"We are very encouraged by the results of this successful collaboration," said Stephen Rose, Foundation Fighting Blindness's chief research officer.
"We created the National Neurovision Research Institute subsidiary to work with both commercial and academic institutions to move promising treatments like RetinoStat into clinical trials.
"As a constituent-driven organisation, we have a strong commitment to getting treatments to market and the people that need them.
"We look forward to further collaborations with Oxford BioMedica on this and other projects to bring treatments and cures to patients with a variety of retinal degenerative diseases." Oxford BioMedica's chief executive officer, Alan Kingsman, commented: "The data presented at Arvo not only demonstrate the potential of RetinoStat as a treatment of retinopathy but also highlight the potential of our LentiVector system to treat other ocular diseases.
"Oxford BioMedica is pleased to be working with Johns Hopkins University and also to have the support of the Foundation Fighting Blindness.
"The new data suggest that a single injection of RetinoStat could provide long-term shut-down of aberrant blood vessel growth, and hence, could offer a safe, effective and more convenient treatment than current approaches for wet AMD as well as diabetic retinopathy, another leading cause of blindness.
"Given the world's aging population, there is a growing need to find better treatments for these conditions."
