Genable Technologies Executes License Agreement With Benitec Biopharma for a Genetic Eye Disease Retinitis Pigmentosa

Source Press Release
Company Genable TechnologiesBenitec Biopharma 
Tags DNA & RNA therapeutics, Sensory Organs, Licensing
Date July 10, 2012

Genable announced today the execution of an exclusive worldwide licensing agreement with Benitec  Biopharma Ltd (ASX:BLT) for the use of  Benitec 's ddRNAi technology for rhodopsin-linked autosomal dominant retinitis  pigmentosa  (RHO -adRP).

RHO -adRP is a designated orphan disease. It affects approximately 1 in 30,000 people; there are around 19,000 patients in the EU alone. Patients with the disease suffer from severe visual dysfunction as a result of the death of the rod and cone photoreceptor  cells. No treatments are currently available. The disease is caused by any one of more than a hundred inherited defects in the rhodopsin gene making a single treatment very difficult until now.

Genable's approach utilises ddRNAi to develop a single therapy that can be used to treat patients with RHO -adRP regardless of which of the RHO  mutations causes the disease. It involves  suppression  of both the mutant and normal genes and replacement with a normal  RHO  gene that has been modified to be resistant to ddRNAi gene silencing. The program has established proof of concept studies in an in vivo model of the disease. Results of the program were reported at the World Gene Therapy Congress in May 2012 in London.

Chief Executive Officer of Genable, Jason Loveridge, commented, "Genable is delighted to have completed this agreement with Benitec  Biopharma which gives us certainty around our freedom to utilise ddRNAi for this purpose. We look forward to continuing the development of our novel therapeutics for the treatment of  RHO -adRP utilising Benitec  Biopharma's ddRNAi-based gene silencing technology."

Chief Executive Officer of Benitec  Biopharma, Dr Peter French, comments, "Benitec  Biopharma is very pleased to have executed this first licensing deal with a small biotech, Genable's novel approach to treatment of a genetic disease based on ddRNAi is further validation of  Benitec 's unique technology. Companies such as Genable and others who see the potential of ddRNAi to transform medical treatment in a range of diseases are great advocates for helping to get the message out that Benitec  Biopharma's ddRNAi technology has the potential to revolutionise treatment of currently untreatable diseases."

Genable Technologies  Limited is a privately held, venture backed, Dublin (Ireland ) based bio- pharmaceutical company developing new gene medicines to treat "dominant" genetic diseases based on the pioneering work of Professor Jane  Farrar, Dr Paul Kenna & Professor Peter Humphries.

Genable utilizes well-established, clinically safe & effective AAV  vectors to obtain expression of RNA interference (RNAi) molecules that suppress the expression of both the faulty and normal gene copies and replaces this with a gene subtly altered to become refractory to  suppression  but still encoding a normal wild type protein.

The combination of suppression  and replacement (S&R) overcomes the significant hurdle in dominant disease of mutation variability by eliminating the need to target specific mutations in a wide range of disorders. Genable's technology is protected by a broad suite of granted patents and patent applications in the USA, EU and worldwide.

Genable's first gene medicine - GT038  - is for treatment of patients with rhodopsin (RHO )-linked autosomal dominant retinitis  pigmentosa  (adRP) - a debilitating form of inherited blindness resulting from a diverse array of mutations in the  RHO  gene.

Source: Evaluate™
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