Genable Technologies Executes License Agreement With Benitec Biopharma for a Genetic Eye Disease Retinitis Pigmentosa
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
-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."
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
(adRP) - a debilitating form of inherited blindness
resulting from a diverse array of mutations in the RHO
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