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UniQure Licenses RNA Interference Technology To Advance Huntington's Disease Program

Stocks in this article: BLT

AMSTERDAM, The Netherlands, December 5, 2012 /PRNewswire/ --

uniQure B.V., a leader in the field of human gene therapy, today announced a non-exclusive cross-licensing agreement with Benitec Biopharma Ltd. (ASX: BLT) giving uniQure access to Benitec's proprietary DNA-directed RNA interference (ddRNAi) technology in Huntington's disease. In return, uniQure granted Benitec non-exclusive access to the Company's AAV5 delivery technology for the development of a ddRNAi therapy for Hepatitis B.

"The cross-licensing agreement with Benitec fully capitalizes on the strength of our advanced AAV platform and our proven ability to deliver therapeutic genes to target cells with high accuracy and efficacy," says Jörn Aldag, CEO of uniQure.  "The agreement with Benitec opens up promising new avenues to develop therapies for high unmet medical needs such as Huntington's disease. While our current programs focus on delivering fully functioning therapeutic genes to remedy faulty or malfunctioning genes, Benitec's ddRNAi technology will allow us to do the opposite - to 'silence' the gene responsible for producing the mutant protein that lies at the basis of Huntington's disease, and to develop a therapy for this devastating disease."

Dr Peter French, CEO of Benitec Biopharma, commented, "Benitec Biopharma is very pleased to have executed this licensing agreement with uniQure, the first company to achieve market approval for a gene therapy product, Glybera, in the West. uniQure have demonstrated their unique ability to take gene therapy-based programs from pre-clinical stages to commercialization, and we are confident that they will be able to achieve a similar outcome in this program. Importantly this agreement also provides Benitec access to uniQure's AAV delivery technology enabling further development of our ddRNAi treatment for Hepatitis B."

About Huntington's disease

Huntington's disease (HD) is a rare, chronic, incurable, progressive and disabling neurological condition, which continues to challenge the medical community. About 30,000 Americans (or 1 in 10,000 people) have Huntington's disease and at least 150,000 individuals have a 50% chance of inheriting the disease. The symptoms of HD become most evident in adulthood, typically 30 to 55 years, characterized by sudden, abnormal, and uncontrolled jerky movements called chorea. Currently, therapeutics for HD are limited only to symptomatic treatments and there are no treatment options with proven safety and efficacy to slow down disease progression or enhance survival rate.

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