Navigation Links
uniQure Licenses RNA Interference Technology to Advance Huntington's Disease Program

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.

About uniQure

uniQure is a world leader in the development of human gene based therapies. uniQure's Glybera, a gene therapy for the treatment of lipoprotein lipase deficiency has been approved in the European Union, and is the first approved gene therapy in the Western world. uniQure's product pipeline of gene therapy products in development comprise hemophilia B, acute intermittent porphyria, Parkinson's disease and SanfilippoB. Using adeno-associated viral (AAV) derived vectors as the delivery vehicle of choice for therapeutic genes, the company has been able to design and validate probably the world's first stable and scalable AAV manufacturing platform. This proprietary platform can be applied to a large number of rare (orphan) diseases caused by one faulty gene, and allows uniQure to pursue its strategy of focusing on this sector of the industry. uniQure's largest shareholders are Forbion Capital Partners and Gilde Healthcare, two of the leading life sciences venture capital firms in the Netherlands. Further information can be found at

About Benitec Biopharma

Benitec Biopharma Ltd is developing novel treatments for chronic and life-threatening conditions based on targeted gene-silencing activity using a transformational technology: DNA-directed RNA interference (ddRNAi) - sometimes called expressed RNAi. The technology's potential to address unmet medical needs and to cure disease results from its demonstrated ability to permanently silence genes which cause the condition. Importantly, this technology's target gene and related gene pathways will rarely have presented as a therapeutic avenue for research for the traditional small molecule agents, currently accounting for the majority of today's pharmaceutical products.

Founded in 1997 and trading publicly since 2001, Benitec Biopharma trades on the Australian Securities Exchange (ASX) under the symbol "BLT".  Benitec aims to deliver a range of novel ddRNAi-based therapeutics to the clinic in partnership with the pharmaceutical industry. Besides a focused R&D strategy in infectious diseases, cancer and chronic cancer-associated pain, Benitec Biopharma is pursuing programs with licensees that have advanced to pre-clinical and/or clinical trials.

SOURCE uniQure B.V.
Copyright©2012 PR Newswire.
All rights reserved

Related medicine technology :

1. MetaStat, Inc. Executes Two New Licenses
2. Cara Therapeutics Licenses Novel Kappa Opioid Agonist, CR845, to Chong Kun Dang Pharmaceutical Corporation For South Korean Market
3. Ventana licenses Gen-Probe IP for measuring ERG protein in prostate cancer patients
4. NTT DATA Licenses DATATRAK ONEā„¢ for China
5. Neuralstem Grants First Licenses For CNS Therapy Surgical Devices
6. West Health Institute licenses Kinect-based physical therapy technology to Reflexion Health; West Health Investment Fund makes $4.25 million investment
7. Parios Regenerative Sciences, Inc. Licenses Exclusive Rights to Organic Nanotechnology for Skin Repair
8. JDRF Announces Research Collaboration with Dexcom, Inc. to Develop "Smart Transmitter" Technology to Accelerate Artificial Pancreas Studies
9. Fujitsu to Showcase Radiation-Hardened RFID Using FRAM Technology at Interphex 2012 in New York City, Booth #2430
10. SolutionStart Corp. Announces John Karagiannis as Vice President of Technology
11. Ultra Clean to Present at the Jefferies 2012 Global Technology, Media & Telcom Conference
Post Your Comments:
(Date:11/26/2015)... UTRECHT, the Netherlands , November 26, 2015 /PRNewswire/ ...   A new combination approach blends immunotherapy ... --> A new combination approach blends immunotherapy with ... A new combination approach blends immunotherapy with ... the Netherlands has found that immunotherapy ...
(Date:11/26/2015)... , November 26, 2015 3D bioprinting ... 2022, according to a new report by Grand View Research ... Disease (CKD) which demands kidney transplantation is expected to boost ... effective substitute for organ transplantation. --> 3D bioprinting ... 2022, according to a new report by Grand View Research ...
(Date:11/26/2015)... ) has ... Future Horizons and Growth Strategies in the ... Shares, Country Segment Forecasts, Competitive Intelligence, Emerging ... --> ) has announced the ... and Growth Strategies in the German Drugs ...
Breaking Medicine Technology:
(Date:11/27/2015)... ... ... A team of Swiss doctors has released a report on mesothelioma relapse and ... findings on the website. Click here to read the details now. ... were treated with chemotherapy followed by EPP surgery. Among the 106 patients who relapsed, ...
(Date:11/27/2015)... CA (PRWEB) , ... November 27, 2015 , ... ... use in Final Cut Pro X. With ProSidebar: Fasion, video editors can easily ... use ProSidebar as a minimalist title opener. Utilize presets featuring self-animating drop zones, ...
(Date:11/27/2015)... ... November 27, 2015 , ... ... has prided itself for not only fulfilling the needs of advisers and clients ... an affordable price and providing top-tier customer service. However, there's always room for ...
(Date:11/27/2015)... Francisco, California (PRWEB) , ... November 27, 2015 , ... ... Janis Joplin Ann Arbor Michigan boxing style concert posters. This is one of Joplin's ... at the Canterbury House at the University of Michigan in Ann Arbor. The According ...
(Date:11/26/2015)... ... November 26, 2015 , ... Somu Sivaramakrishnan announced ... franchise owner, Somu now offers travelers, value and care based Travel Services, including ... sales, as well as, cabin upgrades and special amenities such as, shore excursions, ...
Breaking Medicine News(10 mins):