Benitec Biopharma cross-licenses with uniQure to treat Huntington’s and Hepatitis B

Sydney, Tuesday December 4, 2012. Benitec Biopharma Ltd (ASX: BLT) is pleased to announce that it has entered into a sub-licensing agreement with Amsterdam-based uniQure BV. Benitec’s non-exclusive licence allows uniQure to develop a treatment for Huntington’s Disease using Benitec’s ddRNAi gene silencing technology. In turn, uniQure has granted Benitec a non-exclusive licence to use its AAV 5 (based on Adeno-Associated Viral vectors) delivery technology to assist Benitec’s development of a ddRNAi-based treatment for Hepatitis B.

Benitec’s licence to uniQure covers the application of ddRNAi to target, and thereby silence, key genes identified as significant therapeutic targets in the treatment of Huntington’s Disease. Under the agreement, uniQure has an option to convert the non-exclusive licence to exclusive, based on achievement of certain pre-clinical milestones, and to acquire additional licences to Benitec’s ddRNAi technology for other diseases.

Whilst the terms are commercial in confidence, they are within the expected guidelines for small biotech companies in the early stage of therapeutic development.

Chief Executive Officer of Benitec Biopharma Ltd, Dr Peter French, commented, “Benitec is very pleased to have executed this licensing deal with uniQure, the first company to gain market approval for a gene therapy product (Glybera) in the West. uniQure has demonstrated its unique ability to take gene therapy-based programs from pre-clinical stages to commercialisation, and we’re confident that they’ll achieve a similar outcome in this program. Importantly, this agreement also provides Benitec access to uniQure’s AAV delivery technology, potentially enabling further development of our ddRNAi treatment for Hepatitis B.”

Chief Executive Officer of uniQure, Jorn Aldag, commented, “uniQure is delighted to have completed this agreement with Benitec Biopharma Ltd. It gives us certainty around our freedom to utilise ddRNAi for our Huntington’s program. We look forward to continuing the development of our novel therapeutics for the treatment of Huntington’s Disease, utilising Benitec’s ddRNAi-based gene silencing technology and our proprietary AAV delivery platform.”

Huntington’s Disease (HD) is a rare, chronic, incurable, progressive and disabling neurological condition, which continues to challenge the medical community. In Western countries it is estimated that about five to seven people per 100,000 are affected by HD. About 30,000 Americans have HD and at least 150,000 have a 50% chance of inheriting the disease. In Australia about 1,200 people now have HD and approximately 6,000 are at risk. The symptoms of HD, which become most evident in adulthood, are characterized by sudden, abnormal, and uncontrolled jerky movements called chorea. Currently, therapeutics for HD are limited to treating symptoms. There are no treatment options with proven safety and efficacy to slow down the disease’s progression or to enhance sufferers’ survival rates. A recent market survey by GlobalData estimated that, in 2010, the therapeutics market for HD was valued at $246m, and forecast a Compound Annual Growth Rate of 13.4% to reach $676m by 2018.

For more information please contact:

Carl Stubbings | Chief Business Officer
Phone: +61 (02) 9555 6986 | |

About Benitec Biopharma Limited:

Benitec Biopharma Limited is an ASX-listed biotechnology company (ASX: BLT) based in Sydney, Australia. The company has a pipeline of in-house and partnered therapeutic programs based on its patented gene silencing technology, ddRNAi, also called expressed RNAi. Benitec is developing treatments for chronic and life-threatening human conditions such as cancer-associated pain, Hepatitis B, Hepatitis C, drug resistant lung cancer and oculopharyngeal muscular dystrophy based on this technology. In addition, Benitec has licensed ddRNAi technology to other biopharmaceutical companies for applications including HIV/AIDS and retinitis pigmentosa. For more information on Benitec refer to the Company’s website at

About uniQure

UniQure is a world leader in the development of human gene based therapies. UniQure has a product pipeline of gene therapy products in development for 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


Source: Benitec Biopharma Limited

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