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
http://www.uniqure.com.
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.
