Dutch gene therapy firm UniQure threw its hat into the RNAi ring this week, announcing that it has picked up non-exclusive access to Benitec Biopharma's proprietary expressed RNAi technology for use with its nascent Huntington's disease program.
In doing so, the company hopes to apply its core adeno-associated virus technology with the gene-silencing technology, potentially opening the door to other RNAi programs, Hans Preusting, UniQure senior vice president of business development, told Gene Silencing News.
In exchange for access to the expressed RNAi technology, Benitec has picked up non-exclusive access to UniQure's AAV serotype 5 technology, which was exclusively licensed from the National Institutes of Health for use in treatments for liver and brain diseases, for use with its hepatitis B candidate Hepbarna.
That drug is composed of three shRNA sequences targeting the HBV genome, and is currently in in vivo proof-of-concept testing. Preusting noted that under the cross-licensing deal, UniQure will manufacture Hepbarna, while Benitec will be responsible for the drug's preclinical and clinical development.
Additional terms of the arrangement were not disclosed.
For UniQure, the arrangement marks its expansion beyond gene-replacement therapy, a field in which it recently achieved a key regulatory milestone with the EU approval in October of Glybera for lipoprotein lipase deficiency.
“Manufacturing has long been the issue to getting sufficient product for later-stage trials and commercial applications” of AAV-based drugs, Preusting said. “We have a validated [manufacturing] platform, we have the [intellectual property] around it, and we have now demonstrated that regulators have approved that platform” with the recent decision on Glybera.
“Our aim is to leverage that as broadly as possible,” he added. Thus, the company has turned to RNAi.
Preusting said that UniQure has already conducted early-stage discovery work with expressed RNAi in Huntington's disease, but with the Benitec license it is gearing up to begin research and development efforts in earnest, with testing in animal models of the disease slated for 2013.
He declined to offer a specific research and development timeline for the Huntington's program, but indicated that UniQure expects to move fairly quickly, given the expertise and infrastructure it has in place.
“We know what to do to develop a product for the clinic — the safety studies … the process development, analytical methods,” Preusting said. “Of course, there are always differences when you develop a product for a new indication, but the beauty of our platform is that it is modular and, therefore, requires minimal changes to move new indications rapidly into the clinic.”
Additionally, the company's ongoing collaboration with University of California, San Francisco, researcher Krystof Bankiewicz in Parkinson's disease is expected to give it an edge in Huntington's disease since both programs are focused on AAV delivery directly to the brain, although the Parkinson's effort does not specifically involve RNAi.
“Delivery in the brain is crucial, and we can leverage the know-how that we have in that collaboration for other indications,” namely Huntington's disease, Preusting noted.
And should it find success with its first RNAi program, UniQure anticipates continued work with the technology.
“With RNAi, you can target completely different diseases than with traditional gene therapy,” opening the door to indications previously not available to the company, he said. “Huntington's is also a member of diseases caused by CAG repeats, and those other diseases might make attractive targets.”