AstraZeneca bets on 'genetic scissors' for range of new drugs

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[January 29, 2015]  By Ben Hirschler

LONDON (Reuters) - AstraZeneca said on Thursday it had struck four research agreements in the hot area of genome editing as it bets on a new "genetic scissors" technology to deliver better and more precise drugs for a range of diseases.

The academic and commercial tie-ups will allow British-based AstraZeneca to use so-called CRISPR technology across its entire drug discovery platform in areas such as oncology, cardiovascular, respiratory and immune system medicine.

CRISPR, which stands for clustered regularly interspaced short palindromic repeats, allows scientists to edit the genes of selected cells accurately and efficiently. It has created excitement since emerging two years ago and is already being tipped for a Nobel Prize.

The collaborations with Britain's Wellcome Trust Sanger Institute, the Innovative Genomics Initiative in California, the Broad Institute and Whitehead Institute in Massachusetts, and Thermo Fisher Scientific build on an in-house CRISPR programme at AstraZeneca that has been running for over a year.

Other drugmakers, keen to exploit the potential of a powerful new scientific tool, are also forming alliances.

Earlier this month Switzerland's Novartis struck deals with Intellia Therapeutics and Caribou Biosciences, two unlisted U.S. biotech companies that have been set up to take advantage of CRISPR.

While certain existing techniques already allow scientists to add genes to cells, CRISPR lets them make changes in specific genes far faster and in a much more precise way. It is also much easier to handle in the laboratory.

The technique is based on a naturally occurring system that bacteria use to defend themselves against viral infection and incorporates enzymatic "scissors" that cut DNA in the cell nucleus.

It is already being used by academic researchers seeking to understand how cells work and the hope is that it will now help in the design of improved drugs.

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AstraZeneca believes the technology will let it identify and validate new drug targets more quickly, accelerating the drug development process. It should also reduce the failure rate for experimental compounds as they move from preclinical tests into clinical trials, making drug research more cost-effective.

"CRISPR is a simple yet powerful tool that enables us to manipulate genes of potential importance in disease pathways and examine the impact of these modifications in a highly precise way," said Mene Pangalos, AstraZeneca's head of innovative medicines and early development.

No financial details of the collaborations were disclosed but AstraZeneca said it would share cell lines and compounds with its partners, based on an "open innovation" research model. Neither side will own any targets identified.

(Editing by David Holmes and Keith Weir)

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