AstraZeneca to use CRISPR genome editing technology for key therapeutic areas

PBR Staff Writer Published 30 January 2015

AstraZeneca has entered into four research collaborations to use clustered regularly interspaced short palindromic repeats (CRISPR) technology for genome editing across its discovery platform in key therapeutic areas.

The CRISPR genome-editing tool helps scientists to make changes in specific genes and it has two components, including a homing device to a specific section of DNA (guide-RNA) and enzymatic scissors that cut DNA (Cas9 nuclease).

The company said that in the cell nucleus, the guide-RNA sequence directs the Cas9 nuclease to cause double-stranded breaks in the target DNA sequence.

The CRISPR technology will allow the company to identify and validate new drug targets in preclinical models that closely resemble human disease.

As part of the deal with the Wellcome Trust Sanger Institute, AstraZeneca will focus on deleting specific genes relevant to cancer, cardiovascular, metabolic, respiratory, autoimmune and inflammatory diseases, as well as regenerative medicine to understand their precise role in these conditions.

The company will provide cell lines, which can be targeted using the Sanger Institute's collection of genome-wide CRISPR guide-RNA libraries to generate populations of cells where defined genes are switched off.

In an another deal, the company will work with the Innovative Genomics Initiative (IGI) to identify and validate gene targets relevant to cancer, cardiovascular, metabolic, respiratory, autoimmune and inflammatory diseases and regenerative medicine to understand their precise role in these conditions.

IGI is a joint venture between the University of California, Berkeley and University of California, San Francisco, which will concentrate on either inhibiting (CRISPRi) or activating (CRISPRa) genes to understand their role in disease pathology.

The deal with Thermo Fisher Scientific will see AstraZeneca secure RNA-guide libraries that target individual known human genes and gene families.

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AstraZeneca to use CRISPR genome editing technology for key therapeutic areas