AstraZeneca has announced a research programme that aims to develop new medicines to treat the genetic causes of many debilitating diseases.
The company will use an emerging technology called CRISPR to remove specific disease genes in order to discover new drugs.
The research will be carried out with four leading academic and industrial gene-research centres around the world, including the Wellcome Sanger Institute in Cambridge, UK; the Innovative Genomics Initiative, a joint venture between the University of California, Berkeley, and University of California in San Francisco; the Broad Institute/Whitehead Institute and Thermo Fisher Scientific, which are both based in Massachusetts.
AstraZeneca will share cell lines and compounds with these partners, complementing the company's in-house CRISPR programme and building on its ‘open innovation’ approach to research and development.
CRISPR (clustered regularly interspaced short palindromic repeats) is a genome-editing tool which allows scientists to make changes to specific genes in a faster and much more precise way than current methods.
CRISPR is a simple yet powerful tool that enables us to manipulate genes of potential importance in disease pathways
The technology has two components – a homing device to a specific section of DNA (guide-RNA) and enzymatic ‘scissors’ that cut DNA (Cas9 nuclease). In the cell nucleus, the guide-RNA sequence directs the Cas9 nuclease to cause double-stranded breaks in the target DNA sequence. By harnessing the cell’s own DNA-repair apparatus, the gene being targeted can be altered either by deleting it, adding nucleotides to it, or by turning its activity on or off.
AstraZeneca says in contrast to previous genome-editing techniques, such as zinc-finger nucleases and TALENs, CRISPR is easier to handle in the laboratory.
Mene Pangalos, Executive Vice President, Innovative Medicines & Early Development, AstraZeneca, said: '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.
'By combining the science from our labs with these world-renowned academic and industry partners, we will be able to integrate this ground-breaking technology into our research and help accelerate the discovery of novel treatments for patients.'
Kosuke Yusa, Member of Faculty at the Sanger Institute, said CRISPR has 'transformed the way we study the behaviour of cells' and has the potential to benefit patients.
In addition to the new collaborations, AstraZeneca’s in-house team is currently adapting CRISPR technology to accelerate the production of cell lines and translational models that mimic complex genomic and disease-relevant scenarios.
'Application of the CRISPR technology for precise genome editing in recombinant cell lines and in relevant disease models should enable us to identify novel targets, build better test systems for drug discovery and enhance the translatability of our efficacy and safety models,' said Lorenz Mayr, Vice President, Reagents & Assay Development, AstraZeneca.
