AstraZeneca to employ CRISPR, a new technique for drug discovery

Anglo-Swedish pharma major AstraZeneca (LSE: AZN) has announced a research program to develop new medicines that treat diseases by, in effect, fixing broken genes, marking the first concerted use of an emerging technique called CRISPR (clustered regularly interspaced short palindromic repeats) to snip out specific disease genes to discover new drugs.

AstraZeneca will share cell lines and compounds with its partners and work with them to publish findings of its application of CRISPR technology in peer-reviewed journals, contributing to broader scientific progress in the field. The collaborations complement AstraZeneca’s in-house CRISPR program and will build on the company’s ‘open innovation’ approach to research and development.

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. In contrast to previous genome-editing techniques, such as zinc-finger nucleases and TALENs, CRISPR is easier to handle in the laboratory.