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Randi Hernandez was science editor at BioPharm International from September 2014 to May 2017.
AstraZeneca will team up with various organizations to employ CRISPR technology for precise gene editing in recombinant cell lines.
AstraZeneca is the latest pharmaceutical company interested in harnessing CRISPR (clustered regularly interspaced short palindromic repeats) gene-editing technology for the purpose of drug delivery. The company announced on Jan. 29, 2015 that it is exploring the technology through an in-house program, and will share cell lines and compounds with the Wellcome Trust Sanger Institute, the Innovative Genomics Initiative, Thermo Fisher Scientific, the Broad Institute, and the Whitehead Institute. AstraZeneca will work with these partners to identify novel targets and publish findings of its application of the CRISPR technology.
The CRISPR/Cas9 system-used for gene editing-has a role in adaptive acquired immunity in bacteria and is being investigated for its applications in human cells. The technology consists of a guide-RNA that homes in on a section of DNA and a Cas9 nuclease that cleaves the DNA in a target section.
Scientists can use this technology to find and alter certain DNA targets by harnessing the cell’s own DNA-repair apparatus to silence or activate specific genes in the genome. In the pursuit of this goal, AstraZeneca will provide cell lines to the Sanger Institute, which in return will provide a collection of genome-wide CRISPR guide-RNA for analysis. Thermo Fisher Scientific will provide guide-RNA libraries for the pharma company to screen against its own cell lines. The Broad and Whitehead Institutes will cross-reference a genome-wide CRISPR library with a panel of cancer cell lines, while the Innovative Genomics Inititative will work to identify and validate disease-specific targets by inhibiting or activating CRISPR genes.
“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 Dr. Lorenz Mayr, vice-president, Reagents & Assay Development, AstraZeneca, in a statement.
Novartis recently entered into a five-year partnership with Intellia Therapeutics and Caribou Biosciences to license its proprietary CRISPR/Cas9 gene-editing platform to engineer chimeric antigen receptor (CAR) T cells.