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European Biopharmaceutical Review

Beyond Biopharma




In 2005, the first report of high-efficiency targeted genome editing at an endogenous mammalian locus opened the door to a new chapter in genome biology and researchers’ ability to make bespoke genetic changes in living systems (1). The technology used then was zinc finger nucleases (ZFNs) – a site-specific endonuclease that could be programmed to cut genomic DNA to create a DNA double-strand break in any cell and, in doing so, trigger endogenous DNA repair events that could be manipulated to elicit precise changes in the genome.

Fast-forward seven years, through the nascent era of genome editing, through the evolution of other programmable nuclease platforms (TALENs, MegaTALs), to the year 2012. The research community, now primed with the knowledge of genome editing and what it can bring, yet frustrated with the several-thousand-dollar expense tag and slow throughput of creating custom nucleases for each new gene editing project, was treated to the first glimpse of CRISPR-Cas9: a powerful new genome editing platform that would bring unparalleled speed and cost-effectiveness to genetic engineering (2). Within months of that seminal paper, which defined the functional CRISPR-Cas9 complex that could operate outside of its prokaryotic origin, multiple groups demonstrated its efficacy in an array of hosts: a testament to the simplicity, versatility, and portability of this new tool. The potential for CRISPR-Cas9 as a human therapeutic strategy was immediately recognised and several new companies, including CRISPR Therapeutics, Intellia Therapeutics, and Editas Medicine, were quickly formed to leverage the technology in cell and gene therapy applications.

The use of CRISPR-Cas9 as a direct therapeutic strategy is not the only means by which pharma has adopted the tool. The value of genome editing in creating advanced cell and animalbased models for use in drug discovery, or in the bioproduction of therapeutic proteins, had already been highlighted by the earlier nuclease technologies. However, the arrival of CRISPRCas9 effectively removed many of the technical, temporal, and financial barriers to these endeavours. Moreover, unlike its predecessors, CRISPR-Cas9 and modifications thereof (e.g., CRISPRi and CRISPRa) proved readily adaptable to genomewide library screens wherein the activation, repression, or knockout of endogenous genes can be analysed for phenotypic or transcriptomic consequence (3). This, together with the amenability of CRISPR-Cas9 to multiplexing, opened new possibilities for drug target discovery and pathway analysis (4). The impact of biopharma on what is still a relatively young technology is yet to be fully realised. Nevertheless, the frequency with which publications appear describing new discoveries using this tool demonstrates its remarkable penetration into this field in a short space of time.

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Dr Trevor Collingwood is an independent consultant in life sciences with over 15 years' experience focusing on developments and application of genome editing technologies. He has worked with several pioneering companies in this field including Sangamo Therapeutics, Sigma-Aldrich, and CRISPR Therapeutics and is currently a consultant for ERS Genomics.
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Dr Trevor Collingwood
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