Gene Editing Will Change Everything – Just Not All At One Time
Transformative technology is still in its infancy but great things are expected in human health and industrial and agbio markets. From the discovery in the late 1980s by researchers at Osaka University of strange repeat DNA sequences sitting beside a gene in a common bacterium, to the frenzied deals and financings over CRISPR technology today, gene editing has taken firm hold in the worlds of basic and applied life science. In fact, the variety of gene-editing technologies goes way beyond CRISPR, and its commercial applications go beyond human therapeutics to encompass agriculture, both plants and animals, and a broad array of high-margin industrial products. In short, gene editing holds the promise of transforming the way R&D is conducted and products developed across major sectors of the global life science economy.
Gene editing broadly refers to a suite of methods that use site-specific endonucleases to first target a double-stranded break in the genome and then to repair that gene by disrupting it or by rewriting its sequence. Over the course of the past few decades, the technology has progressed through the use of meganucleases (MEGAs), zinc finger nucleases (ZFNs), transcription activator-like effector nucleases (TALENs), and clustered regularly interspersed short palindromic repeats (CRISPRs). Each specific iteration of the technology has been easier to design and has brought gains in speed and ease-of-use.
Thus far, Sangamo Biosciences is the only company to have applied one of these technologies —ZFNs—to the development of clinical-stage human therapeutics. Other companies such as the start-ups CRISPR Therapeutics and Editas Medicine have focused on CRISPR and have attracted eye-opening investments by elite early-stage VCs. In December, Editas completed multiple licensing deals on the same day for the gene-editing technologies TALEN and CRISPR/Cas9 (Cas9 refers to the protein that binds to RNA molecules which guide it to a specific location on the genome where it triggers a double-stranded break).
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