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As RNA epigenetics continues its rapid progress at the bench, commercial interest in the area is burgeoning—the latest a $54 million series A investment this October in Gotham Therapeutics of New York. “If you are the kind of investor that likes to place big bets on novel areas, then this is an area for you”, says Tony Kouzarides, from the Gurdon Institute in Cambridge, UK and founder of Storm Therapeutics, Cambridge, UK, the first epitranscriptomics firm to emerge from stealth mode. In the first 10 months of 2018, venture capital investors put $168 million into four other early-stage companies (Table 1), in addition to the $21 million that Storm raised back in June 2016. Many of the investors are the venture arms of big pharma: watchful eyes at Pfizer, Merck KGaA, Novartis, GlaxoSmithKline, Taiho, Celgene, Johnson & Johnson and Astellas are trained on the field. But the excitement continues to be tempered by uncertainties in our nascent understanding of RNA biology and technical uncertainties in measuring RNA modifications and their function.
For drug development programs in the field of RNA epigenetics, loosely defined as the mapping and study of post-translational modifications to RNA, one target has already emerged as the lowest-hanging fruit: methyltransferase-like 3 (METTL3). This is an epigenetic 'writer' protein that—together with METTL14 in a complex—methylates adenosine at the N6 position, yielding 6-methyladenosine (m6A). The enzyme is attracting a great deal of interest as potential drug target, at least among academic researchers, because METTL3 levels are often elevated in cancer cells in vitro and in mouse models of human cancer. Indeed, it has clear links to the emergence or maintenance of a range of human cancers, particularly in clinical samples from patients with acute myeloid leukemia.