DUBLIN – Genomics plc raised £25 million (US$32.5 million) in a series B round, led by Vertex Pharmaceuticals Inc., which also signed up for a three- to five-year collaboration in which the Oxford, U.K.-based data science firm will hunt for new drug targets and seek new disease insights by analyzing extensive repositories of human genetic data that are linked to phenotypes and clinical outcomes.

"We'll be focusing our work with Vertex on a small number of diseases with serious unmet need," Genomics founder and CEO Peter Donnelly told BioWorld.

Donnelly, who is also director of the Wellcome Center for Human Genetics (WHG) and professor of statistical science at the University of Oxford, established Genomics four years ago to build a genomic database to power modern discovery biology. It follows a stellar academic career, in which he has played a leading role in several genomics research initiatives, including the HapMap Project and the Wellcome Trust Case Control Consortium. The founding team also includes Gil McVean, professor of statistical genetics at the University of Oxford, company head of innovation Chris Spencer, and Gerton Lunter, who also leads a research group at WHG.

Donnelly likens the company's database to a matrix, comprising 14 million "columns" that correspond to variable loci in the human genome and 7,000 "rows" that correspond to measurable biochemical, biological or clinical parameters, such as from weight, height, lung capacity or blood glucose levels. It gives the company the ability to develop and test numerous biological hypotheses, using analytical approaches like those used in Mendelian randomization studies, in order to predict the consequences of perturbing an aspect of human biology. "It's about doing human biology in humans," Donnelly said. The company has populated its repository with summary statistical data from multiple genomewide association studies – scientists from some 250 research institutions across the world have made statistical data available.

In addition to Cambridge, Mass.-based Vertex, Genomics has also entered collaborations with Biogen, also of Cambridge, on finding new targets in multiple sclerosis and with the Integrated Human Genomics Research Unit of Tokyo-based Eisai Co. Ltd. Big pharma interest in genetics-driven drug and target discovery is on the rise, as evidenced by London-based Glaxosmithkline plc's recent $300 million investment in consumer genomics firm 23andMe, Inc., of Mountain View, Calif., and Tarrytown, N.Y.-based Regeneron Pharmaceuticals Inc.'s $500 million investment in its Regeneron Genetics Centre. Donnelly is on the scientific advisory board of the latter initiative and was previously an adviser to Decode Genetics, Iceland's great pioneer of genetics-driven biological discovery, which is now part of Amgen Inc., of Thousand Oaks, Calif. London-based Benevolent AI Ltd., another contender in this emerging space, recently raised $115 million at a $2 billion valuation, to advance its artificial-intelligence-driven drug discovery and development agenda.

Proprotein convertase subtilisin/kexin type 9 (PCSK9), originally identified by French researchers investigating a family with an unusual, autosomal dominant form of hypercholesterolemia, remains the poster child for genetics-driven drug discovery. The approach, Donnelly contends, will increasingly become a mainstream activity within the biopharma industry.

"I find it really hard to imagine in 10 years' time that no one will do drug discovery in a serious way without having this kind of information at their fingertips," he said. "The current approaches are not working." What's more, the data resources that will become available will be far richer than that is available at present. About 1 billion people will have their genomes sequenced around 10 years from now, he predicted, and that data will be linked to electronic health records and clinical histories.

Genomics' over-subscribed round attracted a syndicate of top-tier U.K. investors. In addition to Vertex, which invested £10.5 million of the total, participants included IP Group, Woodford Investment Management, Invesco Perpetual, Oxford Sciences Innovation, Lansdowne Partners and Australian firm Tanarra. The company raised £10.3 million in 2014.

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