Amgen uncovers trove of gene knockouts in 'molecular national selfie'

DeCODE CEO Kari Stefansson

Amgen's ($AMGN) deCODE Genetics has published a series of papers that hint at how the the Big Biotech could begin to recoup the $415 million it paid for the population-scale sequencing pioneer. For Amgen, the meat of the project lies in the discovery of 8,000 human knockouts, people who lack a working version of one of 1,171 genes.

The sheer number of knockout genes identified by the project--which sequenced the genomes of 2,636 Icelanders and then extrapolated to the broader population--sets it aside from other initiatives. Each of the knockout genes is a possible lead for drug researchers, but separating the signal from the noise will take time. The next step is to get permission to assess the physiological effects of lacking certain genes and explore whether people have biological features that protect them from the deleterious consequences of their knockouts.

Such work could prove a more targeted way to discover targets such as PCSK9, the gene that is at the center of the blockbuster cholesterol drug battle between Amgen and Sanofi ($SNY). DeCODE CEO Kari Stefansson, who was also the senior author of the four Nature Genetics papers, is 100% sure Amgen will discover more protective mutations when it digs into the data and is equally confident of the importance of such findings. "I'm absolutely convinced these protective mutations are going to be important when it comes to pointing us toward good drug targets," Stefansson told Bloomberg.

The history of genetics shows the reality often fails to live up to such confident assertions, something deCODE has seen in its long a turbulent history. DeCODE spotted the potential of population-scale genetic research almost two decades before the U.S. and United Kingdom got their initiatives underway, but in this instance being the first mover resulted in bankruptcy. Within two years of a pair of VCs bailing deCODE out, Amgen bought the company for $415 million in a deal it said would enable it to identify and validate human disease targets.

Amgen's Sean Harper

Over the two years since Amgen struck the deal, little information has leaked out to suggest deCODE has started to live up those early expectations. Amgen and deCODE both provided a rosy, if insubstantial, picture of the arrangement this week. Amgen R&D chief Sean Harper told the New York Times research into a gene linked to cardiovascular disease by deCODE is underway. And Stefansson said Amgen is making go/no-go development decisions based on deCODE's database. It will be years before it is possible to say for certain whether the deal has paid off for Amgen, however.

By then, several other initiatives may have surpassed the scale and scope of deCODE's work. The United Kingdom's 100,000 Genomes Project plans to make data available to drug developers, while Regeneron ($REGN)--another beneficiary of the PCSK9 discovery--has its own sequencing program.

- read the release
- here's the NYT's take
- check out Bloomberg's coverage
- and Bio-IT World's piece

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