Monday, July 27, 2015

Gene therapy: A 20 year Blue Ocean Rollout

This treatment of the emerging gene therapy market as a blue ocean market expansion isn’t focused as much on the cost/value dichotomy as it is on the environmental determinism prevalent in pharmaceutical companies over the last 20-25 years. 

First, what is antisense technology?  Antisense therapy uses RNA sequencing to identify the messenger RNA responsible for activating or deactivating proteins in vivo.  By interrupting this mechanism, a disease (genetic or infectious) related protein can be prohibited from expressing, or encouraged to express (if the interrupted mechanism was the disease cause.)1

Stan Crooke, the pioneer of antisense technology has been advancing this field since the late 1980s.  When he first began researching and publishing on this topic in the late ‘80s, there was little faith in the industry that this type of treatment would be possible to isolate and treat.  (Forbes has a brief overview of the successes and failures of Stan’s company, Isis Pharmaceuticals, over the last several decades.2)

Over the past two decades, Isis Pharmaceuticals has proven that not only is this treatment viable, but that it also opens up treatment possibilities for entire new classes of diseases and genetic disorders.  This is pretty much the definition of a blue ocean market:  An existing, very competitive market, pharmaceuticals, isn’t so much disrupted by new technology, but has had an entire new treatment field opened up within it. 

The interesting blue ocean twist in this case is due to the incredibly lengthy process that all genetic and pharmaceutical research and development must go through.  When you compare Isis Pharmaceuticals to an internet blue ocean technology, like Slack, the progress seems downright glacial.  In two years, Slack’s product has grown into a nearly $3 billion valuation!3  Isis, on the other hand, has taken twenty years to reach a $9 billion valuation.4 

Isis Pharmaceuticals valuation and reach in the ‘new’ (new as in recently proven, with drugs in the field) antisense field isn’t simply due to the few drugs they’ve brought to market.  The real strength of Isis in this new field is their sequencing technology.  By developing and perfecting faster RNA sequencers, Isis has been able to identify dozens of potential antisense disease candidates.  This technology has greatly reduced the dollar, and more importantly, time costs of identifying potential antisense candidates.  By shortening this portion of the new drug pipeline, and by focusing on the emerging field of antisense treatments, Isis has opened up, and proven, an entirely new market for pharmaceutical treatments.

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