One-time treatments that could functionally cure rare diseases. Cyborg T cells trained to kill cancer. A biological hacking method that could allow scientists to rewire errant genes from within. This year, a host of moonshot technologies matured, stoking hopes for real-world applications.
The world of gene therapy–in which single-dose treatments correct debilitating defects–enjoyed something of a renaissance in 2014. Strong clinical results from leaders in the once-maligned field spurred renewed optimism, helping a new generation of startups secure millions in venture financing to develop their next-generation approaches to the field.
And that led to something of a trickle-up phenomenon in the industry, as the innovations of biotechs and academics convinced the world’s biggest players to give the field a second look. Now Bayer, Pfizer (PFE), Biogen Idec (BIIB) and Astellas are among the many companies toiling in gene therapy, joining high-profile biotechs like bluebird bio (BLUE) and uniQure (QURE).
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