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Sunday, July 06, 2014 11:07:19 PM
http://www.bostonglobe.com/business/2013/06/01/after-disappointments-time-now-for-gene-therapy/wMIbxUe3Fc6szlLHccmYMJ/story.html
Alan Smith can still recall his excitement, in the early 1990s, over early experiments in lab rats that demonstrated gene therapy’s potential power to attack diseases such as cystic fibrosis.
Smith, a former chief scientific officer at Genzyme, the Cambridge biotech company, remembers feeling like the experiments were “steppingstones” to developing a whole new wave of medicines for untreatable diseases. Often, they were touted in Genzyme’s annual reports, and the research was funded with tens of millions of dollars from stock market investors.
But more than two decades later, there is still no gene therapy that has won approval from the US Food and Drug Administration, and Genzyme gave up on it as a potential approach to treating cystic fibrosis. What seemed in the 1990s to be a new world just over the horizon kept receding. There were years of “slow, quiet progress,” in Smith’s words, but also setbacks, including deaths and complications for patients who enrolled in early trials.
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