Antisense technology changed one devastating disease. Why hasn't it transformed others?
The 2016 meeting of the Oligonucleotide Therapeutics Society in Montreal began with a rare mix of promise and elation.
That year, the 27-year-old biotech Ionis showed that a drug, later branded Spinraza, could dramatically alter the course of spinal muscular atrophy, a genetic condition that killed most diagnosed children before age 2. Ionis founder and CEO Stanley Crooke, introduced on stage by a video from past colleagues and SMA patients for a lifetime achievement award, was met with standing applause.
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