After decades of research and development, gene therapy is rapidly emerging as one of the most exciting areas in biotechnology—and generating new hope for patients with certain rare and often deadly inherited diseases. Replacement gene therapy that uses neutered viruses to deliver healthy genes to the body is particularly promising, given its potential to cure a range of disorders with the administration of a single treatment.
The first regulatory approval for this technology could come as soon as January, if the Food and Drug Administration gives the go-ahead to Spark Therapeutics (ticker: ONCE) for its one-time treatment that targets a rare, inherited retinal condition leading to blindness.
Other approvals could follow, igniting even greater interest in the field—and in the shares of several biotech companies making impressive headway in clinical trials.
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