Monday, September 30, 2013

When Will Gene Therapy Come to the U.S.?

Several gene therapies are or will soon be in late-stage human trials. One of them could be the first to get FDA approval for sale in the U.S.
There are several groups that could be the first to develop a U.S.-approved gene therapy (see table). High’s team is one; they are enrolling patients in a late-stage trial of a treatment for a disorder that causes blindness at an early age. The patients in this trial have previously been given the gene therapy in one eye, and now the other will be tested.

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