GenSight's gene therapy mystery continues, as patients improve in another failed trial
A three-year-old medical mystery took its latest turn Thursday, when GenSight released data from its third trial for a rare form of blindness: Once again patients saw their vision improve in both eyes. And once again the trial technically failed.
The Phase III study was for Lumevoq, a gene therapy designed to fix Leber hereditary optic neuropathy, or LHON, a rare genetic disease that causes rapid vision loss in patients during adulthood. It had been GenSight’s lead program, lifting the company to a €40 million IPO, but when they completed the pivotal trial in 2018, they found paradoxical results.
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