Promising phase 1/2 gene therapy results for inherited eye disease

We’ve got some early, encouraging results out for a new gene therapy from Biogen subsidiary NightstarX: The treatment, which targets X-linked retinitis pigmentosa, appears to have been well-tolerated in the 18 patients studied, according to a new report in Nature. Some improvements in patient vision were noted about a month after the infusion, and remained the case six month later in six patients. 

Biogen acquired Oxford University spinout NightstarX last year, shelling out $877 million for its eye-focused gene therapy pipeline. It’s currently wrapping up Phase 3 trials for another X-linked retinal disorder called choroideremia — an indication that’s also being pursued hotly by competitor Roche.