Nanoscope’s eye dis­ease gene ther­a­py shows mixed re­sults in PhII

Dal­las-based biotech Nanoscope Ther­a­peu­tics un­veiled Phase II re­sults on its gene ther­a­py for a rare eye dis­ease Thurs­day morn­ing.

In the RE­STORE tri­al, 18 pa­tients with re­tini­tis pig­men­tosa got a gene ther­a­py called MCO-010 while nine got place­bo. On a vi­sion test called the MLYMT, the treat­ment group had a one-point greater change over one year in their score com­pared to the place­bo group, the pri­ma­ry end­point of the study. How­ev­er, the 95% con­fi­dence in­ter­val was 0.0 to 3.0, mean­ing the re­sult was not sta­tis­ti­cal­ly sig­nif­i­cant. The p-val­ue was not pro­vid­ed.

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