Sen­so­ri­on nabs rare pe­di­atric des­ig­na­tion for gene ther­a­py; Ava­lo sells off re­main­ing drug rights for $5M

A French biotech is mov­ing for­ward on its hear­ing loss gene ther­a­py, and the FDA is giv­ing the can­di­date a reg­u­la­to­ry boost.

Sen­so­ri­on put out word Mon­day morn­ing that the US reg­u­la­to­ry agency grant­ed Rare Pe­di­atric Dis­ease Des­ig­na­tion to its lead gene ther­a­py can­di­date, called OTOF-GT. It is be­ing test­ed to treat otofer­lin gene-me­di­at­ed hear­ing loss, a hear­ing loss caused by otofer­lin de­fi­cien­cy that af­fects up to 20,000 peo­ple in the US and Eu­rope, per the biotech.

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