Night­star's drug for rare vi­sion loss ad­mit­ted to FDA's gene ther­a­py speed­way

In the lat­est show of reg­u­la­to­ry sup­port for gene ther­a­pies, Ox­ford spin­out Night­star Ther­a­peu­tics just got ac­cess to a fair­ly new “fast-lane” to speed its reti­nal gene ther­a­py to mar­ket.

The Lon­don-based com­pa­ny scored an FDA des­ig­na­tion called Re­gen­er­a­tive Med­i­cine Ad­vanced Ther­a­py, or RMAT, for its Phase III drug NSR-REP1. The ther­a­py is de­signed to treat choroi­deremia, a rare ge­net­ic con­di­tion that caus­es pro­gres­sive vi­sion loss, by cor­rect­ing mu­ta­tions in the CHM gene.

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