De­railed by the pan­dem­ic, FDA of­fers CRISPR, Ver­tex drug pro­gram VIP sta­tus for its come­back play

CRISPR Ther­a­peu­tics may have run in­to a hur­dle for its clin­i­cal pro­gram to ad­vance CTX001 in se­vere he­mo­glo­binopathies, but the FDA is on record that it will do every­thing it can to get the drug back on track and head­ed to the fin­ish line.

CRISPR and its part­ners at Ver­tex an­nounced Mon­day morn­ing that the FDA had award­ed them the Re­gen­er­a­tive Med­i­cine Ad­vanced Ther­a­py — RMAT — des­ig­na­tion for their pro­gram to treat se­vere sick­le cell dis­ease and trans­fu­sion-de­pen­dent be­ta tha­lassemia. And that should give them an open door as they hus­tle through the clin­i­cal pro­gram.

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