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.

The move comes a lit­tle more than 5 months af­ter re­searchers of­fered a snap­shot look at pos­i­tive re­sults for the first pa­tient in each of those 2 pro­grams. And it fol­lows close on the heels of an SEC fil­ing not­ing that re­searchers here had to stop en­rolling new pa­tients due to the Covid-19 out­break.

In the pro­gram, re­searchers ex­tract hematopoi­et­ic stem and prog­en­i­tor cells from pa­tients and then re-en­gi­neer them us­ing CRISPR/Cas9 tech. Those cells are then in­fused back in­to pa­tients.

At the time, Ver­tex ex­ec­u­tive chair­man Jeff Lei­den her­ald­ed the snap­shot re­sults as ev­i­dence of its “po­ten­tial to be a cu­ra­tive CRISPR/Cas9-based gene-edit­ing ther­a­py.”



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