Ver­tex and CRISPR Ther­a­peu­tics un­veil more pos­i­tive gene ther­a­py da­ta, but busul­fan again casts a shad­ow over the field

Less than 12 hours af­ter re­veal­ing a flop on its sec­ond shot for al­pha-1 an­tit­rypsin de­fi­cien­cy, Ver­tex plowed ahead with an­oth­er da­ta drop from its part­ner­ship with CRISPR Ther­a­peu­tics. And though the topline proved pos­i­tive, con­cerns over con­di­tion­ing agents con­tin­ue to linger over the col­lab­o­ra­tion, as well as the en­tire gene ther­a­py space.

Pre­sent­ing da­ta from two tri­als at the Eu­ro­pean Hema­tol­ogy As­so­ci­a­tion an­nu­al meet­ing, the pair an­nounced that fol­low-up da­ta of at least three months for 22 pa­tients with ge­net­ic blood dis­or­ders in­di­cat­ed a “con­sis­tent and sus­tained” re­sponse to the ex­per­i­men­tal drug CTX001. All 15 pa­tients with trans­fu­sion-de­pen­dent be­ta tha­lassemia did not need fur­ther blood trans­fu­sions and all sev­en with se­vere sick­le cell dis­ease were pain free, the biotechs an­nounced.

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