Lack of ear­ly ef­fi­ca­cy for Trans­late Bio's mR­NA-based cys­tic fi­bro­sis drug rais­es some big ques­tions

Look­ing to bring mR­NA ther­a­peu­tics to cys­tic fi­bro­sis, Trans­late Bio re­vealed its sec­ond in­ter­im analy­sis in a Phase I/II study show­ing their ex­per­i­men­tal drug to …

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