FDA of­fers first thoughts on neu­rode­gen­er­a­tive dis­ease gene ther­a­pies

The FDA has is­sued draft guid­ance on the de­vel­op­ment, test­ing, and tri­al de­sign for hu­man gene ther­a­pies for neu­rode­gen­er­a­tive dis­eases. The doc­u­ment, re­leased on Tues­day, al­so high­lights ap­proval path­ways for these nov­el prod­ucts.

The draft guid­ance, which ap­plies to prod­ucts for both adult and pe­di­atric pop­u­la­tions, em­pha­sizes the im­por­tance of ear­ly com­mu­ni­ca­tion with FDA be­fore the sub­mis­sion of an in­ves­ti­ga­tion­al new drug (IND) ap­pli­ca­tion. The agency point­ed to IN­TER­ACT meet­ings, which can be used to dis­cuss is­sues in a prod­uct’s ear­ly pre­clin­i­cal pro­gram, and pre-IND meet­ings, which oc­cur lat­er in de­vel­op­ment but pri­or to the sub­mis­sion of an ap­pli­ca­tion.

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