Gene ther­a­pies for neu­rode­gen­er­a­tive dis­eases: Pfiz­er, No­var­tis, Bio­gen and oth­ers seek more from FDA

Top bio­phar­ma com­pa­nies are call­ing on the FDA to pro­vide more con­text and ex­am­ples, par­tic­u­lar­ly on ear­ly in­ter­ac­tions with the agency, as part of the com­pa­nies’ feed­back on draft guid­ance from Jan­u­ary re­lat­ed to de­vel­op­ing gene ther­a­pies to treat neu­rode­gen­er­a­tive dis­eases.

The 15-page draft guid­ance out­lines var­i­ous con­sid­er­a­tions on man­u­fac­tur­ing, pre­clin­i­cal and clin­i­cal tri­als for gene ther­a­pies tar­get­ing amy­otroph­ic lat­er­al scle­ro­sis, Alzheimer’s dis­ease and oth­er neu­rode­gen­er­a­tive dis­or­ders that are no­to­ri­ous­ly dif­fi­cult to treat.

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