De­vel­op­ing gene ther­a­pies for neu­rode­gen­er­a­tive dis­eases: FDA fi­nal­izes guid­ance

Bio­phar­ma com­pa­nies de­bat­ing whether or not to start de­vel­op­ing new gene ther­a­pies for neu­rode­gen­er­a­tive dis­eases — a his­tor­i­cal­ly dif­fi­cult field to crack — will now have more ad­vice from the FDA to help on pre­clin­i­cal test­ing and clin­i­cal tri­al de­sign, among oth­er top­ics.

The fi­nal FDA guid­ance, build­ing off a draft from Jan. 2021, in­cludes new clar­i­fi­ca­tions on the rec­om­men­da­tions re­gard­ing the use of tu­mor-form­ing cell lines, com­pa­ra­bil­i­ty stud­ies and crossover de­signs for clin­i­cal tri­als.

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