Two years af­ter in­vestor pan­ic, Avro­bio touts 100% re­sponse as they look to chart a path to­ward ap­proval

Two years af­ter Avro­bio’s ear­ly da­ta pan­icked in­vestors, the gene ther­a­py biotech thinks they have enough da­ta to be­gin plot­ting their bid for ac­cel­er­at­ed ap­proval — and launch a suite of pro­grams be­hind the lead drug that has dom­i­nat­ed the com­pa­ny to date.

Avro­bio an­nounced Mon­day that a year af­ter dos­ing, the first pa­tient giv­en the com­mer­cial form of their Fab­ry dis­ease gene ther­a­py saw 100% re­duc­tion in kid­ney sub­strate, the end­point the FDA us­es for ap­proval. The da­ta are just from one pa­tient but the re­sults add, CEO Ge­off MacK­ay said, to the grow­ing body of ev­i­dence that their gene ther­a­py can sig­nif­i­cant­ly and in­def­i­nite­ly curb the ge­net­ic dis­ease.

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