Point­ing to a cure, Bio­Marin re­ports im­pres­sive tri­al re­sults for he­mo­phil­ia A gene ther­a­py

Bio­Marin says it nailed down hard ev­i­dence that its gene ther­a­py for he­mo­phil­ia A demon­strat­ed a high chance of restor­ing pa­tients’ nat­ur­al clot­ting abil­i­ties, re­duc­ing or elim­i­nat­ing bleed­ing episodes and point­ing them down the road to a nor­mal life. And com­pa­ny $BM­RN in­ves­ti­ga­tors say they are now hus­tling BMN 270 in­to a Phase IIb study that could be used to gain an ac­cel­er­at­ed ap­proval for a po­ten­tial block­buster.

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