The bio­phar­ma week in re­view: Thumbs Up, Thumbs Down

My week­ly take on the top news of the week to hit the bio­phar­ma world. We might be in the mid­dle of a long, hot sum­mer, but there’s been plen­ty of news to liv­en things up. — John Car­roll

Bio­Marin turns in jaw-drop­ping gene ther­a­py re­sults

Ku­dos to Bio­Marin, which just hand­ed in some star­tling good ef­fi­ca­cy da­ta on their gene ther­a­py for he­mo­phil­ia A. We’re still talk­ing small num­bers, but as proof-of-con­cept tri­als go, it’s an eye open­er. Sev­er­al of the pa­tients in­volved re­spond­ed very well to the once-and-done ap­proach to this rare dis­ease. They are clear­ly in the lead in he­mo­phil­ia A gene ther­a­pies, but look for lots of ac­tiv­i­ty from ri­vals in­ter­est­ed in mov­ing ahead on their own. Gene ther­a­py could trans­form lives. It’s great to see it.

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