As Bio­Marin’s lead ri­val wanes, Pfiz­er and Sang­amo post a promis­ing up­date for he­mo­phil­ia A gene ther­a­py

Pfiz­er and its he­mo­phil­ia A gene ther­a­py part­ners at Sang­amo have post­ed the lat­est up­date on their Phase I/II tri­al for SB-525 — and it con­tin­ues to look good as their ri­vals at Bio­Marin cruise to a like­ly ap­proval with a not-so-per­fect leader.

Me­di­an Fac­tor VI­II ex­pres­sion lev­els for their gene ther­a­py are run­ning at 64.2% for the 5 pa­tients now tak­ing the 3e13 vg/kg dose. That in­cludes pa­tients up to 61 weeks af­ter dos­ing, putting the crew on the tri­al at po­ten­tial­ly bet­ter than par with Bio­Marin’s val­rox at a year in, which has at­tract­ed plen­ty of ques­tions as FVI­II lev­els de­clined over 4 years to a me­di­an of 16.4%, as we re­port­ed yes­ter­day.

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