Sang­amo bounces back with up­beat snap­shot on hem A al­lied with Pfiz­er and a plan to beef up AAV man­u­fac­tur­ing

Af­ter get­ting slammed two months ago af­ter rolling out the first in vi­vo hu­man da­ta avail­able from a rare dis­ease study us­ing its gene edit­ing tech, the play­ers at Sang­amo have earned some hard won re­spect on Wall Street to­day af­ter post­ing a snap­shot of promis­ing, though very ear­ly stage, re­sults for their he­mo­phil­ia A gene ther­a­py, part­nered with Pfiz­er.

Fo­cus­ing on the high-dose co­hort — with on­ly 2 pa­tients get­ting a 3e13 vg/kg dose of SB-525 — re­searchers spot­light­ed FVI­II ac­tiv­i­ty of 140% and 94%, or 93% and 65% of nor­mal, de­pend­ing on which as­say they used. That’s very com­pet­i­tive in a field dom­i­nat­ed up to now by Bio­Marin $BM­RN and Spark, now be­ing ac­quired by Roche, so long as it holds up.

One ex vi­vo gene edit­ing pro­gram al­so gath­ered some ini­tial re­spons­es in be­ta tha­lassemia and Sang­amo out­lined plans to beef up its AAV man­u­fac­tur­ing op­er­a­tions with a new con­tract signed with Bram­mer Bio while its own fa­cil­i­ty is un­der con­struc­tion.

Sign up to read this article for free.

Get free access to a limited number of articles, plus choose newsletters to get straight to your inbox.

SIGN UP LOG IN BECOME A PREMIUM SUBSCRIBER