Tuesday, April 02, 2019 9:38:40 AM
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.
Sangamo’s shares $SGMO slowly swelled in pre-market trading as the positive message sunk in. Then it soared. Its stock was up more than 50% before the bell......”
https://www.investorvillage.com/smbd.asp?mb=1933&mn=124063&pt=msg&mid=19300692
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