Gene Editing, Results

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.

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Research Scientist - Immunology
Recursion Pharmaceuticals Salt Lake City, UT
Director of Operations
Atlas Venture Cambridge, MA

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