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I know Sangamo has disappointed in past but they may be putting together a solid validation of their platform with Hemophilia-A and SB-525.

Results presented in Australia this weekend:


The first two patients treated at the 3e13 vg/kg dose level rapidly achieved normal, sustained Factor VIII (FVIII) levels with no reported bleeding events and no factor usage for as long as 24 weeks of follow-up
The two patients more recently treated at the 3e13 vg/kg dose level demonstrated FVIII activity kinetics that appear consistent with the first two patients in this dose cohort at similar early time points
SB-525 showed dose-dependent increases in FVIII activity levels across all dose cohorts evaluated
FDA recently granted regenerative medicine advanced therapy (RMAT) designation for SB-525 gene therapy to treat severe hemophilia A


https://www.pfizer.com/news/press-release/press-release-deta...


Slides: https://investor.sangamo.com/static-files/db8f2f60-1ee7-43f2...

https://finance.yahoo.com/news/sangamo-pfizer-announce-updat...


There are other Hemophilia-A gene treaments from competitors but this may be best in class.

Competitors are Spark's SPK-8011 and BioMarin valrox
https://www.biopharmadive.com/news/biomarin-readies-hemophil...

I heard Pfizer owns most of the commercial rights to this treatment, capping SGMO benefits at about $475 million + double digit royalties (though I don't have link). So this is not the golden ticket for the company but would be a good boost to balance sheet and hopefully a sign of things to come.
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