Skip to main content
Message Font: Serif | Sans-Serif
No. of Recommendations: 1
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


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

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.
Print the post  


What was Your Dumbest Investment?
Share it with us -- and learn from others' stories of flubs.
When Life Gives You Lemons
We all have had hardships and made poor decisions. The important thing is how we respond and grow. Read the story of a Fool who started from nothing, and looks to gain everything.
Contact Us
Contact Customer Service and other Fool departments here.
Work for Fools?
Winner of the Washingtonian great places to work, and Glassdoor #1 Company to Work For 2015! Have access to all of TMF's online and email products for FREE, and be paid for your contributions to TMF! Click the link and start your Fool career.