July 26, 2016 1:18pm

For the treatment of hemophilia A presented at the World Federation of Hemophilia 2016 World Congress

 


 

This new therapeutic comprises an adeno-associated virus cDNA human Factor 8 construct driven by SGMO's synthetic liver specific promoter, which in preclinical studies is at least three times more potent than existing AAV-based cDNA constructs currently under evaluation for the treatment of hemophilia A.

 

The data, presented by Sangamo scientists at the World Federation of Hemophilia 2016 World Congress demonstrated production of supraphysiologic levels of human Factor VIII in a mouse model of the disease and in non-human primates.

  • In animals utilized, mean hFVIII levels ranged from 5 - 230% of normal and were obtained using AAV doses in the 6 x 1011 -- 6 x 1012 vgs/kg range - the most potent dose response in NHPs thus far disclosed for an hF8 cDNA.

 

The Bottom Line: The pre-clinical “compound” may enable clinically relevant levels of hFVIII to be obtained using lower vector doses, which potentially provides a better therapeutic risk/benefit profile for patients. SGMO is conducting additional studies to determine the minimal effective dose necessary to provide therapeutic benefit.

SGMO closed at $5.76 and is UP +$0.17 or +3% to $5.93. It’s slipping already (day's range $5.72 to $5.97) will fall further … it’s still VERY early, an IND application may be filed with the FDA in 2016.