Biotech Values

[rfj1862]

GNT

I may be speaking out of turn because I haven't looked at the data yet, but this strikes me as a particularly bizarre MOA.

>Defibrotide is a single-stranded DNA that protects the vascular endothelial cells, particularly those of small vessels, from damage and activation. After binding to endothelial cells, Defibrotide decreases cell adhesion and pro-coagulant activity of activated endothelial cells, and increases the fibrinolytic potential of endothelial cells. Defibrotide's effects are predominately local within the vascular bed, and there is no significant effect on systemic coagulation. Its beneficial pharmacological effects are due to its anti-thrombotic, anti-inflammatory and anti-ischemic properties.<

Is this just plain ol' ssDNA? Is it a particular sequence? Or perhaps a specific percentage mix of the various bases? Does it have a tertiary structure that allows it to actually activate/inactivate something, or does it passively coat and protect? What protects it from exo- and endonucleases? Naked ssDNA would be chewed apart ASAP if injected. Given that DNA is negatively charged, and the surface charge of RBCs changes with age and disease state, in some cases it might be actively repelled.

Anyway...

One positive: ultra-cheap to make.

John