good point. PGRN systemically is associated with a cancer signal if I remember correctly. But, how long does it take for this to manifest? And what is the effect size? Certainly something worth thinking about. I suppose plasma PGRN is the relevant marker here for systemic exposure.
We may need to wait for the development of superior vectors (relative to AAV9) as a prerequisite to generating unequivocal clinical data. There are at least a couple of public companies who may be relevant in this regard (Sangamo being one).
Alector (INFRONT-3) failed pretty completely so the indirect Sortilin approach is clearly not valid.
Also, given Catalent’s challenges, I’d be a bit concerned relying on them as a sole CDMO.
Do we thing all that excess PGRN is an issue?
good point. PGRN systemically is associated with a cancer signal if I remember correctly. But, how long does it take for this to manifest? And what is the effect size? Certainly something worth thinking about. I suppose plasma PGRN is the relevant marker here for systemic exposure.
We may need to wait for the development of superior vectors (relative to AAV9) as a prerequisite to generating unequivocal clinical data. There are at least a couple of public companies who may be relevant in this regard (Sangamo being one).
Alector (INFRONT-3) failed pretty completely so the indirect Sortilin approach is clearly not valid.
Also, given Catalent’s challenges, I’d be a bit concerned relying on them as a sole CDMO.