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fgimenez | 8 years ago

Yes, you could frame the Voyager treatment as "better levodopa". Though, the mechanism would feasibly mitigate neuro-degeneration and improve the terrible dose escalation of the drug.

Also correct about general delivery difficulties. Hadn't heard of Biontech's method - almost sounds like voodoo by your description.

I think there is interesting blocking and tackling happening on an organ-by-organ basis. E.g. GalNAc for liver hepatocytes, LNPs for systemic mRNA therapies, direct injection for eye or CNS (cheating, but still works). I'm partial to exosome hype...

Also no such discussion is complete without saying CRISPR, but the point remains that you can conjugate it targetted vehicles like antibodies. Conjugating to antibodies seemed to work for Stem :p

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aaavl2821|8 years ago

I dont understand the biology behind biontechs spleen targeting secret sauce (i dont think they did either, but it got them a patent on an otherwise standard lipid formulation). there is a really good nature paper from summer 2016 that outlines a lot of their preclinical dev; the CEO said he sort of regrets publishing that as a lot of competitors sprang up afterwards

out of curiousity, what's the exosome hype? ive heard a few strong life sci VCs mention it, but i talked to a ceo of an exosome company and didnt quite understand the specific therapeutic potential