PYC used to be called Phylogica Limited. The recent SAD study in RP11 was safe and well tolerated- they've commenced to a Multiple Ascending Dose study the other day. Not sure about long-term toxicity of RNA therapies, that's probably a @hottod question in all honesty, but I know that the diseases PYC are targeting lead to either permanent blindness or death via kidney failure, so long-term toxicity effects may be somewhat offset anyway - though as I've said it's not something I've come across. Interestingly, the other condition PYC is targeting is Phelan-McDermid Syndrome, which may make it relevant to NEU holders.
GLA - itching for those Angelman results!
Neuren Media and Analyst Coverage, page-1238
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