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Novel lipids and lipid nanoparticle formulations for delivery of nucleic acids
专利权人:
Acuitas Therapeutics Inc.
发明人:
Ansell, Steven M.,Du, Xinyao
申请号:
AU2020204111
公开号:
AU2020204111A1
申请日:
2020.06.19
申请国别(地区):
AU
年份:
2020
代理人:
摘要:
#$%^&*AU2020204111A120200709.pdf#####H: Interwoven NRPortbl DCC VZS 20339199_I.DOCX-18/06/2020 Abstract Compounds are provided having the following structure:() or a pharmaceutically acceptable salt, tautomer or stereoisomer thereof, wherein Ri, Rb,R2 a, R2B R2 a R3 R 3b R4 , R4 b,Ri R6, R 7' R 8', R, L L2, a,b,c,d and e are as defined herein. Use ofthe compounds as a component of lipid nanoparticle formulations for delivery of a therapeutic agent, compositions comprising the compounds and methods for their use and preparation are also provided. R N R2 R R"b R2b R 3b R44 R 4 N ~R 20000 --- Compound A -a- MC3 -0- Comouwd 5 -z15000 -4- Compound a 10000 d 0.0 0,2 GA 0,6 0.8 1.0 12 mRNA Dose (mg/kg) Figure 3
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