Title:
NOVEL COMPOSITIONS FOR CONJUGATING OLIGONUCLEOTIDES AND CARBOHYDRATES
Document Type and Number:
WIPO Patent Application WO/2024/040531
Kind Code:
A1
Abstract:
The invention provides compositions and linking configurations for an oligonucleotide to be conjugated to a carbohydrate ligand for targeted in vivo delivery of an oligonucleotide. These ligand-conjugated compounds target one or more organs or cell types, e.g., the parenchymal cells of the liver that facilitate endocytic uptake. The compound has structural formula (S-HG1): R206-[A1]a-[A2]b-[A3]c-R205 (S-HG1) R205, R206 are each independently for each occurrence H, OH, a protecting group for OH, a phosphate group, a phosphodiester group, an activated phosphate group, an activated phosphite group, a phosphoramidite, a solid support, -OP(M')(M")O-nucleoside, - OP(M')(M")O-oligonucleotide, a lipid, a PEG, a steroid, a polymer, -O- nucleotide, a nucleoside, -OP(M')( M")OR201-OP(M'")(M"")O- oligonucleotide, or an oligonucleotide; M', M", M'" and M"" are each independently for each occurrence O or S; Al, A2 and A3 are each independently for each occurrence selected from (S-1H) or (S-1G).
Inventors:
LI CHIANG J (US)
CAO CHEN (CN)
GUI LI (CN)
POGULA PRAVEEN (US)
SUN XIANGAO (US)
RAND DANIELLE (US)
CAO CHEN (CN)
GUI LI (CN)
POGULA PRAVEEN (US)
SUN XIANGAO (US)
RAND DANIELLE (US)
Application Number:
PCT/CN2022/114893
Publication Date:
February 29, 2024
Filing Date:
August 25, 2022
Export Citation:
Assignee:
1GLOBE HEALTH INST NANJING (CN)
1GLOBE HEALTH INST LLC (US)
1GLOBE HEALTH INST LLC (US)
International Classes:
C07H15/04; A61K47/54; C07H15/26; C07H21/00; C07H21/02; C12N15/11
Domestic Patent References:
| WO2019128611A1 | 2019-07-04 | |||
| WO2009029688A2 | 2009-03-05 |
Foreign References:
| CN112390835A | 2021-02-23 | |||
| US20090208564A1 | 2009-08-20 |
Other References:
"Lewin's Genes XII", 2017, JONES AND BARTLETT LEARNING
"Molecular Biology and Biotechnology: a Comprehensive Desk Reference", 2011, ANMOL PUBLICATIONS PVT. LTD
BEAUCAGE ET AL., TETRAHEDRON, vol. 48, 1992, pages 2223 - 2311
GREENEWUTS: "Protective Groups in Organic Synthesis", 1991, JOHN WILEY & SONS
"Molecular Biology and Biotechnology: a Comprehensive Desk Reference", 2011, ANMOL PUBLICATIONS PVT. LTD
BEAUCAGE ET AL., TETRAHEDRON, vol. 48, 1992, pages 2223 - 2311
GREENEWUTS: "Protective Groups in Organic Synthesis", 1991, JOHN WILEY & SONS
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