http://rdf.ncbi.nlm.nih.gov/pubchem/patent/WO-2021261998-A1

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classificationCPCAdditional http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A61K2300-00
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filingDate 2021-06-23-04:00^^<http://www.w3.org/2001/XMLSchema#date>
inventor http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_ce7607b775df3167a1cbc787d2e4dc83
http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_0a7f3b7c70f376c16c62069afcdbca95
publicationDate 2021-12-30-04:00^^<http://www.w3.org/2001/XMLSchema#date>
publicationNumber WO-2021261998-A1
titleOfInvention Therapeutic combination of galnac-oligonucleotide conjugate and saponin, and uses thereof
abstract The invention relates to a pharmaceutical combination comprising: a conjugate of an effector molecule and a ligand for ASGPR, wherein the ligand for ASGPR comprises at least one GalNAc moiety; and a saponin of the monodesmosidic or bidesmosidic triterpene glycoside type. The invention also relates to a pharmaceutical composition comprising the conjugate and the saponin. In addition, the invention relates to a pharmaceutical combination or composition of the invention, for use as a medicament, or for use in the treatment or prophylaxis of a disease or health problem in which an expression product is involved of any one or more of genes: apoB, TTR, PCSK9, ALAS1, ATS, GO, CCS, X gene of HBV, S gene of HBV, AAT and LDH, and/or for use in the treatment or prophylaxis of a cancer, an infectious disease, a viral infection, hypercholesterolemia, primary hyperoxaluria, haemophilia A, haemophilia B, AAT related liver disease, acute hepatic porphyria, TTR-mediated amyloidosis, hereditary TTR amyloidosis (hATTR), complement-mediated disease, hepatitis B infection, or an auto-immune disease. Furthermore, the invention relates to an in vitro or ex vivo method for transferring an effector molecule of the invention from outside a cell to inside said cell, preferably into the cytosol of said cell. The invention also relates to an in vitro or ex vivo method for transferring the conjugate of the invention from outside a cell to inside said cell.
priorityDate 2020-06-24-04:00^^<http://www.w3.org/2001/XMLSchema#date>
type http://data.epo.org/linked-data/def/patent/Publication

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