Invention Publication
- Patent Title: CONJUGATE OF SAPONIN, OLIGONUCLEOTIDE AND GALNAC
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Application No.: US18044945Application Date: 2021-09-09
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Publication No.: US20240327829A1Publication Date: 2024-10-03
- Inventor: Guy HERMANS , Ruben POSTEL , Helmus VAN DE LANGEMHEEN , Mazdak ASADIAN BIRJAND
- Applicant: SAPREME TECHNOLOGIES B.V.
- Applicant Address: NL Biltoven
- Assignee: SAPREME TECHNOLOGIES B.V.
- Current Assignee: SAPREME TECHNOLOGIES B.V.
- Current Assignee Address: NL Biltoven
- Priority: NL 26442 2020.09.10 NL 27405 2021.01.26 WO TNL2021050384 2021.06.18
- International Application: PCT/NL2021/050549 2021.09.09
- Date entered country: 2023-03-10
- Main IPC: C12N15/113
- IPC: C12N15/113

Abstract:
The invention relates to a conjugate comprising a saponin covalently linked to a ligand for ASGPR, the ligand comprising at least one GalNAc moiety, and comprising an oligonucleotide covalently linked to the saponin and the ligand for ASGPR. In addition, the invention relates to a pharmaceutical composition comprising the conjugate of the invention. Furthermore, the invention relates to a pharmaceutical composition of the invention, for use as a medicament. The invention also relates to a pharmaceutical composition of the invention, for use in the treatment or prophylaxis of a disease or health problem in which an expression product is involved of for example genes: apoB, HSP17, TTR, PCSK9, ALAS1, AT3, GO, CC5, X gene of HBV, S gene of HBV, AAT and LDH, and for use in the treatment or prophylaxis of for example a cancer, an infectious disease, a viral infection, hypercholesterolemia, primary hyperoxaluria, haemophilia A, haemophilia B, AAT related liver disease, acute hepatic porphyria, TTR amyloidosis, complement-mediated disease, hepatitis B infection, or an auto-immune disease. The invention also relates to a method for producing an oligonucleotide conjugate of the invention. Finally, the invention relates to an in vitro or ex vivo method for transferring the conjugate of the invention from outside a cell to inside said cell.
Information query
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