Conjugate of galnac and saponin, therapeutic composition comprising said conjugate and a galnac-oligonucleotide conjugate
Номер патента: CA3183885A1
Опубликовано: 30-12-2021
Автор(ы): Guy Hermans, Ruben POSTEL
Принадлежит: Sapreme Technologies BV
Опубликовано: 30-12-2021
Автор(ы): Guy Hermans, Ruben POSTEL
Принадлежит: Sapreme Technologies BV
Реферат: The invention relates to a saponin conjugate comprising a saponin covalently linked to a ligand for ASGPR, the ligand comprising at least one GalNAc. The invention also relates to a pharmaceutical combination comprising a first pharmaceutical composition comprising the saponin conjugate of the invention and a second pharmaceutical composition comprising a second conjugate of an effector molecule and a ligand for ASGPR, or a third conjugate of an effector molecule and a binding molecule for binding to a cell-surface molecule. In addition, the invention relates to a pharmaceutical composition comprising the saponin conjugate of the invention and the second conjugate or the third conjugate. Furthermore, the invention relates to a pharmaceutical combination or pharmaceutical composition of the invention, for use as a medicament. The invention also relates to a pharmaceutical combination or 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 genes: apoB, 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 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. Finally, the invention relates to an in vitro or ex vivo method for transferring the second conjugate or the third conjugate of the invention from outside a cell to inside said cell.
Conjugate of galnac and saponin, therapeutic composition comprising said conjugate and a galnac-oligonucleotide conjugate
Номер патента: AU2021295358A1. Автор: Guy Hermans,Ruben POSTEL. Владелец: Sapreme Technologies BV. Дата публикации: 2023-02-16.