The invention relates to a conjugate for transferring an
effector molecule from outside a
cell into said
cell, the conjugate comprising at least one
effector molecule to be transferred into the
cell, at least one
saponin of the mono-desmosidic
triterpene glycoside type or the bi-desmosidic
triterpene glycoside type, and at least one single-domain
antibody (sdAb), covalently bound to each other, wherein the sdAb is capable of binding to a cell-surface molecule of said cell. The invention also 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. In addition, the invention relates to a pharmaceutical composition of the invention, for use in the treatment or the prophylaxis of any one or more of: a
cancer, an auto-immune
disease such as
rheumatoid arthritis, an
enzyme deficiency, a
disease related to an
enzyme deficiency, a
gene defect, a
disease relating to a
gene defect, an infection such as a
viral infection, hypercholesterolemia, primary hyperoxaluria,
haemophilia A,
haemophilia B, alpha-1 antitrypsin related
liver disease, acute hepatic porphyria, an
amyloidosis and
transthyretin-mediated
amyloidosis. The invention also relates to an
in vitro or
ex vivo method for transferring the conjugate from outside a cell to inside said cell or for transferring the
effector molecule comprised by the conjugate of the invention from outside a cell to inside said cell, preferably to the
cytosol of said cell.