Abstract: The invention relates to a Matriptase-2 (MT2) inhibitor, TMPRSS6 inhibitor, Ferroportin blocker, or hepcidin enhancer. In particular, the invention relates to the use of compounds capable of reducing at least one of systemic iron level, Transferrin saturation (Tsat), non-transferrin-bound iron (NTBI), and labile plasma iron (LPI). This may take place by inhibiting the expression of a target gene, wherein the target gene may be Transmembrane protease, serine 6 (TMPRSS6). Further, the invention relates to compositions comprising said compound and to methods for using such compounds and/or compositions. The uses may comprise therapeutic uses such as for reduction or iron overload and the prevention of iron related toxicity before, during or after chemotherapeutic conditioning, stem cell transplantation and engraftment.

Abstract: The present invention relates to products and compositions and their uses. In particular the invention relates to nucleic acid products that interfere with the TMPRSS6 gene expression or inhibits its expression in combination with one or more iron chelators and possibly other active agents, as well as therapeutic uses such as for the treatment of hemochromatosis, porphyria and blood disorders such as beta-thalassemia, sickle cell disease and transfusional iron overload or myelodysplastic syndrome, and infections and non-relapse related mortality associated with bone marrow transplantation.

Abstract: The present invention relates to products and compositions and their uses. In particular the invention relates to nucleic acid products that interfere with the TMPRSS6 gene expression or inhibits its expression in combination with one or more iron chelators and possibly other active agents, as well as therapeutic uses such as for the treatment of hemochromatosis, porphyria and blood disorders such as beta-thalassemia, sickle cell disease and transfusional iron overload or myelodysplastic syndrome, and infections and non-relapse related mortality associated with bone marrow transplantation.

Abstract: The invention relates to a Matriptase-2 (MT2) and/or TMPRSS6 inhibitor for the prevention, decrease of the risk of suffering from or treatment of a myeloproliferative disorder.

Abstract: The invention relates to nucleic acid products that interfere with or inhibit CNNM4 (Cyclin M4) gene expression. It further relates to therapeutic uses of CNNM4 inhibition for the treatment of diseases, such as liver diseases including non-alcoholic fatty liver disease (NAFLD), non-alcoholic steatohepatitis (NASH), liver steatosis, liver fibrosis, liver cirrhosis, liver cancer and other diseases associated with magnesium dysregulation.

Abstract: The invention relates to nucleic acid products that interfere with or inhibit PROS1 gene expression. It further relates to therapeutic uses of PROS1 inhibition for the treatment of bleeding disorders.

Abstract: The invention relates to nucleic acid products that interfere with or inhibit CNNM4 (Cyclin M4) gene expression. It further relates to therapeutic uses of CNNM4 inhibition for the treatment of diseases, such as liver diseases including non-alcoholic fatty liver disease (NAFLD), non-alcoholic steatohepatitis (NASH), liver steatosis, liver fibrosis, liver cirrhosis, liver cancer and other diseases associated with magnesium dysregulation.

Abstract: The invention relates to nucleic acid products that interfere with or inhibit PROS1 gene expression. It further relates to therapeutic uses of PROS1 inhibition for the treatment of bleeding disorders.

Abstract: The present invention relates to products and compositions and their uses. In particular the invention relates to nucleic acid products that interfere with the TMPRSS6 gene expression or inhibits its expression in combination with one or more iron chelators and possibly other active agents, as well as therapeutic uses such as for the treatment of hemochromatosis, porphyria and blood disorders such as beta-thalassemia, sickle cell disease and transfusional iron overload or myelodysplastic syndrome, and infections and non-relapse related mortality associated with bone marrow transplantation.

Abstract: The invention relates to nucleic acid products that interfere with PROS1 gene expression or inhibit its expression. The nucleic acids are particularly for use in the treatment, prevention or reduction of risk of suffering from a bleeding disorder.

Abstract: The invention relates to nucleic acid products that interfere with or inhibit PROS1 gene expression. It further relates to therapeutic uses of PROS1 inhibition for the treatment of bleeding disorders.

Abstract: The present invention relates to products and compositions and their uses. In particular the invention relates to nucleic acid products that interfere with the TMPRSS6 gene expression or inhibits its expression and therapeutic uses such as for the treatment of hemochromatosis, porphyria and blood disorders such as ?-thalassemias, sickle cell disease and transfusional iron overload or myelodysplastic syndrome.

Abstract: The present invention relates to products and compositions and their uses. In particular the invention relates to nucleic acid products that interfere with the TMPRSS6 gene expression or inhibits its expression and therapeutic uses such as for the treatment of hemochromatosis, porphyria and blood disorders such as ?-thalassemias, sickle cell disease and transfusional iron overload or myelodysplastic syndrome.

Abstract: The present invention relates to products and compositions and their uses. In particular the invention relates to nucleic acid products that interfere with the TMPRSS6 gene expression or inhibits its expression and therapeutic uses such as for the treatment of hemochromatosis, porphyria and blood disorders such as ?-thalassemias, sickle cell disease and transfusional iron overload or myelodysplastic syndrome.

Abstract: The present invention is related to composition comprising a lipid composition, wherein the lipid composition consists of a cationic lipid of formula (I) wherein n is any one of 1, 2, 3, and 4, wherein m is any one of 1, 2 and 3, Y? is an anion, wherein each of R1 and R2 is individually and independently selected from the group consisting of linear C12-C18 alkyl and linear C12-C18 alkenyl; a sterol compound, wherein the sterol compound is selected from the group consisting of cholesterol and stigmasterol; and a PEGylated lipid, wherein the PEGylated lipid comprises a PEG moiety and wherein the PEGylated lipid is selected from the group consisting of a PEGylated phosphoethanolamine of formula (II) wherein each of R3 and R4 is individually and independently linear C13-C17 alkyl, and p is any integer from 15 to 130; a PEGylated ceramide of formula (III) wherein R5 is linear C7-C15 alkyl, and q is any integer from 15 to 130; and a PEGylated diacylglycerol of formula (IV) wherein each of R6 and R7 is individually

Abstract: The present invention is related to composition comprising a lipid composition, wherein the lipid composition consists of a cationic lipid of formula (I) wherein n is any one of 1, 2, 3, and 4, wherein m is any one of 1, 2 and 3, Y? is an anion, wherein each of R1 and R2 is individually and independently selected from the group consisting of linear C12-C18 alkyl and linear C12-C18 alkenyl; a sterol compound, wherein the sterol compound is selected from the group consisting of cholesterol and stigmasterol; and a PEGylated lipid, wherein the PEGylated lipid comprises a PEG moiety and wherein the PEGylated lipid is selected from the group consisting of a PEGylated phosphoethanolamine of formula (II) wherein each of R3 and R4 is individually and independently linear C13-C17 alkyl, and p is any integer from 15 to 130; a PEGylated ceramide of formula (III) wherein R5 is linear C7-C15 alkyl, and q is any integer from 15 to 130; and a PEGylated diacylglycerol of formula (IV) wherein each of R6 and R7 is individually