Silence Therapeutics Gmbh

Universitât Bern

Ute Schaeper

Sibylle Dames

Lucas Bethge

Christian Frauendorf

Judith Hauptmann

Adrien Weingärtner

Anne Angelillo-Scherrer

Eliot Morrison

Raja Prince Eladnani

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.

Nucleic acids for inhibiting expression of TMPRSS6 and iron chelators

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.

Nucleic acids for inhibiting expression of PROS1 in a cell

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 &#x3b2;-thalassemias, sickle cell disease and transfusional iron overload or myelodysplastic syndrome.

Products and compositions

Growing community of inventors

Berlin, Germany

Ute Schaeper