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AssigneesSilence Therapeutics GmbH, New York Blood Center, Inc.
InventorsUte Schaeper, Francesca Vinchi
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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.
A therapeutic agent for use in the treatment of an iron metabolism disease or condition wherein the therapeutic agent comprises an inhibitor selected from:
The therapeutic agent for use according to any one of statements 1 to 8, wherein the nucleic acid is EU401 or EU402.
A pharmaceutical composition for use in the treatment of an iron metabolism disease or condition, the pharmaceutical composition comprising an effective amount of the therapeutic agent as defined in any of the preceding claims, further comprising a pharmaceutically acceptable diluent, carrier, or excipient.
The therapeutic agent for use or the pharmaceutical composition for use of any of the preceding claims, wherein the disease or condition is
A therapeutic agent for use or the pharmaceutical composition for use of any of the preceding claims, wherein the treatment comprises or consists of the step of:
A therapeutic agent for use or the pharmaceutical composition for use of any of the preceding claims, wherein the treatment further comprises co-administration of one or more chemotherapeutic agent, radiotherapy, and/or immunotherapy.
A therapeutic agent for use or the pharmaceutical composition for use of any of the preceding claims, wherein the cell explantation, implantation, or transplantation treatment comprises hematopoietic stem cell transplantation (HSCT), optionally wherein the therapeutic agent or the pharmaceutical composition is administered to the subject in need thereof prior to, during, or after conditioning for HSCT,
A method of treating an iron metabolism disease or condition in a subject in need thereof, comprising administering an effective amount of the pharmaceutical composition or the therapeutic agent defined in any of the preceding claims, wherein the treatment comprises cell explantation, implantation, or transplantation.
Use of a therapeutic agent or a pharmaceutical composition according to the preceding claims for the manufacture of a medicament for the treatment of an iron metabolism disease or condition, wherein the treatment comprises cell explantation, implantation, or transplantation.
Dependencies are inferred from the claim text.