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Last Updated: December 17, 2025

Claims for Patent: 8,236,773


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Summary for Patent: 8,236,773
Title:Aptamer therapeutics useful in the treatment of complement-related disorders
Abstract:The invention provides nucleic acid therapeutics and methods for using these nucleic acid therapeutics in the treatment of complement-related disorders.
Inventor(s):Claude Benedict, David Epstein, Charles Wilson, Dilara McCauley, Jeffrey Kurz, Markus Kurz, Thomas Greene McCauley, James Rottman
Assignee:Astellas US LLC
Application Number:US11/884,411
Patent Claims: 1. A method of treating a C5 complement protein, C5a and/or C5b-9-mediated disorder, the method comprising the step of administering to a patient in need thereof a therapeutically effective amount of a compound having the structure set forth below or a salt thereof: where, indicates a linker Aptamer=fCmGfCfCGfCmGmGfUfCfUfCmAmGmGfCGfCfUmGmAmGfUfCfUmGmAmGfUfUfUAfCfCfUmGfCmG-3T (SEQ ID NO:4), wherein fC and fU=2′-fluoro nucleotides, and mG and rnA=2′-OMe nucleotides and all other nucleotides are 2′-OH and 3T indicates an inverted deoxy thymidine.

2. The method of claim 1, wherein the linker is an alkyl linker.

3. The method of claim 2, wherein the alkyl linker comprises 2 to 18 consecutive CH2 groups.

4. The method of claim 1, wherein the compound has the structure set forth below or a salt thereof: where Aptamer=fCmGfCfCGfCmGmGfUfCfUfCmAmGmGfCGfCfUmGmAmGfUfCfUmGmAmGfUfUfUAfCfCfUmGf CmG-3T (SEQ ID NO 4), wherein fC and fU=2′-fluoro nucleotides, and mG and rnA=2′-OMe nucleotides and all other nucleotides are 2′-OH and 3T indicates an inverted deoxy thymidine.

5. The method of claim 1 or 4, wherein the disorder to be treated is myocardial injury relating to CABG surgery, myocardial injury relating to balloon angioplasty or myocardial injury relating to restenosis.

6. The method of claim 1 or 4, wherein the disorder to be treated is C5, C5a and/or C5b-9 complement protein mediated complications relating to CABG surgery.

7. The method of claim 1 or 4, wherein the disorder to be treated is C5 mediated complement complications relating to percutaneous coronary intervention.

8. The method of claim 1 or 4, wherein the disorder to be treated is paroxysomal nocturnal hemoglobinuria.

9. The method of claim 1 or 4, wherein the disorder to be treated is acute transplant rejection, hyperacute transplant rejection, subacute transplant rejection or chronic transplant rejection.

10. The method of claim 1 or 4, wherein the disorder to be treated is complement mediated ocular tissue damage.

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