Claims for Patent: 8,436,049

Summary for Patent: 8,436,049

Title:	Uses of pentamidine and related compounds
Abstract:	Methods are provided herein for treatment of myotonic dystrophy and other toxic RNA diseases in a subject. In some examples, the method comprises administration of a compound that binds a nucleotide repeat expansion in a ribonucleic acid molecule, thereby treating the disease. In additional examples, the method comprises administration of a compound that disrupts binding of muscleblind-like proteins to an RNA nucleotide repeat expansion. Compounds for use in the disclosed method include pentamidine or heptamidine or derivatives thereof. Representative compounds are described herein.
Inventor(s):	Berglund; John Andrew (Eugene, OR), Warf; M. Bryan (Eugene, OR), Matthys; Catherine (Eugene, OR), Haley; Michael M. (Eugene, OR), Hilton; Cameron L. (Eugene, OR)
Assignee:	State of Oregon Acting By and Through the State Board of Higher Education on behalf of the University of Oregon (Eugene, OR) N/A (N/A)
Application Number:	12/918,696
Patent Claims:	1. A method of treating myotonic dystrophy in a subject, comprising administering to the subject a compound, diamidine having the structure ##STR00020## wherein n=2-7, that binds a nucleotide repeat expansion in a ribonucleic acid molecule, thereby treating myotonic dystrophy. 2. The method of claim 1, wherein the nucleotide repeat expansion comprises a trinucleotide repeat expansion. 3. The method of claim 2, wherein the trinucleotide repeat expansion comprises a CUG repeat. 4. The method of claim 1, wherein the nucleotide repeat expansion comprises a trinucleotide repeat expansion in the pre-mRNA or mRNA of the dystrophia myotonica-protein kinase gene. 5. The method of claim 1, wherein the nucleotide repeat expansion comprises a tetranucleotide repeat expansion. 6. The method of claim 5, wherein the tetranucleotide repeat expansion comprises a CCUG repeat. 7. The method of claim 1, wherein the nucleotide repeat expansion comprises a tetranucleotide repeat expansion in a zinc finger 9 ribonucleic acid. 8. The method of claim 1, wherein the diamidine comprises pentamidine or heptamidine. 9. The method of claim 1, wherein the compound diamidine having the structure ##STR00021## wherein n=2-7, that binds the nucleotide repeat expansion disrupts the binding of muscleblind-like proteins to CUG repeats of dystrophia myotonica-protein kinase. 10. The method of claim 1, wherein the compound diamidine having the structure ##STR00022## wherein n=2-7, that binds the nucleotide repeat expansion disrupts binding of muscleblind-like proteins to CCUG repeats within intron 1 of zinc finger 9 pre-mRNA. 11. A method of treating myotonic dystrophy caused by toxic RNA in a subject, comprising administering to the subject a compound, diamidine having the structure ##STR00023## wherein n=2-7, that binds a nucleotide repeat expansion in a ribonucleic acid molecule, thereby treating the disease caused by toxic RNA. 12. A method of treating myotonic dystrophy caused by toxic RNA in a subject, comprising administering to the subject a compound, diamidine having the structure ##STR00024## wherein n=2-7, that displaces muscleblind-like (MBNL) protein from a nucleotide repeat expansion in a ribonucleic acid molecule, thereby treating the disease caused by toxic RNA. 13. The method of claim 11, wherein the diamidine comprises pentamidine or heptamidine.

Details for Patent 8,436,049

Applicant	Tradename	Biologic Ingredient	Dosage Form	BLA	Approval Date	Patent No.	Expiredate
Merck Sharp & Dohme Corp.	INTRON A	interferon alfa-2b	For Injection	103132	06/04/1986		2028-02-21
Merck Sharp & Dohme Corp.	INTRON A	interferon alfa-2b	For Injection	103132			2028-02-21
Merck Sharp & Dohme Corp.	INTRON A	interferon alfa-2b	Injection	103132			2028-02-21