Claims for Patent: 9,371,527

Summary for Patent: 9,371,527

Title:	Compositions and methods related to protein displacement therapy for myotonic distrophy
Abstract:	Disclosed are compositions and methods related to the interaction of polyCUG and polyCCUG repeat RNA and proteins that bind to these repetitive RNA sequences. Also disclosed are methods of treating DM1 or DM2 comprising inhibiting the interaction of poly(CUG).sup.exp or poly(CCUG).sup.exp RNA with muscleblind proteins, or by causing improvement of spliceopathy in myotonic dystrophy.
Inventor(s):	Thornton; Charles A. (Rochester, NY), Wheeler; Thurman (Rochester, NY), Sobczak; Krzysztof (Poznan, PL), Osborne; Robert (Essex, GB), Miller; Jill (Rockport, NY), Swanson; Maurice Scott (Gainsville, FL)
Assignee:	University of Rochester (Rochester, NY)
Application Number:	14/297,137
Patent Claims:	1. A method of treating myotonic dystrophy comprising administering to a subject with myotonic dystrophy an agent that inhibits the interaction of MBNL1 with poly(CUG).sup.exp mRNA, wherein the agent comprises an antisense oligonucleotide, and wherein the antisense oligonucleotide has a nucleobase sequence consisting of 2, 3, 4, 5, 6, 7, 8, 9, 10, 11, or 12 CAG repeats that are fully complementary to the poly(CUG).sup.exp. 2. The method of claim 1, wherein the antisense oligonucleotide is a morpholino. 3. The method of claim 1, wherein the antisense oligonucleotide sequence is SEQ ID NO: 3. 4. The method of claim 3, wherein the antisense oligonucleotide is a morpholino. 5. The method of claim 1, wherein the agent comprises a conjugate. 6. The method of claim 1, wherein the antisense oligonucleotide is a peptide nucleic acid. 7. A method of treating myotonic dystrophy in a subject in need thereof comprising administering to the subject an agent that corrects spliceopathy, wherein the agent comprises an antisense oligonucleotide, wherein the antisense oligonucleotide corrects spliceopathy of chloride ion channel ClC-1 in the subject, wherein the antisense oligonucleotide has a nucleobase sequence that is complementary to either the junction between intron 6 and exon 7a of ClC-1 pre-mRNA or the junction between exon 7a and intron 7a of ClC-1 pre-mRNA. 8. The method of claim 7, wherein the antisense oligonucleotide is a morpholino. 9. The method of claim 7, wherein the antisense oligonucleotide has the nucleobase sequence of SEQ ID NO: 4. 10. The method of claim 9, wherein the antisense oligonucleotide is a morpholino. 11. The method of claim 7, wherein the agent is a peptide nucleic acid. 12. The method of claim 7, wherein the agent comprises a conjugate. 13. The method of claim 7, wherein the antisense oligonucleotide has a nucleobase sequence of SEQ ID NO: 6.

Details for Patent 9,371,527

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		2026-09-21
Merck Sharp & Dohme Corp.	INTRON A	interferon alfa-2b	For Injection	103132			2026-09-21
Merck Sharp & Dohme Corp.	INTRON A	interferon alfa-2b	Injection	103132			2026-09-21