COMPOSITIONS FOR TREATING MUSCULAR DYSTROPHY

• US 20140315862A1
• Filed: 03/14/2014
• Published: 10/23/2014
• Est. Priority Date: 03/15/2013
• Status: Active Grant

• Current Assignee: Sarepta Therapeutics, Inc.
• Original Assignee: Sarepta Therapeutics, Inc.
• Inventors: Kaye, Edward M.
• Granted Patent: US 9,506,058 B2
• Time in Patent Office: Days
• US Class Current: 514/81
• CPC Class Codes: A61K 31/7125 Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
• CPC Class Codes: A61P 1/00 Drugs for disorders of the alimentary tract or the digestive system
• CPC Class Codes: A61P 21/00 Drugs for disorders of the muscular or neuromuscular system
• CPC Class Codes: A61P 21/02 Muscle relaxants, e.g. for tetanus or cramps
• CPC Class Codes: C12N 15/113 Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing when used in plants C12N15/8218
• CPC Class Codes: C12N 2310/33 of the base
• CPC Class Codes: C12N 2320/30 Special therapeutic applications

Abstract

Improved compositions and methods for treating muscular dystrophy by administering antisense molecules capable of binding to a selected target site in the human dystrophin gene to induce exon skipping are described.

Claims

1. A method for treating Duchenne muscular dystrophy in a patient in need thereof comprising administering to the patient a dose of eteplirsen of about 30 mg/kg once a week.

2. The method according to claim 1, wherein eteplirsen is administered in a single dose.

3. The method according to claim 1, wherein eteplirsen is administered intravenously.

4. The method according to claim 1, wherein the patient has an out-of-frame deletion(s) that may be corrected by skipping exon 51 of the dystrophin gene.

5. The method according to claim 1, wherein the patient is a pediatric patient.

6. The method according to claim 1, wherein the patient is administered an oral corticosteroid for at least 24 weeks prior to the first dose of eteplirsen.

7. A method for treating Duchenne muscular dystrophy in a patient in need thereof comprising administering intravenously to the patient a single dose of eteplirsen of about 30 mg/kg once a week, wherein the patient has an out-of-frame deletion(s) that may be corrected by skipping exon 51 of the dystrophin gene.

8. A method for treating Duchenne muscular dystrophy a patient in need thereof comprising administering to the patient a dose of eteplirsen of about 50 mg/kg once a week.

9. The method according to claim 8, wherein eteplirsen is administered in a single dose.

10. The method according to claim 8, wherein eteplirsen is administered intravenously.

11. The method according to claim 8, wherein the patient has an out-of-frame deletion(s) that may be corrected by skipping exon 51 of the dystrophin gene.

12. The method according to claim 8, wherein the patient is a pediatric patient.

13. The method according to claim 8, wherein the patient is administered an oral corticosteroid for at least 24 weeks prior to the first dose of eteplirsen.

14. A method for treating Duchenne muscular dystrophy a patient in need thereof comprising administering intravenously to the patient a single dose of eteplirsen of about 50 mg/kg once a week, wherein the patient has an out-of-frame deletion(s) that may be corrected by skipping exon 51 of the dystrophin gene.

15. A method for increasing dystrophin production in a patient having Duchenne muscular dystrophy comprising administering to the patient a dose of eteplirsen of about 30 mg/kg once a week.

16. The method according to claim 15, wherein eteplirsen is administered in a single dose.

17. The method according to claim 15, wherein eteplirsen is administered intravenously.

18. The method according to claim 15, wherein the patient has an out-of-frame deletion(s) that may be corrected by skipping exon 51 of the dystrophin gene.

19. The method according to claim 15, wherein the patient is a pediatric patient.

20. The method according to claim 15, wherein the patient is administered an oral corticosteroid for at least 24 weeks prior to the first dose of eteplirsen.

21. A method for increasing dystrophin production in a patient having Duchenne muscular dystrophy comprising administering to the patient a dose of eteplirsen of about 50 mg/kg once a week.

22. The method according to claim 21, wherein eteplirsen is administered in a single dose.

23. The method according to claim 21, wherein eteplirsen is administered intravenously.

24. The method according to claim 21, wherein the patient has an out-of-frame deletion(s) that may be corrected by skipping exon 51 of the dystrophin gene.

25. The method according to claim 21, wherein the patient is a pediatric patient.

26. The method according to claim 21, wherein the patient is administered an oral corticosteroid for at least 24 weeks prior to the first dose of eteplirsen.