Compound and method for treating myotonic dystrophy

a myotonic dystrophy and compound technology, applied in the direction of drug compositions, specific cell targeting, peptide/protein ingredients, etc., can solve the problems of often hindering the practical utility of many drugs with potentially useful biological activity, and achieve the effect of blocking the sequestration of muscleblind-like 1 protein
US20100016215A1Inactive Publication Date: 2010-01-21SAREPTA THERAPEUTICS INC
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Patent Information

Authority / Receiving Office
US · United States
Patent Type
Applications(United States)
Current Assignee / Owner
SAREPTA THERAPEUTICS INC
Publication Date
2010-01-21
Estimated Expiration
Not applicable · inactive patent

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Abstract

An antisense compound for use in treating myotonic dystrophy DM1 or DM2, a method of enhancing antisense targeting to heart and quadricep muscles, and a method for treating DM1 or DM2 in a mammalian subject are disclosed. The oligonucleotide has 8-30 bases, with at least 8 contiguous bases being complementary to the polyCUG or polyCCUG repeats in the 3′UTR region of dystrophia myotonica protein kinase (DMPK) mRNA in DM1 or DM2, respectively. Conjugated to the oligonucleotide is a cell-penetrating peptide having the sequence (RXRR(B / X)R)2XB, where R is arginine; B is β-alanine; and each X is —C(O)—(CH2)n—NH—, where n is 4-6. The antisense compound is effective to selectively block the sequestration of muscleblind-like 1 protein (MBNL1) and / or CUGBP, in heart and quadricep muscle in a myotonic dystrophy animal model.
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Description

[0001] This application claims priority to U.S. provisional application Ser. No. 60 / 937,725, filed Jun. 29, 2007, and to U.S. patent application Ser. No. 12 / 217,040, filed Jun. 30, 2008, both of which are incorporated by reference herein in their entirety.FIELD OF THE INVENTION

[0002] The invention relates to an antisense compound and method for treating myotonic dystrophy DM1 and DM2.REFERENCES

[0003] Abes, S., H. M. Moulton et al. (2006). “Vectorization of morpholino oligomers by the (R-Ahx-R)4 peptide allows efficient splicing correction in the absence of endosomolytic agents.”J Control Release 116(3): 304-13.

[0004] Arap, W. et al. (2004). “Human and mouse targeting peptides identified by phage display.” U.S. Appn. Pubn. No. 20040170955.

[0005] Behlke, M. A. (2006). “Progress towards in vivo use of siRNAs.”Mol Ther 13(4): 644-70.

[0006] Alter, J., F. Lou et al. (2006). “Systemic delivery of morpholino oligonucleotide restores dystrophin expression bodywide and improves dystrophic pathology...

Claims

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