Compound and method for treating myotonic dystrophy
US8741863B2 · kind B2 · utility
Assignee
Inventors
Key dates
| Filing date | Aug 26, 2011 |
| Grant date | Jun 3, 2014 |
| Priority date | — |
| Expiry date | Oct 14, 2031 |
Classification
- Technology area (CPC C)Chemistry; Metallurgy
- CPC primaryC12N2810/40
- WIPO fieldPharmaceuticals
- WIPO sectorChemistry
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.
Source: USPTO / EPO open patent data. Objective bibliographic and citation counts.