Title:
PHARMACEUTICAL COMPOSITION FOR MUSCLE DISEASE TREATMENT
Document Type and Number:
WIPO Patent Application WO/2020/209285
Kind Code:
A1
Abstract:
Developed were: a nucleic acid complex that exhibits an excellent anti-sense effect in skeletal muscle and/or cardiac muscle; and a therapeutic or prophylactic composition that contains said nucleic acid complex as the active ingredient, and that is for muscle disease with the onset in skeletal muscle, cardiac muscle, or the like. Provided is a double-stranded nucleic acid complex obtained by annealing a first nucleic acid strand, which hybridizes with a transcription product of a target gene and has an antisense effect on the transcription product, and a second nucleic acid strand, which includes a base sequence complementary to the first nucleic acid strand and binds with cholesterol or an analog thereof.
Inventors:
YOKOTA TAKANORI (JP)
NAGATA TETSUYA (JP)
NAGATA TETSUYA (JP)
Application Number:
PCT/JP2020/015818
Publication Date:
October 15, 2020
Filing Date:
April 08, 2020
Export Citation:
Assignee:
UNIV NAT CORP TOKYO MEDICAL & DENTAL (JP)
International Classes:
A61K48/00; A61K31/713; A61K47/56; A61P3/10; A61P7/00; A61P9/00; A61P9/06; A61P21/00; A61P21/04; A61P25/28; A61P29/00; C12N15/113
Domestic Patent References:
| WO2018056442A1 | 2018-03-29 | |||
| WO2017068790A1 | 2017-04-27 | |||
| WO2013089283A1 | 2013-06-20 | |||
| WO2014203518A1 | 2014-12-24 | |||
| WO2014192310A1 | 2014-12-04 | |||
| WO2007143315A2 | 2007-12-13 | |||
| WO2008043753A2 | 2008-04-17 | |||
| WO2008049085A1 | 2008-04-24 |
Foreign References:
| JP2015502134A | 2015-01-22 | |||
| JP2018530560A | 2018-10-18 | |||
| JP2018528783A | 2018-10-04 | |||
| JP2019073832A | 2019-05-16 |
Other References:
NAGATA T.; OHYAGI M.; IHARA K.; KABURAGI H.; NISHINA K.; PIAO W.; YOSHIDA-TANAKA K.; KUWAHARA H.; YOSHIOKA K.; YOKOTA T.: "The effect of DNA/RNA heteroduplex oligonucleotides on muscle", JOURNAL OF NEUROLOGICAL SCIENCES, vol. 381, 1 January 1900 (1900-01-01), NL , pages 846, XP085295674, ISSN: 0022-510X, DOI: 10.1016/j.jns.2017.08.2383
NAGATA, TETSUYA: "HT-02-3 Recent progress of DNA/RNA heteroduplex oligonucleotide", CLINICAL NEUROLOGY, vol. 58, no. Suppl. 1, 30 November 2017 (2017-11-30), JP , pages S47, XP009531868, ISSN: 0009-918X
NISHINA, KAZUTAKA ET AL.: "DNA/RNA heteroduplex oligonucleotide for highly efficient gene silencing", NATURE COMMUNICATIONS, vol. 6, no. 7969, 2015, XP055333717, DOI: 10.1038/ncomms8969
MAJIMA, TAKAMASA ET AL.: "DNA/RNA Heteroduplex Oligonucleotide Has a High Efficacy for Gene Slicing", THE MEDICAL FRONTLINE, vol. 73, no. 6, 30 November 2017 (2017-11-30), JP , pages 781 - 786, XP009531801, ISSN: 0370-8241
NAGATA, TETSUYA: "Gene therapy with therapeutic oligonucleotide", CLINICAL NEUROLOGY, vol. 59, no. S13, XP009531797
"Principle of Myology", 1995, NANKODO CO., LTD., pages: 469 - 550
"GenBank", Database accession no. NC_000086.7
See also references of EP 3954395A4
NAGATA, TETSUYA: "HT-02-3 Recent progress of DNA/RNA heteroduplex oligonucleotide", CLINICAL NEUROLOGY, vol. 58, no. Suppl. 1, 30 November 2017 (2017-11-30), JP , pages S47, XP009531868, ISSN: 0009-918X
NISHINA, KAZUTAKA ET AL.: "DNA/RNA heteroduplex oligonucleotide for highly efficient gene silencing", NATURE COMMUNICATIONS, vol. 6, no. 7969, 2015, XP055333717, DOI: 10.1038/ncomms8969
MAJIMA, TAKAMASA ET AL.: "DNA/RNA Heteroduplex Oligonucleotide Has a High Efficacy for Gene Slicing", THE MEDICAL FRONTLINE, vol. 73, no. 6, 30 November 2017 (2017-11-30), JP , pages 781 - 786, XP009531801, ISSN: 0370-8241
NAGATA, TETSUYA: "Gene therapy with therapeutic oligonucleotide", CLINICAL NEUROLOGY, vol. 59, no. S13, XP009531797
"Principle of Myology", 1995, NANKODO CO., LTD., pages: 469 - 550
"GenBank", Database accession no. NC_000086.7
See also references of EP 3954395A4
Attorney, Agent or Firm:
HIRAKI & ASSOCIATES (JP)
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