Title:
COMPOUND, METHOD, AND PHARMACEUTICAL COMPOSITION FOR REGULATING PLP1 EXPRESSION
Document Type and Number:
WIPO Patent Application WO/2021/261538
Kind Code:
A1
Abstract:
The present invention pertains to: a modified oligonucleotide that is formed of 12-30 residues, that has at least eight consecutive nucleic acid base sequences complementary to an equal length portion at, from the 5'-end in the nucleic acid base sequence indicated in SEQ ID NO: 1 of the sequence listing, positions 9964-10286, positions 140007-14152, positions 4439-4470, positions 4569-4616, positions 9061-9164, positions 11926-12015, positions 12817-12874, positions 14839-14920, or positions 15088-15114, that has a total length nucleic acid base sequence being at least 85% complementation to an equal length portion of the nucleic acid base sequence indicated in SEQ ID NO: 1 of the sequence listing, and that inhibits expression of proteolipid protein 1 (PLP1); and a medicinal drug that contains the modified oligonucleotide and that is for treating, preventing, delaying, or improving PLP1-related diseases.
Inventors:
KUMAGAI SHINJI (JP)
KANAGAWA TAKAYUKI (JP)
SAWAMOTO HIROAKI (JP)
KANAGAWA TAKAYUKI (JP)
SAWAMOTO HIROAKI (JP)
Application Number:
PCT/JP2021/023883
Publication Date:
December 30, 2021
Filing Date:
June 24, 2021
Export Citation:
Assignee:
MITSUBISHI TANABE PHARMA CORP (JP)
International Classes:
C12N15/113; A61K31/7088; A61P25/00; A61P25/08; A61P27/02; A61P43/00; C12N15/12
Domestic Patent References:
| WO2019156115A1 | 2019-08-15 | |||
| WO2017047816A1 | 2017-03-23 | |||
| WO2020100826A1 | 2020-05-22 |
Foreign References:
| JP2016530882A | 2016-10-06 | |||
| JP2018511307A | 2018-04-26 | |||
| JP2020500537A | 2020-01-16 |
Other References:
ELITT MATTHEW S., BARBAR LILIANNE, SHICK H. ELIZABETH, POWERS BERIT E., MAENO-HIKICHI YUKA, MADHAVAN MAYUR, ALLAN KEVIN C., NAWASH: "Therapeutic suppression of proteolipid protein rescues Pelizaeus-Merzbacher Disease in mice", BIORXIV, 31 December 2018 (2018-12-31), pages 1 - 54, XP055893692, DOI: 10.1101/508192
Attorney, Agent or Firm:
IP FIRM SHUWA (JP)
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