Title:
NUCLEIC ACID COMPLEX
Document Type and Number:
WIPO Patent Application WO/2021/054370
Kind Code:
A1
Abstract:
The present invention addresses the problem of providing a nucleic acid agent, which is delivered efficiently to a nervous system such as the central nervous system where drug delivery might be impeded by BBB, etc. and which exerts an antisense effect on a target transcript at the delivery site, and a composition comprising the nucleic acid agent. In one embodiment, the present invention provides a double-stranded nucleic acid complex obtained by annealing a first nucleic acid strand, which hybridizes with a part of a target transcript and has an antisense effect on the target transcript, and a second nucleic acid strand which contains a base sequence complementary to the first nucleic acid strand and binds to a C22-35 alkyl group optionally substituted by a hydroxy group or an analog thereof.
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Inventors:
YOKOTA TAKANORI (JP)
NAGATA TETSUYA (JP)
FURUKAWA HIDEKI (JP)
YOGO TAKATOSHI (JP)
NAKAGAWA YASUO (JP)
SASAKI SHIGEKAZU (JP)
TOKUNOH RYOSUKE (JP)
SEKI TOMOHIRO (JP)
HIDAKA KOSUKE (JP)
KIKUCHI FUMIAKI (JP)
KUBO OSAMU (JP)
KASAHARA TAKAHITO (JP)
KOJIMA TAKUTO (JP)
WANG JUNSI (JP)
TOKUNAGA NORIHITO (JP)
NAGATA TETSUYA (JP)
FURUKAWA HIDEKI (JP)
YOGO TAKATOSHI (JP)
NAKAGAWA YASUO (JP)
SASAKI SHIGEKAZU (JP)
TOKUNOH RYOSUKE (JP)
SEKI TOMOHIRO (JP)
HIDAKA KOSUKE (JP)
KIKUCHI FUMIAKI (JP)
KUBO OSAMU (JP)
KASAHARA TAKAHITO (JP)
KOJIMA TAKUTO (JP)
WANG JUNSI (JP)
TOKUNAGA NORIHITO (JP)
Application Number:
PCT/JP2020/035117
Publication Date:
March 25, 2021
Filing Date:
September 16, 2020
Export Citation:
Assignee:
UNIV NAT CORP TOKYO MEDICAL & DENTAL (JP)
TAKEDA PHARMACEUTICALS CO (JP)
TAKEDA PHARMACEUTICALS CO (JP)
International Classes:
A61K48/00; A61K31/713; A61K47/54; A61P25/00; A61P43/00; C12N5/079; C12N5/10; C12N15/113
Domestic Patent References:
| WO2018056442A1 | 2018-03-29 | |||
| WO2019182109A1 | 2019-09-26 | |||
| WO2017053999A1 | 2017-03-30 | |||
| WO2015168172A1 | 2015-11-05 | |||
| WO2016081600A1 | 2016-05-26 | |||
| WO2007143315A2 | 2007-12-13 | |||
| WO2008043753A2 | 2008-04-17 | |||
| WO2008049085A1 | 2008-04-24 |
Foreign References:
| JP2018528783A | 2018-10-04 | |||
| JP2019168930A | 2019-10-03 | |||
| US5955599A | 1999-09-21 | |||
| US5264423A | 1993-11-23 | |||
| US5286717A | 1994-02-15 |
Other References:
NAOKI IWAMOTO ET AL., ANGEW. CHEM. INT. ED. ENGL., vol. 48, no. 3, 2009, pages 496 - 9
NATSUHISA OKA ET AL., J. AM. CHEM. SOC., vol. 125, 2003, pages 8307 - 8317
NATSUHISA OKA ET AL., J. AM. CHEM. SOC., vol. 130, 2008, pages 16031 - 16037
YOHEI NUKAGA ET AL., J. ORG. CHEM., vol. 81, 2016, pages 2753 - 2762
YOHEI NUKAGA ET AL., J. ORG. CHEM., vol. 77, 2012, pages 7913 - 7922
NAOKI IWAMOTO ET AL., NAT. BIOTECHNOL, vol. 35, no. 9, 2017, pages 845 - 851
ANASTASIA KHVOROVA ET AL., NAT. BIOTECHNOL., vol. 35, no. 3, 2017, pages 238 - 248
"GenBank", Database accession no. 002847
See also references of EP 4032551A4
NATSUHISA OKA ET AL., J. AM. CHEM. SOC., vol. 125, 2003, pages 8307 - 8317
NATSUHISA OKA ET AL., J. AM. CHEM. SOC., vol. 130, 2008, pages 16031 - 16037
YOHEI NUKAGA ET AL., J. ORG. CHEM., vol. 81, 2016, pages 2753 - 2762
YOHEI NUKAGA ET AL., J. ORG. CHEM., vol. 77, 2012, pages 7913 - 7922
NAOKI IWAMOTO ET AL., NAT. BIOTECHNOL, vol. 35, no. 9, 2017, pages 845 - 851
ANASTASIA KHVOROVA ET AL., NAT. BIOTECHNOL., vol. 35, no. 3, 2017, pages 238 - 248
"GenBank", Database accession no. 002847
See also references of EP 4032551A4
Attorney, Agent or Firm:
HIRAKI & ASSOCIATES (JP)
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