Title:
BLOOD-BRAIN BARRIER PENETRATING APTAMER, AND USE THEREOF
Document Type and Number:
WIPO Patent Application WO/2022/005179
Kind Code:
A1
Abstract:
The present invention relates to an aptamer penetrating the blood-brain barrier (BBB) to enable receptor-mediated transcytosis, and a use thereof. The aptamer of the present invention selectively targets transferrin receptor proteins only, rapidly passes through BBB by means of receptor-mediated transcytosis, can effectively transport and deliver various therapeutic agents such as proteins, antibodies, nucleic acids and low molecular weight compounds through simple chemical bonds, exhibits interspecific cross-reactivity in transferrin receptors, and is easily chemically modified, and thus is applicable as a drug carrier for nervous system diseases, especially brain and central nervous system diseases.
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Inventors:
KIM YOUNDONG (KR)
LEE DA SOM (KR)
KIM SOYEON (KR)
LIM SO RYONG (KR)
SHIN EUISU (KR)
KANG NAYE (KR)
HAN DONGIL (KR)
LEE DA SOM (KR)
KIM SOYEON (KR)
LIM SO RYONG (KR)
SHIN EUISU (KR)
KANG NAYE (KR)
HAN DONGIL (KR)
Application Number:
PCT/KR2021/008212
Publication Date:
January 06, 2022
Filing Date:
June 29, 2021
Export Citation:
Assignee:
APTAMER SCIENCES INC (KR)
International Classes:
C12N15/115; A61K47/18
Domestic Patent References:
| WO2013163303A2 | 2013-10-31 |
Foreign References:
| KR20170107958A | 2017-09-26 | |||
| CN106191069A | 2016-12-07 |
Other References:
KEITH E MAIER, ROHIT K JANGRA, KEVIN R SHIEH, DAVID K CURETON, HUI XIAO, ERIK L SNAPP, SEAN P WHELAN, KARTIK CHANDRAN, MATTHEW LEV: "A New Transferrin Receptor Aptamer Inhibits New World Hemorrhagic Fever Mammarenavirus Entry", MOLECULAR THERAPY-NUCLEIC ACIDS, CELL PRESS, US, vol. 5, 1 January 2016 (2016-01-01), US , pages e321, XP055715282, ISSN: 2162-2531, DOI: 10.1038/mtna.2016.32
JOANNA MACDONALD, PATRICK HOUGHTON, DONGXI XIANG, WEI DUAN, SARAH SHIGDAR: "Truncation and Mutation of a Transferrin Receptor Aptamer Enhances Binding Affinity", NUCLEIC ACID THERAPEUTICS, MARY ANN LIEBERT, INC. PUBLISHERS, US, vol. 26, no. 6, 1 December 2016 (2016-12-01), US , pages 348 - 354, XP055490746, ISSN: 2159-3337, DOI: 10.1089/nat.2015.0585
PEARSON ET AL., PROC. NATL. ACAD. SCI. USA, vol. 85, 1988, pages 2444
NEEDLEMANWUNSCH, J. MOL. BIOL., vol. 48, 1970, pages 443 - 453
RICE ET AL.: "EMBOSS: The European Molecular Biology Open Software Suite", TRENDS GENET., vol. 16, 2000, pages 276 - 277, XP004200114, DOI: 10.1016/S0168-9525(00)02024-2
DEVEREUX, J. ET AL., NUCLEIC ACIDS RESEARCH, vol. 12, 1984, pages 387
ATSCHUL, S. F. ET AL., J MOLEC BIOL, vol. 215, 1990, pages 403
"Guide to Huge Computers", 1994, ACADEMIC PRESS
CARILLO ET AL., SIAM J APPLIED MATH, vol. 48, 1988, pages 1073
NEEDLEMAN ET AL., J MOL BIOL., vol. 48, 1970, pages 443
SMITHWATERMAN, ADV. APPL. MATH, vol. 2, 1981, pages 482
GRIBSKOV ET AL., NUCL. ACIDS RES., vol. 14, 1986, pages 6745
"Atlas Of Protein Sequence and Structure", 1979, NATIONAL BIOMEDICAL RESEARCH FOUNDATION, pages: 353 - 358
J. SAMBROOK ET AL.: "Molecular Cloning, A Laboratory Manual", 1989, COLD SPRING HARBOR LABORATORY PRESS
J. A. KIM ET AL., BIOMICROFLUIDICS, 2015
JOANNA MACDONALD, PATRICK HOUGHTON, DONGXI XIANG, WEI DUAN, SARAH SHIGDAR: "Truncation and Mutation of a Transferrin Receptor Aptamer Enhances Binding Affinity", NUCLEIC ACID THERAPEUTICS, MARY ANN LIEBERT, INC. PUBLISHERS, US, vol. 26, no. 6, 1 December 2016 (2016-12-01), US , pages 348 - 354, XP055490746, ISSN: 2159-3337, DOI: 10.1089/nat.2015.0585
PEARSON ET AL., PROC. NATL. ACAD. SCI. USA, vol. 85, 1988, pages 2444
NEEDLEMANWUNSCH, J. MOL. BIOL., vol. 48, 1970, pages 443 - 453
RICE ET AL.: "EMBOSS: The European Molecular Biology Open Software Suite", TRENDS GENET., vol. 16, 2000, pages 276 - 277, XP004200114, DOI: 10.1016/S0168-9525(00)02024-2
DEVEREUX, J. ET AL., NUCLEIC ACIDS RESEARCH, vol. 12, 1984, pages 387
ATSCHUL, S. F. ET AL., J MOLEC BIOL, vol. 215, 1990, pages 403
"Guide to Huge Computers", 1994, ACADEMIC PRESS
CARILLO ET AL., SIAM J APPLIED MATH, vol. 48, 1988, pages 1073
NEEDLEMAN ET AL., J MOL BIOL., vol. 48, 1970, pages 443
SMITHWATERMAN, ADV. APPL. MATH, vol. 2, 1981, pages 482
GRIBSKOV ET AL., NUCL. ACIDS RES., vol. 14, 1986, pages 6745
"Atlas Of Protein Sequence and Structure", 1979, NATIONAL BIOMEDICAL RESEARCH FOUNDATION, pages: 353 - 358
J. SAMBROOK ET AL.: "Molecular Cloning, A Laboratory Manual", 1989, COLD SPRING HARBOR LABORATORY PRESS
J. A. KIM ET AL., BIOMICROFLUIDICS, 2015
Attorney, Agent or Firm:
HANOL INTELLECTUAL PROPERTY AND LAW (KR)
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