Title:
COMPOSITION AND METHOD FOR TREATING LEBER'S HEREDITARY OPTIC NEUROPATHY CAUSED BY ND4 MUTATION
Document Type and Number:
WIPO Patent Application WO/2023/011632
Kind Code:
A1
Abstract:
Provided is a recombinant nucleic acid, comprising: a mitochondrial targeting sequence, a mitochondrial protein coding sequence, a 3'UTR nucleic acid sequence, and a polyadenylate tail sequence, wherein the mitochondrial protein coding sequence encodes a polypeptide comprising a mitochondrial protein. Further provided are a pharmaceutical composition comprising the recombinant nucleic acid and a method for treating Leber's hereditary optic neuropathy (LHON) by using the pharmaceutical composition.
Inventors:
LI BIN (CN)
Application Number:
PCT/CN2022/110545
Publication Date:
February 09, 2023
Filing Date:
August 05, 2022
Export Citation:
Assignee:
WUHAN NEUROPHTH BIOTECHNOLOGY LTD COMPANY (CN)
International Classes:
C12N15/11; A61K48/00; A61P27/02; C12N15/864
Domestic Patent References:
| WO2020001657A1 | 2020-01-02 |
Foreign References:
| CN112584874A | 2021-03-30 | |||
| CN111068071A | 2020-04-28 | |||
| CN110876269A | 2020-03-10 | |||
| CN102634527A | 2012-08-15 | |||
| US6207392B1 | 2001-03-27 | |||
| US4897268A | 1990-01-30 | |||
| US5075109A | 1991-12-24 | |||
| US5928647A | 1999-07-27 | |||
| US5811128A | 1998-09-22 | |||
| US5820883A | 1998-10-13 | |||
| US5853763A | 1998-12-29 | |||
| US5814344A | 1998-09-29 | |||
| US5942252A | 1999-08-24 | |||
| US4789734A | 1988-12-06 | |||
| US4906474A | 1990-03-06 | |||
| US4925673A | 1990-05-15 | |||
| US3625214A | 1971-12-07 |
Other References:
DU HAO, WANG GUI-PAN, PEI HAN, LIU RONG, HU WEI-KUN, LI TAO, LI BIN: "Constructing and detecting of rAAV2 /2-ND4 in gene therapy of leber hereditary optic neuropathy", RECENT ADVANCES IN OPHTHALMOLOGY, vol. 32, no. 3, 5 March 2012 (2012-03-05), pages 201 - 203, XP093031790, ISSN: 1003-5141, DOI: 10.13389/j.cnki.rao.2012.03.004
CHOI ET AL., CURR GENE THER, vol. 5, no. 3, 2005, pages 299 - 310
WU ET AL., MOLECULAR THERAPY, vol. 14, no. 3, 2006, pages 316 - 327
MANCUSO ET AL., NATURE, vol. 461, 2009, pages 784 - 7
LAUGHLIN ET AL., PNAS, vol. 76, 1979, pages 5567 - 5571
COURANARDI, VIROLOGY JOURNAL, vol. 4, 2007, pages 99
CRONIN ET AL., ARVO ABSTRACT: D1048, 2008
DEVEREUX ET AL., NUCLEIC ACIDS RESEARCH, vol. 12, 1984, pages 387 - 395
ALTSCHUL S. F, J MOL EVOL, vol. 36, 1993, pages 290 - 300
ALTSCHUL, S, F ET AL., J MOL BIOL, vol. 215, 1990, pages 403 - 10
ALLOCCA, J VIOL, vol. 81, 2007, pages 11372 - 80
BAINBRIDGE, N ENG J MED, 2008
GUY, IN OPHTHALMOLOGY, vol. 124, 2017, pages 1621 - 1634
VIGNAL, OPHTHALMOLOGY, vol. 6, 2018, pages 945 - 947
AUSUBEL, F ET AL.: "Current Protocols in Molecular Biology", 1998, JOHN WILEY & SONS
KRAUS, MAARONSON, S., METHODS ENZYMOL., vol. 200, 1991, pages 546 - 556
KARLIN, SALTSCHUL, S., PROC. NATL. ACAD. SCI. USA, vol. 90, 1993, pages 5873 - 5877
ALTSCHUL, S ET AL., NUCLEIC ACIDS RES., vol. 25, 1997, pages 3389 - 3402
NIELSEN, P ET AL., SCIENCE, vol. 254, 1991, pages 1497 - 1500
"Principles of Fluorescence Spectroscopy", July 1999, LENUM PUB CORP
ANSEL ET AL.: "Pharmaceutical Dosage Forms and Drug Delivery Systems", 1999, LIPPENCOTT, WILLIAMS, & WILKINS
G. GREGORIADIS: "Drug Carriers in Biology and Medicine", 1979, ACADEMIC PRESS, article "Liposomes", pages: 2
JEIN, TIPS, vol. 19, pages 155 - 157
BANGHAM ET AL., J. MOL. BIOL., vol. 23, 1965, pages 238 - 252
SZOKA ET AL., PROC. NATL ACAD. SCI. USA, vol. 75, 1978, pages 4194 - 4198
"Remington's Pharmaceutical Sciences", MACK PUBLISHING
CHOI ET AL., CURR GENE THER, vol. 5, no. 3, 2005, pages 299 - 310
WU ET AL., MOLECULAR THERAPY, vol. 14, no. 3, 2006, pages 316 - 327
MANCUSO ET AL., NATURE, vol. 461, 2009, pages 784 - 7
LAUGHLIN ET AL., PNAS, vol. 76, 1979, pages 5567 - 5571
COURANARDI, VIROLOGY JOURNAL, vol. 4, 2007, pages 99
CRONIN ET AL., ARVO ABSTRACT: D1048, 2008
DEVEREUX ET AL., NUCLEIC ACIDS RESEARCH, vol. 12, 1984, pages 387 - 395
ALTSCHUL S. F, J MOL EVOL, vol. 36, 1993, pages 290 - 300
ALTSCHUL, S, F ET AL., J MOL BIOL, vol. 215, 1990, pages 403 - 10
ALLOCCA, J VIOL, vol. 81, 2007, pages 11372 - 80
BAINBRIDGE, N ENG J MED, 2008
GUY, IN OPHTHALMOLOGY, vol. 124, 2017, pages 1621 - 1634
VIGNAL, OPHTHALMOLOGY, vol. 6, 2018, pages 945 - 947
AUSUBEL, F ET AL.: "Current Protocols in Molecular Biology", 1998, JOHN WILEY & SONS
KRAUS, MAARONSON, S., METHODS ENZYMOL., vol. 200, 1991, pages 546 - 556
KARLIN, SALTSCHUL, S., PROC. NATL. ACAD. SCI. USA, vol. 90, 1993, pages 5873 - 5877
ALTSCHUL, S ET AL., NUCLEIC ACIDS RES., vol. 25, 1997, pages 3389 - 3402
NIELSEN, P ET AL., SCIENCE, vol. 254, 1991, pages 1497 - 1500
"Principles of Fluorescence Spectroscopy", July 1999, LENUM PUB CORP
ANSEL ET AL.: "Pharmaceutical Dosage Forms and Drug Delivery Systems", 1999, LIPPENCOTT, WILLIAMS, & WILKINS
G. GREGORIADIS: "Drug Carriers in Biology and Medicine", 1979, ACADEMIC PRESS, article "Liposomes", pages: 2
JEIN, TIPS, vol. 19, pages 155 - 157
BANGHAM ET AL., J. MOL. BIOL., vol. 23, 1965, pages 238 - 252
SZOKA ET AL., PROC. NATL ACAD. SCI. USA, vol. 75, 1978, pages 4194 - 4198
"Remington's Pharmaceutical Sciences", MACK PUBLISHING
Attorney, Agent or Firm:
METIS IP (CHENGDU) LLC (CN)
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