Title:
NOVEL ADENO-ASSOCIATED VIRUS VIRION FOR TREATING TAY-SACHS DISEASE AND SANDHOFF DISEASE
Document Type and Number:
WIPO Patent Application WO/2019/146745
Kind Code:
A1
Abstract:
In a conventional method for treating Sandhoff disease and Tay-Sachs disease by administering to a patient a modified β-subunit in the form of a protein, the administration needs to be performed at a high frequency.
The present invention pertains to a recombinant adeno-associated virus virion comprising a capsomer which contains a protein capable of forming a virus virion and a polynucleotide which is packaged within the capsomer and comprises a promoter sequence and a base sequence operably linked to the promoter sequence, said base sequence encoding a first amino acid sequence, which is derived from the amino acid sequence of the 55th-556th amino acid sequence of the β-subunit of wild type human β-hexosaminidase (SEQ ID NO: 28) by substitution of the 312nd-318th amino acids into glycine, serine, glutamic acid, proline, serine, glycine and threonine in this order, and a second amino acid sequence which is the amino acid sequence of a signal peptide linked to the N-terminal side of the first amino acid sequence.
Inventors:
ITOH KOHJI (JP)
TSUJI DAISUKE (JP)
MURAMATSU SHINICHI (JP)
ASAI KATSUHITO (JP)
TSUJI DAISUKE (JP)
MURAMATSU SHINICHI (JP)
ASAI KATSUHITO (JP)
Application Number:
PCT/JP2019/002428
Publication Date:
August 01, 2019
Filing Date:
January 25, 2019
Export Citation:
Assignee:
UNIV TOKUSHIMA (JP)
UNIV JICHI MEDICAL (JP)
GENE THERAPY RES INSTITUTION CO LTD (JP)
UNIV JICHI MEDICAL (JP)
GENE THERAPY RES INSTITUTION CO LTD (JP)
International Classes:
C12N15/35; A61K35/76; A61P25/28; C12N7/01
Domestic Patent References:
| WO2014061735A1 | 2014-04-24 | |||
| WO2012057363A1 | 2012-05-03 | |||
| WO2010082622A1 | 2010-07-22 | |||
| WO2014061735A1 | 2014-04-24 | |||
| WO2012057363A1 | 2012-05-03 | |||
| WO2003018821A2 | 2003-03-06 | |||
| WO2003053476A1 | 2003-07-03 | |||
| WO2007001010A1 | 2007-01-04 |
Foreign References:
| JP2015523998A | 2015-08-20 | |||
| JP2016526045A | 2016-09-01 | |||
| US20030219414A1 | 2003-11-27 | |||
| JP2002369692A | 2002-12-24 | |||
| JP2018011705A | 2018-01-25 |
Other References:
TSUJI, DAISUKE: "Gene therapy by intracerebral AAV injection for inborn errors of metabolism", FARUMASHIA, vol. 43, no. 6, 2007, pages 571 - 572, XP009522033, DOI: 10.14894/faruawpsj.43.6_571
TSUJI D ET AL., ANN. NEUROL., vol. 69, no. 4, April 2011 (2011-04-01), pages 691 - 701
MATSUOKA K ET AL., MOL. THER., vol. 19, no. 6, June 2011 (2011-06-01), pages 1017 - 1024
"Molecular Cloning, A Laboratory Manual", 1989, COLD SPRING HARBOR LABORATORY PRESS
"Current Protocols in Molecular Biology", 1987, JOHN WILEY & SONS, article "Current Protocols in Molecular Biology"
JOURNAL OF THE PHARMACEUTICAL SOCIETY OF JAPAN, vol. 126, no. 11, pages 1021 - 1028
HUMAN GENE THERAPY, vol. 13, 2002, pages 345 - 354
NEURONAL DEVELOPMENT, vol. 45, 2001, pages 92 - 103
EXPERIMENTAL MEDICINE, vol. 20, 2002, pages 1296 - 1300
HUM. GENE THER., vol. 16, 2005, pages 541 - 550
GAO, G ET AL., CURR. GENE THER., vol. 5, 2005, pages 285 - 297
XIN, K-Q ET AL., J. VIROL., vol. 80, 2006, pages 11899 - 910
HELLSTROEM, M ET AL., GENE THER., vol. 16, 2009, pages 521 - 32
TAYMANS ET AL., HUM. GENE THER., vol. 18, 2007, pages 195 - 206
KAPLITT ET AL., LANCET, vol. 369, 2007, pages 2097 - 2105
MARKS ET AL., LANCET NEUROL., vol. 7, 2008, pages 400 - 408
CHRISTINE ET AL., NEUROLOGY, vol. 73, 2009, pages 1662 - 1669
MURAMATSU ET AL., MOL. THER., vol. 18, 2010, pages 1731 - 1735
J. SAMBROOK ET AL.: "Molecular Cloning", 2001, COLD SPRING HARBOR LAB. PRESS
CARLINALTUR, PROC. NATL. ACAD. SCI., U.S.A., vol. 87, 1990, pages 2264 - 2268
PROC. NATL. ACAD. SCI., U.S.A., vol. 90, 1993, pages 5873
ALTSCHUL SF ET AL., J. MOL. BIOL., vol. 215, 1990, pages 403
FOUST KD ET AL., NAT. BIOTECHNOL., vol. 27, no. 1, January 2009 (2009-01-01), pages 59 - 65
HANDA ET AL., J. GEN. VIROL., vol. 81, 2000, pages 2077 - 2084
MURAMATSU ET AL., VIROLOGY, vol. 221, 1996, pages 208 - 217
KONDO T. ET AL., CELL STEM CELL, vol. 12, no. 4, 2013, pages 487 - 496
LI ET AL., MOL. THER., vol. 13, 2006, pages 160 - 166
See also references of EP 3744846A4
TSUJI D ET AL., ANN. NEUROL., vol. 69, no. 4, April 2011 (2011-04-01), pages 691 - 701
MATSUOKA K ET AL., MOL. THER., vol. 19, no. 6, June 2011 (2011-06-01), pages 1017 - 1024
"Molecular Cloning, A Laboratory Manual", 1989, COLD SPRING HARBOR LABORATORY PRESS
"Current Protocols in Molecular Biology", 1987, JOHN WILEY & SONS, article "Current Protocols in Molecular Biology"
JOURNAL OF THE PHARMACEUTICAL SOCIETY OF JAPAN, vol. 126, no. 11, pages 1021 - 1028
HUMAN GENE THERAPY, vol. 13, 2002, pages 345 - 354
NEURONAL DEVELOPMENT, vol. 45, 2001, pages 92 - 103
EXPERIMENTAL MEDICINE, vol. 20, 2002, pages 1296 - 1300
HUM. GENE THER., vol. 16, 2005, pages 541 - 550
GAO, G ET AL., CURR. GENE THER., vol. 5, 2005, pages 285 - 297
XIN, K-Q ET AL., J. VIROL., vol. 80, 2006, pages 11899 - 910
HELLSTROEM, M ET AL., GENE THER., vol. 16, 2009, pages 521 - 32
TAYMANS ET AL., HUM. GENE THER., vol. 18, 2007, pages 195 - 206
KAPLITT ET AL., LANCET, vol. 369, 2007, pages 2097 - 2105
MARKS ET AL., LANCET NEUROL., vol. 7, 2008, pages 400 - 408
CHRISTINE ET AL., NEUROLOGY, vol. 73, 2009, pages 1662 - 1669
MURAMATSU ET AL., MOL. THER., vol. 18, 2010, pages 1731 - 1735
J. SAMBROOK ET AL.: "Molecular Cloning", 2001, COLD SPRING HARBOR LAB. PRESS
CARLINALTUR, PROC. NATL. ACAD. SCI., U.S.A., vol. 87, 1990, pages 2264 - 2268
PROC. NATL. ACAD. SCI., U.S.A., vol. 90, 1993, pages 5873
ALTSCHUL SF ET AL., J. MOL. BIOL., vol. 215, 1990, pages 403
FOUST KD ET AL., NAT. BIOTECHNOL., vol. 27, no. 1, January 2009 (2009-01-01), pages 59 - 65
HANDA ET AL., J. GEN. VIROL., vol. 81, 2000, pages 2077 - 2084
MURAMATSU ET AL., VIROLOGY, vol. 221, 1996, pages 208 - 217
KONDO T. ET AL., CELL STEM CELL, vol. 12, no. 4, 2013, pages 487 - 496
LI ET AL., MOL. THER., vol. 13, 2006, pages 160 - 166
See also references of EP 3744846A4
Attorney, Agent or Firm:
HIRAKI & ASSOCIATES (JP)
Download PDF:
Previous Patent: MOTOR CONTROL DEVICE, INTEGRATED VALVE DEVICE, AND HEAT EXCHANGER
Next Patent: ESTIMATION DEVICE AND ESTIMATION METHOD
Next Patent: ESTIMATION DEVICE AND ESTIMATION METHOD