Title:
ADENO-ASSOCIATED VIRUS VIRION FOR TREATING ORNITHINE TRANSCARBAMYLASE DEFICIENCY
Document Type and Number:
WIPO Patent Application WO/2022/224372
Kind Code:
A1
Abstract:
The present application provides a novel means for treating ornithine transcarbamylase deficiency. More specifically, the present application provides a modified adeno-associated virus vector which expresses ornithine transcarbamylase so that an attack from a neutralizing antibody in blood is reduced and a gene is efficiently introduced into a liver of a living body (for example, human). The present application provides, for example, a modified adeno-associated virus vector which expresses ornithine transcarbamylase, which contains a modified VP1 protein that contains, for example, an amino acid sequence obtained by substituting at least one of the 472th amino acid, the 587th amino acid, and the 706th amino acid in the amino acid sequence of the VP1 protein with another amino acid, and which does not cross-react with a neutralizing antibody against AAV2.
Inventors:
MURAMATSU SHIN-ICHI (JP)
TAKINO NAOMI (JP)
ITO MIKA (JP)
YAMAGATA TAKANORI (JP)
MURAMATSU KAZUHIRO (JP)
TAKINO NAOMI (JP)
ITO MIKA (JP)
YAMAGATA TAKANORI (JP)
MURAMATSU KAZUHIRO (JP)
Application Number:
PCT/JP2021/016141
Publication Date:
October 27, 2022
Filing Date:
April 21, 2021
Export Citation:
Assignee:
UNIV JICHI MEDICAL (JP)
GENE THERAPY RES INSTITUTION CO LTD (JP)
GENE THERAPY RES INSTITUTION CO LTD (JP)
International Classes:
C12N15/864; A61K35/76; A61K38/45; A61K48/00; A61P43/00
Domestic Patent References:
| WO2021009805A1 | 2021-01-21 | |||
| WO2008124724A1 | 2008-10-16 | |||
| WO2012057363A1 | 2012-05-03 | |||
| WO2018131551A1 | 2018-07-19 | |||
| WO2003018821A2 | 2003-03-06 | |||
| WO2003053476A1 | 2003-07-03 | |||
| WO2007001010A1 | 2007-01-04 | |||
| WO2006073073A1 | 2006-07-13 |
Foreign References:
| JP2019216667A | 2019-12-26 | |||
| JP2017512466A | 2017-05-25 | |||
| JP2021097617A | 2021-07-01 | |||
| JP2002516062A | 2002-06-04 | |||
| US20020086356A1 | 2002-07-04 |
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DUNBER CE ET AL., SCIENCE, vol. 359, 2018, pages 4672
HASTIE ESAMULSKI RJ, HUM GENE THER, vol. 26, 2015, pages 257 - 265
OHMORI T ET AL., SCI REP, vol. 7, 2017, pages 4159
RANGARAJAN S ET AL., N ENGL J MED, vol. 377, 2017, pages 2519 - 2530
WANG L. ET AL., GENE THERAPY, vol. 19, 2012, pages 404 - 410
MIMURO J ET AL., J MED VIROL, vol. 86, 2014, pages 1990 - 1997
LING C ET AL., J INTEGR MED, vol. 13, 2015, pages 341 - 346
MELIANI A ET AL., HUM GENE THER METHODS, vol. 26, 2015, pages 45 - 53
YAKUGAKU ZASSHI, 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
HU, C ET AL., MOLECULAR THERAPY, vol. 22, no. 10, October 2014 (2014-10-01), pages 1792 - 1802
"GenBank", Database accession no. NC_006261.1
LI C ET AL., GENE THER, vol. 19, 2012, pages 288 - 294
FOUST K.D. ET AL., NAT BIOTECHNOL., vol. 27, no. 1, January 2009 (2009-01-01), pages 1987 - 2018
KARLINALTSCHUL, PROC. NATL. ACAD. SCI. USA, vol. 87, 1990, pages 2264 - 2268
PROC. NATL. ACAD. SCI USA, vol. 90, 1993, pages 5873
ALTSCHUL SF ET AL., J MOL BIOL, vol. 215, 1990, pages 403
CHIRMULE N ET AL., GENE THER, vol. 6, 1999, pages 1574 - 1583
MOSKALENKO, M ET AL., J VIROL, vol. 74, 2000, pages 1761 - 1766
FU H ET AL., HUM GENE THER CLIN DEV, vol. 28, 2017, pages 187 - 196
LING CJ ET AL., INTEGR MED, vol. 13, 2015, pages 341 - 346
ITO ET AL., ANN CLIN BIOCHEM, vol. 46, 2009, pages 508 - 510
MELIANI A ET AL., HUM GENE THER METHOS, vol. 26, 2015, pages 45 - 53
SUMMERFOLD ET AL., MOL THER, vol. 24, pages 2016
PILLAY ET AL., NATURE, vol. 530, 2016, pages 108 - 112
RAN FA ET AL., NATURE, vol. 520, no. 7546, 2015, pages 186 - 91
PIERSON ET AL., J. BIOL. CHEM., vol. 252, 1977, pages 6464 - 6469
NATURE GENETICS, vol. 24, no. 2, February 2000 (2000-02-01), pages 180 - 183
"GenBank Accession", Database accession no. #NC_001401.2
LISOWSKI L ET AL., NATURE, vol. 506, no. 7488, 20 February 2014 (2014-02-20), pages 382 - 386
MELIAN A ET AL., HUMAN GENE THERAPY METHODS, vol. 26, 2015, pages 45 - 52
PEROCHEAU, D.P ET AL., HUMAN GENE THERAPY, vol. 30, no. 1, 2018, pages 79 - 87
SUGAHARA, G ET AL., J INHERIT METAB DIS, vol. 2020, pages 1 - 11
Attorney, Agent or Firm:
KOBAYASHI Hiroshi et al. (JP)
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