Title:
PHARMACEUTICAL COMPOSITION UTILIZING BORNAVIRUS VECTOR
Document Type and Number:
WIPO Patent Application WO/2023/033050
Kind Code:
A1
Abstract:
Disclosed are: a bornavirus vector capable of binding to a mutant SOD1 protein and carrying a nucleic acid encoding an antibody or antibody fragment, in which the antibody or antibody fragment contains a heavy-chain variable region that may have substitution by 3 or less amino acid residues and comprises a heavy-chain CDR1 comprising the amino acid sequence of GFSLNTSGMG (SEQ ID NO:1), a heavy-chain CDR2 comprising the amino acid sequence of IWWDDDK (SEQ ID NO:2) and a heavy-chain CDR3 comprising the amino acid sequence of ARLGYAMDY (SEQ ID NO:3) and/or a light-chain variable region that may have substitution by 3 or less amino acid residues and comprises a light-chain CDR1 comprising the amino acid sequence of QNVGGTN (SEQ ID NO:4), a light-chain CDR2 comprising the amino acid sequence for SAS and a light-chain CDR3 comprising the amino acid sequence of QQYYIYPYT (SEQ ID NO:5); a recombinant virus which contains RNA encoded in the bornavirus vector; a cell infected by the recombinant virus; and a pharmaceutical composition containing the cell.
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Inventors:
TOMONAGA KEIZO (JP)
MAKINO AKIKO (JP)
SAKAI MADOKA (JP)
KOMORIZONO RYO (JP)
URUSHITANI MAKOTO (JP)
MORIMURA TOSHIFUMI (JP)
MINAMIYAMA SUMIO (JP)
MAKINO AKIKO (JP)
SAKAI MADOKA (JP)
KOMORIZONO RYO (JP)
URUSHITANI MAKOTO (JP)
MORIMURA TOSHIFUMI (JP)
MINAMIYAMA SUMIO (JP)
Application Number:
PCT/JP2022/032784
Publication Date:
March 09, 2023
Filing Date:
August 31, 2022
Export Citation:
Assignee:
UNIV KYOTO (JP)
NATIONAL UNIV CORPORATION SHIGA UNIV OF MEDICAL SCIENCE (JP)
NATIONAL UNIV CORPORATION SHIGA UNIV OF MEDICAL SCIENCE (JP)
International Classes:
C12N15/86; A61K35/76; A61P21/02; C12N5/10; C12N7/01; C07K16/40; C07K16/46; C12N15/13
Domestic Patent References:
| WO2010113647A1 | 2010-10-07 | |||
| WO2010113647A1 | 2010-10-07 |
Foreign References:
| JP2009507475A | 2009-02-26 | |||
| JP2014504156A | 2014-02-20 | |||
| JP2009529498A | 2009-08-20 | |||
| JP2018148865A | 2018-09-27 | |||
| JP2018148865A | 2018-09-27 |
Other References:
SUMIO MINAMIYAMA , MADOKA SAKAI , YUKO YAMAGUCHI , RYOTA HIKIAMI , HIDEKI WADA , MAKIKO HASHI , AKEMI SHODAI , AKIKO MAKINO, TAKAK: "AO-01-1 A single Intrathecal injection of OPC expressing scFv against misfolded SOD1 improved ALS model rats", CLINICAL NEUROLOGY, vol. 61, no. S1, 10 April 2021 (2021-04-10), JP , pages S221, XP009544134, ISSN: 0009-918X, DOI: 10.5692/clinicalneurol.61_supplement_S221
MINAMIYAMA, SUMIO; SAKAI, MADOKA; YAMAGUCHI, YUKO; HIKIAMI, RYOTA; TAMAKI, YOSHITAKA; SHODAI, AKEMI; MAKINO, AKIKO; MAKI, TAKAKUNI: "TST-13 A novel cell transplantation therapy for familial ALS using oligodendrocyte precursor cells expressing scFv specific for misfolded SOD1", AMYOTROPHIC LATERAL SCLEROSIS AND FRONTOTEMPORAL DEGENERATION, vol. 20, no. Suppl. 1, 1 January 2019 (2019-01-01), pages 224 - 225, XP009544538, ISSN: 2167-8421
SUMIO MINAMIYAMA, MADOKA SAKAI, YUKO YAMAGUCHI, RYOTA HIKIAMI, YOSHITAKA TAMAKI, AKEMI SHODAI, AKIKO MAKINO, TAKAKUNI MAKI, KEIZO : "AO-02-4: A novel cell transplantation therapy for ALS using OPCs expressing scFv recognizing misfolded SOD1", 60TH ANNUAL MEETING OF THE JAPANESE SOCIETY OF NEUROLOGY; MAY 22-25, 2019, vol. 59, no. S213, 1 January 2019 (2019-01-01) - 25 May 2019 (2019-05-25), JP, pages 340, XP009544096
PRIYANKA PATEL: "Adeno-associated Virus–mediated Delivery of a Recombinant Single-chain Antibody Against Misfolded Superoxide Dismutase for Treatment of Amyotrophic Lateral Sclerosis", MOLECULAR THERAPY, vol. 22, no. 3, 1 March 2014 (2014-03-01), US , pages 498 - 510, XP055510935, ISSN: 1525-0016, DOI: 10.1038/mt.2013.239
MICROBIOL IMMUNOL, vol. 61, no. 9, September 2017 (2017-09-01), pages 380 - 386
J VIROL., vol. 85, no. 23, December 2011 (2011-12-01), pages 12170 - 8
SCI REP., vol. 6, 18 May 2016 (2016-05-18), pages 26154
FUJINO K ET AL., MICROBIOL IMMUNOL., vol. 61, no. 9, September 2017 (2017-09-01), pages 380 - 386
MIYOSHI, H ET AL.: "Gene delivery to hematopoietic stem cells using lentiviral vectors", METHODS MOL. BIOL., vol. 246, 2004, pages 429 - 38, XP055877454
MINAMIYAMA, SUMIO; SAKAI, MADOKA; YAMAGUCHI, YUKO; HIKIAMI, RYOTA; TAMAKI, YOSHITAKA; SHODAI, AKEMI; MAKINO, AKIKO; MAKI, TAKAKUNI: "TST-13 A novel cell transplantation therapy for familial ALS using oligodendrocyte precursor cells expressing scFv specific for misfolded SOD1", AMYOTROPHIC LATERAL SCLEROSIS AND FRONTOTEMPORAL DEGENERATION, vol. 20, no. Suppl. 1, 1 January 2019 (2019-01-01), pages 224 - 225, XP009544538, ISSN: 2167-8421
SUMIO MINAMIYAMA, MADOKA SAKAI, YUKO YAMAGUCHI, RYOTA HIKIAMI, YOSHITAKA TAMAKI, AKEMI SHODAI, AKIKO MAKINO, TAKAKUNI MAKI, KEIZO : "AO-02-4: A novel cell transplantation therapy for ALS using OPCs expressing scFv recognizing misfolded SOD1", 60TH ANNUAL MEETING OF THE JAPANESE SOCIETY OF NEUROLOGY; MAY 22-25, 2019, vol. 59, no. S213, 1 January 2019 (2019-01-01) - 25 May 2019 (2019-05-25), JP, pages 340, XP009544096
PRIYANKA PATEL: "Adeno-associated Virus–mediated Delivery of a Recombinant Single-chain Antibody Against Misfolded Superoxide Dismutase for Treatment of Amyotrophic Lateral Sclerosis", MOLECULAR THERAPY, vol. 22, no. 3, 1 March 2014 (2014-03-01), US , pages 498 - 510, XP055510935, ISSN: 1525-0016, DOI: 10.1038/mt.2013.239
MICROBIOL IMMUNOL, vol. 61, no. 9, September 2017 (2017-09-01), pages 380 - 386
J VIROL., vol. 85, no. 23, December 2011 (2011-12-01), pages 12170 - 8
SCI REP., vol. 6, 18 May 2016 (2016-05-18), pages 26154
FUJINO K ET AL., MICROBIOL IMMUNOL., vol. 61, no. 9, September 2017 (2017-09-01), pages 380 - 386
MIYOSHI, H ET AL.: "Gene delivery to hematopoietic stem cells using lentiviral vectors", METHODS MOL. BIOL., vol. 246, 2004, pages 429 - 38, XP055877454
Attorney, Agent or Firm:
SAEGUSA & PARTNERS (JP)
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