Title:
ANTI-TRANSTHYRETIN HUMAN ANTIBODY
Document Type and Number:
WIPO Patent Application WO/2015/115332
Kind Code:
A1
Abstract:
A human antibody including an H chain complementarity determining region comprising an amino acid sequence shown by SEQ ID NOS: 1-3 and an L chain complementarity determining region comprising an amino acid sequence shown by SEQ ID NOS: 4-6. This human antibody is suited to use in humans, and exhibits activity for binding specifically to transthyretin (TTR) which has undergone a structural change and activity for inhibiting fibrosis of TTR.
Inventors:
TORIKAI MASAHARU (JP)
HOSOI AKIHIKO (JP)
TAKEO TOMOYO (JP)
UENO MASAYO (JP)
SOEJIMA KENJI (JP)
NAKASHIMA TOSHIHIRO (JP)
ANDO YUKIO (JP)
JONO HIROFUMI (JP)
SU YU (JP)
MIZUGUCHI MINEYUKI (JP)
HOSOI AKIHIKO (JP)
TAKEO TOMOYO (JP)
UENO MASAYO (JP)
SOEJIMA KENJI (JP)
NAKASHIMA TOSHIHIRO (JP)
ANDO YUKIO (JP)
JONO HIROFUMI (JP)
SU YU (JP)
MIZUGUCHI MINEYUKI (JP)
Application Number:
PCT/JP2015/051860
Publication Date:
August 06, 2015
Filing Date:
January 23, 2015
Export Citation:
Assignee:
CHEMO SERO THERAPEUT RES INST (JP)
International Classes:
A61K39/395; C12N15/09; A61P25/28; C07K16/18; C12M1/34; C12N15/02; C12P21/08; G01N33/53
Domestic Patent References:
| WO2010030203A1 | 2010-03-18 |
Foreign References:
| JP2010195710A | 2010-09-09 | |||
| JP2010195710A | 2010-09-09 |
Other References:
AKIHIKO HOSOI: "Kazokusei Amyloid Polyneuropathy no Kotai Ryoho", REIMEI, vol. 21, 2012, pages 47 - 51, XP008184732
MITSUHARU UEDA ET AL.: "Saishin no Shinkei Nanbyo Chiryo to Kongo no Tenbo 6. Amyloidosis", MEDICINE AND DRUG JOURNAL, vol. 48, no. 5, 2012, pages 1307 - 1314, XP008184689
YUKIO ANDO: "Kazokusei Amyloid Polyneuropathy (FAP) no Kan Ishoku to Aratana Chiryo no Kaihatsu", ANNUAL REVIEW SHINKEI, vol. 2011, 2011, pages 310 - 317, XP008184906
HIROYOSHI OBA: "Jinko Kotai Library ni yoru Kotai Iyaku Kaihatsu", KOTAI IYAKU NO SAIZENSEN, 2007, pages 157 - 169, XP008184908
GLENNER, G.G.: "Amyloid deposits and amyloidosis: the beta-fibrilloses (second of two parts).", N ENGL J MED, vol. 302, 1980, pages 1333 - 1343
ANDO, Y.; JONO, H.: "Rinsho yori", vol. 56, 2008, article "Pathogenesis and therapy for transthyretin related amyloidosis", pages: 114 - 120
YOSHIKI SEKIJIMA: "Molecular mechanism of TTR amyloid deposition and its control", IGAKU-NO-AYUMI, vol. 229, 2009, pages 349 - 356
YUKO MOTOZAKI; SHOJI YAMADA: "Molecular epidemiology of familial amyloidotic polyneuropathy (FAP", IGAKU-NO-AYUMI, vol. 229, 2009, pages 357 - 362
HOU, X.; AGUILAR, M.I.; SMALL, D.H.: "Transthyretin and familial amyloidotic polyneuropathy. Recent progress in understanding the molecular mechanism . of neurodegeneration.", FEBS J, vol. 274, 2007, pages 1637 - 1650
ARAKI, S.; ANDO, Y.: "Transthyretin-related familial amyloidotic polyneuropathy - Progress in Kumamoto", PROC JPN ACAD SER B PHYS BIOL SCI, vol. 86, 2010, pages 694 - 706
EPISKOPOU, V.; MAEDA, S.; NISHIGUCHI, S.; SHIMADA, K.; GAITANARIS, .G.A.; GOTTESMAN, M.E.; ROBERTSON, E.J.: "Disruption of the transthyretin gene results in mice with depressed levels of plasma retinol and thyroid hormone.", PROC NATL ACAD SCI U S A, vol. 90, 1993, pages 2375 - 2379, XP001093658, DOI: doi:10.1073/pnas.90.6.2375
YUKIO ANDO: "Liver transplantation and other treatments for familial amyloidotic polyneuropathy (FAP", IGAKU-NO-AYUMI, vol. 229, 2009, pages 363 - 368
SAID, G.; GRIPPON, S.; KIRKPATRICK, P.: "Tafamidis", NAT REV DRUG DISCOV, vol. 11, 2012, pages 185 - 186
GOLDSTEINS, G.; PERSSON, H.; ANDERSSON, K.; OLOFSSON, A.; DACKLIN, I.; EDVINSSON, A.; SARAIVA, M.J.; LUNDGREN, E.: "Exposure of cryptic epitopes on transthyretin only in amyloid and in amyloidogenic mutants.", PROC NATL ACAD SCI U S A, vol. 96, 1999, pages 3108 - 3113, XP002987251, DOI: doi:10.1073/pnas.96.6.3108
TERAZAKI, H.; ANDO, Y.; FERNANDES, R.; YAMAMURA, K.; MAEDA, S.; & SARAIVA, M.J.: "Immunization in familial amyloidotic polyneuropathy: counteracting deposition by immunization with a Y78F TTR mutant.", LAB INVEST, vol. 86, 2006, pages 23 - 31, XP002531715, DOI: doi:10.1038/LABINVEST.3700365
BERGSTROEM, J.; ENGSTROEM, U.; YAMASHITA, T.; ANDO, Y.; WESTERMARK, P.: "Surface exposed epitopes and structural heterogeneity of in vivo formed transthyretin amyloid fibrils.", BIOCHEM BIOPHYS RES CORAMUN, vol. 348, 2006, pages 532 - 539
MATSUBARA, K.; MIZUGUCHI, M.; KAWANO, K.: "Expression of a synthetic gene encoding human transthyretin in Escherichia coli.", PROTEIN EXPR PURIF, vol. 30, 2003, pages 55 - 61, XP004432804, DOI: doi:10.1016/S1046-5928(03)00069-X
UEDA, M.; ANDO, Y.; HAKAMATA, Y.; NAKAMURA, M.; YAMASHITA, T.; OBAYASHI, K.; HIMENO, S.; INOUE, S.; SATO, Y.; KANEKO, T.: "A transgenic rat with the human ATTR V30M: a novel tool for analyses of ATTR metabolisms.", BIOCHEM BIOPHYS RES COMMUN, vol. 352, 2007, pages 299 - 304, XP029000099, DOI: doi:10.1016/j.bbrc.2006.11.045
MATSUBARA, K.; MIZUGUCHI, M.; IGARASHI, K.; SHINOHARA, Y.; TAKEUCHI, M.; MATSUURA, A.; SAITOH, T.; MORI, Y.; SHINODA, H.; KAWANO,: "Dimeric transthyretin variant assembles into spherical neurotoxins.", BIOCHEMISTRY, vol. 44, 2005, pages 3280 - 3288
SENJU, S.; HARUTA, M.; MATSUMURA, K.; MATSUNAGA, Y.; FUKUSHIMA, S.; IKEDA, T.; TAKAMATSU, K.; IRIE, A.; NISHIMURA, Y.: "Generation of dendritic cells and macrophages from human induced pluripotent stem cells aiming at cell therapy.", GENE THER, vol. 18, 2011, pages 874 - 883, XP055027827, DOI: doi:10.1038/gt.2011.22
"Phage Display of Peptides and Proteins: A Laboratory Manual"
"Antibody Engineering: A PRACTICAL APPROACH"
"ANTIBODY ENGINEERING"
See also references of EP 3101132A4
MITSUHARU UEDA ET AL.: "Saishin no Shinkei Nanbyo Chiryo to Kongo no Tenbo 6. Amyloidosis", MEDICINE AND DRUG JOURNAL, vol. 48, no. 5, 2012, pages 1307 - 1314, XP008184689
YUKIO ANDO: "Kazokusei Amyloid Polyneuropathy (FAP) no Kan Ishoku to Aratana Chiryo no Kaihatsu", ANNUAL REVIEW SHINKEI, vol. 2011, 2011, pages 310 - 317, XP008184906
HIROYOSHI OBA: "Jinko Kotai Library ni yoru Kotai Iyaku Kaihatsu", KOTAI IYAKU NO SAIZENSEN, 2007, pages 157 - 169, XP008184908
GLENNER, G.G.: "Amyloid deposits and amyloidosis: the beta-fibrilloses (second of two parts).", N ENGL J MED, vol. 302, 1980, pages 1333 - 1343
ANDO, Y.; JONO, H.: "Rinsho yori", vol. 56, 2008, article "Pathogenesis and therapy for transthyretin related amyloidosis", pages: 114 - 120
YOSHIKI SEKIJIMA: "Molecular mechanism of TTR amyloid deposition and its control", IGAKU-NO-AYUMI, vol. 229, 2009, pages 349 - 356
YUKO MOTOZAKI; SHOJI YAMADA: "Molecular epidemiology of familial amyloidotic polyneuropathy (FAP", IGAKU-NO-AYUMI, vol. 229, 2009, pages 357 - 362
HOU, X.; AGUILAR, M.I.; SMALL, D.H.: "Transthyretin and familial amyloidotic polyneuropathy. Recent progress in understanding the molecular mechanism . of neurodegeneration.", FEBS J, vol. 274, 2007, pages 1637 - 1650
ARAKI, S.; ANDO, Y.: "Transthyretin-related familial amyloidotic polyneuropathy - Progress in Kumamoto", PROC JPN ACAD SER B PHYS BIOL SCI, vol. 86, 2010, pages 694 - 706
EPISKOPOU, V.; MAEDA, S.; NISHIGUCHI, S.; SHIMADA, K.; GAITANARIS, .G.A.; GOTTESMAN, M.E.; ROBERTSON, E.J.: "Disruption of the transthyretin gene results in mice with depressed levels of plasma retinol and thyroid hormone.", PROC NATL ACAD SCI U S A, vol. 90, 1993, pages 2375 - 2379, XP001093658, DOI: doi:10.1073/pnas.90.6.2375
YUKIO ANDO: "Liver transplantation and other treatments for familial amyloidotic polyneuropathy (FAP", IGAKU-NO-AYUMI, vol. 229, 2009, pages 363 - 368
SAID, G.; GRIPPON, S.; KIRKPATRICK, P.: "Tafamidis", NAT REV DRUG DISCOV, vol. 11, 2012, pages 185 - 186
GOLDSTEINS, G.; PERSSON, H.; ANDERSSON, K.; OLOFSSON, A.; DACKLIN, I.; EDVINSSON, A.; SARAIVA, M.J.; LUNDGREN, E.: "Exposure of cryptic epitopes on transthyretin only in amyloid and in amyloidogenic mutants.", PROC NATL ACAD SCI U S A, vol. 96, 1999, pages 3108 - 3113, XP002987251, DOI: doi:10.1073/pnas.96.6.3108
TERAZAKI, H.; ANDO, Y.; FERNANDES, R.; YAMAMURA, K.; MAEDA, S.; & SARAIVA, M.J.: "Immunization in familial amyloidotic polyneuropathy: counteracting deposition by immunization with a Y78F TTR mutant.", LAB INVEST, vol. 86, 2006, pages 23 - 31, XP002531715, DOI: doi:10.1038/LABINVEST.3700365
BERGSTROEM, J.; ENGSTROEM, U.; YAMASHITA, T.; ANDO, Y.; WESTERMARK, P.: "Surface exposed epitopes and structural heterogeneity of in vivo formed transthyretin amyloid fibrils.", BIOCHEM BIOPHYS RES CORAMUN, vol. 348, 2006, pages 532 - 539
MATSUBARA, K.; MIZUGUCHI, M.; KAWANO, K.: "Expression of a synthetic gene encoding human transthyretin in Escherichia coli.", PROTEIN EXPR PURIF, vol. 30, 2003, pages 55 - 61, XP004432804, DOI: doi:10.1016/S1046-5928(03)00069-X
UEDA, M.; ANDO, Y.; HAKAMATA, Y.; NAKAMURA, M.; YAMASHITA, T.; OBAYASHI, K.; HIMENO, S.; INOUE, S.; SATO, Y.; KANEKO, T.: "A transgenic rat with the human ATTR V30M: a novel tool for analyses of ATTR metabolisms.", BIOCHEM BIOPHYS RES COMMUN, vol. 352, 2007, pages 299 - 304, XP029000099, DOI: doi:10.1016/j.bbrc.2006.11.045
MATSUBARA, K.; MIZUGUCHI, M.; IGARASHI, K.; SHINOHARA, Y.; TAKEUCHI, M.; MATSUURA, A.; SAITOH, T.; MORI, Y.; SHINODA, H.; KAWANO,: "Dimeric transthyretin variant assembles into spherical neurotoxins.", BIOCHEMISTRY, vol. 44, 2005, pages 3280 - 3288
SENJU, S.; HARUTA, M.; MATSUMURA, K.; MATSUNAGA, Y.; FUKUSHIMA, S.; IKEDA, T.; TAKAMATSU, K.; IRIE, A.; NISHIMURA, Y.: "Generation of dendritic cells and macrophages from human induced pluripotent stem cells aiming at cell therapy.", GENE THER, vol. 18, 2011, pages 874 - 883, XP055027827, DOI: doi:10.1038/gt.2011.22
"Phage Display of Peptides and Proteins: A Laboratory Manual"
"Antibody Engineering: A PRACTICAL APPROACH"
"ANTIBODY ENGINEERING"
See also references of EP 3101132A4
Attorney, Agent or Firm:
SAMEJIMA, Mutsumi et al. (JP)
Mutsumi Sameshima (JP)
Mutsumi Sameshima (JP)
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