Title:
METHOD FOR ENHANCING INNATE IMMUNE RESPONSE
Document Type and Number:
WIPO Patent Application WO/2022/255411
Kind Code:
A1
Abstract:
Provided is a method for enhancing the innate immune response. The present disclosure pertains, in one embodiment, to a method for enhancing the innate immune response of a subject or a cell, the subject or the cell being a subject or a cell having an innate immune response involving a gene subject to splicing, the method including administering a compound having an effect on selective splicing to the subject or the cell or bringing such a compound into contact with the subject or the cell.
Inventors:
HAGIWARA MASATOSHI
IIDA KEI
NODA TAKESHI
AJIRO MASAHIKO
IIDA KEI
NODA TAKESHI
AJIRO MASAHIKO
Application Number:
PCT/JP2022/022350
Publication Date:
December 08, 2022
Filing Date:
June 01, 2022
Export Citation:
Assignee:
UNIV KYOTO (JP)
International Classes:
A61K45/00; A61K31/41; A61K31/425; A61K31/433; A61K31/437; A61K48/00
Domestic Patent References:
| WO2014021337A1 | 2014-02-06 | |||
| WO2018043674A1 | 2018-03-08 | |||
| WO2015083750A1 | 2015-06-11 | |||
| WO2010010797A1 | 2010-01-28 | |||
| WO2012001941A1 | 2012-01-05 | |||
| WO2014021337A1 | 2014-02-06 | |||
| WO2018043674A1 | 2018-03-08 | |||
| WO2015083750A1 | 2015-06-11 | |||
| WO2010010797A1 | 2010-01-28 |
Other References:
ARTARINI ANITA; MEYER MICHAEL; SHIN YU JIN; HUBER KILIAN; HILZ NIKOLAUS; BRACHER FRANZ; EROS DANIEL; ORFI LASZLO; KERI GYORGY; GOE: "Regulation of influenza A virus mRNA splicing by CLK1", ANTIVIRAL RESEARCH, vol. 168, 1 August 2019 (2019-08-01), NL , pages 187 - 196, XP085730457, ISSN: 0166-3542, DOI: 10.1016/j.antiviral.2019.06.003
LI CHAO; XU LV-JIE; LIAN WEN-WEN; PANG XIAO-CONG; JIA HAO; LIU AI-LIN; DU GUAN-HUA: "Anti-influenza effect and action mechanisms of the chemical constituent gallocatechin-7-gallate fromPithecellobium clypeariaBenth", ACTA PHARMACOLOGICA SINICA, vol. 39, no. 12, 25 May 2018 (2018-05-25), GB , pages 1913 - 1922, XP036651746, ISSN: 1671-4083, DOI: 10.1038/s41401-018-0030-x
DRAPPIER MELISSA, MICHIELS THOMAS: "Inhibition of the OAS/RNase L pathway by viruses", CURRENT OPINION IN VIROLOGY, vol. 15, 1 December 2015 (2015-12-01), United Kingdom , pages 19 - 26, XP093011849, ISSN: 1879-6257, DOI: 10.1016/j.coviro.2015.07.002
AJIRO, MASAHIKO : "O-45 Therapeutic Strategy for Pseudo Exon-Related Diseases by Targeting CLK-SRSF Axis", PROCEEDINGS OF THE 21ST ANNUAL MEETING OF THE RNA SOCIETY OF JAPAN; JULY 17-19, 2019, vol. 21, 17 July 2019 (2019-07-17) - 19 July 2019 (2019-07-19), JP, pages 47, XP009541765
IIDA KEI, AJIRO MASAHIKO, MURAMOTO YUKIKO, TAKENAGA TORU, DENAWA MASATSUGU, KUROSAWA RYO, NODA TAKESHI, HAGIWARA MASATOSHI: "Switching of OAS1 splicing isoforms mitigates SARS-CoV-2 infection", BIORXIV, 23 August 2021 (2021-08-23), pages 1 - 32, XP093011850, DOI: 10.1101/2021.08.23.457314
PAIRO-CASTINEIRA E, CLOHISEY S, KLARIC L, BRETHERICK AD, RAWLIK K, PASKO D, WALKER S, PARKINSON N, FOURMAN MH, RUSSELL CD, FURNISS: "Genetic mechanisms of critical illness in COVID-19", NATURE, vol. 591, 2021, pages 92 - 98, XP037386549, DOI: 10.1038/s41586-020-03065-y
THE COVID-19 HOST GENETICS INITIATIVE, 2021
BLANCO-MELO DNILSSON-PAYANT BELIU WUHL SHOAGLAND DMOLLER RJORDAN TXOISHI KPANIS MSACHS D: "Imbalanced Host Response to SARS-CoV-2 Drives Development of COVID-19", CELL, vol. 181, 2020, pages 1036 - 1045, XP055857047, DOI: 10.1016/j.cell.2020.04.026
HAMANO EHIJIKATA MITOYAMA SQUY TPHI NCLONG HTHA LDBAN V VANMATSUSHITA IYANAI H: "Polymorphisms of interferon-inducible genes OAS-1 and MxA associated with SARS in the Vietnamese population", BIOCHEM BIOPHYS RES COMMUN, vol. 329, 2005, pages 1234 - 9, XP004784864, DOI: 10.1016/j.bbrc.2005.02.101
HE JFENG DDE VLAS SJWANG HFONTANET AZHANG PPLANCOULAINE STANG FZHAN LYANG H: "Association of SARS susceptibility with single nucleic acid polymorphisms of OAS1 and MxA genes: a case-control study", BMC INFECT DIS, vol. 6, 2006, pages 106, XP021015633, DOI: 10.1186/1471-2334-6-106
LIN R-JYU H-PCHANG B-LTANG W-CLIAO C-LLIN Y-L: "Distinct antiviral roles for human 2',5'-oligoadenylate synthetase family members against dengue virus infection", J IMMUNOL, vol. 183, 2009, pages 8035 - 43
PROG. MED., vol. 5, 1985, pages 2157 - 2161
ADAM D.: "A guide to R - the pandemic's misunderstood metric", NATURE, vol. 583, 2020, pages 346 - 348, XP037189469, DOI: 10.1038/d41586-020-02009-w
CAREY CMGOVANDE AACOOPER JMHARTLEY MKKRANZUSCH PJELDE NC: "Recurrent Loss-of-Function Mutations Reveal Costs to OAS1 Antiviral Activity in Primates", CELL HOST MICROBE, vol. 25, 2019, pages 336 - 343
CARTEGNI LWANG JZHU ZZHANG MQKRAINER AR: "ESEfinder: A web resource to identify exonic splicing enhancers", NUCLEIC ACIDS RES, vol. 31, 2003, pages 3568 - 71, XP055358041, DOI: 10.1093/nar/gkg616
CHOI UYKANG J-SHWANG YSKIM Y J: "Oligoadenylate synthase-like (OASL) proteins: dual functions and associations with diseases", EXP MOL MED, vol. 47, 2015, pages e144
DI HELBAHESH HBRINTON MA: "Characteristics of Human OAS 1 Isoform Proteins", VIRUSES, vol. 12, 2020
FORD ETHANOS D: "The transcriptional code of human IFN-beta gene expression", BIOCHIM BIOPHYS ACTA, vol. 1799, 2010, pages 328 - 36, XP026965631
HARKO TLOBO FSNMAK MK: "Exact analytical solutions of the Susceptible-Infected-Recovered (SIR) epidemic model and of the SIR model with equal death and birth rates", APPL MATH COMPUT, vol. 236, 2014, pages 184 - 194, XP028644772, DOI: 10.1016/j.amc.2014.03.030
HUNG IF-NLUNG K-CTSO EY-KLIU RCHUNG TW-HCHU M-YNG Y YLO JCHAN JTAM AR: "Triple combination of interferon beta-lb, lopinavir-ritonavir, and ribavirin in the treatment of patients admitted to hospital with COVID-19: an open-label, randomised, phase 2 trial", LANCET (LONDON, ENGLAND, vol. 395, 2020, pages 1695 - 1704, XP055849472, DOI: 10.1016/S0140-6736(20)31042-4
IBSEN MS, GAD HH, ANDERSEN LL, HORNUNG V, JULKUNEN I, SARKAR SN, HARTMANN R.: "Structural and functional analysis reveals that human OASL binds dsRNA to enhance RIG-I signaling", NUCLEIC ACIDS RES, vol. 43, 2015, pages 5236 - 48
KJAER KHPAHUS JHANSEN MFPOULSEN JBCHRISTENSEN EIJUSTESEN JMARTENSEN PM: "Mitochondrial localization of the OAS1 p46 isoform associated with a common single nucleotide polymorphism", BMC CELL BIOL, vol. 15, 2014, pages 1 - 14
MARCUS JHNOVEMBRE J: "Visualizing the geography of genetic variants", BIOINFORMATICS, vol. 33, 2017, pages 594 - 595
MENDEZ FLWATKINS JCHAMMER MF: "Neandertal origin of genetic variation at the cluster of OAS immunity genes", MOL BIOL EVOL, vol. 30, 2013, pages 798 - 801
MONK PDMARSDEN RJTEAR VJBROOKES JBATTEN TNMANKOWSKI MGABBAY FJDAVIES DEHOLGATE STHO L-P: "Safety and efficacy of inhaled nebulised interferon beta-la (SNG001) for treatment of SARS-CoV-2 infection: a randomised, double-blind, placebo-controlled, phase 2 trial", LANCET RESPIR MED, vol. 9, 2021, pages 196 - 206, XP055822196, DOI: 10.1016/S2213-2600(20)30511-7
NILE SHNILE AQ JLI LJIA XKAI G: "COVID-19: Pathogenesis, cytokine storm and therapeutic potential of interferons", CYTOKINE GROWTH FACTOR REV, vol. 53, 2020, pages 66 - 70, XP086180312, DOI: 10.1016/j.cytogfr.2020.05.002
NOGUCHI SHAMANO EMATSUSHITA IHIJIKATA MITO HNAGASE TKEICHO N: "Differential effects of a common splice site polymorphism on the generation of OAS 1 variants in human bronchial epithelial cells", HUM IMMUNOL, vol. 74, 2013, pages 395 - 401
SADLER AJWILLIAMS BRG: "Interferon-inducible antiviral effectors", NAT REV IMMUNOL, vol. 8, 2008, pages 559 - 68, XP055229143, DOI: 10.1038/nri2314
SAKUMA MIIDA KHAGIWARA M: "Deciphering targeting rules of splicing modulator compounds: case of TG003", BMC MOL BIOL, vol. 16, 2015, pages 16
SCHLEE MHARTMANN G: "Discriminating self from non-self in nucleic acid sensing", NAT REV IMMUNOL, vol. 16, 2016, pages 566 - 80, XP037065556, DOI: 10.1038/nri.2016.78
SCHWANKE HSTEMPEL MBRINKMANN MM: "Of Keeping and Tipping the Balance: Host Regulation and Viral Modulation of IRF3-Dependent IFNB1 Expression", VIRUSES, vol. 12, 2020
SHALHOUB S.: "Interferon beta-lb for COVID-19", LANCET (LONDON, ENGLAND, vol. 395, 2020, pages 1670 - 1671
SHIBATA SAJIRO MHAGIWARA M: "Mechanism-Based Personalized Medicine for Cystic Fibrosis by Suppressing Pseudo Exon Inclusion", CELL CHEM BIOL, vol. 27, 2020, pages 1472, XP086414564, DOI: 10.1016/j.chembiol.2020.08.013
SOETAERT KPETZOLDT TSETZER RW: "Solving Differential Equations in R: Package deSolve", J STAT SOFTW, vol. 33, 2010
THE COVID-19 HOST GENETICS INITIATIVE: "Mapping the human genetic architecture of COVID-19 by worldwide meta-analysis", MEDRXIV 2021.03.10.21252820, 2021
ZEBERG HPAABO S: "A genomic region associated with protection against severe COVID-19 is inherited from Neandertals", PROC NATL ACAD SCI U S A, vol. 118, 2021
ZHOU SBUTLER-LAPORTE GNAKANISHI TMORRISON DRAFILALO JAFILALO MLAURENT LPIETZNER MKERRISON NZHAO K: "A Neanderthal OAS1 isoform protects individuals of European ancestry against COVID-19 susceptibility and severity", NAT MED. D I: 10.1038/ -021-01281-1, 2021
LI CHAO; XU LV-JIE; LIAN WEN-WEN; PANG XIAO-CONG; JIA HAO; LIU AI-LIN; DU GUAN-HUA: "Anti-influenza effect and action mechanisms of the chemical constituent gallocatechin-7-gallate fromPithecellobium clypeariaBenth", ACTA PHARMACOLOGICA SINICA, vol. 39, no. 12, 25 May 2018 (2018-05-25), GB , pages 1913 - 1922, XP036651746, ISSN: 1671-4083, DOI: 10.1038/s41401-018-0030-x
DRAPPIER MELISSA, MICHIELS THOMAS: "Inhibition of the OAS/RNase L pathway by viruses", CURRENT OPINION IN VIROLOGY, vol. 15, 1 December 2015 (2015-12-01), United Kingdom , pages 19 - 26, XP093011849, ISSN: 1879-6257, DOI: 10.1016/j.coviro.2015.07.002
AJIRO, MASAHIKO : "O-45 Therapeutic Strategy for Pseudo Exon-Related Diseases by Targeting CLK-SRSF Axis", PROCEEDINGS OF THE 21ST ANNUAL MEETING OF THE RNA SOCIETY OF JAPAN; JULY 17-19, 2019, vol. 21, 17 July 2019 (2019-07-17) - 19 July 2019 (2019-07-19), JP, pages 47, XP009541765
IIDA KEI, AJIRO MASAHIKO, MURAMOTO YUKIKO, TAKENAGA TORU, DENAWA MASATSUGU, KUROSAWA RYO, NODA TAKESHI, HAGIWARA MASATOSHI: "Switching of OAS1 splicing isoforms mitigates SARS-CoV-2 infection", BIORXIV, 23 August 2021 (2021-08-23), pages 1 - 32, XP093011850, DOI: 10.1101/2021.08.23.457314
PAIRO-CASTINEIRA E, CLOHISEY S, KLARIC L, BRETHERICK AD, RAWLIK K, PASKO D, WALKER S, PARKINSON N, FOURMAN MH, RUSSELL CD, FURNISS: "Genetic mechanisms of critical illness in COVID-19", NATURE, vol. 591, 2021, pages 92 - 98, XP037386549, DOI: 10.1038/s41586-020-03065-y
THE COVID-19 HOST GENETICS INITIATIVE, 2021
BLANCO-MELO DNILSSON-PAYANT BELIU WUHL SHOAGLAND DMOLLER RJORDAN TXOISHI KPANIS MSACHS D: "Imbalanced Host Response to SARS-CoV-2 Drives Development of COVID-19", CELL, vol. 181, 2020, pages 1036 - 1045, XP055857047, DOI: 10.1016/j.cell.2020.04.026
HAMANO EHIJIKATA MITOYAMA SQUY TPHI NCLONG HTHA LDBAN V VANMATSUSHITA IYANAI H: "Polymorphisms of interferon-inducible genes OAS-1 and MxA associated with SARS in the Vietnamese population", BIOCHEM BIOPHYS RES COMMUN, vol. 329, 2005, pages 1234 - 9, XP004784864, DOI: 10.1016/j.bbrc.2005.02.101
HE JFENG DDE VLAS SJWANG HFONTANET AZHANG PPLANCOULAINE STANG FZHAN LYANG H: "Association of SARS susceptibility with single nucleic acid polymorphisms of OAS1 and MxA genes: a case-control study", BMC INFECT DIS, vol. 6, 2006, pages 106, XP021015633, DOI: 10.1186/1471-2334-6-106
LIN R-JYU H-PCHANG B-LTANG W-CLIAO C-LLIN Y-L: "Distinct antiviral roles for human 2',5'-oligoadenylate synthetase family members against dengue virus infection", J IMMUNOL, vol. 183, 2009, pages 8035 - 43
PROG. MED., vol. 5, 1985, pages 2157 - 2161
ADAM D.: "A guide to R - the pandemic's misunderstood metric", NATURE, vol. 583, 2020, pages 346 - 348, XP037189469, DOI: 10.1038/d41586-020-02009-w
CAREY CMGOVANDE AACOOPER JMHARTLEY MKKRANZUSCH PJELDE NC: "Recurrent Loss-of-Function Mutations Reveal Costs to OAS1 Antiviral Activity in Primates", CELL HOST MICROBE, vol. 25, 2019, pages 336 - 343
CARTEGNI LWANG JZHU ZZHANG MQKRAINER AR: "ESEfinder: A web resource to identify exonic splicing enhancers", NUCLEIC ACIDS RES, vol. 31, 2003, pages 3568 - 71, XP055358041, DOI: 10.1093/nar/gkg616
CHOI UYKANG J-SHWANG YSKIM Y J: "Oligoadenylate synthase-like (OASL) proteins: dual functions and associations with diseases", EXP MOL MED, vol. 47, 2015, pages e144
DI HELBAHESH HBRINTON MA: "Characteristics of Human OAS 1 Isoform Proteins", VIRUSES, vol. 12, 2020
FORD ETHANOS D: "The transcriptional code of human IFN-beta gene expression", BIOCHIM BIOPHYS ACTA, vol. 1799, 2010, pages 328 - 36, XP026965631
HARKO TLOBO FSNMAK MK: "Exact analytical solutions of the Susceptible-Infected-Recovered (SIR) epidemic model and of the SIR model with equal death and birth rates", APPL MATH COMPUT, vol. 236, 2014, pages 184 - 194, XP028644772, DOI: 10.1016/j.amc.2014.03.030
HUNG IF-NLUNG K-CTSO EY-KLIU RCHUNG TW-HCHU M-YNG Y YLO JCHAN JTAM AR: "Triple combination of interferon beta-lb, lopinavir-ritonavir, and ribavirin in the treatment of patients admitted to hospital with COVID-19: an open-label, randomised, phase 2 trial", LANCET (LONDON, ENGLAND, vol. 395, 2020, pages 1695 - 1704, XP055849472, DOI: 10.1016/S0140-6736(20)31042-4
IBSEN MS, GAD HH, ANDERSEN LL, HORNUNG V, JULKUNEN I, SARKAR SN, HARTMANN R.: "Structural and functional analysis reveals that human OASL binds dsRNA to enhance RIG-I signaling", NUCLEIC ACIDS RES, vol. 43, 2015, pages 5236 - 48
KJAER KHPAHUS JHANSEN MFPOULSEN JBCHRISTENSEN EIJUSTESEN JMARTENSEN PM: "Mitochondrial localization of the OAS1 p46 isoform associated with a common single nucleotide polymorphism", BMC CELL BIOL, vol. 15, 2014, pages 1 - 14
MARCUS JHNOVEMBRE J: "Visualizing the geography of genetic variants", BIOINFORMATICS, vol. 33, 2017, pages 594 - 595
MENDEZ FLWATKINS JCHAMMER MF: "Neandertal origin of genetic variation at the cluster of OAS immunity genes", MOL BIOL EVOL, vol. 30, 2013, pages 798 - 801
MONK PDMARSDEN RJTEAR VJBROOKES JBATTEN TNMANKOWSKI MGABBAY FJDAVIES DEHOLGATE STHO L-P: "Safety and efficacy of inhaled nebulised interferon beta-la (SNG001) for treatment of SARS-CoV-2 infection: a randomised, double-blind, placebo-controlled, phase 2 trial", LANCET RESPIR MED, vol. 9, 2021, pages 196 - 206, XP055822196, DOI: 10.1016/S2213-2600(20)30511-7
NILE SHNILE AQ JLI LJIA XKAI G: "COVID-19: Pathogenesis, cytokine storm and therapeutic potential of interferons", CYTOKINE GROWTH FACTOR REV, vol. 53, 2020, pages 66 - 70, XP086180312, DOI: 10.1016/j.cytogfr.2020.05.002
NOGUCHI SHAMANO EMATSUSHITA IHIJIKATA MITO HNAGASE TKEICHO N: "Differential effects of a common splice site polymorphism on the generation of OAS 1 variants in human bronchial epithelial cells", HUM IMMUNOL, vol. 74, 2013, pages 395 - 401
SADLER AJWILLIAMS BRG: "Interferon-inducible antiviral effectors", NAT REV IMMUNOL, vol. 8, 2008, pages 559 - 68, XP055229143, DOI: 10.1038/nri2314
SAKUMA MIIDA KHAGIWARA M: "Deciphering targeting rules of splicing modulator compounds: case of TG003", BMC MOL BIOL, vol. 16, 2015, pages 16
SCHLEE MHARTMANN G: "Discriminating self from non-self in nucleic acid sensing", NAT REV IMMUNOL, vol. 16, 2016, pages 566 - 80, XP037065556, DOI: 10.1038/nri.2016.78
SCHWANKE HSTEMPEL MBRINKMANN MM: "Of Keeping and Tipping the Balance: Host Regulation and Viral Modulation of IRF3-Dependent IFNB1 Expression", VIRUSES, vol. 12, 2020
SHALHOUB S.: "Interferon beta-lb for COVID-19", LANCET (LONDON, ENGLAND, vol. 395, 2020, pages 1670 - 1671
SHIBATA SAJIRO MHAGIWARA M: "Mechanism-Based Personalized Medicine for Cystic Fibrosis by Suppressing Pseudo Exon Inclusion", CELL CHEM BIOL, vol. 27, 2020, pages 1472, XP086414564, DOI: 10.1016/j.chembiol.2020.08.013
SOETAERT KPETZOLDT TSETZER RW: "Solving Differential Equations in R: Package deSolve", J STAT SOFTW, vol. 33, 2010
THE COVID-19 HOST GENETICS INITIATIVE: "Mapping the human genetic architecture of COVID-19 by worldwide meta-analysis", MEDRXIV 2021.03.10.21252820, 2021
ZEBERG HPAABO S: "A genomic region associated with protection against severe COVID-19 is inherited from Neandertals", PROC NATL ACAD SCI U S A, vol. 118, 2021
ZHOU SBUTLER-LAPORTE GNAKANISHI TMORRISON DRAFILALO JAFILALO MLAURENT LPIETZNER MKERRISON NZHAO K: "A Neanderthal OAS1 isoform protects individuals of European ancestry against COVID-19 susceptibility and severity", NAT MED. D I: 10.1038/ -021-01281-1, 2021
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IKEUCHI & PARTNERS (JP)
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