SG11201909520VA - Rna for treatment of autoimmune diseases - Google Patents

Rna for treatment of autoimmune diseases

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Publication number
SG11201909520VA
SG11201909520VA SG11201909520VA SG11201909520VA SG 11201909520V A SG11201909520V A SG 11201909520VA SG 11201909520V A SG11201909520V A SG 11201909520VA SG 11201909520V A SG11201909520V A SG 11201909520VA
Authority
SG
Singapore
Prior art keywords
mainz
international
der
rna
pct
Prior art date
Application number
Inventor
Ugur Sahin
Sebastian Kreiter
Christina Krienke
Jutta Petschenka
Lena Mareen Kranz
Mustafa Diken
Original Assignee
Biontech Rna Pharmaceuticals Gmbh
Tron Translationale Onkologie An Der Univ Der Johannes Gutenberg Univ Mainz Gemeinnuetzige Gmbh
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
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Publication date
Application filed by Biontech Rna Pharmaceuticals Gmbh, Tron Translationale Onkologie An Der Univ Der Johannes Gutenberg Univ Mainz Gemeinnuetzige Gmbh filed Critical Biontech Rna Pharmaceuticals Gmbh
Publication of SG11201909520VA publication Critical patent/SG11201909520VA/en

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    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00Medicinal preparations containing antigens or antibodies
    • A61K39/0005Vertebrate antigens
    • A61K39/0008Antigens related to auto-immune diseases; Preparations to induce self-tolerance
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0008Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • A61K48/0025Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid
    • A61K48/0033Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid the non-active part being non-polymeric
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/10Dispersions; Emulsions
    • A61K9/127Liposomes
    • A61K9/1271Non-conventional liposomes, e.g. PEGylated liposomes, liposomes coated with polymers
    • A61K9/1272Non-conventional liposomes, e.g. PEGylated liposomes, liposomes coated with polymers with substantial amounts of non-phosphatidyl, i.e. non-acylglycerophosphate, surfactants as bilayer-forming substances, e.g. cationic lipids
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00Drugs for immunological or allergic disorders
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00Drugs for immunological or allergic disorders
    • A61P37/02Immunomodulators
    • A61P37/06Immunosuppressants, e.g. drugs for graft rejection
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/10Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/1003Extracting or separating nucleic acids from biological samples, e.g. pure separation or isolation methods; Conditions, buffers or apparatuses therefor
    • C12N15/1006Extracting or separating nucleic acids from biological samples, e.g. pure separation or isolation methods; Conditions, buffers or apparatuses therefor by means of a solid support carrier, e.g. particles, polymers
    • C12N15/101Extracting or separating nucleic acids from biological samples, e.g. pure separation or isolation methods; Conditions, buffers or apparatuses therefor by means of a solid support carrier, e.g. particles, polymers by chromatography, e.g. electrophoresis, ion-exchange, reverse phase
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/117Nucleic acids having immunomodulatory properties, e.g. containing CpG-motifs
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00Medicinal preparations containing antigens or antibodies
    • A61K2039/51Medicinal preparations containing antigens or antibodies comprising whole cells, viruses or DNA/RNA
    • A61K2039/53DNA (RNA) vaccination
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00Medicinal preparations containing antigens or antibodies
    • A61K2039/555Medicinal preparations containing antigens or antibodies characterised by a specific combination antigen/adjuvant
    • A61K2039/55511Organic adjuvants
    • A61K2039/55555Liposomes; Vesicles, e.g. nanoparticles; Spheres, e.g. nanospheres; Polymers
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00Medicinal preparations containing antigens or antibodies
    • A61K2039/60Medicinal preparations containing antigens or antibodies characteristics by the carrier linked to the antigen
    • A61K2039/6018Lipids, e.g. in lipopeptides
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/33Chemical structure of the base
    • C12N2310/335Modified T or U

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Immunology (AREA)
  • Organic Chemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Epidemiology (AREA)
  • Microbiology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Rheumatology (AREA)
  • Mycology (AREA)
  • Transplantation (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Analytical Chemistry (AREA)
  • Dispersion Chemistry (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)

Abstract

INTERNATIONAL APPLICATION PUBLISHED UNDER THE PATENT COOPERATION TREATY (PCT) (19) World Intellectual Property Organization International Bureau (43) International Publication Date 18 October 2018 (18.10.2018) W I P0 I P C T omit OH Elm Him °ow° lII ow (10) International Publication Number WO 2018/189193 Al (51) International Patent Classification: A61K 39/00 (2006.01) A61P 37/00 (2006.01) A61K 48/00 (2006.01) (21) International Application Number: PCT/EP2018/059188 (22) International Filing Date: 10 April 2018 (10.04.2018) (25) Filing Language: English (26) Publication Language: English (30) Priority Data: PCT/EP2017/058651 11 April 2017 (11.04.2017) EP (71) Applicants: BIONTECH RNA PHARMACEUTICALS GMBH [DE/DE]; An der Goldgrube 12, 55131 Mainz (DE). TRON - TRANSLATIONALE ONKOLOGIE AN DER UNIVERSITATSMEDIZIN DER JOHANNES GUTENBERG-UNIVERSITAT MAINZ GEMEIN- NUTZIGE GMBH [DE/DE]; FreiligrathstraBe 12, 55131 Mainz (DE). UNIVERSITATSMEDIZIN DER JOHAN- NES GUTENBERG-UNIVERSITAT MAINZ [DE/DE]; LangenbeckstraBe 1, 55131 Mainz (DE). (72) Inventors: SAHIN, Ugur; c/o BioNTech AG, An der Goldgrube 12, 55131 Mainz (DE). KREITER, Sebast- ian; Niklas-Vogt-StraBe 3, 55131 Mainz (DE). KRIENKE, Christina; SilvanerstraBe 6, 55276 Dienheim (DE). PETSCHENKA, Jutta; Am Schinnergraben 37, 55129 Mainz (DE). KRANZ, Lena Mareen; FeldbergstraBe 28, 55118 Mainz (DE). DIKEN, Mustafa; Bert-Brecht-StraBe 5, 55128 Mainz (DE). (74) Agent: MULLER, Christian; ZSP Patentanwalte PartG mbB, Hansastr. 32, 80686 Munich (DE). (81) Designated States (unless otherwise indicated, for every kind of national protection available): AE, AG, AL, AM, AO, AT, AU, AZ, BA, BB, BG, BH, BN, BR, BW, BY, BZ, CA, CH, CL, CN, CO, CR, CU, CZ, DE, DJ, DK, DM, DO, DZ, EC, EE, EG, ES, FI, GB, GD, GE, GH, GM, GT, HN, HR, HU, ID, IL, IN, IR, IS, JO, JP, KE, KG, KH, KN, KP, KR, KW, KZ, LA, LC, LK, LR, LS, LU, LY, MA, MD, ME, MG, MK, MN, MW, MX, MY, MZ, NA, NG, NI, NO, NZ, OM, PA, PE, PG, PH, PL, PT, QA, RO, RS, RU, RW, SA, SC, SD, SE, SG, SK, SL, SM, ST, SV, SY, TH, TJ, TM, TN, TR, TT, TZ, UA, UG, US, UZ, VC, VN, ZA, ZM, ZW. (84) Designated States (unless otherwise indicated, for every kind of regional protection available): ARIPO (BW, GH, GM, KE, LR, LS, MW, MZ, NA, RW, SD, SL, ST, SZ, TZ, UG, ZM, ZW), Eurasian (AM, AZ, BY, KG, KZ, RU, TJ, TM), European (AL, AT, BE, BG, CH, CY, CZ, DE, DK, EE, ES, FI, FR, GB, GR, HR, HU, IE, IS, IT, LT, LU, LV, MC, MK, MT, NL, NO, PL, PT, RO, RS, SE, SI, SK, SM, TR), OAPI (BF, BJ, CF, CG, CI, CM, GA, GN, GQ, GW, KM, ML, MR, NE, SN, TD, TG). Published: with international search report (Art. 21(3)) with sequence listing part of description (Rule 5.2(a)) 1-1 O \" (57) : The present invention relates to non-immunogenic RNA. This RNA forms the basis for the development of therapeutic 0 agents for inducing tolerance towards an autoantigen and thus, for the treatment of autoimmune diseases. (54) Title: RNA FOR TREATMENT OF AUTOIMMUNE DISEASES
SG11201909520V 2017-04-11 2018-04-10 Rna for treatment of autoimmune diseases SG11201909520VA (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
PCT/EP2017/058651 WO2018188730A1 (en) 2017-04-11 2017-04-11 Rna for treatment of autoimmune diseases
PCT/EP2018/059188 WO2018189193A1 (en) 2017-04-11 2018-04-10 Rna for treatment of autoimmune diseases

Publications (1)

Publication Number Publication Date
SG11201909520VA true SG11201909520VA (en) 2019-11-28

Family

ID=58632945

Family Applications (2)

Application Number Title Priority Date Filing Date
SG11201909520V SG11201909520VA (en) 2017-04-11 2018-04-10 Rna for treatment of autoimmune diseases
SG10202111163VA SG10202111163VA (en) 2017-04-11 2018-04-10 Rna for treatment of autoimmune diseases

Family Applications After (1)

Application Number Title Priority Date Filing Date
SG10202111163VA SG10202111163VA (en) 2017-04-11 2018-04-10 Rna for treatment of autoimmune diseases

Country Status (22)

Country Link
US (2) US11701413B2 (en)
EP (2) EP3981424A1 (en)
JP (3) JP7078641B2 (en)
KR (2) KR20190134765A (en)
CN (2) CN118161602A (en)
AU (1) AU2018253352A1 (en)
BR (1) BR112019020667A8 (en)
CA (1) CA3059505A1 (en)
CY (1) CY1124767T1 (en)
DK (1) DK3609529T3 (en)
ES (1) ES2893451T3 (en)
HR (1) HRP20211559T1 (en)
HU (1) HUE056871T2 (en)
IL (2) IL269793B (en)
LT (1) LT3609529T (en)
MX (1) MX2019012144A (en)
PL (1) PL3609529T3 (en)
PT (1) PT3609529T (en)
RS (1) RS62582B1 (en)
SG (2) SG11201909520VA (en)
SI (1) SI3609529T1 (en)
WO (2) WO2018188730A1 (en)

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EP3880174A2 (en) * 2018-11-12 2021-09-22 Translate Bio, Inc. Methods for inducing immune tolerance
US11161892B1 (en) 2020-12-07 2021-11-02 Think Therapeutics, Inc. Method of compact peptide vaccines using residue optimization
US11058751B1 (en) 2020-11-20 2021-07-13 Think Therapeutics, Inc. Compositions for optimized RAS peptide vaccines
US11421015B2 (en) 2020-12-07 2022-08-23 Think Therapeutics, Inc. Method of compact peptide vaccines using residue optimization
CN115232825B (en) * 2021-04-22 2023-09-26 中国人民解放军军事科学院军事医学研究院 New coronavirus vaccine based on 1083 skeleton
US11464842B1 (en) 2021-04-28 2022-10-11 Think Therapeutics, Inc. Compositions and method for optimized peptide vaccines using residue optimization
WO2022248525A1 (en) 2021-05-26 2022-12-01 Imcyse Sa Methods of treating or preventing autoimmune diseases
AR126654A1 (en) 2021-06-29 2023-11-01 Imcyse Sa PEPTIDES AND METHODS FOR THE TREATMENT OF OPTICAL NEUROMYELITIS
CN113336862B (en) * 2021-07-05 2022-03-01 广东省科学院动物研究所 Recombinant protein for resisting multiple sclerosis and preparation method and application thereof
WO2023030635A1 (en) 2021-09-02 2023-03-09 BioNTech SE Potency assay for therapeutic potential of coding nucleic acid
WO2023131254A1 (en) * 2022-01-06 2023-07-13 上海吉量医药工程有限公司 Pseudouridine modified at n1 position and use thereof in mrna synthesis

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US7030098B2 (en) * 1999-03-12 2006-04-18 The Board Of Trustees Of The Leland Stanford Junior University DNA vaccination for treatment of autoimmune disease
AU2002952834A0 (en) 2002-09-16 2002-12-05 The Walter And Eliza Hall Institute Of Medical Research A method of treating an autoimmune disease
ES2735531T3 (en) 2005-08-23 2019-12-19 Univ Pennsylvania RNA containing modified nucleosides and methods of use thereof
US9012219B2 (en) 2005-08-23 2015-04-21 The Trustees Of The University Of Pennsylvania RNA preparations comprising purified modified RNA for reprogramming cells
WO2009030254A1 (en) * 2007-09-04 2009-03-12 Curevac Gmbh Complexes of rna and cationic peptides for transfection and for immunostimulation
EP2050814A1 (en) 2007-10-17 2009-04-22 Txcell Compositions for treating multiple sclerosis
EP3623474A1 (en) * 2009-12-07 2020-03-18 The Trustees of The University of Pennsylvania Rna preparations comprising purified modified rna for reprogramming cells
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WO2013044419A1 (en) * 2011-09-30 2013-04-04 Beijing Advaccine Biotechnology Co., Ltd. Combined facilitator, antigen and dna vaccine for preventing and treating autoimmune diseases
EP4372081A2 (en) 2011-12-30 2024-05-22 Cellscript, Llc Making and using in vitro-synthesized ssrna for introducing into mammalian cells to induce a biological or biochemical effect
WO2013120498A1 (en) 2012-02-15 2013-08-22 Curevac Gmbh Nucleic acid comprising or coding for a histone stem-loop and a poly(a) sequence or a polyadenylation signal for increasing the expression of an encoded allergenic antigen or an autoimmune self-antigen
US20160032316A1 (en) 2013-03-14 2016-02-04 The Trustees Of The University Of Pennsylvania Purification and Purity Assessment of RNA Molecules Synthesized with Modified Nucleosides
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AU2016315444B2 (en) * 2015-08-28 2023-02-23 BioNTech SE Method for reducing immunogenicity of RNA
AU2017251983B2 (en) 2016-04-22 2023-07-20 BioNTech SE Methods for providing single-stranded RNA

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Publication number Publication date
JP2024059794A (en) 2024-05-01
BR112019020667A8 (en) 2022-08-16
IL290980A (en) 2022-05-01
CA3059505A1 (en) 2018-10-18
EP3609529A1 (en) 2020-02-19
KR20190134765A (en) 2019-12-04
BR112019020667A2 (en) 2020-05-05
RU2019135808A (en) 2021-05-11
HUE056871T2 (en) 2022-04-28
DK3609529T3 (en) 2021-10-11
US20200061166A1 (en) 2020-02-27
JP7444924B2 (en) 2024-03-06
US20230330198A1 (en) 2023-10-19
AU2018253352A1 (en) 2019-10-17
IL269793A (en) 2019-11-28
IL269793B (en) 2022-04-01
JP2022110127A (en) 2022-07-28
WO2018189193A1 (en) 2018-10-18
RS62582B1 (en) 2021-12-31
EP3609529B1 (en) 2021-09-08
CN110719787B (en) 2024-03-15
RU2019135808A3 (en) 2021-08-10
PT3609529T (en) 2021-10-14
US11701413B2 (en) 2023-07-18
PL3609529T3 (en) 2021-12-13
WO2018188730A1 (en) 2018-10-18
CN118161602A (en) 2024-06-11
SI3609529T1 (en) 2021-11-30
HRP20211559T1 (en) 2022-01-21
ES2893451T3 (en) 2022-02-09
MX2019012144A (en) 2019-11-21
SG10202111163VA (en) 2021-11-29
JP2020516658A (en) 2020-06-11
LT3609529T (en) 2021-10-25
CN110719787A (en) 2020-01-21
EP3981424A1 (en) 2022-04-13
KR20240042138A (en) 2024-04-01
CY1124767T1 (en) 2022-11-25
JP7078641B2 (en) 2022-05-31

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