CA3019981A1 - Nucleic acid linked to a trivalent glycoconjugate - Google Patents

Nucleic acid linked to a trivalent glycoconjugate Download PDF

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Publication number
CA3019981A1
CA3019981A1 CA3019981A CA3019981A CA3019981A1 CA 3019981 A1 CA3019981 A1 CA 3019981A1 CA 3019981 A CA3019981 A CA 3019981A CA 3019981 A CA3019981 A CA 3019981A CA 3019981 A1 CA3019981 A1 CA 3019981A1
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CA
Canada
Prior art keywords
nucleic acid
glycoconjugate
trivalent
acid linked
compound
Prior art date
Legal status (The legal status 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 status listed.)
Pending
Application number
CA3019981A
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English (en)
French (fr)
Inventor
Christian Frauendorf
Mark Cameron
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Silence Therapeutics GmbH
Original Assignee
Silence Therapeutics 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.)
Filing date
Publication date
Application filed by Silence Therapeutics GmbH filed Critical Silence Therapeutics GmbH
Publication of CA3019981A1 publication Critical patent/CA3019981A1/en
Pending legal-status Critical Current

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    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7125—Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/54—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound
    • A61K47/549—Sugars, nucleosides, nucleotides or nucleic acids
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/56—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic macromolecular compound, e.g. an oligomeric, polymeric or dendrimeric molecule
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P29/00—Non-central analgesic, antipyretic or antiinflammatory agents, e.g. antirheumatic agents; Non-steroidal antiinflammatory drugs [NSAID]
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/04—Anorexiants; Antiobesity agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/06—Antihyperlipidemics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12—Antivirals
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P5/00—Drugs for disorders of the endocrine system
    • A61P5/06—Drugs for disorders of the endocrine system of the anterior pituitary hormones, e.g. TSH, ACTH, FSH, LH, PRL, GH
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P5/00—Drugs for disorders of the endocrine system
    • A61P5/06—Drugs for disorders of the endocrine system of the anterior pituitary hormones, e.g. TSH, ACTH, FSH, LH, PRL, GH
    • A61P5/08—Drugs for disorders of the endocrine system of the anterior pituitary hormones, e.g. TSH, ACTH, FSH, LH, PRL, GH for decreasing, blocking or antagonising the activity of the anterior pituitary hormones
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A61P7/04—Antihaemorrhagics; Procoagulants; Haemostatic agents; Antifibrinolytic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A61P7/06—Antianaemics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/32—Special delivery means, e.g. tissue-specific

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Diabetes (AREA)
  • Molecular Biology (AREA)
  • Epidemiology (AREA)
  • Hematology (AREA)
  • Genetics & Genomics (AREA)
  • Biochemistry (AREA)
  • Biomedical Technology (AREA)
  • Obesity (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Cardiology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Endocrinology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Pain & Pain Management (AREA)
  • Rheumatology (AREA)
  • Child & Adolescent Psychology (AREA)
  • Virology (AREA)
  • Communicable Diseases (AREA)
CA3019981A 2016-04-05 2017-04-05 Nucleic acid linked to a trivalent glycoconjugate Pending CA3019981A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
EP16163939.8 2016-04-05
EP16163939.8A EP3228326A1 (en) 2016-04-05 2016-04-05 Nucleic acid linked to a trivalent glycoconjugate
PCT/EP2017/058112 WO2017174657A1 (en) 2016-04-05 2017-04-05 Nucleic acid linked to a trivalent glycoconjugate

Publications (1)

Publication Number Publication Date
CA3019981A1 true CA3019981A1 (en) 2017-10-12

Family

ID=55697082

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3019981A Pending CA3019981A1 (en) 2016-04-05 2017-04-05 Nucleic acid linked to a trivalent glycoconjugate

Country Status (11)

Country Link
US (2) US10913945B2 (enExample)
EP (3) EP3228326A1 (enExample)
JP (1) JP7120925B2 (enExample)
CN (2) CN116036117A (enExample)
AU (1) AU2017245687B2 (enExample)
CA (1) CA3019981A1 (enExample)
DK (1) DK3439703T3 (enExample)
ES (1) ES2805026T3 (enExample)
HU (1) HUE049931T2 (enExample)
PL (1) PL3439703T3 (enExample)
WO (1) WO2017174657A1 (enExample)

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MA45478A (fr) * 2016-04-11 2019-02-20 Arbutus Biopharma Corp Compositions de conjugués d'acides nucléiques ciblés
EP3385272A1 (en) * 2017-04-05 2018-10-10 Silence Therapeutics GmbH Further novel oligonucleotide-ligand conjugates
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EP3483269A1 (en) 2017-11-13 2019-05-15 Silence Therapeutics GmbH Products and compositions
WO2018185252A1 (en) 2017-04-05 2018-10-11 Silence Therapeutics Gmbh Nucleic acid conjugates
CA3056179A1 (en) 2017-04-05 2018-10-11 Silence Therapeutics Gmbh Products and compositions
WO2018185253A1 (en) * 2017-04-05 2018-10-11 Silence Therapeutics Gmbh Ligand modified double-stranded nucleic acids
CN110520531A (zh) 2017-04-05 2019-11-29 赛伦斯治疗有限责任公司 产品和组合物
EP3550022A1 (en) 2018-04-05 2019-10-09 Silence Therapeutics GmbH Products and compositions
WO2019092282A1 (en) 2017-11-13 2019-05-16 Silence Therapeutics Gmbh Nucleic acids for inhibiting expression of aldh2 in a cell
CA3081910A1 (en) * 2017-11-13 2019-05-16 Silence Therapeutics Gmbh Nucleic acids for inhibiting expression of a target gene comprising phosphorodithioate linkages
EP3483270A1 (en) 2017-11-13 2019-05-15 Silence Therapeutics GmbH Products and compositions
FI3710586T3 (fi) 2017-11-13 2023-03-02 Nukleiinihappoja LPA:n ekspression inhiboimiseksi solussa
EP3549610A1 (en) 2018-04-05 2019-10-09 Silence Therapeutics GmbH Nucleic acid conjugates
EP3775207A1 (en) 2018-04-05 2021-02-17 Silence Therapeutics GmbH Sirnas with vinylphosphonate at the 5' end of the antisense strand
EP3550021A1 (en) 2018-04-05 2019-10-09 Silence Therapeutics GmbH Products and compositions for inhibiting expression of a target gene
EP3598995A1 (en) 2018-07-26 2020-01-29 Silence Therapeutics GmbH Products and compositions
WO2020099476A1 (en) * 2018-11-13 2020-05-22 Silence Therapeutics Gmbh Nucleic acids for inhibiting expression of lpa in a cell
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US20220243202A1 (en) 2019-05-07 2022-08-04 Universität Bern Nucleic acids for inhibiting expression of pros1 in a cell
CN114981430A (zh) * 2019-08-27 2022-08-30 赛伦斯治疗有限责任公司 用于抑制细胞中的c3表达的核酸
KR20220058578A (ko) 2019-09-03 2022-05-09 아크투루스 쎄라퓨틱스, 인크. 치료 활성 접합체의 아시알로당단백질 수용체 매개 전달
IL292391A (en) * 2019-10-24 2022-06-01 Genevant Sciences Gmbh Bracelets and methods for treating acromegaly
US20240229041A2 (en) 2020-05-27 2024-07-11 Asociación Centro De Investigación Cooperativa En Biociencias-Cic Biogune Nucleic acids for inhibiting expression of cnnm4 in a cell
WO2022096424A1 (en) 2020-11-04 2022-05-12 Universität Bern Nucleic acids for inhibiting expression of pros1 in a cell
JP2023547512A (ja) 2020-11-04 2023-11-10 ウニベルジテート ベルン 細胞におけるｐｒｏｓ１の発現を阻害するための核酸
WO2022184852A1 (en) 2021-03-03 2022-09-09 Silence Therapeutics Gmbh Conjugated nucleic acids comprising a phosphorodithioate for inhibiting gene expression in a cell
WO2022223557A1 (en) 2021-04-20 2022-10-27 Silence Therapeutics Gmbh Nucleic acids for inhibiting expression of xdh in a cell
AU2022265493A1 (en) 2021-04-27 2023-09-21 Silence Therapeutics Gmbh Sirna targeting tmprss6 for the treatment of myeloproliferative disorders
CN118234861A (zh) 2021-09-02 2024-06-21 赛伦斯治疗有限责任公司 用于抑制细胞中的补体因子b(cfb)表达的核酸
US20240271141A1 (en) * 2021-11-29 2024-08-15 Shanghai Argo Biopharmaceutical Co., Ltd. Composition and method for inhibiting expression of hepatitis b virus (hbv)protein
WO2023208106A1 (zh) * 2022-04-29 2023-11-02 石药集团中奇制药技术（石家庄）有限公司 具有肝靶向递送效应的化合物及其寡聚核苷酸缀合物
WO2024013334A1 (en) * 2022-07-15 2024-01-18 Silence Therapeutics Gmbh Nucleic acids for inhibiting expression of agt in a cell
WO2024126687A1 (en) 2022-12-16 2024-06-20 Silence Therapeutics Gmbh Nucleic acids conjugated to an albumin binding moiety
CN120981247A (zh) 2023-03-27 2025-11-18 赛伦斯治疗有限责任公司 用于干细胞移植的化合物和组合物
CN116444589B (zh) * 2023-06-15 2023-09-19 北京悦康科创医药科技股份有限公司 新型GalNAc化合物及其硫代寡核苷酸缀合物和偶联方法
GB202311334D0 (en) 2023-07-24 2023-09-06 Astrazeneca Ab Multivalent cargo-carrying complexes and uses thereof
GB202311324D0 (en) 2023-07-24 2023-09-06 Astrazeneca Ab Multivalent cargo-carrying complexes and uses thereof
WO2025021831A1 (en) 2023-07-24 2025-01-30 Astrazeneca Ab Multivalent cargo-carrying complexes and uses thereof
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WO2025162939A1 (en) 2024-01-29 2025-08-07 Silence Therapeutics Gmbh Nucleic acids for inhibiting expression of gpr146 in a cell
WO2025163186A1 (en) 2024-02-01 2025-08-07 Silence Therapeutics Gmbh Conjugated nucleic acids and nucleic acids comprising locked nucleosides and inverted nucleotides for inhibiting gene expression in a cell
WO2025169133A1 (en) 2024-02-09 2025-08-14 Astrazeneca Ab Double-stranded rnai agents and compositions for reducing expression of angiopoietin-like 3 (angptl3) and methods of use thereof

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Also Published As

Publication number Publication date
PL3439703T3 (pl) 2020-10-19
AU2017245687B2 (en) 2023-03-09
EP3439703B1 (en) 2020-04-29
WO2017174657A1 (en) 2017-10-12
CN109562188B (zh) 2022-12-06
US20190119676A1 (en) 2019-04-25
JP2019510793A (ja) 2019-04-18
EP3744350A1 (en) 2020-12-02
EP3439703A1 (en) 2019-02-13
US20210079397A1 (en) 2021-03-18
HUE049931T2 (hu) 2020-11-30
ES2805026T3 (es) 2021-02-10
CN116036117A (zh) 2023-05-02
US10913945B2 (en) 2021-02-09
AU2017245687A1 (en) 2018-10-25
EP3228326A1 (en) 2017-10-11
DK3439703T3 (da) 2020-06-29
JP7120925B2 (ja) 2022-08-17
CN109562188A (zh) 2019-04-02
US11912993B2 (en) 2024-02-27

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EEER Examination request

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EEER Examination request

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EEER Examination request

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EEER Examination request

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EEER Examination request

Effective date: 20220331

EEER Examination request

Effective date: 20220331

EEER Examination request

Effective date: 20220331