HUE050678T2 - Deoxinukleozid terápia ingadozó nukleotid pool által okozott betegségekhez, ideértve a mitokondriális DNS depléciós szindrómát - Google Patents

Deoxinukleozid terápia ingadozó nukleotid pool által okozott betegségekhez, ideértve a mitokondriális DNS depléciós szindrómát

Info

Publication number
HUE050678T2
HUE050678T2 HUE19156021A HUE19156021A HUE050678T2 HU E050678 T2 HUE050678 T2 HU E050678T2 HU E19156021 A HUE19156021 A HU E19156021A HU E19156021 A HUE19156021 A HU E19156021A HU E050678 T2 HUE050678 T2 HU E050678T2
Authority
HU
Hungary
Prior art keywords
diseases caused
mitochondrial dna
dna depletion
including mitochondrial
nucleotide pools
Prior art date
Application number
HUE19156021A
Other languages
English (en)
Hungarian (hu)
Inventor
Michio Hirano
Caterina Garone
Ramon Marti
Original Assignee
Univ Columbia
Fundacio Hospital Univ Vall Dhebron Institut De Recerca
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 Univ Columbia, Fundacio Hospital Univ Vall Dhebron Institut De Recerca filed Critical Univ Columbia
Publication of HUE050678T2 publication Critical patent/HUE050678T2/hu

Links

Classifications

    • 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
    • A61K31/7042—Compounds having saccharide radicals and heterocyclic rings
    • A61K31/7052—Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides
    • A61K31/706—Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides containing six-membered rings with nitrogen as a ring hetero atom
    • A61K31/7064—Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides containing six-membered rings with nitrogen as a ring hetero atom containing condensed or non-condensed pyrimidines
    • A61K31/7068—Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides containing six-membered rings with nitrogen as a ring hetero atom containing condensed or non-condensed pyrimidines having oxo groups directly attached to the pyrimidine ring, e.g. cytidine, cytidylic acid
    • 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
    • A61K31/33—Heterocyclic compounds
    • A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/495—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with two or more nitrogen atoms as the only ring heteroatoms, e.g. piperazine or tetrazines
    • A61K31/505—Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim
    • A61K31/513—Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim having oxo groups directly attached to the heterocyclic ring, e.g. cytosine
    • 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
    • A61K31/7042—Compounds having saccharide radicals and heterocyclic rings
    • A61K31/7052—Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides
    • A61K31/706—Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides containing six-membered rings with nitrogen as a ring hetero atom
    • A61K31/7064—Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides containing six-membered rings with nitrogen as a ring hetero atom containing condensed or non-condensed pyrimidines
    • A61K31/7068—Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides containing six-membered rings with nitrogen as a ring hetero atom containing condensed or non-condensed pyrimidines having oxo groups directly attached to the pyrimidine ring, e.g. cytidine, cytidylic acid
    • A61K31/7072—Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides containing six-membered rings with nitrogen as a ring hetero atom containing condensed or non-condensed pyrimidines having oxo groups directly attached to the pyrimidine ring, e.g. cytidine, cytidylic acid having two oxo groups directly attached to the pyrimidine ring, e.g. uridine, uridylic acid, thymidine, zidovudine
    • 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
    • A61K31/7042—Compounds having saccharide radicals and heterocyclic rings
    • A61K31/7052—Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides
    • A61K31/706—Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides containing six-membered rings with nitrogen as a ring hetero atom
    • A61K31/7064—Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides containing six-membered rings with nitrogen as a ring hetero atom containing condensed or non-condensed pyrimidines
    • A61K31/7076—Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides containing six-membered rings with nitrogen as a ring hetero atom containing condensed or non-condensed pyrimidines containing purines, e.g. adenosine, adenylic acid
    • 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
    • A61K31/7042—Compounds having saccharide radicals and heterocyclic rings
    • A61K31/7052—Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides
    • A61K31/706—Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides containing six-membered rings with nitrogen as a ring hetero atom
    • A61K31/7064—Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides containing six-membered rings with nitrogen as a ring hetero atom containing condensed or non-condensed pyrimidines
    • A61K31/7076—Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides containing six-membered rings with nitrogen as a ring hetero atom containing condensed or non-condensed pyrimidines containing purines, e.g. adenosine, adenylic acid
    • A61K31/708—Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides containing six-membered rings with nitrogen as a ring hetero atom containing condensed or non-condensed pyrimidines containing purines, e.g. adenosine, adenylic acid having oxo groups directly attached to the purine ring system, e.g. guanosine, guanylic acid
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0053—Mouth and digestive tract, i.e. intraoral and peroral administration
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • 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
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Q—MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
    • C12Q1/00—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
    • C12Q1/68—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids
    • C12Q1/6813—Hybridisation assays
    • C12Q1/6827—Hybridisation assays for detection of mutation or polymorphism
    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N33/00—Investigating or analysing materials by specific methods not covered by groups G01N1/00 - G01N31/00
    • G01N33/48—Biological material, e.g. blood, urine; Haemocytometers
    • G01N33/50—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K2300/00—Mixtures or combinations of active ingredients, wherein at least one active ingredient is fully defined in groups A61K31/00 - A61K41/00

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Molecular Biology (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Neurology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Hematology (AREA)
  • Biotechnology (AREA)
  • Immunology (AREA)
  • Nutrition Science (AREA)
  • Physiology (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Zoology (AREA)
  • Analytical Chemistry (AREA)
  • Wood Science & Technology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Microbiology (AREA)
  • Urology & Nephrology (AREA)
  • Physics & Mathematics (AREA)
  • Biochemistry (AREA)
  • Obesity (AREA)
  • Child & Adolescent Psychology (AREA)
  • Diabetes (AREA)
  • Biophysics (AREA)
  • General Physics & Mathematics (AREA)
HUE19156021A 2015-06-17 2016-06-17 Deoxinukleozid terápia ingadozó nukleotid pool által okozott betegségekhez, ideértve a mitokondriális DNS depléciós szindrómát HUE050678T2 (hu)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US201562180914P 2015-06-17 2015-06-17

Publications (1)

Publication Number Publication Date
HUE050678T2 true HUE050678T2 (hu) 2020-12-28

Family

ID=57546446

Family Applications (2)

Application Number Title Priority Date Filing Date
HUE19156021A HUE050678T2 (hu) 2015-06-17 2016-06-17 Deoxinukleozid terápia ingadozó nukleotid pool által okozott betegségekhez, ideértve a mitokondriális DNS depléciós szindrómát
HUE16812537A HUE046399T2 (hu) 2015-06-17 2016-06-17 Deoxinukleozid terápia kiegyensúlyozatlan nukleotid pool által okozott betegségek, ideértve a mitokondriális DNS depléciós szindrómát, kezelésére

Family Applications After (1)

Application Number Title Priority Date Filing Date
HUE16812537A HUE046399T2 (hu) 2015-06-17 2016-06-17 Deoxinukleozid terápia kiegyensúlyozatlan nukleotid pool által okozott betegségek, ideértve a mitokondriális DNS depléciós szindrómát, kezelésére

Country Status (24)

Country Link
US (5) US10471087B2 (enExample)
EP (3) EP3505174B1 (enExample)
JP (3) JP6599484B2 (enExample)
KR (3) KR20220018623A (enExample)
CN (2) CN116726035A (enExample)
AU (3) AU2016280293B2 (enExample)
BR (2) BR112017027079B1 (enExample)
CA (1) CA2989653A1 (enExample)
CY (2) CY1122605T1 (enExample)
DK (2) DK3505174T3 (enExample)
ES (2) ES2808148T3 (enExample)
HK (1) HK1252133A1 (enExample)
HR (2) HRP20191794T1 (enExample)
HU (2) HUE050678T2 (enExample)
IL (3) IL275256B2 (enExample)
LT (2) LT3505174T (enExample)
MX (2) MX389016B (enExample)
PL (2) PL3310362T3 (enExample)
PT (2) PT3505174T (enExample)
RS (2) RS60572B1 (enExample)
RU (1) RU2721492C2 (enExample)
SI (2) SI3505174T1 (enExample)
SM (2) SMT202000399T1 (enExample)
WO (1) WO2016205671A1 (enExample)

Families Citing this family (22)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US9549909B2 (en) 2013-05-03 2017-01-24 The Katholieke Universiteit Leuven Method for the treatment of dravet syndrome
US10292996B2 (en) 2015-03-26 2019-05-21 The Trustees Of Columbia University In The City Of New York Deoxyribonucleoside monophospate bypass therapy for mitochondrial DNA depletion syndrome
LT3505174T (lt) * 2015-06-17 2020-09-10 The Trustees Of Columbia University In The City Of New York Deoksinukleozidų terapija, skirta gydyti ligas, kurias sukelia nesubalansuoti nukleotidų kiekiai, įskaitant mitochondrinės dnr išsekimo sindromus
US20170174614A1 (en) 2015-12-22 2017-06-22 Zogenix International Limited Metabolism resistant fenfluramine analogs and methods of using the same
CN108883399B (zh) 2015-12-22 2021-06-22 周格尼克斯国际有限公司 芬氟拉明组合物及其制备方法
RU2021106383A (ru) 2016-08-24 2021-04-05 Зодженикс Интернэшнл Лимитед Состав для ингибирования образования агонистов 5-ht2b и способы его применения
WO2019051097A1 (en) 2017-09-08 2019-03-14 The Regents Of The University Of California RNA-GUIDED ENDONUCLEASE FUSION POLYPEPTIDES AND METHODS OF USING SAME
US10682317B2 (en) 2017-09-26 2020-06-16 Zogenix International Limited Ketogenic diet compatible fenfluramine formulation
US11628182B2 (en) * 2018-04-12 2023-04-18 Zogenix Mds, Inc. Prodrugs of deoxynucleosides for treatment of diseases cased by unbalanced nucleotide pools
BR112020021324A2 (pt) 2018-04-18 2021-01-19 The Trustees Of Columbia University In The City Of New York Terapia gênica para doenças causadas por pools de nucleotídeos em desequilíbrio incluindo síndromes de depleção do dna mitocondrial
WO2019216919A1 (en) 2018-05-11 2019-11-14 Zogenix International Limited Compositions and methods for treating seizure-induced sudden death
US12144787B2 (en) 2018-11-19 2024-11-19 Zogenix International Limited Method of treating patients with a mutation in cyclin-dependent kinase-like 5 (CDKL5)
JP7769603B2 (ja) * 2019-08-19 2025-11-13 ユーシービー バイオサイエンシズ，インコーポレイテッド デオキシシチジンの多形形態、それを含む組成物および使用
JP2022546611A (ja) * 2019-09-05 2022-11-04 ミトレインボー セラピューティクス，インコーポレーテッド ミトコンドリアｄｎａ枯渇障害の処置
CN115667505A (zh) 2020-03-19 2023-01-31 因特利亚治疗公司 用于定向基因组编辑的方法和组合物
US11612574B2 (en) 2020-07-17 2023-03-28 Zogenix International Limited Method of treating patients infected with severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2)
US20220096514A1 (en) * 2020-09-29 2022-03-31 Zogenix International Limited Methods of treating thymidine kinase 2 deficiency by administering deoxycytidine and deoxythymidine
WO2022145439A1 (ja) * 2020-12-28 2022-07-07 ヤマサ醤油株式会社 筋萎縮抑制剤及び筋萎縮を抑制させる方法
JP2024515025A (ja) 2021-03-26 2024-04-04 ゾジェニックス，インコーポレイテッド プリン及びピリミジンを含有する水性溶液及びその使用
AU2022291783A1 (en) * 2021-06-18 2024-02-01 Ucb Biosciences, Inc. Prodrugs of deoxynucleosides for treatment of mitochondrial diseases caused by unbalanced nucleotide pools
JP2025522100A (ja) * 2022-07-12 2025-07-10 ユーシービー バイオサイエンシス、インコーポレイテッド ヌクレオシド薬剤のバイオアベイラビリティを増加させる方法
WO2024206595A2 (en) * 2023-03-30 2024-10-03 The Trustees Of Columbia University In The City Of New York Pharmacological therapy for mitochondrial dna depletion deletions syndrome involving mutations in the guk1 gene

Family Cites Families (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN101120947B (zh) * 1998-08-10 2010-11-24 艾丹尼克斯(开曼)有限公司 用于治疗乙型肝炎的β-L-2′-脱氧-核苷
ES2298130T3 (es) * 1999-02-23 2008-05-16 The Regents Of The University Of California Uso de triacetiluridina para el tratamiento de trastornos mitocondriales.
ATE275406T1 (de) * 2000-05-26 2004-09-15 Idenix Cayman Ltd Methoden zur behandlung von delta hepatitis virus infektionen mit beta-l-2' deoxy-nucleosiden
WO2012125848A2 (en) 2011-03-16 2012-09-20 Baylor College Of Medicine A method for comprehensive sequence analysis using deep sequencing technology
US10292996B2 (en) 2015-03-26 2019-05-21 The Trustees Of Columbia University In The City Of New York Deoxyribonucleoside monophospate bypass therapy for mitochondrial DNA depletion syndrome
IL289747B2 (en) * 2015-06-05 2025-10-01 Fundacio Hospital Univ Vall Dhebron Institut De Recerca Combination of deoxyribonucleosides for the treatment of mitochondrial DNA depletion syndrome and/or deletion syndrome
LT3505174T (lt) * 2015-06-17 2020-09-10 The Trustees Of Columbia University In The City Of New York Deoksinukleozidų terapija, skirta gydyti ligas, kurias sukelia nesubalansuoti nukleotidų kiekiai, įskaitant mitochondrinės dnr išsekimo sindromus

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Publication number Publication date
JP2020019792A (ja) 2020-02-06
CY1122605T1 (el) 2021-03-12
AU2020204042A1 (en) 2020-07-09
PL3310362T3 (pl) 2020-04-30
DK3505174T3 (da) 2020-07-13
SMT201900571T1 (it) 2019-11-13
EP3310362B1 (en) 2019-08-28
AU2020204042B2 (en) 2021-07-15
RU2721492C2 (ru) 2020-05-19
JP6675037B2 (ja) 2020-04-01
RS59724B1 (sr) 2020-01-31
US12251392B2 (en) 2025-03-18
RU2018101305A (ru) 2019-07-17
EP3310362A1 (en) 2018-04-25
IL275256B2 (en) 2023-09-01
HRP20200949T1 (hr) 2020-10-02
US20250360154A1 (en) 2025-11-27
EP3505174B1 (en) 2020-04-29
IL275256B1 (en) 2023-05-01
EP3310362A4 (en) 2018-12-19
WO2016205671A1 (en) 2016-12-22
PL3505174T3 (pl) 2020-08-24
EP3505174A1 (en) 2019-07-03
HK1252133A1 (zh) 2019-05-17
PT3505174T (pt) 2020-07-06
US20210244754A1 (en) 2021-08-12
SI3505174T1 (sl) 2021-02-26
US20200038424A1 (en) 2020-02-06
JP7036782B2 (ja) 2022-03-15
US20230277577A1 (en) 2023-09-07
PT3310362T (pt) 2019-10-29
ES2748556T3 (es) 2020-03-17
CY1123107T1 (el) 2021-10-29
JP2020019791A (ja) 2020-02-06
JP2018521993A (ja) 2018-08-09
ES2808148T3 (es) 2021-02-25
IL275256A (en) 2020-07-30
RU2020112901A (ru) 2020-06-05
HUE046399T2 (hu) 2020-03-30
EP3569236A1 (en) 2019-11-20
KR20250048602A (ko) 2025-04-09
IL275255A (en) 2020-07-30
IL256331A (en) 2018-02-28
CN116726035A (zh) 2023-09-12
AU2016280293B2 (en) 2020-07-02
BR122020021913B1 (pt) 2021-07-06
SMT202000399T1 (it) 2020-09-10
AU2021250841B2 (en) 2023-02-09
RU2018101305A3 (enExample) 2019-10-31
BR112017027079B1 (pt) 2021-04-27
DK3310362T3 (da) 2019-10-07
IL256331B (en) 2020-08-31
WO2016205671A9 (en) 2018-01-04
HRP20191794T1 (hr) 2020-02-07
US11110111B2 (en) 2021-09-07
RS60572B1 (sr) 2020-08-31
IL275255B (en) 2021-04-29
AU2016280293A1 (en) 2018-02-08
LT3505174T (lt) 2020-09-10
LT3310362T (lt) 2019-11-11
KR20220018623A (ko) 2022-02-15
US20180133241A1 (en) 2018-05-17
US10471087B2 (en) 2019-11-12
MX2020000269A (es) 2022-01-03
SI3310362T1 (sl) 2020-02-28
AU2021250841A1 (en) 2021-11-04
MX2017016425A (es) 2018-12-11
JP6599484B2 (ja) 2019-10-30
CA2989653A1 (en) 2016-12-22
US11666592B2 (en) 2023-06-06
KR20180039624A (ko) 2018-04-18
MX389016B (es) 2025-03-20
CN107847512A (zh) 2018-03-27

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