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SMT201900571T1 - Terapia con deossinucleosidi per malattie causate da pool di nucleotidi sbilanciati tra cui le sindromi da deplezione del dna mitocondriale - Google Patents

Terapia con deossinucleosidi per malattie causate da pool di nucleotidi sbilanciati tra cui le sindromi da deplezione del dna mitocondriale

Info

Publication number
SMT201900571T1
SMT201900571T1 SM20190571T SMT201900571T SMT201900571T1 SM T201900571 T1 SMT201900571 T1 SM T201900571T1 SM 20190571 T SM20190571 T SM 20190571T SM T201900571 T SMT201900571 T SM T201900571T SM T201900571 T1 SMT201900571 T1 SM T201900571T1
Authority
SM
San Marino
Prior art keywords
diseases caused
mitochondrial dna
dna depletion
including mitochondrial
nucleotide pools
Prior art date
Application number
SM20190571T
Other languages
English (en)
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 SMT201900571T1 publication Critical patent/SMT201900571T1/it

Links

Classifications

    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7042Compounds having saccharide radicals and heterocyclic rings
    • A61K31/7052Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides
    • A61K31/706Compounds 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
    • A61K31/7064Compounds 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
    • A61K31/7068Compounds 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/33Heterocyclic compounds
    • A61K31/395Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/495Heterocyclic 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
    • A61K31/505Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim
    • A61K31/513Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim having oxo groups directly attached to the heterocyclic ring, e.g. cytosine
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7042Compounds having saccharide radicals and heterocyclic rings
    • A61K31/7052Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides
    • A61K31/706Compounds 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
    • A61K31/7064Compounds 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
    • A61K31/7068Compounds 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
    • A61K31/7072Compounds 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7042Compounds having saccharide radicals and heterocyclic rings
    • A61K31/7052Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides
    • A61K31/706Compounds 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
    • A61K31/7064Compounds 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
    • A61K31/7076Compounds 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7042Compounds having saccharide radicals and heterocyclic rings
    • A61K31/7052Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides
    • A61K31/706Compounds 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
    • A61K31/7064Compounds 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
    • A61K31/7076Compounds 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
    • A61K31/708Compounds 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • 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
    • AHUMAN NECESSITIES
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    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/0012Galenical forms characterised by the site of application
    • A61K9/0053Mouth and digestive tract, i.e. intraoral and peroral administration
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00Drugs for disorders of the muscular or neuromuscular system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00Drugs for disorders of the metabolism
    • A61P3/04Anorexiants; Antiobesity agents
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
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    • C12QMEASURING 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
    • C12Q1/00Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
    • C12Q1/68Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids
    • C12Q1/6813Hybridisation assays
    • C12Q1/6827Hybridisation assays for detection of mutation or polymorphism
    • GPHYSICS
    • G01MEASURING; TESTING
    • G01NINVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N33/00Investigating or analysing materials by specific methods not covered by groups G01N1/00 - G01N31/00
    • G01N33/48Biological material, e.g. blood, urine; Haemocytometers
    • G01N33/50Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K2300/00Mixtures or combinations of active ingredients, wherein at least one active ingredient is fully defined in groups A61K31/00 - A61K41/00

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  • Urology & Nephrology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Microbiology (AREA)
  • Wood Science & Technology (AREA)
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  • Physics & Mathematics (AREA)
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  • General Physics & Mathematics (AREA)
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SM20190571T 2015-06-17 2016-06-17 Terapia con deossinucleosidi per malattie causate da pool di nucleotidi sbilanciati tra cui le sindromi da deplezione del dna mitocondriale SMT201900571T1 (it)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201562180914P 2015-06-17 2015-06-17
EP16812537.5A EP3310362B1 (en) 2015-06-17 2016-06-17 Deoxynucleoside therapy for diseases caused by unbalanced nucleotide pools including mitochondrial dna depletion syndromes
PCT/US2016/038110 WO2016205671A1 (en) 2015-06-17 2016-06-17 Deoxynucleoside therapy for diseases caused by unbalanced nucleotide pools including mitochondrial dna depletion syndromes

Publications (1)

Publication Number Publication Date
SMT201900571T1 true SMT201900571T1 (it) 2019-11-13

Family

ID=57546446

Family Applications (2)

Application Number Title Priority Date Filing Date
SM20200399T SMT202000399T1 (it) 2015-06-17 2016-06-17 Terapia con deossinucleosidi per malattie causate da pool di nucleotidi sbilanciati tra cui le sindromi da deplezione del dna mitocondriale
SM20190571T SMT201900571T1 (it) 2015-06-17 2016-06-17 Terapia con deossinucleosidi per malattie causate da pool di nucleotidi sbilanciati tra cui le sindromi da deplezione del dna mitocondriale

Family Applications Before (1)

Application Number Title Priority Date Filing Date
SM20200399T SMT202000399T1 (it) 2015-06-17 2016-06-17 Terapia con deossinucleosidi per malattie causate da pool di nucleotidi sbilanciati tra cui le sindromi da deplezione del dna mitocondriale

Country Status (24)

Country Link
US (5) US10471087B2 (it)
EP (3) EP3569236A1 (it)
JP (3) JP6599484B2 (it)
KR (3) KR20180039624A (it)
CN (2) CN116726035A (it)
AU (3) AU2016280293B2 (it)
BR (2) BR112017027079B1 (it)
CA (1) CA2989653A1 (it)
CY (2) CY1122605T1 (it)
DK (2) DK3310362T3 (it)
ES (2) ES2808148T3 (it)
HK (1) HK1252133A1 (it)
HR (2) HRP20191794T1 (it)
HU (2) HUE046399T2 (it)
IL (3) IL275256B2 (it)
LT (2) LT3310362T (it)
MX (2) MX389016B (it)
PL (2) PL3310362T3 (it)
PT (2) PT3505174T (it)
RS (2) RS59724B1 (it)
RU (1) RU2721492C2 (it)
SI (2) SI3310362T1 (it)
SM (2) SMT202000399T1 (it)
WO (1) WO2016205671A1 (it)

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
PL3310362T3 (pl) * 2015-06-17 2020-04-30 The Trustees Of Columbia University In The City Of New York Terapia deoksynukleozydowa chorób spowodowanych przez niezrównoważoną pulę nukleotydów włączając zespół deplecji mitochondrialnego DNA
KR20180095674A (ko) 2015-12-22 2018-08-27 조게닉스 인터내셔널 리미티드 대사 저항성 펜플루라민 유사체 및 그 사용 방법
SG11201804811WA (en) 2015-12-22 2018-07-30 Zogenix International Ltd Fenfluramine compositions and methods of preparing the same
RU2746000C2 (ru) 2016-08-24 2021-04-05 Зодженикс Интернэшнл Лимитед Состав для ингибирования образования агонистов 5-ht2b и способы его применения
US11649442B2 (en) 2017-09-08 2023-05-16 The Regents Of The University Of California RNA-guided endonuclease fusion polypeptides and methods of use thereof
US10682317B2 (en) 2017-09-26 2020-06-16 Zogenix International Limited Ketogenic diet compatible fenfluramine formulation
IL277725B2 (en) * 2018-04-12 2024-02-01 Modis Therapeutics Inc Pro-drugs of deoxynucleosides for the treatment of diseases resulting from an imbalance in the nucleotide reserves
KR20210009317A (ko) 2018-04-18 2021-01-26 더 트러스티스 오브 콜롬비아 유니버시티 인 더 시티 오브 뉴욕 미토콘드리아 dna 고갈 증후군을 포함하는 불균형 뉴클레오타이드 풀에 의해 초래된 질환에 대한 유전자 요법
JP2021526507A (ja) 2018-05-11 2021-10-07 ゾゲニクス インターナショナル リミテッド 発作により誘発される突然死を処置するための組成物および方法
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
WO2021046355A1 (en) * 2019-09-05 2021-03-11 Mitorainbow Therapeutics, Inc. Treating mitochondrial dna depletion disorders
WO2021188840A1 (en) * 2020-03-19 2021-09-23 Rewrite Therapeutics, Inc. Methods and compositions for directed genome editing
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
BR112023017619A2 (pt) * 2021-03-26 2023-12-05 Zogenix Mds Inc Soluções aquosas contendo purinas e pirimidinas e seus usos
AU2022291783A1 (en) * 2021-06-18 2024-02-01 Ucb Biosciences, Inc. Prodrugs of deoxynucleosides for treatment of mitochondrial diseases caused by unbalanced nucleotide pools
CN119730857A (zh) * 2022-07-12 2025-03-28 优西比生物科学股份有限公司 提高核苷类药物物质的生物利用度的方法
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
RU2300381C2 (ru) * 1998-08-10 2007-06-10 Айденикс (Кайман) Лимитед β-L-2'-ДЕЗОКСИНУКЛЕОЗИДЫ ДЛЯ ЛЕЧЕНИЯ ГЕПАТИТА В
KR100879559B1 (ko) * 1999-02-23 2009-01-22 더 리젠츠 오브 더 유니버시티 오브 캘리포니아 미토콘드리아 장애의 치료 또는 예방용 제약조성물
CN100490818C (zh) * 2000-05-26 2009-05-27 诺瓦蒂斯有限公司 β-L-2′-脱氧核苷在制备用于治疗丁型肝炎病毒感染的药物中的用途
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
PT3302499T (pt) * 2015-06-05 2023-05-12 Fundacio Hospital Univ Vall Dhebron Institut De Recerca Tratamento de doenças mitocondriais
PL3310362T3 (pl) * 2015-06-17 2020-04-30 The Trustees Of Columbia University In The City Of New York Terapia deoksynukleozydowa chorób spowodowanych przez niezrównoważoną pulę nukleotydów włączając zespół deplecji mitochondrialnego DNA

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PL3310362T3 (pl) 2020-04-30
BR112017027079B1 (pt) 2021-04-27
CA2989653A1 (en) 2016-12-22
AU2021250841A1 (en) 2021-11-04
PT3310362T (pt) 2019-10-29
RS59724B1 (sr) 2020-01-31
IL275255B (en) 2021-04-29
EP3310362A1 (en) 2018-04-25
EP3310362B1 (en) 2019-08-28
US20200038424A1 (en) 2020-02-06
EP3505174A1 (en) 2019-07-03
US20230277577A1 (en) 2023-09-07
HRP20191794T1 (hr) 2020-02-07
ES2808148T3 (es) 2021-02-25
RU2020112901A (ru) 2020-06-05
KR20180039624A (ko) 2018-04-18
WO2016205671A9 (en) 2018-01-04
HRP20200949T1 (hr) 2020-10-02
AU2016280293A1 (en) 2018-02-08
KR20220018623A (ko) 2022-02-15
PT3505174T (pt) 2020-07-06
DK3505174T3 (da) 2020-07-13
JP6599484B2 (ja) 2019-10-30
LT3505174T (lt) 2020-09-10
US20210244754A1 (en) 2021-08-12
US20250360154A1 (en) 2025-11-27
EP3505174B1 (en) 2020-04-29
BR122020021913B1 (pt) 2021-07-06
US10471087B2 (en) 2019-11-12
IL275256A (en) 2020-07-30
PL3505174T3 (pl) 2020-08-24
EP3310362A4 (en) 2018-12-19
MX2017016425A (es) 2018-12-11
RS60572B1 (sr) 2020-08-31
SI3505174T1 (sl) 2021-02-26
DK3310362T3 (da) 2019-10-07
HUE046399T2 (hu) 2020-03-30
EP3569236A1 (en) 2019-11-20
HUE050678T2 (hu) 2020-12-28
JP7036782B2 (ja) 2022-03-15
CY1122605T1 (el) 2021-03-12
JP6675037B2 (ja) 2020-04-01
JP2020019791A (ja) 2020-02-06
IL256331B (en) 2020-08-31
US20180133241A1 (en) 2018-05-17
IL275255A (en) 2020-07-30
JP2020019792A (ja) 2020-02-06
IL256331A (en) 2018-02-28
MX389016B (es) 2025-03-20
KR20250048602A (ko) 2025-04-09
AU2021250841B2 (en) 2023-02-09
IL275256B1 (en) 2023-05-01
CN107847512A (zh) 2018-03-27
SI3310362T1 (sl) 2020-02-28
US12251392B2 (en) 2025-03-18
AU2020204042B2 (en) 2021-07-15
HK1252133A1 (zh) 2019-05-17
RU2018101305A3 (it) 2019-10-31
RU2721492C2 (ru) 2020-05-19
MX2020000269A (es) 2022-01-03
JP2018521993A (ja) 2018-08-09
ES2748556T3 (es) 2020-03-17
AU2020204042A1 (en) 2020-07-09
LT3310362T (lt) 2019-11-11
US11110111B2 (en) 2021-09-07
RU2018101305A (ru) 2019-07-17
CY1123107T1 (el) 2021-10-29
AU2016280293B2 (en) 2020-07-02
WO2016205671A1 (en) 2016-12-22
CN116726035A (zh) 2023-09-12
US11666592B2 (en) 2023-06-06
SMT202000399T1 (it) 2020-09-10
IL275256B2 (en) 2023-09-01

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