LT3717646T - Angelmano sindromo gydymas priešprasme nukleorūgštimi - Google Patents

Angelmano sindromo gydymas priešprasme nukleorūgštimi

Info

Publication number
LT3717646T
LT3717646T LTEPPCT/US2018/063416T LTUS2018063416T LT3717646T LT 3717646 T LT3717646 T LT 3717646T LT US2018063416 T LTUS2018063416 T LT US2018063416T LT 3717646 T LT3717646 T LT 3717646T
Authority
LT
Lithuania
Prior art keywords
industry
treatment
nucleic acid
angelman syndrome
angelman
Prior art date
Application number
LTEPPCT/US2018/063416T
Other languages
English (en)
Lithuanian (lt)
Inventor
Scott Victor DINDOT
Original Assignee
The Texas A&M University System
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 The Texas A&M University System filed Critical The Texas A&M University System
Publication of LT3717646T publication Critical patent/LT3717646T/lt

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    • C—CHEMISTRY; METALLURGY
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    • 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
    • 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
    • A61K48/005—Medicinal 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • 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
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C07H21/02—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with ribosyl as saccharide radical
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C07H21/04—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with deoxyribosyl as saccharide radical
    • 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
    • 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
    • C12N2310/11—Antisense
    • C12N2310/113—Antisense targeting other non-coding nucleic acids, e.g. antagomirs
    • 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
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/322—2'-R Modification
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/33—Chemical structure of the base
    • C12N2310/334—Modified C
    • C12N2310/3341—5-Methylcytosine
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/341—Gapmers, i.e. of the type ===---===
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3521—Methyl
    • 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
    • C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3525—MOE, methoxyethoxy
    • 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

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  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Molecular Biology (AREA)
  • Organic Chemistry (AREA)
  • Biochemistry (AREA)
  • Biotechnology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Epidemiology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Medicinal Preparation (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
LTEPPCT/US2018/063416T 2017-12-01 2018-11-30 Angelmano sindromo gydymas priešprasme nukleorūgštimi LT3717646T (lt)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201762593431P 2017-12-01 2017-12-01
US201862676034P 2018-05-24 2018-05-24
PCT/US2018/063416 WO2019109001A1 (en) 2017-12-01 2018-11-30 Angelman syndrome antisense treatment

Publications (1)

Publication Number Publication Date
LT3717646T true LT3717646T (lt) 2025-06-10

Family

ID=66664231

Family Applications (1)

Application Number Title Priority Date Filing Date
LTEPPCT/US2018/063416T LT3717646T (lt) 2017-12-01 2018-11-30 Angelmano sindromo gydymas priešprasme nukleorūgštimi

Country Status (27)

Country Link
US (5) US11198869B2 (https=)
EP (2) EP4328306A3 (https=)
JP (3) JP7297320B2 (https=)
KR (2) KR20240161203A (https=)
CN (2) CN111433361B (https=)
AU (1) AU2018375807B2 (https=)
BR (1) BR112020009431A2 (https=)
CA (1) CA3079755A1 (https=)
CL (1) CL2020001344A1 (https=)
CO (1) CO2020006361A2 (https=)
DK (1) DK3717646T3 (https=)
ES (1) ES3032315T3 (https=)
FI (1) FI3717646T3 (https=)
HR (1) HRP20250804T1 (https=)
HU (1) HUE072645T2 (https=)
IL (1) IL274146B2 (https=)
LT (1) LT3717646T (https=)
MX (2) MX2020005680A (https=)
PE (1) PE20211238A1 (https=)
PL (1) PL3717646T3 (https=)
PT (1) PT3717646T (https=)
RS (1) RS66986B1 (https=)
RU (1) RU2020121752A (https=)
SG (1) SG11202003596WA (https=)
SI (1) SI3717646T1 (https=)
SM (1) SMT202500334T1 (https=)
WO (1) WO2019109001A1 (https=)

Families Citing this family (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
HUE067905T2 (hu) 2015-11-12 2024-11-28 Hoffmann La Roche Oligonukleotidok paternális UBE3A expresszió indukálására
CN111770758A (zh) * 2018-02-27 2020-10-13 北卡罗来纳-查佩尔山大学 用于治疗Angelman综合征的方法和组合物
FI3947684T3 (fi) 2019-03-29 2025-05-27 Ionis Pharmaceuticals Inc Yhdisteitä ja menetelmiä ube3a-ats:n moduloimiseksi
BR112022024206A2 (pt) * 2020-06-29 2023-01-03 Ionis Pharmaceuticals Inc Compostos e métodos para modular plp1
US20230416851A1 (en) * 2020-10-06 2023-12-28 University Of Maryland, Baltimore Rapid diagnostic electrochemical biosensing targeted with antisense oligonucleotides
IL315359A (en) * 2022-03-07 2024-11-01 Univ Connecticut shRNA TARGETING SNORD115 LOCATIONS TO RESTORE PATERNAL UBE3A GENE EXPRESSION IN ANGELMAN SYNDROME
WO2024162126A1 (ja) 2023-01-31 2024-08-08 富士フイルム株式会社 硬化性組成物、硬化物、光学材料、回折光学素子及び化合物
KR20250068540A (ko) * 2023-11-08 2025-05-16 한국과학기술원 Ube3a를 표적으로 하여 발현증강을 유도하는 안티센스 올리고뉴클레오티드 및 이의 용도
TW202548015A (zh) * 2024-04-11 2025-12-16 美商奧崔基尼克斯製藥公司 天使症候群反義治療的給藥方案

Family Cites Families (29)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1993007883A1 (en) 1991-10-24 1993-04-29 Isis Pharmaceuticals, Inc. Derivatized oligonucleotides having improved uptake and other properties
US20040259247A1 (en) * 2000-12-01 2004-12-23 Thomas Tuschl Rna interference mediating small rna molecules
EP2397563A3 (en) 2004-09-17 2012-07-18 Isis Pharmaceuticals, Inc. Enhanced antisense oligonucleotides
EP1676834A1 (en) 2004-12-30 2006-07-05 Sanofi-Aventis Deutschland GmbH Fused bicyclic carboxamide derivates for use as CXCR2 inhibitors in the treatment of inflammation
WO2007031091A2 (en) 2005-09-15 2007-03-22 Santaris Pharma A/S Rna antagonist compounds for the modulation of p21 ras expression
US8178503B2 (en) * 2006-03-03 2012-05-15 International Business Machines Corporation Ribonucleic acid interference molecules and binding sites derived by analyzing intergenic and intronic regions of genomes
US20080299659A1 (en) * 2007-03-02 2008-12-04 Nastech Pharmaceutical Company Inc. Nucleic acid compounds for inhibiting apob gene expression and uses thereof
WO2008113832A2 (en) 2007-03-22 2008-09-25 Santaris Pharma A/S SHORT RNA ANTAGONIST COMPOUNDS FOR THE MODULATION OF TARGET mRNA
DK2285819T3 (da) 2008-04-04 2013-12-02 Isis Pharmaceuticals Inc Oligomere forbindelser omfattende neutralt bundne, terminale bicykliske nukleosider
RU2572826C2 (ru) 2008-12-02 2016-01-20 Чиралджен, Лтд. Способ синтеза модифицированных по атому фосфора нуклеиновых кислот
EP2462153B1 (en) 2009-08-06 2015-07-29 Isis Pharmaceuticals, Inc. Bicyclic cyclohexose nucleic acid analogs
US20120282176A1 (en) 2011-04-20 2012-11-08 Roche Glycart Ag Method and Constructs for the pH Dependent Passage of the Blood-brain-barrier
EP3453761A1 (en) 2011-08-29 2019-03-13 Ionis Pharmaceuticals, Inc. Oligomer-conjugate complexes and their use
EP2850092B1 (en) 2012-04-09 2017-03-01 Ionis Pharmaceuticals, Inc. Tricyclic nucleic acid analogs
US20150141320A1 (en) * 2012-05-16 2015-05-21 Rana Therapeutics, Inc. Compositions and methods for modulating gene expression
BR112014028634A2 (pt) * 2012-05-16 2017-06-27 Rana Therapeutics Inc composições e métodos para modulação da expressão de utrn
ES2809199T3 (es) 2012-06-25 2021-03-03 Ionis Pharmaceuticals Inc Modulación de la expresión de UBE3A-ATS
JP6453212B2 (ja) 2012-07-13 2019-01-16 ウェイブ ライフ サイエンシズ リミテッドＷａｖｅ Ｌｉｆｅ Ｓｃｉｅｎｃｅｓ Ｌｔｄ． キラル制御
ES2917473T3 (es) 2014-01-16 2022-07-08 Wave Life Sciences Ltd Diseño quiral
JP2017536366A (ja) 2014-11-19 2017-12-07 ロシュ イノベーション センター コペンハーゲン エーエス Ｌｎａキラルホスホロチオエート
WO2016086104A1 (en) 2014-11-25 2016-06-02 Ionis Pharmaceuticals, Inc. Modulation of ube3a-ats expression
CN107208092B (zh) 2014-12-16 2021-09-10 罗氏创新中心哥本哈根有限公司 手性毒性筛选方法
AU2016215155A1 (en) * 2015-02-04 2017-08-17 F. Hoffmann-La Roche Ag Tau antisense oligomers and uses thereof
EP3286318A2 (en) * 2015-04-22 2018-02-28 Mina Therapeutics Limited Sarna compositions and methods of use
HUE067905T2 (hu) 2015-11-12 2024-11-28 Hoffmann La Roche Oligonukleotidok paternális UBE3A expresszió indukálására
CN109311925B (zh) 2016-05-12 2022-06-03 罗氏创新中心哥本哈根有限公司 立体限定的氧杂氮杂磷杂环戊烷亚磷酰胺单体与核苷或寡核苷酸的增强的偶联
US10414747B2 (en) 2016-10-04 2019-09-17 Merck Sharp & Dohme Corp. Benzo[b]thiophene compounds as sting agonists
JP7164540B2 (ja) 2017-03-29 2022-11-01 ロシュ イノベーション センター コペンハーゲン エーエス 立体的に規定されたホスホロチオエートオリゴヌクレオチドの調製のための直交保護基
JP2020524718A (ja) 2017-06-22 2020-08-20 キュラデブ・ファーマ・リミテッドＣｕｒａｄｅｖ Ｐｈａｒｍａ Ｌｉｍｉｔｅｄ ヒトｓｔｉｎｇの複素環式小分子調節因子

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KR102723989B1 (ko) 2024-11-01
CO2020006361A2 (es) 2020-08-10
JP7297320B2 (ja) 2023-06-26
JP2025041632A (ja) 2025-03-26
JP7601441B2 (ja) 2024-12-17
ES3032315T3 (en) 2025-07-17
US11685920B2 (en) 2023-06-27
EP3717646A1 (en) 2020-10-07
SG11202003596WA (en) 2020-05-28
AU2018375807B2 (en) 2025-04-10
CN111433361A (zh) 2020-07-17
EP4328306A2 (en) 2024-02-28
JP2023123517A (ja) 2023-09-05
US20200370046A1 (en) 2020-11-26
US20220090075A1 (en) 2022-03-24
US20250066786A1 (en) 2025-02-27
KR20200094152A (ko) 2020-08-06
MX2025009778A (es) 2025-09-02
CA3079755A1 (en) 2019-06-06
BR112020009431A2 (pt) 2020-10-13
AU2018375807A1 (en) 2020-05-07
EP3717646B1 (en) 2025-04-23
RU2020121752A (ru) 2021-12-30
US12180480B2 (en) 2024-12-31
US11739328B2 (en) 2023-08-29
HRP20250804T1 (hr) 2025-09-12
PE20211238A1 (es) 2021-07-09
WO2019109001A1 (en) 2019-06-06
CL2020001344A1 (es) 2020-09-21
JP2021505129A (ja) 2021-02-18
CN118291458A (zh) 2024-07-05
IL274146B2 (en) 2024-11-01
DK3717646T3 (en) 2025-05-19
IL274146A (en) 2020-06-30
HUE072645T2 (hu) 2025-12-28
SMT202500334T1 (it) 2025-11-10
RS66986B1 (sr) 2025-07-31
CN111433361B (zh) 2024-03-29
PT3717646T (pt) 2025-07-28
US20230332152A1 (en) 2023-10-19
PL3717646T3 (pl) 2025-10-27
MX2020005680A (es) 2020-08-20
US11198869B2 (en) 2021-12-14
KR20240161203A (ko) 2024-11-12
FI3717646T3 (fi) 2025-07-17
EP4328306A3 (en) 2025-05-21
EP3717646A4 (en) 2021-12-01
US20230112629A1 (en) 2023-04-13
IL274146B1 (en) 2024-07-01
SI3717646T1 (sl) 2025-08-29

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