HRP20231515T1 - Postupci liječenja ili prevencije spinalne mišićne atrofije - Google Patents
Postupci liječenja ili prevencije spinalne mišićne atrofije Download PDFInfo
- Publication number
- HRP20231515T1 HRP20231515T1 HRP20231515TT HRP20231515T HRP20231515T1 HR P20231515 T1 HRP20231515 T1 HR P20231515T1 HR P20231515T T HRP20231515T T HR P20231515TT HR P20231515 T HRP20231515 T HR P20231515T HR P20231515 T1 HRP20231515 T1 HR P20231515T1
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- Croatia
- Prior art keywords
- antisense compound
- salt
- use according
- doses
- human subject
- Prior art date
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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
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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
- C12N15/1136—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 against growth factors, growth regulators, cytokines, lymphokines or hormones
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- 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
- A61K31/712—Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
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- 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
- 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
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- 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
- A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
- A61K9/0021—Intradermal administration, e.g. through microneedle arrays or needleless injectors
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- 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
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- 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
- A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
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- 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
- G01N33/68—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving proteins, peptides or amino acids
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- 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
- G01N33/68—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving proteins, peptides or amino acids
- G01N33/6893—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving proteins, peptides or amino acids related to diseases not provided for elsewhere
- G01N33/6896—Neurological disorders, e.g. Alzheimer's disease
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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
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/11—Antisense
-
- 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
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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
- 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
- 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
- C12N2310/34—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
- 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
- C12N2320/33—Alteration of splicing
-
- 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
- C12N2320/34—Allele or polymorphism specific uses
-
- 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
- C12N2320/35—Special therapeutic applications based on a specific dosage / administration regimen
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- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Biomedical Technology (AREA)
- Molecular Biology (AREA)
- General Health & Medical Sciences (AREA)
- Genetics & Genomics (AREA)
- Medicinal Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Public Health (AREA)
- Organic Chemistry (AREA)
- Veterinary Medicine (AREA)
- Biochemistry (AREA)
- Biotechnology (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Epidemiology (AREA)
- General Engineering & Computer Science (AREA)
- Physics & Mathematics (AREA)
- Microbiology (AREA)
- Neurology (AREA)
- Hematology (AREA)
- Urology & Nephrology (AREA)
- Immunology (AREA)
- Biophysics (AREA)
- Plant Pathology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Neurosurgery (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Physical Education & Sports Medicine (AREA)
- Dermatology (AREA)
- General Physics & Mathematics (AREA)
- Pathology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Cell Biology (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201962876360P | 2019-07-19 | 2019-07-19 | |
| EP20753556.8A EP3999643B1 (en) | 2019-07-19 | 2020-07-16 | Methods of treating or preventing spinal muscular atrophy |
| PCT/US2020/042312 WO2021016032A1 (en) | 2019-07-19 | 2020-07-16 | Methods of treating or preventing spinal muscular atrophy |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| HRP20231515T1 true HRP20231515T1 (hr) | 2024-03-01 |
Family
ID=71995097
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| HRP20231515TT HRP20231515T1 (hr) | 2019-07-19 | 2020-07-16 | Postupci liječenja ili prevencije spinalne mišićne atrofije |
Country Status (33)
| Country | Link |
|---|---|
| US (3) | US20220325282A1 (https=) |
| EP (2) | EP4335503A3 (https=) |
| JP (3) | JP7279253B2 (https=) |
| KR (1) | KR20220042113A (https=) |
| CN (1) | CN114302727A (https=) |
| AR (1) | AR119430A1 (https=) |
| AU (1) | AU2020316997A1 (https=) |
| BR (1) | BR112022000870A2 (https=) |
| CA (1) | CA3149906A1 (https=) |
| CL (1) | CL2021003602A1 (https=) |
| CO (1) | CO2022001354A2 (https=) |
| CR (1) | CR20220071A (https=) |
| DK (1) | DK3999643T5 (https=) |
| ES (1) | ES2966791T3 (https=) |
| FI (1) | FI3999643T3 (https=) |
| HR (1) | HRP20231515T1 (https=) |
| HU (1) | HUE064071T2 (https=) |
| IL (1) | IL289745A (https=) |
| JO (2) | JOP20220007A1 (https=) |
| LT (1) | LT3999643T (https=) |
| MA (1) | MA62935B1 (https=) |
| MD (1) | MD3999643T2 (https=) |
| MX (1) | MX2022000441A (https=) |
| PE (1) | PE20220941A1 (https=) |
| PH (1) | PH12022550114A1 (https=) |
| PL (1) | PL3999643T3 (https=) |
| PT (1) | PT3999643T (https=) |
| RS (1) | RS64879B1 (https=) |
| SI (1) | SI3999643T1 (https=) |
| SM (1) | SMT202300442T1 (https=) |
| TW (2) | TW202438086A (https=) |
| UY (1) | UY38790A (https=) |
| WO (1) | WO2021016032A1 (https=) |
Families Citing this family (4)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP3797780B1 (en) | 2014-04-17 | 2022-09-14 | Biogen MA Inc. | Compositions and methods for modulation of smn2 splicing in a subject |
| US10436802B2 (en) | 2014-09-12 | 2019-10-08 | Biogen Ma Inc. | Methods for treating spinal muscular atrophy |
| PT3999643T (pt) | 2019-07-19 | 2023-12-11 | Biogen Ma Inc | Métodos de tratamento da atrofia muscular espinhal |
| WO2024138018A1 (en) * | 2022-12-23 | 2024-06-27 | University Of Pittsburgh - Of The Commonwealth System Of Higher Education | Treatment of gemin5-mediated disorders |
Family Cites Families (13)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP3470072A1 (en) * | 2005-06-23 | 2019-04-17 | Biogen MA Inc. | Compositions and methods for modulation of smn2 splicing |
| WO2008157753A1 (en) * | 2007-06-21 | 2008-12-24 | Sma Foundation | Methods of treatment for spinal muscular atrophy |
| ES2699827T3 (es) | 2009-06-17 | 2019-02-13 | Biogen Ma Inc | Composiciones y métodos para la modulación de corte y empalme de SMN2 en un sujeto |
| WO2011032109A1 (en) | 2009-09-11 | 2011-03-17 | Sma Foundation | Biomarkers for spinal muscular atrophy |
| EP2714037B1 (en) * | 2011-05-25 | 2016-07-13 | Université Paris Descartes | Erk inhibitors for use in treating spinal muscular atrophy |
| WO2014110291A1 (en) | 2013-01-09 | 2014-07-17 | Isis Pharmaceuticals, Inc. | Compositions and methods for modulation of smn2 splicing in a subject |
| US10436802B2 (en) | 2014-09-12 | 2019-10-08 | Biogen Ma Inc. | Methods for treating spinal muscular atrophy |
| EP3484524B1 (en) * | 2016-07-15 | 2022-11-09 | Ionis Pharmaceuticals, Inc. | Compounds and methods for modulation of smn2 |
| TW201927241A (zh) | 2017-12-21 | 2019-07-16 | 瑞士商赫孚孟拉羅股份公司 | 用於肌肉失能之數位生物標記 |
| CN119679944A (zh) | 2018-01-25 | 2025-03-25 | 渤健马萨诸塞州股份有限公司 | 治疗脊髓性肌萎缩症的方法 |
| IT201800004359A1 (it) | 2018-04-10 | 2019-10-10 | IDENTIFICAZIONE DI miRNA MUSCOLARI COME BIOMARCATORI E TRATTAMENTO CO-ADIUVANTE PER L’ATROFIA MUSCOLARE SPINALE | |
| US20210308281A1 (en) * | 2018-08-15 | 2021-10-07 | Biogen Ma Inc. | Combination therapy for spinal muscular atrophy |
| PT3999643T (pt) | 2019-07-19 | 2023-12-11 | Biogen Ma Inc | Métodos de tratamento da atrofia muscular espinhal |
-
2020
- 2020-07-16 PT PT207535568T patent/PT3999643T/pt unknown
- 2020-07-16 EP EP23210526.2A patent/EP4335503A3/en active Pending
- 2020-07-16 BR BR112022000870A patent/BR112022000870A2/pt unknown
- 2020-07-16 ES ES20753556T patent/ES2966791T3/es active Active
- 2020-07-16 PH PH1/2022/550114A patent/PH12022550114A1/en unknown
- 2020-07-16 SI SI202030323T patent/SI3999643T1/sl unknown
- 2020-07-16 DK DK20753556.8T patent/DK3999643T5/da active
- 2020-07-16 MX MX2022000441A patent/MX2022000441A/es unknown
- 2020-07-16 LT LTEPPCT/US2020/042312T patent/LT3999643T/lt unknown
- 2020-07-16 JP JP2022500671A patent/JP7279253B2/ja active Active
- 2020-07-16 FI FIEP20753556.8T patent/FI3999643T3/fi active
- 2020-07-16 HR HRP20231515TT patent/HRP20231515T1/hr unknown
- 2020-07-16 US US17/626,324 patent/US20220325282A1/en not_active Abandoned
- 2020-07-16 AU AU2020316997A patent/AU2020316997A1/en active Pending
- 2020-07-16 HU HUE20753556A patent/HUE064071T2/hu unknown
- 2020-07-16 EP EP20753556.8A patent/EP3999643B1/en active Active
- 2020-07-16 PL PL20753556.8T patent/PL3999643T3/pl unknown
- 2020-07-16 RS RS20231128A patent/RS64879B1/sr unknown
- 2020-07-16 UY UY0001038790A patent/UY38790A/es not_active Application Discontinuation
- 2020-07-16 WO PCT/US2020/042312 patent/WO2021016032A1/en not_active Ceased
- 2020-07-16 SM SM20230442T patent/SMT202300442T1/it unknown
- 2020-07-16 MA MA62935A patent/MA62935B1/fr unknown
- 2020-07-16 CR CR20220071A patent/CR20220071A/es unknown
- 2020-07-16 CN CN202080049461.1A patent/CN114302727A/zh active Pending
- 2020-07-16 MD MDE20220574T patent/MD3999643T2/ro unknown
- 2020-07-16 CA CA3149906A patent/CA3149906A1/en active Pending
- 2020-07-16 KR KR1020227001151A patent/KR20220042113A/ko active Pending
- 2020-07-16 PE PE2021002322A patent/PE20220941A1/es unknown
- 2020-07-17 TW TW113114478A patent/TW202438086A/zh unknown
- 2020-07-17 AR ARP200102019A patent/AR119430A1/es unknown
- 2020-07-17 TW TW109124224A patent/TWI841762B/zh active
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2021
- 2021-12-31 CL CL2021003602A patent/CL2021003602A1/es unknown
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2022
- 2022-01-10 IL IL289745A patent/IL289745A/en unknown
- 2022-01-16 JO JOP/2022/0007A patent/JOP20220007A1/ar unknown
- 2022-02-10 CO CONC2022/0001354A patent/CO2022001354A2/es unknown
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2023
- 2023-05-10 JP JP2023077805A patent/JP7665678B2/ja active Active
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2024
- 2024-05-10 US US18/661,339 patent/US20240318182A1/en not_active Abandoned
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2025
- 2025-04-07 US US19/172,111 patent/US20250230440A1/en active Pending
- 2025-04-09 JP JP2025064592A patent/JP2025096486A/ja active Pending
- 2025-05-05 JO JOJO/P/2025/0103A patent/JOP20250103A1/ar unknown
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