IL260959B2 - Gene therapy for treating mucopolysaccharidosis type i - Google Patents
Gene therapy for treating mucopolysaccharidosis type iInfo
- Publication number
- IL260959B2 IL260959B2 IL260959A IL26095918A IL260959B2 IL 260959 B2 IL260959 B2 IL 260959B2 IL 260959 A IL260959 A IL 260959A IL 26095918 A IL26095918 A IL 26095918A IL 260959 B2 IL260959 B2 IL 260959B2
- Authority
- IL
- Israel
- Prior art keywords
- suspension
- hidua
- vector
- mps
- raav
- Prior art date
Links
Classifications
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- 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
- 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
- A61K48/0075—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 delivery route, e.g. oral, subcutaneous
-
- 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
-
- 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
- A61K9/10—Dispersions; Emulsions
-
- 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
- A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
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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
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
- C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
- C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
- C12N15/86—Viral vectors
-
- 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
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
- C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
- C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
- C12N15/86—Viral vectors
- C12N15/864—Parvoviral vectors, e.g. parvovirus, densovirus
- C12N15/8645—Adeno-associated virus
-
- 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
- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/14—Hydrolases (3)
- C12N9/24—Hydrolases (3) acting on glycosyl compounds (3.2)
- C12N9/2402—Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1)
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y302/00—Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
- C12Y302/01—Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
- C12Y302/01031—Beta-glucuronidase (3.2.1.31)
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y302/00—Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
- C12Y302/01—Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
- C12Y302/01076—L-Iduronidase (3.2.1.76)
-
- 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
- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14141—Use of virus, viral particle or viral elements as a vector
- C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
-
- 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
- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/15—Vector systems having a special element relevant for transcription chimeric enhancer/promoter combination
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Genetics & Genomics (AREA)
- Engineering & Computer Science (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- General Health & Medical Sciences (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Medicinal Chemistry (AREA)
- Molecular Biology (AREA)
- Biomedical Technology (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Biochemistry (AREA)
- Microbiology (AREA)
- Epidemiology (AREA)
- Plant Pathology (AREA)
- Virology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Obesity (AREA)
- Hematology (AREA)
- Diabetes (AREA)
- Dermatology (AREA)
- Dispersion Chemistry (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Enzymes And Modification Thereof (AREA)
- Medicinal Preparation (AREA)
- Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
Applications Claiming Priority (6)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201662290547P | 2016-02-03 | 2016-02-03 | |
| US201662323271P | 2016-04-15 | 2016-04-15 | |
| US201662337178P | 2016-05-16 | 2016-05-16 | |
| US201662367798P | 2016-07-28 | 2016-07-28 | |
| US201762452560P | 2017-01-31 | 2017-01-31 | |
| PCT/US2017/016133 WO2017136500A1 (en) | 2016-02-03 | 2017-02-02 | Gene therapy for treating mucopolysaccharidosis type i |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| IL260959A IL260959A (en) | 2018-09-20 |
| IL260959B1 IL260959B1 (en) | 2024-05-01 |
| IL260959B2 true IL260959B2 (en) | 2024-09-01 |
Family
ID=58108727
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| IL260959A IL260959B2 (en) | 2016-02-03 | 2017-02-02 | Gene therapy for treating mucopolysaccharidosis type i |
Country Status (11)
| Country | Link |
|---|---|
| US (3) | US10973929B2 (https=) |
| EP (1) | EP3411488A1 (https=) |
| JP (2) | JP7360241B2 (https=) |
| KR (2) | KR20180121899A (https=) |
| AU (3) | AU2017215211C1 (https=) |
| BR (1) | BR112018015751A2 (https=) |
| CA (1) | CA3012195A1 (https=) |
| IL (1) | IL260959B2 (https=) |
| MA (1) | MA43968A (https=) |
| SG (2) | SG10201912758TA (https=) |
| WO (1) | WO2017136500A1 (https=) |
Families Citing this family (37)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| AU2014235096B2 (en) | 2013-03-15 | 2020-03-19 | The Trustees Of The University Of Pennsylvania | Compositions and methods for treating MPSI |
| CN115120746A (zh) | 2013-05-15 | 2022-09-30 | 明尼苏达大学董事会 | 腺相关病毒介导的基因向中枢神经系统转移 |
| CA3012195A1 (en) | 2016-02-03 | 2017-08-10 | The Trustees Of The University Of Pennsylvania | Gene therapy for treating mucopolysaccharidosis type i |
| JOP20190200A1 (ar) | 2017-02-28 | 2019-08-27 | Univ Pennsylvania | تركيبات نافعة في معالجة ضمور العضل النخاعي |
| PT3589730T (pt) | 2017-02-28 | 2024-02-28 | Univ Pennsylvania | Vetor de vírus adenoassociado (aav) de clado f e suas utilizações |
| IL316926A (en) | 2017-05-11 | 2025-01-01 | Univ Pennsylvania | Gene therapy for neuronal ceroid lipofuscinosis |
| CA3068328A1 (en) * | 2017-07-06 | 2019-01-10 | The Trustees Of The University Of Pennsylvania | Aav9-mediated gene therapy for treating mucopolysaccharidosis type i |
| KR102883778B1 (ko) | 2017-09-22 | 2025-11-12 | 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 | Ii형 점액다당류증의 치료를 위한 유전자 요법 |
| CN107699590A (zh) * | 2017-10-13 | 2018-02-16 | 成都中医药大学 | 一种制备重组人α‑L‑艾杜糖醛酸酶的方法 |
| CN111989396A (zh) | 2017-11-30 | 2020-11-24 | 宾夕法尼亚州大学信托人 | 用于iiib型粘多糖贮积病的基因疗法 |
| AU2018375164B2 (en) | 2017-11-30 | 2025-05-15 | The Trustees Of The University Of Pennsylvania | Gene therapy for Mucopolysaccharidosis IIIA |
| IL280198B2 (en) * | 2018-07-18 | 2026-01-01 | Regenxbio Inc | Treatment of mucopolysaccharidosis I with fully glycosylated human iduronidase-alpha-L (idus) |
| CA3114175A1 (en) | 2018-10-01 | 2020-04-09 | The Trustees Of The University Of Pennsylvania | Beta-galactosidase based aav for treating gm1 gangliosidosis |
| IL282411B2 (en) | 2018-10-25 | 2025-09-01 | Regeneron Pharma | Methods for analyzing viral capsid protein composition |
| US20220025065A1 (en) * | 2018-12-10 | 2022-01-27 | Denali Therapeutics Inc. | Lysosomal storage disorder biomarkers and methods of use thereof |
| WO2020132455A1 (en) * | 2018-12-21 | 2020-06-25 | The Trustees Of The University Of Pennsylvania | Compositions for drg-specific reduction of transgene expression |
| AU2019428629A1 (en) * | 2019-02-06 | 2021-01-28 | Sangamo Therapeutics, Inc. | Method for the treatment of mucopolysaccharidosis type I |
| BR112021015817A2 (pt) | 2019-02-22 | 2021-10-13 | The Trustees Of The University Of Pennsylvania | Vírus adeno-associado recombinante para tratamento de neurodegeneração com início na idade adulta associado a grn |
| WO2021081217A1 (en) * | 2019-10-23 | 2021-04-29 | The Trustees Of The University Of Pennsylvania | Compositions for drg-specific reduction of transgene expression |
| KR20220131522A (ko) * | 2020-01-22 | 2022-09-28 | 리젠엑스바이오 인크. | 완전-인간 글리코실화된 인간 알파-l-이두로니다아제 (idua)를 사용한 점액다당류증 i의 치료 |
| KR20220145838A (ko) * | 2020-02-02 | 2022-10-31 | 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 | Gm1 강글리오사이드증을 치료하는 데 유용한 조성물 |
| MX2022014258A (es) | 2020-05-12 | 2023-02-22 | Univ Pennsylvania | Composiciones para la reducción específica de un fármaco de la expresión de transgén. |
| EP4204014A1 (en) * | 2020-08-26 | 2023-07-05 | The Trustees of The University of Pennsylvania | Recombinant adeno-associated virus for treatment of grn-associated adult-onset neurodegeneration |
| AU2021356684A1 (en) | 2020-10-09 | 2023-05-11 | The Trustees Of The University Of Pennsylvania | Compositions and methods for treatment of fabry disease |
| WO2022094284A1 (en) * | 2020-10-30 | 2022-05-05 | Immusoft Corporation | Methods of administering genetically modified b cells for in vivo delivery of therapeutic agents |
| WO2022165313A1 (en) | 2021-02-01 | 2022-08-04 | Regenxbio Inc. | Gene therapy for neuronal ceroid lipofuscinoses |
| WO2023049846A1 (en) * | 2021-09-24 | 2023-03-30 | The Trustees Of The University Of Pennsylvania | Compositions useful for treatment of charcot-marie-tooth disease |
| EP4423264A4 (en) * | 2021-10-27 | 2025-12-17 | Univ North Carolina Chapel Hill | AAV-IDUA VECTOR FOR TREATING MPS I |
| US20250295807A1 (en) | 2021-11-15 | 2025-09-25 | The Trustees Of The University Of Pennsylvania | Compositions for drg-specific reduction of transgene expression |
| US20240408157A1 (en) | 2021-12-02 | 2024-12-12 | The Trustees Of The University Of Pennsylvania | Compositions and methods for treatment of fabry disease |
| WO2023172975A1 (en) * | 2022-03-08 | 2023-09-14 | University Of Massachusetts | Methods of raav packaging |
| WO2023196892A1 (en) | 2022-04-06 | 2023-10-12 | The Trustees Of The University Of Pennsylvania | Passive immunization with anti- aav neutralizing antibodies to prevent off-target transduction of intrathecally delivered aav vectors |
| EP4504797A1 (en) | 2022-04-06 | 2025-02-12 | The Trustees of The University of Pennsylvania | Compositions and methods for treating her2 positive metastatic breast cancer and other cancers |
| CN115838709A (zh) * | 2022-11-29 | 2023-03-24 | 武汉禾元生物科技股份有限公司 | 以基因工程水稻表达和制备重组人艾杜糖苷酸酶的方法 |
| WO2024258961A1 (en) | 2023-06-12 | 2024-12-19 | The Trustees Of The University Of Pennsylvania | Aav gene therapy for mucopolysaccharidosis iiib |
| TW202516019A (zh) | 2023-06-29 | 2025-04-16 | 賓州大學委員會 | 具中樞神經系統靶向模體的突變aav及含有其之組成物 |
| IL326390A (en) | 2023-08-10 | 2026-04-01 | Univ Pennsylvania | Compositions and methods for treating spinal muscular atrophy |
Citations (2)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2014151341A1 (en) * | 2013-03-15 | 2014-09-25 | The Trustees Of The University Of Pennsylvania | Compositions and methods for treating mpsi |
| WO2014186579A1 (en) * | 2013-05-15 | 2014-11-20 | Regents Of The University Of Minnesota | Adeno-associated virus mediated gene transfer to the central nervous system |
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| US5478745A (en) | 1992-12-04 | 1995-12-26 | University Of Pittsburgh | Recombinant viral vector system |
| EP0931158A1 (en) | 1996-09-06 | 1999-07-28 | The Trustees Of The University Of Pennsylvania | An inducible method for production of recombinant adeno-associated viruses utilizing t7 polymerase |
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| ES2525067T3 (es) | 2005-04-07 | 2014-12-17 | The Trustees Of The University Of Pennsylvania | Método de incremento de la función de un vector de AAV |
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| WO2011126808A2 (en) | 2010-03-29 | 2011-10-13 | The Trustees Of The University Of Pennsylvania | Pharmacologically induced transgene ablation system |
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| TWI617573B (zh) | 2015-08-06 | 2018-03-11 | 中央研究院 | 用於酵素置換療法的改造酵素 |
| KR102738860B1 (ko) | 2015-10-23 | 2024-12-04 | 유니버시티 오브 아이오와 리써치 파운데이션 | 리소좀 축적 질환 (lsd)을 갖는 포유동물의 치료 방법에 사용하기 위한 aav 입자를 포함하는 약제학적 조성물 |
| ES2934848T3 (es) | 2015-12-11 | 2023-02-27 | Univ Pennsylvania | Método de purificación escalable para AAV8 |
| US11028372B2 (en) | 2015-12-11 | 2021-06-08 | The Trustees Of The University Of Pennsylvania | Scalable purification method for AAVRH10 |
| EP3387138B1 (en) | 2015-12-11 | 2022-01-26 | The Trustees Of The University Of Pennsylvania | Scalable purification method for aav9 |
| WO2017100674A1 (en) | 2015-12-11 | 2017-06-15 | The Trustees Of The University Of Pennsylvania | Scalable purification method for aav1 |
| WO2017123757A1 (en) | 2016-01-15 | 2017-07-20 | Sangamo Therapeutics, Inc. | Methods and compositions for the treatment of neurologic disease |
| CA3012195A1 (en) | 2016-02-03 | 2017-08-10 | The Trustees Of The University Of Pennsylvania | Gene therapy for treating mucopolysaccharidosis type i |
| CA3068328A1 (en) | 2017-07-06 | 2019-01-10 | The Trustees Of The University Of Pennsylvania | Aav9-mediated gene therapy for treating mucopolysaccharidosis type i |
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2017
- 2017-02-02 CA CA3012195A patent/CA3012195A1/en active Pending
- 2017-02-02 JP JP2018540838A patent/JP7360241B2/ja active Active
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2021
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2023
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| WO2014151341A1 (en) * | 2013-03-15 | 2014-09-25 | The Trustees Of The University Of Pennsylvania | Compositions and methods for treating mpsi |
| WO2014186579A1 (en) * | 2013-05-15 | 2014-11-20 | Regents Of The University Of Minnesota | Adeno-associated virus mediated gene transfer to the central nervous system |
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| CHRISTIAN HINDERER ET AL,, NEONATAL SYSTEMIC AAV INDUCES TOLERANCE TO CNS GENE THERAPY IN MPS I DOGS AND NONHUMAN PRIMATES, 31 August 2015 (2015-08-31) * |
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Also Published As
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| WO2017136500A1 (en) | 2017-08-10 |
| AU2026201588A1 (en) | 2026-03-26 |
| EP3411488A1 (en) | 2018-12-12 |
| JP7360241B2 (ja) | 2023-10-12 |
| US20190038772A1 (en) | 2019-02-07 |
| MA43968A (fr) | 2018-12-12 |
| US10973929B2 (en) | 2021-04-13 |
| SG11201806270XA (en) | 2018-08-30 |
| AU2023202680A1 (en) | 2023-07-13 |
| US20240024510A1 (en) | 2024-01-25 |
| BR112018015751A2 (pt) | 2019-02-05 |
| JP2023062088A (ja) | 2023-05-02 |
| IL260959B1 (en) | 2024-05-01 |
| JP2019512005A (ja) | 2019-05-09 |
| KR20250171426A (ko) | 2025-12-08 |
| KR20180121899A (ko) | 2018-11-09 |
| AU2017215211A1 (en) | 2018-08-09 |
| IL260959A (en) | 2018-09-20 |
| CA3012195A1 (en) | 2017-08-10 |
| AU2017215211C1 (en) | 2023-11-16 |
| SG10201912758TA (en) | 2020-02-27 |
| US20210228740A1 (en) | 2021-07-29 |
| AU2017215211B2 (en) | 2023-05-18 |
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