SG11201912631PA - Aav9-mediated gene therapy for treating mucopolysaccharidosis type i - Google Patents
Aav9-mediated gene therapy for treating mucopolysaccharidosis type iInfo
- Publication number
- SG11201912631PA SG11201912631PA SG11201912631PA SG11201912631PA SG11201912631PA SG 11201912631P A SG11201912631P A SG 11201912631PA SG 11201912631P A SG11201912631P A SG 11201912631PA SG 11201912631P A SG11201912631P A SG 11201912631PA SG 11201912631P A SG11201912631P A SG 11201912631PA
- Authority
- SG
- Singapore
- Prior art keywords
- aav9
- gene therapy
- mediated gene
- mucopolysaccharidosis type
- treating mucopolysaccharidosis
- Prior art date
Links
- 102100035028 Alpha-L-iduronidase Human genes 0.000 title 1
- 101001019502 Homo sapiens Alpha-L-iduronidase Proteins 0.000 title 1
- 206010056886 Mucopolysaccharidosis I Diseases 0.000 title 1
- 208000028781 Mucopolysaccharidosis type 1 Diseases 0.000 title 1
- 238000001415 gene therapy Methods 0.000 title 1
- 230000001404 mediated effect Effects 0.000 title 1
Classifications
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
- A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- A61K38/43—Enzymes; Proenzymes; Derivatives thereof
- A61K38/46—Hydrolases (3)
- A61K38/47—Hydrolases (3) acting on glycosyl compounds (3.2), e.g. cellulases, lactases
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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
- A61K31/33—Heterocyclic compounds
- A61K31/335—Heterocyclic compounds having oxygen as the only ring hetero atom, e.g. fungichromin
- A61K31/34—Heterocyclic compounds having oxygen as the only ring hetero atom, e.g. fungichromin having five-membered rings with one oxygen as the only ring hetero atom, e.g. isosorbide
- A61K31/343—Heterocyclic compounds having oxygen as the only ring hetero atom, e.g. fungichromin having five-membered rings with one oxygen as the only ring hetero atom, e.g. isosorbide condensed with a carbocyclic ring, e.g. coumaran, bufuralol, befunolol, clobenfurol, amiodarone
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- A—HUMAN NECESSITIES
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- A61K31/33—Heterocyclic compounds
- A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
- A61K31/435—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom
- A61K31/4353—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom ortho- or peri-condensed with heterocyclic ring systems
- A61K31/436—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom ortho- or peri-condensed with heterocyclic ring systems the heterocyclic ring system containing a six-membered ring having oxygen as a ring hetero atom, e.g. rapamycin
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- A—HUMAN NECESSITIES
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- 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
- A61K31/435—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom
- A61K31/439—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom the ring forming part of a bridged ring system, e.g. quinuclidine
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- A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
- A61K31/535—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with at least one nitrogen and one oxygen as the ring hetero atoms, e.g. 1,2-oxazines
- A61K31/5375—1,4-Oxazines, e.g. morpholine
- A61K31/5377—1,4-Oxazines, e.g. morpholine not condensed and containing further heterocyclic rings, e.g. timolol
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- A61K31/57—Compounds containing cyclopenta[a]hydrophenanthrene ring systems; Derivatives thereof, e.g. steroids substituted in position 17 beta by a chain of two carbon atoms, e.g. pregnane or progesterone
- A61K31/573—Compounds containing cyclopenta[a]hydrophenanthrene ring systems; Derivatives thereof, e.g. steroids substituted in position 17 beta by a chain of two carbon atoms, e.g. pregnane or progesterone substituted in position 21, e.g. cortisone, dexamethasone, prednisone or aldosterone
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- 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
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- 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
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- C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
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- 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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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
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- C12Y302/01—Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
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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
- A61K48/0083—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 administration regime
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- 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
- C12N2750/14121—Viruses as such, e.g. new isolates, mutants or their genomic sequences
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- 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
- C12N2750/14132—Use of virus as therapeutic agent, other than vaccine, e.g. as cytolytic agent
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- 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
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- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- General Health & Medical Sciences (AREA)
- Genetics & Genomics (AREA)
- Medicinal Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Veterinary Medicine (AREA)
- Organic Chemistry (AREA)
- Epidemiology (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- Biomedical Technology (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Biochemistry (AREA)
- Microbiology (AREA)
- Molecular Biology (AREA)
- Virology (AREA)
- Immunology (AREA)
- Gastroenterology & Hepatology (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Mycology (AREA)
- Dermatology (AREA)
- Hematology (AREA)
- Diabetes (AREA)
- Obesity (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
Applications Claiming Priority (4)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201762529385P | 2017-07-06 | 2017-07-06 | |
| US201762530614P | 2017-07-10 | 2017-07-10 | |
| US201862616106P | 2018-01-11 | 2018-01-11 | |
| PCT/US2018/040957 WO2019010335A1 (en) | 2017-07-06 | 2018-07-05 | Aav9-mediated gene therapy for treating mucopolysaccharidosis type i |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| SG11201912631PA true SG11201912631PA (en) | 2020-01-30 |
Family
ID=63036365
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| SG11201912631PA SG11201912631PA (en) | 2017-07-06 | 2018-07-05 | Aav9-mediated gene therapy for treating mucopolysaccharidosis type i |
Country Status (11)
| Country | Link |
|---|---|
| US (2) | US11890329B2 (en) |
| EP (1) | EP3676385A1 (en) |
| JP (2) | JP2020526514A (en) |
| KR (2) | KR20240063170A (en) |
| AU (1) | AU2018298133A1 (en) |
| BR (1) | BR112020000063A2 (en) |
| CA (1) | CA3068328A1 (en) |
| IL (1) | IL271780A (en) |
| MA (1) | MA50016A (en) |
| SG (1) | SG11201912631PA (en) |
| WO (1) | WO2019010335A1 (en) |
Families Citing this family (14)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP6591956B2 (en) | 2013-03-15 | 2019-10-16 | ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア | Compositions and methods for treating MPS1 |
| CA2912678C (en) | 2013-05-15 | 2023-10-10 | Regents Of The University Of Minnesota | Adeno-associated virus mediated gene transfer to the central nervous system |
| EP3411488A1 (en) | 2016-02-03 | 2018-12-12 | The Trustees Of The University Of Pennsylvania | Gene therapy for treating mucopolysaccharidosis type i |
| AU2018298133A1 (en) * | 2017-07-06 | 2020-01-23 | The Trustees Of The University Of Pennsylvania | AAV9-mediated gene therapy for treating mucopolysaccharidosis type I |
| SG11202002457RA (en) | 2017-09-22 | 2020-04-29 | Univ Pennsylvania | Gene therapy for treating mucopolysaccharidosis type ii |
| EP3886919A1 (en) * | 2018-11-30 | 2021-10-06 | Novartis AG | Aav viral vectors and uses thereof |
| IL292372A (en) * | 2019-10-23 | 2022-06-01 | Univ Pennsylvania | Preparations for the reduction of transgene expression specifically drg |
| WO2021150570A1 (en) * | 2020-01-22 | 2021-07-29 | Regenxbio Inc. | Treatment of mucopolysaccharidosis i with fully-human glycosylated human alpha-l-iduronidase (idua) |
| US20230310654A1 (en) * | 2020-08-10 | 2023-10-05 | Prevail Therapeutics, Inc. | Gene therapies for lysosomal disorders |
| IL300409A (en) * | 2020-08-10 | 2023-04-01 | Prevail Therapeutics Inc | Gene therapies for neurodegenerative disorders |
| AU2022214429A1 (en) | 2021-02-01 | 2023-09-14 | Tern Therapeutics, Llc | Gene therapy for neuronal ceroid lipofuscinoses |
| US20240316217A1 (en) * | 2021-02-05 | 2024-09-26 | Regents Of The University Of Minnesota | Methods for preventing cardiac or skeletal defects in diseases including mucopolysaccharidoses |
| US20240226331A9 (en) * | 2021-02-22 | 2024-07-11 | Decibel Therapeutics, Inc. | Methods and compositions for reducing nucleic acid vector-induced toxicity in the inner ear |
| WO2024205528A1 (en) * | 2023-03-31 | 2024-10-03 | Erci̇yes Üni̇versi̇tesi̇ Strateji̇ Geli̇şti̇rme Dai̇re Başkanliği | A multi-head for use in electrohydrodynamic |
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| JPH06504449A (en) | 1991-11-14 | 1994-05-26 | ウイミンズ・アンド・チルドレンズ・ホスピタル | Synthetic alpha-L-iduronidase and the gene sequence encoding it |
| US6149909A (en) | 1995-06-23 | 2000-11-21 | Women's And Children's Hospital | Synthetic α-L-iduronidase and genetic sequences encoding same |
| US5478745A (en) | 1992-12-04 | 1995-12-26 | University Of Pittsburgh | Recombinant viral vector system |
| JP2001500015A (en) | 1996-09-06 | 2001-01-09 | トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア | Method for producing inducible recombinant adeno-associated virus using T7 polymerase |
| CA2328518A1 (en) | 1998-05-13 | 1999-11-18 | Harbor-Ucla | Recombinant (alpha)-l-iduronidase, methods for producing and purifying the same and methods for treating diseases caused by deficiencies thereof |
| JP4827353B2 (en) | 1999-08-09 | 2011-11-30 | ターゲティッド ジェネティクス コーポレイション | Increased expression of single-stranded heterologous nucleotide sequences from recombinant viral vectors by designing sequences to form intrastrand base pairs |
| US6426208B1 (en) | 1999-11-12 | 2002-07-30 | Harbor-Ucla Research And Education Institute | Recombinant α-L-iduronidase, methods for producing and purifying the same and methods for treating diseases caused by deficiencies thereof |
| RU2196988C2 (en) | 2000-04-19 | 2003-01-20 | Государственный новосибирский областной клинический диагностический центр | Method for diagnostics of mucopolysaccharidosis |
| JP2004504016A (en) | 2000-06-30 | 2004-02-12 | マキシゲン・エイピーエス | Peptide-extended glycosylated polypeptides |
| SG168422A1 (en) | 2001-11-13 | 2011-02-28 | Univ Pennsylvania | A method of detecting and/or identifying adeno-associated virus (aav) sequences and isolating novel sequences identified thereby |
| JP4810062B2 (en) | 2001-12-17 | 2011-11-09 | ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア | Sequence of adeno-associated virus (AAV) serotype 8 |
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| SG10202007803QA (en) | 2011-02-17 | 2020-09-29 | Univ Pennsylvania | Compositions and methods for altering tissue specificity and improving aav9-mediated gene transfer |
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| JP6591956B2 (en) | 2013-03-15 | 2019-10-16 | ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア | Compositions and methods for treating MPS1 |
| CA2912678C (en) | 2013-05-15 | 2023-10-10 | Regents Of The University Of Minnesota | Adeno-associated virus mediated gene transfer to the central nervous system |
| DK3581207T5 (en) | 2013-06-11 | 2024-09-02 | Ppl Bvi Ltd | FUSION PROTEINS COMPRISING HOXD12 AND NEMO BINDING DOMAIN AND USES THEREOF |
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| EP3800260A1 (en) | 2014-09-24 | 2021-04-07 | City of Hope | Adeno-associated virus vector variants for high efficiency genome editing and methods thereof |
| AU2015330092A1 (en) * | 2014-10-06 | 2017-04-13 | Arthrogen B.V. | AAV-based gene therapy |
| SI3294323T1 (en) * | 2015-05-15 | 2022-06-30 | Regenxbio Inc. | Adeno-associated virus for therapeutic delivery to the central nervous system |
| HK1256909A1 (en) | 2015-08-06 | 2019-10-04 | 宾夕法尼亚大学 | Glp-1 and use thereof in compositions for treating metabolic diseases |
| WO2017024204A1 (en) * | 2015-08-06 | 2017-02-09 | Academia Sinica | Engineered enzyme for enzyme replacement therapy |
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| SG11201803218PA (en) * | 2015-10-23 | 2018-05-30 | Univ Iowa Res Found | Methods for treating neurodegenerative diseases using gene therapy to delay disease onset and progression while providing cognitive protection |
| EP3387117B1 (en) | 2015-12-11 | 2022-11-23 | The Trustees Of The University Of Pennsylvania | Scalable purification method for aav8 |
| WO2017100674A1 (en) | 2015-12-11 | 2017-06-15 | The Trustees Of The University Of Pennsylvania | Scalable purification method for aav1 |
| EP3387138B1 (en) | 2015-12-11 | 2022-01-26 | The Trustees Of The University Of Pennsylvania | Scalable purification method for aav9 |
| ES2918998T3 (en) | 2015-12-11 | 2022-07-21 | Univ Pennsylvania | Scalable purification method for AAVrh10 |
| WO2017123757A1 (en) | 2016-01-15 | 2017-07-20 | Sangamo Therapeutics, Inc. | Methods and compositions for the treatment of neurologic disease |
| EP3411488A1 (en) | 2016-02-03 | 2018-12-12 | The Trustees Of The University Of Pennsylvania | Gene therapy for treating mucopolysaccharidosis type i |
| AU2017362969A1 (en) | 2016-11-15 | 2019-06-20 | Regents Of The University Of Minnesota | Method for improving neurological function in MPSI and MPSII and other neurological disorders |
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| AU2018298133A1 (en) | 2017-07-06 | 2020-01-23 | The Trustees Of The University Of Pennsylvania | AAV9-mediated gene therapy for treating mucopolysaccharidosis type I |
| IL280198B1 (en) | 2018-07-18 | 2025-09-01 | Regenxbio Inc | Treatment of mucopolysaccharidosis i with fully-human glycosylated human alpha-l-iduronidase (idus) |
| WO2021150570A1 (en) | 2020-01-22 | 2021-07-29 | Regenxbio Inc. | Treatment of mucopolysaccharidosis i with fully-human glycosylated human alpha-l-iduronidase (idua) |
| US20240316217A1 (en) | 2021-02-05 | 2024-09-26 | Regents Of The University Of Minnesota | Methods for preventing cardiac or skeletal defects in diseases including mucopolysaccharidoses |
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2018
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- 2018-07-05 MA MA050016A patent/MA50016A/en unknown
- 2018-07-05 KR KR1020247013510A patent/KR20240063170A/en active Pending
- 2018-07-05 KR KR1020207003304A patent/KR20200053468A/en not_active Ceased
- 2018-07-05 BR BR112020000063-9A patent/BR112020000063A2/en unknown
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- 2018-07-05 WO PCT/US2018/040957 patent/WO2019010335A1/en not_active Ceased
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| EP3676385A1 (en) | 2020-07-08 |
| JP7670759B2 (en) | 2025-04-30 |
| JP2023123760A (en) | 2023-09-05 |
| CA3068328A1 (en) | 2019-01-10 |
| US20200147185A1 (en) | 2020-05-14 |
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| MA50016A (en) | 2020-07-08 |
| BR112020000063A2 (en) | 2020-07-14 |
| KR20200053468A (en) | 2020-05-18 |
| KR20240063170A (en) | 2024-05-09 |
| JP2020526514A (en) | 2020-08-31 |
| WO2019010335A1 (en) | 2019-01-10 |
| US11890329B2 (en) | 2024-02-06 |
| US20240307511A1 (en) | 2024-09-19 |
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