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
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
- 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
- A61K31/395—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
- 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
- A61K31/395—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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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
- A61K35/66—Microorganisms or materials therefrom
- A61K35/76—Viruses; Subviral particles; Bacteriophages
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
- A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
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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
- A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
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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
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- A—HUMAN NECESSITIES
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- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P3/00—Drugs for disorders of the metabolism
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- 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
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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
- C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
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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
- 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)
- C12Y302/01076—L-Iduronidase (3.2.1.76)
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K2300/00—Mixtures or combinations of active ingredients, wherein at least one active ingredient is fully defined in groups A61K31/00 - A61K41/00
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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
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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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- 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
- C12N2750/14121—Viruses as such, e.g. new isolates, mutants or their genomic sequences
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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
- 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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- 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
Landscapes
- 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)
- Bioinformatics & Cheminformatics (AREA)
- Medicinal Chemistry (AREA)
- Organic Chemistry (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Pharmacology & Pharmacy (AREA)
- Epidemiology (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Biomedical Technology (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Biochemistry (AREA)
- Microbiology (AREA)
- Molecular Biology (AREA)
- Virology (AREA)
- Immunology (AREA)
- Biophysics (AREA)
- Gastroenterology & Hepatology (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Dermatology (AREA)
- Mycology (AREA)
- Diabetes (AREA)
- Hematology (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 (fr) | 2017-07-06 | 2018-07-05 | Thérapie génique médiée par aav9 pour traiter la mucopolysaccharidose de 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 (1) | US11890329B2 (fr) |
EP (1) | EP3676385A1 (fr) |
JP (2) | JP2020526514A (fr) |
KR (2) | KR20240063170A (fr) |
AU (1) | AU2018298133A1 (fr) |
BR (1) | BR112020000063A2 (fr) |
CA (1) | CA3068328A1 (fr) |
IL (1) | IL271780A (fr) |
MA (1) | MA50016A (fr) |
SG (1) | SG11201912631PA (fr) |
WO (1) | WO2019010335A1 (fr) |
Families Citing this family (11)
Publication number | Priority date | Publication date | Assignee | Title |
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PT2984166T (pt) | 2013-03-15 | 2020-07-16 | Univ Pennsylvania | Composições para tratar mps i |
BR112018015751A2 (pt) | 2016-02-03 | 2019-02-05 | Univ Pennsylvania | terapia gênica para tratamento de mucopolissacaridose tipo i |
EP3676385A1 (fr) * | 2017-07-06 | 2020-07-08 | The Trustees of The University of Pennsylvania | Thérapie génique médiée par aav9 pour traiter la mucopolysaccharidose de type i |
EP3684938A1 (fr) | 2017-09-22 | 2020-07-29 | The Trustees of the University of Pennsylvania | Thérapie génique pour le traitement de la mucopolysaccharidose de type ii |
AU2020369570A1 (en) * | 2019-10-23 | 2022-05-12 | 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 |
US20230346979A1 (en) * | 2020-08-10 | 2023-11-02 | Prevail Therapeutics, Inc. | Gene therapies for neurodegenerative disorders |
IL300407A (en) * | 2020-08-10 | 2023-04-01 | Prevail Therapeutics Inc | Gene therapies for lysosomal disorders |
WO2022165313A1 (fr) | 2021-02-01 | 2022-08-04 | Regenxbio Inc. | Thérapie génique de céroïdes-lipofuscinoses neuronales |
EP4288556A1 (fr) * | 2021-02-05 | 2023-12-13 | Regents of the University of Minnesota | Méthodes de prévention de défauts cardiaques ou squelettiques dans des maladies comprenant des mucopolysaccharidoses |
EP4294461A1 (fr) * | 2021-02-22 | 2023-12-27 | Decibel Therapeutics, Inc. | Méthodes et compositions pour réduire la toxicité induite par un vecteur d'acide nucléique dans l'oreille interne |
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WO2017024198A1 (fr) | 2015-08-06 | 2017-02-09 | The Trustees Of The University Of Pennsylvania | Glp-1 et son utilisation dans des compositions pour traiter des maladies métaboliques |
US11117942B2 (en) * | 2015-08-31 | 2021-09-14 | The Trustees Of The University Of Pennsylvania | AAV-EPO for treating companion animals |
CN116650668A (zh) * | 2015-10-23 | 2023-08-29 | 衣阿华大学研究基金会 | 使用基因疗法以推迟疾病发生和发展同时提供认知保护来治疗神经变性疾病的方法 |
US11098286B2 (en) | 2015-12-11 | 2021-08-24 | The Trustees Of The University Of Pennsylvania | Scalable purification method for AAV9 |
ES2934848T3 (es) | 2015-12-11 | 2023-02-27 | Univ Pennsylvania | Método de purificación escalable para AAV8 |
US11015173B2 (en) | 2015-12-11 | 2021-05-25 | The Trustees Of The University Of Pennsylvania | Scalable purification method for AAV1 |
US11028372B2 (en) | 2015-12-11 | 2021-06-08 | The Trustees Of The University Of Pennsylvania | Scalable purification method for AAVRH10 |
AU2017207818B2 (en) | 2016-01-15 | 2024-03-28 | Regents Of The University Of Minnesota | Methods and compositions for the treatment of neurologic disease |
BR112018015751A2 (pt) | 2016-02-03 | 2019-02-05 | Univ Pennsylvania | terapia gênica para tratamento de mucopolissacaridose tipo i |
IL266639B1 (en) | 2016-11-15 | 2024-02-01 | Univ Minnesota | A method to improve neurological function in MPSI and MPSII and other neurological disorders |
KR20190109506A (ko) | 2017-01-31 | 2019-09-25 | 리젠엑스바이오 인크. | 완전히-인간 글리코실화된 인간 알파-l-이두로니다아제(idua)를 이용한 뮤코다당증 1형의 치료 |
EP3676385A1 (fr) | 2017-07-06 | 2020-07-08 | The Trustees of The University of Pennsylvania | Thérapie génique médiée par aav9 pour traiter la mucopolysaccharidose de type i |
JP2021531276A (ja) | 2018-07-18 | 2021-11-18 | レジェンクスバイオ インコーポレーテッド | 完全ヒトグリコシル化ヒトα−L−イズロニダーゼ(IDUA)を用いたムコ多糖症I型の治療 |
KR20220131522A (ko) | 2020-01-22 | 2022-09-28 | 리젠엑스바이오 인크. | 완전-인간 글리코실화된 인간 알파-l-이두로니다아제 (idua)를 사용한 점액다당류증 i의 치료 |
EP4288556A1 (fr) | 2021-02-05 | 2023-12-13 | Regents of the University of Minnesota | Méthodes de prévention de défauts cardiaques ou squelettiques dans des maladies comprenant des mucopolysaccharidoses |
-
2018
- 2018-07-05 EP EP18746390.6A patent/EP3676385A1/fr active Pending
- 2018-07-05 MA MA050016A patent/MA50016A/fr unknown
- 2018-07-05 SG SG11201912631PA patent/SG11201912631PA/en unknown
- 2018-07-05 KR KR1020247013510A patent/KR20240063170A/ko active Search and Examination
- 2018-07-05 BR BR112020000063-9A patent/BR112020000063A2/pt unknown
- 2018-07-05 US US16/624,084 patent/US11890329B2/en active Active
- 2018-07-05 JP JP2020500131A patent/JP2020526514A/ja active Pending
- 2018-07-05 AU AU2018298133A patent/AU2018298133A1/en active Pending
- 2018-07-05 KR KR1020207003304A patent/KR20200053468A/ko not_active Application Discontinuation
- 2018-07-05 WO PCT/US2018/040957 patent/WO2019010335A1/fr unknown
- 2018-07-05 CA CA3068328A patent/CA3068328A1/fr active Pending
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2019
- 2019-12-31 IL IL271780A patent/IL271780A/en unknown
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2023
- 2023-06-30 JP JP2023108011A patent/JP2023123760A/ja active Pending
Also Published As
Publication number | Publication date |
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WO2019010335A1 (fr) | 2019-01-10 |
MA50016A (fr) | 2020-07-08 |
US11890329B2 (en) | 2024-02-06 |
AU2018298133A1 (en) | 2020-01-23 |
BR112020000063A2 (pt) | 2020-07-14 |
CA3068328A1 (fr) | 2019-01-10 |
JP2023123760A (ja) | 2023-09-05 |
US20200147185A1 (en) | 2020-05-14 |
KR20200053468A (ko) | 2020-05-18 |
EP3676385A1 (fr) | 2020-07-08 |
JP2020526514A (ja) | 2020-08-31 |
IL271780A (en) | 2020-02-27 |
KR20240063170A (ko) | 2024-05-09 |
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