MA50016A - Thérapie génique médiée par aav9 pour traiter la mucopolysaccharidose de type i - Google Patents
Thérapie génique médiée par aav9 pour traiter la mucopolysaccharidose de type iInfo
- Publication number
- MA50016A MA50016A MA050016A MA50016A MA50016A MA 50016 A MA50016 A MA 50016A MA 050016 A MA050016 A MA 050016A MA 50016 A MA50016 A MA 50016A MA 50016 A MA50016 A MA 50016A
- Authority
- MA
- Morocco
- Prior art keywords
- aav9
- gene therapy
- mucopolysaccharidosis type
- media
- treat 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
- 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
- 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
- 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
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- 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
-
- 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
-
- 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
-
- 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
Applications Claiming Priority (3)
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 |
Publications (1)
Publication Number | Publication Date |
---|---|
MA50016A true MA50016A (fr) | 2020-07-08 |
Family
ID=63036365
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
MA050016A MA50016A (fr) | 2017-07-06 | 2018-07-05 | Thérapie génique médiée par aav9 pour traiter la mucopolysaccharidose de type i |
Country Status (11)
Country | Link |
---|---|
US (1) | US11890329B2 (fr) |
EP (1) | EP3676385A1 (fr) |
JP (2) | JP2020526514A (fr) |
KR (1) | KR20200053468A (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 |
---|---|---|---|---|
JP6591956B2 (ja) | 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 | Mps1を治療するための組成物および方法 |
CA3012195A1 (fr) | 2016-02-03 | 2017-08-10 | The Trustees Of The University Of Pennsylvania | Therapie genique pour traiter la mucopolysaccharidose de 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 |
US11819539B2 (en) | 2017-09-22 | 2023-11-21 | The Trustees Of The University Of Pennsylvania | Gene therapy for treating Mucopolysaccharidosis type II |
IL292372A (en) * | 2019-10-23 | 2022-06-01 | Univ Pennsylvania | Preparations for the reduction of transgene expression specifically drg |
BR112022014563A2 (pt) * | 2020-01-22 | 2022-09-13 | Regenxbio Inc | Método para tratamento de um sujeito humano diagnosticado com mucopolissacaridose i (mps i) |
US20230346979A1 (en) * | 2020-08-10 | 2023-11-02 | Prevail Therapeutics, Inc. | Gene therapies for neurodegenerative disorders |
CA3190866A1 (fr) * | 2020-08-10 | 2022-02-17 | Prevail Therapeutics, Inc. | Therapies geniques contre les troubles lysosomaux |
US20240091380A1 (en) | 2021-02-01 | 2024-03-21 | Regenxbio Inc. | Gene therapy for neuronal ceroid lipofuscinoses |
WO2022170082A1 (fr) * | 2021-02-05 | 2022-08-11 | Regents Of The University Of Minnesota | Méthodes de prévention de défauts cardiaques ou squelettiques dans des maladies comprenant des mucopolysaccharidoses |
WO2022178419A1 (fr) * | 2021-02-22 | 2022-08-25 | 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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CA3012195A1 (fr) | 2016-02-03 | 2017-08-10 | The Trustees Of The University Of Pennsylvania | Therapie genique pour traiter la mucopolysaccharidose de type i |
EP3541946A1 (fr) | 2016-11-15 | 2019-09-25 | Regents Of The University Of Minnesota | Procédé d'amélioration de la fonction neurologique dans la mpsi et la mpsii et d'autres troubles neurologiques |
JP2020508289A (ja) | 2017-01-31 | 2020-03-19 | 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 |
AU2018298133A1 (en) | 2017-07-06 | 2020-01-23 | The Trustees Of The University Of Pennsylvania | AAV9-mediated gene therapy for treating mucopolysaccharidosis type I |
SG11202100423YA (en) | 2018-07-18 | 2021-02-25 | Regenxbio Inc | Treatment of mucopolysaccharidosis i with fully-human glycosylated human alpha-l-iduronidase (idus) |
BR112022014563A2 (pt) | 2020-01-22 | 2022-09-13 | Regenxbio Inc | Método para tratamento de um sujeito humano diagnosticado com mucopolissacaridose i (mps i) |
WO2022170082A1 (fr) | 2021-02-05 | 2022-08-11 | Regents Of The University Of Minnesota | Méthodes de prévention de défauts cardiaques ou squelettiques dans des maladies comprenant des mucopolysaccharidoses |
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2018
- 2018-07-05 AU AU2018298133A patent/AU2018298133A1/en active Pending
- 2018-07-05 SG SG11201912631PA patent/SG11201912631PA/en unknown
- 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 US US16/624,084 patent/US11890329B2/en active Active
- 2018-07-05 MA MA050016A patent/MA50016A/fr unknown
- 2018-07-05 EP EP18746390.6A patent/EP3676385A1/fr active Pending
- 2018-07-05 CA CA3068328A patent/CA3068328A1/fr active Pending
- 2018-07-05 BR BR112020000063-9A patent/BR112020000063A2/pt unknown
- 2018-07-05 JP JP2020500131A patent/JP2020526514A/ja 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
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Publication number | Publication date |
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KR20200053468A (ko) | 2020-05-18 |
JP2023123760A (ja) | 2023-09-05 |
WO2019010335A1 (fr) | 2019-01-10 |
BR112020000063A2 (pt) | 2020-07-14 |
EP3676385A1 (fr) | 2020-07-08 |
CA3068328A1 (fr) | 2019-01-10 |
US11890329B2 (en) | 2024-02-06 |
US20200147185A1 (en) | 2020-05-14 |
SG11201912631PA (en) | 2020-01-30 |
JP2020526514A (ja) | 2020-08-31 |
AU2018298133A1 (en) | 2020-01-23 |
IL271780A (en) | 2020-02-27 |
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