CO2021002505A2 - Tratamiento de mucopolisacaridosis iva - Google Patents
Tratamiento de mucopolisacaridosis ivaInfo
- Publication number
- CO2021002505A2 CO2021002505A2 CONC2021/0002505A CO2021002505A CO2021002505A2 CO 2021002505 A2 CO2021002505 A2 CO 2021002505A2 CO 2021002505 A CO2021002505 A CO 2021002505A CO 2021002505 A2 CO2021002505 A2 CO 2021002505A2
- Authority
- CO
- Colombia
- Prior art keywords
- treatment
- iva
- raavs
- gene therapy
- methods
- Prior art date
Links
- 208000012253 mucopolysaccharidosis IVA Diseases 0.000 title abstract 3
- 208000025797 Mucopolysaccharidosis type 4A Diseases 0.000 title abstract 2
- 206010028095 Mucopolysaccharidosis IV Diseases 0.000 abstract 3
- 238000000034 method Methods 0.000 abstract 3
- 101001066305 Homo sapiens N-acetylgalactosamine-6-sulfatase Proteins 0.000 abstract 2
- 238000001415 gene therapy Methods 0.000 abstract 2
- 241000702421 Dependoparvovirus Species 0.000 abstract 1
- 102100031688 N-acetylgalactosamine-6-sulfatase Human genes 0.000 abstract 1
- 101150010487 are gene Proteins 0.000 abstract 1
- 210000000988 bone and bone Anatomy 0.000 abstract 1
- 208000010978 mucopolysaccharidosis type 4 Diseases 0.000 abstract 1
Classifications
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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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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
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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
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P19/00—Drugs for skeletal disorders
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61Q—SPECIFIC USE OF COSMETICS OR SIMILAR TOILETRY PREPARATIONS
- A61Q19/00—Preparations for care of the skin
-
- 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)
- C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
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- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K2217/00—Genetically modified animals
- A01K2217/05—Animals comprising random inserted nucleic acids (transgenic)
- A01K2217/054—Animals comprising random inserted nucleic acids (transgenic) inducing loss of function
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- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K2227/00—Animals characterised by species
- A01K2227/10—Mammal
- A01K2227/105—Murine
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- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K2267/00—Animals characterised by purpose
- A01K2267/03—Animal model, e.g. for test or diseases
- A01K2267/0306—Animal model for genetic diseases
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2319/00—Fusion polypeptide
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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
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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
- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y301/00—Hydrolases acting on ester bonds (3.1)
- C12Y301/06—Sulfuric ester hydrolases (3.1.6)
- C12Y301/06004—N-Acetylgalactosamine-6-sulfatase (3.1.6.4)
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- General Health & Medical Sciences (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- Biotechnology (AREA)
- Biomedical Technology (AREA)
- Biochemistry (AREA)
- General Engineering & Computer Science (AREA)
- Molecular Biology (AREA)
- Medicinal Chemistry (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Pharmacology & Pharmacy (AREA)
- Microbiology (AREA)
- Epidemiology (AREA)
- Plant Pathology (AREA)
- Virology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Immunology (AREA)
- Physical Education & Sports Medicine (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Dermatology (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Enzymes And Modification Thereof (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Peptides Or Proteins (AREA)
Abstract
En la presente se proporcionan métodos de terapia génica para el tratamiento de la mucopolisacaridosis tipo IVA (MPS IVA) que implican el uso de virus adenoasociados recombinantes (rAAV) para administrar N-acetilgalactosamina-6-sulfato sulfatasa humana (hGALNS) al hueso de un sujeto humano al que se le diagnosticó MPS IVA. También se proporcionan en la presente rAAV que pueden utilizarse en los métodos de terapia génica y en métodos para elaborar tales rAAV.
Applications Claiming Priority (4)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US201862711238P | 2018-07-27 | 2018-07-27 | |
US201862756880P | 2018-11-07 | 2018-11-07 | |
US201962799834P | 2019-02-01 | 2019-02-01 | |
PCT/US2019/043631 WO2020023857A1 (en) | 2018-07-27 | 2019-07-26 | Treatment of mucopolysaccharidosis iva |
Publications (1)
Publication Number | Publication Date |
---|---|
CO2021002505A2 true CO2021002505A2 (es) | 2021-03-08 |
Family
ID=69180546
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
CONC2021/0002505A CO2021002505A2 (es) | 2018-07-27 | 2021-02-25 | Tratamiento de mucopolisacaridosis iva |
Country Status (12)
Country | Link |
---|---|
US (1) | US20210292789A1 (es) |
EP (1) | EP3829619A4 (es) |
JP (1) | JP2021531044A (es) |
KR (1) | KR20210040984A (es) |
AU (1) | AU2019312301A1 (es) |
BR (1) | BR112021001498A2 (es) |
CA (1) | CA3107800A1 (es) |
CO (1) | CO2021002505A2 (es) |
IL (1) | IL280343A (es) |
SG (1) | SG11202100781VA (es) |
TW (1) | TW202039857A (es) |
WO (1) | WO2020023857A1 (es) |
Families Citing this family (3)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
WO2021021661A1 (en) * | 2019-07-26 | 2021-02-04 | Regenxbio Inc. | Engineered nucleic acid regulatory element and methods of uses thereof |
US20230064077A1 (en) * | 2020-01-29 | 2023-03-02 | Regenxbio Inc. | Treatment of mucopolysaccharidosis iva |
WO2023077092A1 (en) * | 2021-10-28 | 2023-05-04 | Regenxbio Inc. | Engineered nucleic acid regulatory elements and methods and uses thereof |
Family Cites Families (9)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
US20070207139A1 (en) * | 2004-06-10 | 2007-09-06 | Saint Louis University, A Non-Profit Organization | Enhancing the effect of therapeutic proteins on the central nervous system |
US7863238B2 (en) * | 2004-06-10 | 2011-01-04 | Saint Louis University | Proteins with an attached short peptide of acidic amino acids |
US7972593B2 (en) * | 2004-06-10 | 2011-07-05 | Saint Louis University | Delivery of therapeutic agents to the bone |
JP5728389B2 (ja) * | 2009-01-30 | 2015-06-03 | ユニキュアー アイピー ビー.ブイ. | アラニン−グリオキシル酸アミノトランスフェラーゼ治療薬 |
US20140271550A1 (en) * | 2013-03-14 | 2014-09-18 | The Trustees Of The University Of Pennsylvania | Constructs and Methods for Delivering Molecules via Viral Vectors with Blunted Innate Immune Responses |
US20160243260A1 (en) * | 2013-10-24 | 2016-08-25 | Uniqure Ip B.V. | Treatment of neurological diseases using adeno-associated virus (AAV) comprising AAV-5 capsid proteins |
EP3101125A1 (en) * | 2015-06-05 | 2016-12-07 | Laboratorios Del Dr. Esteve, S.A. | Adenoassociated virus vectors for the treatment of mucopolysaccharidoses |
JP6877408B2 (ja) * | 2015-08-31 | 2021-05-26 | 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 | ペット治療用aav−epo |
KR20210027266A (ko) * | 2018-05-30 | 2021-03-10 | 에스테베 파마슈티칼스 에스에이 | 뮤코다당질축적증 iv a형의 치료를 위한 아데노관련 바이러스 벡터 |
-
2019
- 2019-07-26 BR BR112021001498-5A patent/BR112021001498A2/pt unknown
- 2019-07-26 JP JP2021527020A patent/JP2021531044A/ja active Pending
- 2019-07-26 CA CA3107800A patent/CA3107800A1/en active Pending
- 2019-07-26 EP EP19841900.4A patent/EP3829619A4/en active Pending
- 2019-07-26 KR KR1020217004895A patent/KR20210040984A/ko not_active Application Discontinuation
- 2019-07-26 TW TW108126686A patent/TW202039857A/zh unknown
- 2019-07-26 US US17/263,049 patent/US20210292789A1/en active Pending
- 2019-07-26 SG SG11202100781VA patent/SG11202100781VA/en unknown
- 2019-07-26 AU AU2019312301A patent/AU2019312301A1/en active Pending
- 2019-07-26 WO PCT/US2019/043631 patent/WO2020023857A1/en unknown
-
2021
- 2021-01-21 IL IL280343A patent/IL280343A/en unknown
- 2021-02-25 CO CONC2021/0002505A patent/CO2021002505A2/es unknown
Also Published As
Publication number | Publication date |
---|---|
TW202039857A (zh) | 2020-11-01 |
WO2020023857A1 (en) | 2020-01-30 |
BR112021001498A2 (pt) | 2021-04-27 |
AU2019312301A1 (en) | 2021-02-18 |
IL280343A (en) | 2021-03-25 |
JP2021531044A (ja) | 2021-11-18 |
EP3829619A1 (en) | 2021-06-09 |
SG11202100781VA (en) | 2021-02-25 |
EP3829619A4 (en) | 2022-05-18 |
KR20210040984A (ko) | 2021-04-14 |
CA3107800A1 (en) | 2020-01-30 |
US20210292789A1 (en) | 2021-09-23 |
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