IL280343A - Treatment of mucopolysaccharidosis iva - Google Patents
Treatment of mucopolysaccharidosis ivaInfo
- Publication number
- IL280343A IL280343A IL280343A IL28034321A IL280343A IL 280343 A IL280343 A IL 280343A IL 280343 A IL280343 A IL 280343A IL 28034321 A IL28034321 A IL 28034321A IL 280343 A IL280343 A IL 280343A
- Authority
- IL
- Israel
- Prior art keywords
- treatment
- mucopolysaccharidosis iva
- mucopolysaccharidosis
- iva
- Prior art date
Links
- 208000025797 Mucopolysaccharidosis type 4A Diseases 0.000 title 1
- 208000012253 mucopolysaccharidosis IVA Diseases 0.000 title 1
Classifications
-
- 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
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
-
- 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
-
- 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)
-
- 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)
-
- 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
-
- 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
-
- 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
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2319/00—Fusion polypeptide
-
- 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
- C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination
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)
- Biomedical Technology (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Biochemistry (AREA)
- Molecular Biology (AREA)
- Medicinal Chemistry (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Microbiology (AREA)
- Epidemiology (AREA)
- Virology (AREA)
- Biophysics (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Immunology (AREA)
- General Chemical & Material Sciences (AREA)
- Physical Education & Sports Medicine (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)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Peptides Or Proteins (AREA)
- Enzymes And Modification Thereof (AREA)
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 |
|---|---|
| IL280343A true IL280343A (en) | 2021-03-25 |
Family
ID=69180546
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| IL280343A IL280343A (en) | 2018-07-27 | 2021-01-21 | Treatment of mucopolysaccharidosis iva |
Country Status (12)
| Country | Link |
|---|---|
| US (1) | US12247213B2 (enExample) |
| EP (1) | EP3829619A4 (enExample) |
| JP (1) | JP2021531044A (enExample) |
| KR (1) | KR20210040984A (enExample) |
| AU (1) | AU2019312301A1 (enExample) |
| BR (1) | BR112021001498A2 (enExample) |
| CA (1) | CA3107800A1 (enExample) |
| CO (1) | CO2021002505A2 (enExample) |
| IL (1) | IL280343A (enExample) |
| SG (1) | SG11202100781VA (enExample) |
| TW (1) | TW202039857A (enExample) |
| WO (1) | WO2020023857A1 (enExample) |
Families Citing this family (5)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| BR112021001498A2 (pt) | 2018-07-27 | 2021-04-27 | Regenxbio Inc. | vírus adeno-associado recombinante (raav), composição farmacêutica, polinucleotídeo, plasmídeo de raav, célula ex vivo, método de produção de um raav e métodos para tratar um sujeito humano diagnosticado com mucopolissacaridose tipo iva (mps iva) |
| CN113226352B (zh) | 2018-08-24 | 2025-08-26 | 星火治疗有限公司 | 最优化启动子序列、无内含子的表达结构及使用方法 |
| EP4004214A1 (en) * | 2019-07-26 | 2022-06-01 | RegenxBio Inc. | Engineered nucleic acid regulatory element and methods of uses thereof |
| BR112022015036A2 (pt) * | 2020-01-29 | 2022-10-11 | Regenxbio Inc | Vírus adenoassociado recombinante (raav), composição farmacêutica, polinucleotídeo, plasmídeo de raav, célula ex vivo, método de produção de um raav, método para tratar um sujeito humano diagnosticado com mucopolissacaridose tipo iva (mps iva) |
| EP4423285A1 (en) * | 2021-10-28 | 2024-09-04 | RegenxBio Inc. | Engineered nucleic acid regulatory elements and methods and uses thereof |
Family Cites Families (11)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US7972593B2 (en) | 2004-06-10 | 2011-07-05 | Saint Louis University | Delivery of therapeutic agents to the bone |
| 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 |
| EP2384200B1 (en) | 2009-01-30 | 2013-07-10 | UniQure IP B.V. | Alanine-glyoxylate aminotransferase therapeutics |
| 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 |
| WO2015060722A1 (en) * | 2013-10-24 | 2015-04-30 | Uniqure Ip B.V. | Aav-5 pseudotyped vector for gene therapy for neurological diseases |
| EP3101125A1 (en) * | 2015-06-05 | 2016-12-07 | Laboratorios Del Dr. Esteve, S.A. | Adenoassociated virus vectors for the treatment of mucopolysaccharidoses |
| HK1257024A1 (zh) | 2015-08-31 | 2019-10-11 | 宾夕法尼亚大学 | 用於治疗伴侣动物的aav-epo |
| MY204731A (en) * | 2018-05-30 | 2024-09-11 | Esteve Pharmaceuticals Sa | Adenoassociated virus vectors for the treatment of mucopolysaccharidoses type iv a |
| BR112021001498A2 (pt) | 2018-07-27 | 2021-04-27 | Regenxbio Inc. | vírus adeno-associado recombinante (raav), composição farmacêutica, polinucleotídeo, plasmídeo de raav, célula ex vivo, método de produção de um raav e métodos para tratar um sujeito humano diagnosticado com mucopolissacaridose tipo iva (mps iva) |
| BR112022015036A2 (pt) | 2020-01-29 | 2022-10-11 | Regenxbio Inc | Vírus adenoassociado recombinante (raav), composição farmacêutica, polinucleotídeo, plasmídeo de raav, célula ex vivo, método de produção de um raav, método para tratar um sujeito humano diagnosticado com mucopolissacaridose tipo iva (mps iva) |
-
2019
- 2019-07-26 BR BR112021001498-5A patent/BR112021001498A2/pt not_active IP Right Cessation
- 2019-07-26 KR KR1020217004895A patent/KR20210040984A/ko not_active Withdrawn
- 2019-07-26 CA CA3107800A patent/CA3107800A1/en active Pending
- 2019-07-26 JP JP2021527020A patent/JP2021531044A/ja active Pending
- 2019-07-26 TW TW108126686A patent/TW202039857A/zh unknown
- 2019-07-26 EP EP19841900.4A patent/EP3829619A4/en not_active Withdrawn
- 2019-07-26 WO PCT/US2019/043631 patent/WO2020023857A1/en not_active Ceased
- 2019-07-26 AU AU2019312301A patent/AU2019312301A1/en not_active Abandoned
- 2019-07-26 US US17/263,049 patent/US12247213B2/en active Active
- 2019-07-26 SG SG11202100781VA patent/SG11202100781VA/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 |
|---|---|
| CA3107800A1 (en) | 2020-01-30 |
| US12247213B2 (en) | 2025-03-11 |
| TW202039857A (zh) | 2020-11-01 |
| CO2021002505A2 (es) | 2021-03-08 |
| WO2020023857A1 (en) | 2020-01-30 |
| EP3829619A1 (en) | 2021-06-09 |
| EP3829619A4 (en) | 2022-05-18 |
| AU2019312301A1 (en) | 2021-02-18 |
| KR20210040984A (ko) | 2021-04-14 |
| US20210292789A1 (en) | 2021-09-23 |
| BR112021001498A2 (pt) | 2021-04-27 |
| JP2021531044A (ja) | 2021-11-18 |
| SG11202100781VA (en) | 2021-02-25 |
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