AR121201A1 - Tratamiento de mucopolisacaridosis iva - Google Patents
Tratamiento de mucopolisacaridosis ivaInfo
- Publication number
- AR121201A1 AR121201A1 ARP210100217A ARP210100217A AR121201A1 AR 121201 A1 AR121201 A1 AR 121201A1 AR P210100217 A ARP210100217 A AR P210100217A AR P210100217 A ARP210100217 A AR P210100217A AR 121201 A1 AR121201 A1 AR 121201A1
- Authority
- AR
- Argentina
- Prior art keywords
- treatment
- iva
- raavs
- gene therapy
- mps
- Prior art date
Links
- 208000025797 Mucopolysaccharidosis type 4A Diseases 0.000 title abstract 2
- 208000012253 mucopolysaccharidosis IVA Diseases 0.000 title abstract 2
- 238000000034 method Methods 0.000 abstract 3
- 101001066305 Homo sapiens N-acetylgalactosamine-6-sulfatase Proteins 0.000 abstract 2
- 206010028095 Mucopolysaccharidosis IV Diseases 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
- 210000000988 bone and bone Anatomy 0.000 abstract 1
- 208000010978 mucopolysaccharidosis type 4 Diseases 0.000 abstract 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
- 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
-
- 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
- A01K2217/07—Animals genetically altered by homologous recombination
- A01K2217/075—Animals genetically altered by homologous recombination inducing loss of function, i.e. knock out
-
- 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
- C07K2319/33—Fusion polypeptide fusions for targeting to specific cell types, e.g. tissue specific targeting, targeting of a bacterial subspecies
-
- 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
- C12N2800/00—Nucleic acids vectors
- C12N2800/22—Vectors comprising a coding region that has been codon optimised for expression in a respective host
-
- 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
-
- 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
- C12N2830/15—Vector systems having a special element relevant for transcription chimeric enhancer/promoter combination
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Chemical & Material Sciences (AREA)
- Biotechnology (AREA)
- Biomedical Technology (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- General Health & Medical Sciences (AREA)
- Biochemistry (AREA)
- General Engineering & Computer Science (AREA)
- Molecular Biology (AREA)
- Microbiology (AREA)
- Medicinal Chemistry (AREA)
- Epidemiology (AREA)
- Public Health (AREA)
- Biophysics (AREA)
- Virology (AREA)
- Pharmacology & Pharmacy (AREA)
- Plant Pathology (AREA)
- Animal Behavior & Ethology (AREA)
- Physics & Mathematics (AREA)
- Veterinary Medicine (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines Containing Plant Substances (AREA)
- Polysaccharides And Polysaccharide Derivatives (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
Applications Claiming Priority (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202062967499P | 2020-01-29 | 2020-01-29 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| AR121201A1 true AR121201A1 (es) | 2022-04-27 |
Family
ID=74673384
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| ARP210100217A AR121201A1 (es) | 2020-01-29 | 2021-01-28 | Tratamiento de mucopolisacaridosis iva |
Country Status (11)
| Country | Link |
|---|---|
| US (1) | US20230064077A1 (enExample) |
| EP (1) | EP4097238A1 (enExample) |
| JP (1) | JP2023512043A (enExample) |
| KR (1) | KR20220133900A (enExample) |
| AR (1) | AR121201A1 (enExample) |
| AU (1) | AU2021212749A1 (enExample) |
| BR (1) | BR112022015036A2 (enExample) |
| CA (1) | CA3168251A1 (enExample) |
| IL (1) | IL294638A (enExample) |
| TW (1) | TW202136516A (enExample) |
| WO (1) | WO2021154956A1 (enExample) |
Families Citing this family (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| TW202039857A (zh) | 2018-07-27 | 2020-11-01 | 美商銳進科斯生物股份有限公司 | 黏多醣病iva之治療 |
Family Cites Families (21)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP2369002A1 (en) | 1999-08-09 | 2011-09-28 | Targeted Genetics Corporation | Enhancement of expression of a single-stranded, heterologous nucleotide sequence from recombinant viral vectors by designing the sequence such that it forms intrastrand base pairs |
| SG10202108118RA (en) | 2001-11-13 | 2021-08-30 | Univ Pennsylvania | A method of detecting and/or identifying adeno-associated virus (aav) sequences and isolating novel sequences identified thereby |
| ES2975413T3 (es) | 2001-12-17 | 2024-07-05 | Univ Pennsylvania | Secuencias de serotipo 8 de virus adenoasociado (AAV), vectores que las contienen y usos de las mismas |
| HUE054805T2 (hu) | 2003-09-30 | 2021-09-28 | Univ Pennsylvania | Adeno-asszociált vírus (AAV) kládok, szekvenciák, az azokat tartalmazó vektorok és alkalmazásaik |
| EP3085389A1 (en) | 2005-04-07 | 2016-10-26 | The Trustees Of The University Of Pennsylvania | Method of increasing the function of an aav vector |
| WO2006132118A1 (ja) | 2005-06-09 | 2006-12-14 | Matsushita Electric Industrial Co., Ltd. | 振幅誤差補償装置及び直交度誤差補償装置 |
| WO2010138263A2 (en) | 2009-05-28 | 2010-12-02 | University Of Massachusetts | Novel aav 's and uses thereof |
| US8927514B2 (en) | 2010-04-30 | 2015-01-06 | City Of Hope | Recombinant adeno-associated vectors for targeted treatment |
| US8628966B2 (en) | 2010-04-30 | 2014-01-14 | City Of Hope | CD34-derived recombinant adeno-associated vectors for stem cell transduction and systemic therapeutic gene transfer |
| CN107828820B (zh) | 2010-10-27 | 2022-06-07 | 学校法人自治医科大学 | 用于向神经系统细胞导入基因的腺相关病毒粒子 |
| US9409953B2 (en) | 2011-02-10 | 2016-08-09 | The University Of North Carolina At Chapel Hill | Viral vectors with modified transduction profiles and methods of making and using the same |
| CN107012171B (zh) | 2011-04-22 | 2021-07-13 | 加利福尼亚大学董事会 | 具有变异衣壳的腺相关病毒病毒体及其使用方法 |
| EP3147295B2 (en) | 2011-08-24 | 2023-11-22 | The Board of Trustees of the Leland Stanford Junior University | New avv capsid proteins for nucleic acid transfer |
| EP3470523A1 (en) | 2012-05-09 | 2019-04-17 | Oregon Health & Science University | Adeno associated virus plasmids and vectors |
| CA2905952A1 (en) | 2013-03-13 | 2014-10-02 | The Children's Hospital Of Philadelphia | Adeno-associated virus vectors and methods of use thereof |
| PT3024498T (pt) | 2013-07-22 | 2020-03-06 | Childrens Hospital Philadelphia | Variante aav e composições, métodos e usos para transferência genética em células, órgãos e tecidos |
| EP3564379A1 (en) | 2013-09-13 | 2019-11-06 | California Institute of Technology | Selective recovery |
| NZ718926A (en) | 2013-10-11 | 2021-12-24 | Massachusetts Eye & Ear Infirmary | Methods of predicting ancestral virus sequences and uses thereof |
| US10746742B2 (en) | 2014-04-25 | 2020-08-18 | Oregon Health & Science University | Methods of viral neutralizing antibody epitope mapping |
| US12146169B2 (en) * | 2018-05-30 | 2024-11-19 | Esteve Pharmaceuticals, S.A. | Adenoassociated virus vectors for the treatment of mucopolysaccharidoses type IV A |
| TW202039857A (zh) * | 2018-07-27 | 2020-11-01 | 美商銳進科斯生物股份有限公司 | 黏多醣病iva之治療 |
-
2021
- 2021-01-28 WO PCT/US2021/015436 patent/WO2021154956A1/en not_active Ceased
- 2021-01-28 EP EP21707517.5A patent/EP4097238A1/en not_active Withdrawn
- 2021-01-28 KR KR1020227027208A patent/KR20220133900A/ko not_active Withdrawn
- 2021-01-28 CA CA3168251A patent/CA3168251A1/en active Pending
- 2021-01-28 JP JP2022546078A patent/JP2023512043A/ja active Pending
- 2021-01-28 TW TW110103306A patent/TW202136516A/zh unknown
- 2021-01-28 US US17/792,969 patent/US20230064077A1/en not_active Abandoned
- 2021-01-28 AR ARP210100217A patent/AR121201A1/es unknown
- 2021-01-28 IL IL294638A patent/IL294638A/en unknown
- 2021-01-28 AU AU2021212749A patent/AU2021212749A1/en not_active Abandoned
- 2021-01-28 BR BR112022015036A patent/BR112022015036A2/pt not_active Application Discontinuation
Also Published As
| Publication number | Publication date |
|---|---|
| KR20220133900A (ko) | 2022-10-05 |
| WO2021154956A1 (en) | 2021-08-05 |
| EP4097238A1 (en) | 2022-12-07 |
| JP2023512043A (ja) | 2023-03-23 |
| CA3168251A1 (en) | 2021-08-05 |
| IL294638A (en) | 2022-09-01 |
| US20230064077A1 (en) | 2023-03-02 |
| BR112022015036A2 (pt) | 2022-10-11 |
| AU2021212749A1 (en) | 2022-08-18 |
| TW202136516A (zh) | 2021-10-01 |
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