CL2023002530A1 - Composición y métodos para el tratamiento de la enfermedad de fabry - Google Patents
Composición y métodos para el tratamiento de la enfermedad de fabryInfo
- Publication number
- CL2023002530A1 CL2023002530A1 CL2023002530A CL2023002530A CL2023002530A1 CL 2023002530 A1 CL2023002530 A1 CL 2023002530A1 CL 2023002530 A CL2023002530 A CL 2023002530A CL 2023002530 A CL2023002530 A CL 2023002530A CL 2023002530 A1 CL2023002530 A1 CL 2023002530A1
- Authority
- CL
- Chile
- Prior art keywords
- fabry disease
- subject
- treatment
- composition
- methods
- Prior art date
Links
- 208000024720 Fabry Disease Diseases 0.000 title abstract 2
- 241000283923 Marmota monax Species 0.000 abstract 1
- 101000718529 Saccharolobus solfataricus (strain ATCC 35092 / DSM 1617 / JCM 11322 / P2) Alpha-galactosidase Proteins 0.000 abstract 1
- 241000700605 Viruses Species 0.000 abstract 1
- 108091093126 WHP Posttrascriptional Response Element Proteins 0.000 abstract 1
- 210000000234 capsid Anatomy 0.000 abstract 1
- 208000006454 hepatitis Diseases 0.000 abstract 1
- 231100000283 hepatitis Toxicity 0.000 abstract 1
- 239000002773 nucleotide Substances 0.000 abstract 1
- 125000003729 nucleotide group Chemical group 0.000 abstract 1
- 230000001124 posttranscriptional effect Effects 0.000 abstract 1
- 230000001105 regulatory effect Effects 0.000 abstract 1
- 230000005100 tissue tropism Effects 0.000 abstract 1
- 239000013598 vector Substances 0.000 abstract 1
- 239000013603 viral vector Substances 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
-
- 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
-
- 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
- A61P3/00—Drugs for disorders of the metabolism
-
- 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
-
- 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)
- C12N9/2465—Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1) acting on alpha-galactose-glycoside bonds, e.g. alpha-galactosidase (3.2.1.22)
-
- 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)
- C12Y302/01022—Alpha-galactosidase (3.2.1.22)
-
- 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
- C12N2750/14122—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
-
- 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
-
- 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
- C12N2830/42—Vector systems having a special element relevant for transcription being an intron or intervening sequence for splicing and/or stability of RNA
-
- 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
- C12N2830/48—Vector systems having a special element relevant for transcription regulating transport or export of RNA, e.g. RRE, PRE, WPRE, CTE
-
- 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
- C12N2830/50—Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal
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)
- Medicinal Chemistry (AREA)
- General Engineering & Computer Science (AREA)
- Molecular Biology (AREA)
- Biochemistry (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Microbiology (AREA)
- Epidemiology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Virology (AREA)
- Physics & Mathematics (AREA)
- Obesity (AREA)
- Hematology (AREA)
- Diabetes (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
Applications Claiming Priority (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202163154485P | 2021-02-26 | 2021-02-26 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| CL2023002530A1 true CL2023002530A1 (es) | 2024-04-19 |
Family
ID=80786597
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CL2023002530A CL2023002530A1 (es) | 2021-02-26 | 2023-08-25 | Composición y métodos para el tratamiento de la enfermedad de fabry |
Country Status (17)
| Country | Link |
|---|---|
| US (1) | US20240175054A1 (https=) |
| EP (1) | EP4298227A1 (https=) |
| JP (1) | JP2024509792A (https=) |
| KR (1) | KR20230150836A (https=) |
| CN (1) | CN117321212A (https=) |
| AR (1) | AR124981A1 (https=) |
| AU (1) | AU2022227017A1 (https=) |
| BR (1) | BR112023016983A2 (https=) |
| CA (1) | CA3209673A1 (https=) |
| CL (1) | CL2023002530A1 (https=) |
| CO (1) | CO2023011012A2 (https=) |
| EC (1) | ECSP23072174A (https=) |
| IL (1) | IL305440A (https=) |
| MX (1) | MX2023009978A (https=) |
| PE (1) | PE20240806A1 (https=) |
| TW (1) | TW202302855A (https=) |
| WO (1) | WO2022183052A1 (https=) |
Families Citing this family (3)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP2025529884A (ja) * | 2022-08-25 | 2025-09-09 | 武田薬品工業株式会社 | ファブリー病の治療に使用するための組成物 |
| CN121002183A (zh) * | 2023-04-17 | 2025-11-21 | 杭州复因生物科技有限公司 | 用于基因治疗的组合物和方法 |
| WO2024236544A1 (en) | 2023-05-17 | 2024-11-21 | Takeda Pharmaceutical Company Limited | Pet tracer compounds for tracing alpha-galactosidase a and reaction intermediates, and methods of use |
Family Cites Families (26)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5139941A (en) | 1985-10-31 | 1992-08-18 | University Of Florida Research Foundation, Inc. | AAV transduction vectors |
| US5436146A (en) | 1989-09-07 | 1995-07-25 | The Trustees Of Princeton University | Helper-free stocks of recombinant adeno-associated virus vectors |
| US6268213B1 (en) | 1992-06-03 | 2001-07-31 | Richard Jude Samulski | Adeno-associated virus vector and cis-acting regulatory and promoter elements capable of expressing at least one gene and method of using same for gene therapy |
| US5869305A (en) | 1992-12-04 | 1999-02-09 | The University Of Pittsburgh | Recombinant viral vector system |
| US5478745A (en) | 1992-12-04 | 1995-12-26 | University Of Pittsburgh | Recombinant viral vector system |
| US6204059B1 (en) | 1994-06-30 | 2001-03-20 | University Of Pittsburgh | AAV capsid vehicles for molecular transfer |
| US6093570A (en) | 1995-06-07 | 2000-07-25 | The University Of North Carolina At Chapel Hill | Helper virus-free AAV production |
| US5741683A (en) | 1995-06-07 | 1998-04-21 | The Research Foundation Of State University Of New York | In vitro packaging of adeno-associated virus DNA |
| WO1998046728A1 (en) | 1997-04-14 | 1998-10-22 | Cell Genesys, Inc. | Methods for increasing the efficiency of recombinant aav product |
| WO1999061643A1 (en) | 1998-05-27 | 1999-12-02 | University Of Florida | Method of preparing recombinant adeno-associated virus compositions by using an iodixananol gradient |
| ES2340230T3 (es) | 1998-11-10 | 2010-05-31 | University Of North Carolina At Chapel Hill | Vectores viricos y sus procedimientos de preparacion y administracion. |
| US7201898B2 (en) | 2000-06-01 | 2007-04-10 | The University Of North Carolina At Chapel Hill | Methods and compounds for controlled release of recombinant parvovirus vectors |
| NZ532635A (en) | 2001-11-13 | 2007-05-31 | Univ Pennsylvania | A method of identifying unknown adeno-associated virus (AAV) sequences and a kit for the method |
| DK2359869T3 (en) | 2001-12-17 | 2019-04-15 | Univ Pennsylvania | Sequences of adeno-associated virus (AAV) serotype 8, vectors containing these, and uses thereof |
| AU2003274397A1 (en) | 2002-06-05 | 2003-12-22 | University Of Florida | Production of pseudotyped recombinant aav virions |
| CN1856576B (zh) | 2003-09-30 | 2011-05-04 | 宾夕法尼亚州立大学托管会 | 腺伴随病毒(aav)进化支、序列、含有这些序列的载体及它们的应用 |
| WO2006110689A2 (en) | 2005-04-07 | 2006-10-19 | The Trustees Of The University Of Pennsylvania | Method of increasing the function of an aav vector |
| US7588772B2 (en) | 2006-03-30 | 2009-09-15 | Board Of Trustees Of The Leland Stamford Junior University | AAV capsid library and AAV capsid proteins |
| US20120322861A1 (en) | 2007-02-23 | 2012-12-20 | Barry John Byrne | Compositions and Methods for Treating Diseases |
| US9249425B2 (en) | 2011-05-16 | 2016-02-02 | The Trustees Of The University Of Pennslyvania | Proviral plasmids and production of recombinant adeno-associated virus |
| EP3794131A4 (en) * | 2018-05-15 | 2022-01-26 | University of Massachusetts | RAAV VECTORS ENCODING FOR LYSOSOMAL BETA-GALACTOSIDASE (GLB1) AND CATHEPSIN A |
| AR116624A1 (es) * | 2018-10-10 | 2021-05-26 | Amicus Therapeutics Inc | Composiciones de polipéptidos estabilizados por enlaces disulfuro y métodos de uso |
| US12454701B2 (en) * | 2018-12-05 | 2025-10-28 | Abeona Therapeutics Inc. | Recombinant adeno-associated viral vector for gene delivery |
| SG11202106348QA (en) * | 2018-12-20 | 2021-07-29 | Codexis Inc | Human alpha-galactosidase variants |
| JP7716338B2 (ja) * | 2019-01-04 | 2025-07-31 | サンガモ セラピューティクス， インコーポレイテッド | ファブリー病の処置のための方法及び組成物 |
| AU2021356684A1 (en) * | 2020-10-09 | 2023-05-11 | The Trustees Of The University Of Pennsylvania | Compositions and methods for treatment of fabry disease |
-
2022
- 2022-02-25 KR KR1020237032457A patent/KR20230150836A/ko active Pending
- 2022-02-25 IL IL305440A patent/IL305440A/en unknown
- 2022-02-25 PE PE2023002431A patent/PE20240806A1/es unknown
- 2022-02-25 CN CN202280030576.5A patent/CN117321212A/zh active Pending
- 2022-02-25 AR ARP220100423A patent/AR124981A1/es unknown
- 2022-02-25 CA CA3209673A patent/CA3209673A1/en active Pending
- 2022-02-25 EP EP22711380.0A patent/EP4298227A1/en active Pending
- 2022-02-25 MX MX2023009978A patent/MX2023009978A/es unknown
- 2022-02-25 WO PCT/US2022/017998 patent/WO2022183052A1/en not_active Ceased
- 2022-02-25 BR BR112023016983A patent/BR112023016983A2/pt not_active Application Discontinuation
- 2022-02-25 US US18/547,809 patent/US20240175054A1/en active Pending
- 2022-02-25 TW TW111107143A patent/TW202302855A/zh unknown
- 2022-02-25 JP JP2023552146A patent/JP2024509792A/ja not_active Withdrawn
- 2022-02-25 AU AU2022227017A patent/AU2022227017A1/en not_active Abandoned
-
2023
- 2023-08-24 CO CONC2023/0011012A patent/CO2023011012A2/es unknown
- 2023-08-25 CL CL2023002530A patent/CL2023002530A1/es unknown
- 2023-09-22 EC ECSENADI202372174A patent/ECSP23072174A/es unknown
Also Published As
| Publication number | Publication date |
|---|---|
| CO2023011012A2 (es) | 2023-08-28 |
| PE20240806A1 (es) | 2024-04-18 |
| MX2023009978A (es) | 2023-09-06 |
| US20240175054A1 (en) | 2024-05-30 |
| AU2022227017A1 (en) | 2023-09-07 |
| ECSP23072174A (es) | 2023-10-31 |
| AR124981A1 (es) | 2023-05-24 |
| KR20230150836A (ko) | 2023-10-31 |
| IL305440A (en) | 2023-10-01 |
| EP4298227A1 (en) | 2024-01-03 |
| CN117321212A (zh) | 2023-12-29 |
| JP2024509792A (ja) | 2024-03-05 |
| CA3209673A1 (en) | 2022-09-01 |
| TW202302855A (zh) | 2023-01-16 |
| BR112023016983A2 (pt) | 2023-11-07 |
| WO2022183052A1 (en) | 2022-09-01 |
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