CL2019002474A1 - Vector clado f de virus adeno-asociado (vaa) y usos relacionados. - Google Patents
Vector clado f de virus adeno-asociado (vaa) y usos relacionados.Info
- Publication number
- CL2019002474A1 CL2019002474A1 CL2019002474A CL2019002474A CL2019002474A1 CL 2019002474 A1 CL2019002474 A1 CL 2019002474A1 CL 2019002474 A CL2019002474 A CL 2019002474A CL 2019002474 A CL2019002474 A CL 2019002474A CL 2019002474 A1 CL2019002474 A1 CL 2019002474A1
- Authority
- CL
- Chile
- Prior art keywords
- vector
- associated virus
- raav
- seq
- aavhu68
- Prior art date
Links
- 241000702421 Dependoparvovirus Species 0.000 title abstract 2
- 210000000234 capsid Anatomy 0.000 abstract 3
- FWMNVWWHGCHHJJ-SKKKGAJSSA-N 4-amino-1-[(2r)-6-amino-2-[[(2r)-2-[[(2r)-2-[[(2r)-2-amino-3-phenylpropanoyl]amino]-3-phenylpropanoyl]amino]-4-methylpentanoyl]amino]hexanoyl]piperidine-4-carboxylic acid Chemical class C([C@H](C(=O)N[C@H](CC(C)C)C(=O)N[C@H](CCCCN)C(=O)N1CCC(N)(CC1)C(O)=O)NC(=O)[C@H](N)CC=1C=CC=CC=1)C1=CC=CC=C1 FWMNVWWHGCHHJJ-SKKKGAJSSA-N 0.000 abstract 2
- 150000001413 amino acids Chemical group 0.000 abstract 2
- 239000004471 Glycine Substances 0.000 abstract 1
- 125000000010 L-asparaginyl group Chemical group O=C([*])[C@](N([H])[H])([H])C([H])([H])C(=O)N([H])[H] 0.000 abstract 1
- 235000001014 amino acid Nutrition 0.000 abstract 1
- 239000000203 mixture Substances 0.000 abstract 1
- 239000002773 nucleotide Substances 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
- C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
-
- 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
-
- 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
- A61K35/761—Adenovirus
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K16/00—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies
- C07K16/18—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans
- C07K16/32—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans against translation products of oncogenes
-
- 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
-
- 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
-
- 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
-
- 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
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- Genetics & Genomics (AREA)
- General Health & Medical Sciences (AREA)
- Virology (AREA)
- Medicinal Chemistry (AREA)
- Engineering & Computer Science (AREA)
- Zoology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Wood Science & Technology (AREA)
- Biochemistry (AREA)
- Microbiology (AREA)
- Immunology (AREA)
- General Engineering & Computer Science (AREA)
- Biomedical Technology (AREA)
- Biotechnology (AREA)
- Molecular Biology (AREA)
- Biophysics (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Mycology (AREA)
- Epidemiology (AREA)
- Gastroenterology & Hepatology (AREA)
- Oncology (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Peptides Or Proteins (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
Abstract
UN VECTOR DE VIRUS ADENOASOCIADO RECOMBINANTE (RAAV), QUE COMPRENDE UNA CÁPSIDE DE AAVHU68 PRODUCIDA EN UN SISTEMA DE PRODUCCIÓN QUE COMPRENDE UNA SECUENCIA DE NUCLEÓTIDOS DE LA SEQ ID NO: 1, O UNA SECUENCIA AL MENOS 75 % IDÉNTICA A ESTA, QUE CODIFICA LA SEQ ID NO:2. LA CÁPSIDE DE AAVHU68 COMPRENDE SUBPOBLACIONES DE RESIDUOS DE ASPARAGINA MUY DESAMIDADOS EN LOS PARES DE ASPARAGINA-GLICINA EN LA SECUENCIA DE AMINOÁCIDOS DE LA SEQ ID NO: 2. TAMBIÉN SE PROPORCIONAN COMPOSICIONES QUE CONTIENEN EL RAAV Y USOS DE ESTAS. ADEMÁS, SE PROPORCIONAN RAAV QUE TIENEN UNA CÁPSIDE DE AAV MODIFICADA, QUE COMPRENDE AL MENOS UNA SUBPOBLACIÓN DE PROTEÍNAS VP1 O VP2 CON UNA VAL EN LA POSICIÓN DE AMINOÁCIDOS 157, CON REFERENCIA A LA NUMERACIÓN DE VP1 DE AAVHU68.
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201762464748P | 2017-02-28 | 2017-02-28 | |
| US201762591002P | 2017-11-27 | 2017-11-27 | |
| US201862614002P | 2018-01-05 | 2018-01-05 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| CL2019002474A1 true CL2019002474A1 (es) | 2019-11-29 |
Family
ID=61628475
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CL2019002474A CL2019002474A1 (es) | 2017-02-28 | 2019-08-27 | Vector clado f de virus adeno-asociado (vaa) y usos relacionados. |
Country Status (29)
| Country | Link |
|---|---|
| US (2) | US11827906B2 (es) |
| EP (2) | EP3589730B1 (es) |
| JP (3) | JP7455579B2 (es) |
| KR (1) | KR102775350B1 (es) |
| CN (2) | CN110741082B (es) |
| AU (2) | AU2018227440B2 (es) |
| BR (1) | BR112019017839A2 (es) |
| CA (1) | CA3053399A1 (es) |
| CL (1) | CL2019002474A1 (es) |
| CO (1) | CO2019009525A2 (es) |
| DK (1) | DK3589730T5 (es) |
| ES (1) | ES2971872T3 (es) |
| FI (1) | FI3589730T3 (es) |
| HR (1) | HRP20240257T1 (es) |
| HU (1) | HUE065885T2 (es) |
| IL (1) | IL268644B2 (es) |
| LT (1) | LT3589730T (es) |
| MD (1) | MD3589730T2 (es) |
| MX (2) | MX2019010275A (es) |
| PE (1) | PE20191362A1 (es) |
| PL (1) | PL3589730T3 (es) |
| PT (1) | PT3589730T (es) |
| RS (1) | RS65241B1 (es) |
| SA (1) | SA519402565B1 (es) |
| SG (4) | SG10201912973XA (es) |
| SI (1) | SI3589730T1 (es) |
| SM (1) | SMT202400080T1 (es) |
| WO (1) | WO2018160582A1 (es) |
| ZA (1) | ZA201905407B (es) |
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| EP3211085B1 (en) | 2003-09-30 | 2021-03-31 | The Trustees of The University of Pennsylvania | Adeno-associated virus (aav) clades, sequences, vectors containing same, and uses therefor |
| USRE50283E1 (en) | 2013-04-18 | 2025-01-28 | Fondazione Telethon Ets | Effective delivery of large genes by dual AAV vectors |
| EP3831842A1 (en) | 2015-09-28 | 2021-06-09 | The University of North Carolina at Chapel Hill | Methods and compositions for antibody-evading virus vectors |
| AU2017207906B2 (en) * | 2016-01-15 | 2021-03-11 | American Gene Technologies International Inc. | Methods and compositions for the activation of gamma-delta T-cells |
| SI3589730T1 (sl) * | 2017-02-28 | 2024-04-30 | The Trustees Of The University Of Pennsylvania | Adeno-povezan virus (AAV) clade F vektor in njihova uporaba |
| JOP20190200A1 (ar) | 2017-02-28 | 2019-08-27 | Univ Pennsylvania | تركيبات نافعة في معالجة ضمور العضل النخاعي |
| SG10201912401QA (en) | 2017-05-11 | 2020-02-27 | Univ Pennsylvania | Gene therapy for neuronal ceroid lipofuscinoses |
| MX2020005663A (es) | 2017-11-30 | 2020-08-20 | Univ Pennsylvania | Terapia genica para mucopolisacaridosis iii a. |
| US11723989B2 (en) | 2017-11-30 | 2023-08-15 | The Trustees Of The University Of Pennsylvania | Gene therapy for mucopolysaccharidosis IIIB |
| MX2020008932A (es) * | 2018-02-27 | 2020-10-01 | Univ Pennsylvania | Novedosos vectores de virus adenoasociado (aav), vectores de aav que tienen una desamidacion reducida de la capside y usos de estos. |
| MX2020008933A (es) * | 2018-02-27 | 2021-01-15 | Univ Pennsylvania | Novedosos vectores de virus adenoasociado (aav), vectores de aav que tienen una desamidacion reducida de la capside y usos de estos. |
| US12091435B2 (en) | 2018-04-03 | 2024-09-17 | Ginkgo Bioworks, Inc. | Antibody-evading virus vectors |
| SG11202009450SA (en) | 2018-04-03 | 2020-10-29 | Stridebio Inc | Virus vectors for targeting ophthalmic tissues |
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| AU2020229772A1 (en) * | 2019-02-26 | 2021-08-26 | The Trustees Of The University Of Pennsylvania | Compositions useful in treatment of krabbe disease |
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