BR112014025985A2 - composição e métodos para transferência de gene altamente eficaz com o uso de variantes de capsídeo de aav - Google Patents
composição e métodos para transferência de gene altamente eficaz com o uso de variantes de capsídeo de aavInfo
- Publication number
- BR112014025985A2 BR112014025985A2 BR112014025985A BR112014025985A BR112014025985A2 BR 112014025985 A2 BR112014025985 A2 BR 112014025985A2 BR 112014025985 A BR112014025985 A BR 112014025985A BR 112014025985 A BR112014025985 A BR 112014025985A BR 112014025985 A2 BR112014025985 A2 BR 112014025985A2
- Authority
- BR
- Brazil
- Prior art keywords
- aav
- methods
- composition
- gene transfer
- vector
- Prior art date
Links
- 210000000234 capsid Anatomy 0.000 title abstract 3
- 238000000034 method Methods 0.000 title abstract 3
- 108090000623 proteins and genes Proteins 0.000 title abstract 3
- 239000000203 mixture Substances 0.000 title abstract 2
- KDXKERNSBIXSRK-UHFFFAOYSA-N Lysine Natural products NCCCCC(N)C(O)=O KDXKERNSBIXSRK-UHFFFAOYSA-N 0.000 abstract 2
- 239000004472 Lysine Substances 0.000 abstract 2
- 108091028043 Nucleic acid sequence Proteins 0.000 abstract 2
- 125000003588 lysine group Chemical group [H]N([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])(N([H])[H])C(*)=O 0.000 abstract 2
- 150000007523 nucleic acids Chemical group 0.000 abstract 2
- 238000006467 substitution reaction Methods 0.000 abstract 2
- 108090000565 Capsid Proteins Proteins 0.000 abstract 1
- 102100023321 Ceruloplasmin Human genes 0.000 abstract 1
- 241000702421 Dependoparvovirus Species 0.000 abstract 1
- 238000001415 gene therapy Methods 0.000 abstract 1
- 230000001404 mediated effect Effects 0.000 abstract 1
- 239000008194 pharmaceutical composition Substances 0.000 abstract 1
- 102000004169 proteins and genes Human genes 0.000 abstract 1
- 230000001105 regulatory effect Effects 0.000 abstract 1
- 230000026683 transduction Effects 0.000 abstract 1
- 238000010361 transduction Methods 0.000 abstract 1
- 230000034512 ubiquitination Effects 0.000 abstract 1
- 238000010798 ubiquitination Methods 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
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
- A61P31/12—Antivirals
- A61P31/14—Antivirals for RNA viruses
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P7/00—Drugs for disorders of the blood or the extracellular fluid
- A61P7/04—Antihaemorrhagics; Procoagulants; Haemostatic agents; Antifibrinolytic agents
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
- C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
- C07H21/04—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with deoxyribosyl as saccharide radical
-
- 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
- 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
- C07K14/01—DNA viruses
- C07K14/015—Parvoviridae, e.g. feline panleukopenia virus, human parvovirus
-
- 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
- C12N15/864—Parvoviral vectors, e.g. parvovirus, densovirus
-
- 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
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Genetics & Genomics (AREA)
- Organic Chemistry (AREA)
- Engineering & Computer Science (AREA)
- General Health & Medical Sciences (AREA)
- Molecular Biology (AREA)
- Biotechnology (AREA)
- Virology (AREA)
- Biochemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Medicinal Chemistry (AREA)
- General Engineering & Computer Science (AREA)
- Biomedical Technology (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Biophysics (AREA)
- Pharmacology & Pharmacy (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Microbiology (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Gastroenterology & Hepatology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Epidemiology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Hematology (AREA)
- Diabetes (AREA)
- Oncology (AREA)
- Communicable Diseases (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)
- Peptides Or Proteins (AREA)
- Preparation Of Compounds By Using Micro-Organisms (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201261635273P | 2012-04-18 | 2012-04-18 | |
| US201361794995P | 2013-03-15 | 2013-03-15 | |
| PCT/US2013/037170 WO2013158879A1 (en) | 2012-04-18 | 2013-04-18 | Composition and methods for highly efficient gene transfer using aav capsid variants |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| BR112014025985A2 true BR112014025985A2 (pt) | 2017-07-11 |
Family
ID=49384070
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| BR122021020419-5A BR122021020419B1 (pt) | 2012-04-18 | 2013-04-18 | Vetor de vírus adeno-associado (aav) que compreende uma proteína de capsídeo vp1, composições farmacêuticas compreendendo o mesmo, e seus usos |
| BR112014025985A BR112014025985A2 (pt) | 2012-04-18 | 2013-04-18 | composição e métodos para transferência de gene altamente eficaz com o uso de variantes de capsídeo de aav |
Family Applications Before (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| BR122021020419-5A BR122021020419B1 (pt) | 2012-04-18 | 2013-04-18 | Vetor de vírus adeno-associado (aav) que compreende uma proteína de capsídeo vp1, composições farmacêuticas compreendendo o mesmo, e seus usos |
Country Status (26)
| Country | Link |
|---|---|
| US (2) | US9909142B2 (instruction) |
| EP (1) | EP2839014B1 (instruction) |
| JP (1) | JP6342886B2 (instruction) |
| KR (1) | KR102063483B1 (instruction) |
| CN (1) | CN104487579B (instruction) |
| AU (1) | AU2013249202B2 (instruction) |
| BR (2) | BR122021020419B1 (instruction) |
| CA (1) | CA2870736C (instruction) |
| CO (1) | CO7200251A2 (instruction) |
| DK (1) | DK2839014T3 (instruction) |
| ES (1) | ES2862912T3 (instruction) |
| HU (1) | HUE054087T2 (instruction) |
| IL (1) | IL235102B (instruction) |
| IN (1) | IN2014DN08812A (instruction) |
| MX (1) | MX359518B (instruction) |
| MY (1) | MY172457A (instruction) |
| NZ (1) | NZ701693A (instruction) |
| PE (1) | PE20150163A1 (instruction) |
| PH (1) | PH12014502347A1 (instruction) |
| PL (1) | PL2839014T3 (instruction) |
| PT (1) | PT2839014T (instruction) |
| RU (1) | RU2683497C2 (instruction) |
| SG (1) | SG11201406776TA (instruction) |
| SI (1) | SI2839014T1 (instruction) |
| WO (1) | WO2013158879A1 (instruction) |
| ZA (1) | ZA201407582B (instruction) |
Families Citing this family (128)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| ES2428218T3 (es) * | 2005-04-07 | 2013-11-06 | The Trustees Of The University Of Pennsylvania | Cápsides de AAV rh48 modificadas, composiciones que las contienen y usos de las mismas |
| HUE030719T2 (en) | 2007-04-09 | 2017-05-29 | Univ Florida | RAAV vector formulations and methods comprising tyrosine-modified capsid proteins for their use |
| US9725485B2 (en) | 2012-05-15 | 2017-08-08 | University Of Florida Research Foundation, Inc. | AAV vectors with high transduction efficiency and uses thereof for gene therapy |
| US8734809B2 (en) | 2009-05-28 | 2014-05-27 | University Of Massachusetts | AAV's and uses thereof |
| CA3182519A1 (en) | 2011-04-29 | 2012-11-01 | Selecta Biosciences, Inc. | Tolerogenic synthetic nanocarriers for antigen-specific deletion of t effector cells |
| ES2862912T3 (es) | 2012-04-18 | 2021-10-08 | Childrens Hospital Philadelphia | Composición y procedimientos para la transferencia genética de alta eficiencia utilizando variantes de cápside de VAA |
| US10294281B2 (en) | 2012-05-15 | 2019-05-21 | University Of Florida Research Foundation, Incorporated | High-transduction-efficiency rAAV vectors, compositions, and methods of use |
| US12358954B2 (en) | 2012-05-15 | 2025-07-15 | University Of Florida Research Foundation, Incorporated | Capsid-modified rAAV vector compositions and methods therefor |
| ES2897508T3 (es) * | 2013-05-31 | 2022-03-01 | Univ California | Variantes de virus adenoasociados y métodos de uso de las mismas |
| PE20160188A1 (es) * | 2013-07-22 | 2016-04-27 | Philadelphia Children Hospital | Variantes aav y composiciones, metodos y usos para transferencia de gen a celulas, organos y tejidos |
| US20160369298A1 (en) | 2013-09-26 | 2016-12-22 | University Of Florida Research Foundation, Inc. | Synthetic combinatorial aav capsid library for targeted gene therapy |
| GB201403684D0 (en) * | 2014-03-03 | 2014-04-16 | King S College London | Vector |
| WO2015134643A1 (en) * | 2014-03-04 | 2015-09-11 | University Of Florida Research Foundation, Inc. | Improved raav vectors and methods for transduction of photoreceptors and rpe cells |
| US10577627B2 (en) | 2014-06-09 | 2020-03-03 | Voyager Therapeutics, Inc. | Chimeric capsids |
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