BR112021021200A2 - Células expressando um receptor quimérico de um locus cd247 modificado, polinucleotídeos relacionados e métodos - Google Patents
Células expressando um receptor quimérico de um locus cd247 modificado, polinucleotídeos relacionados e métodosInfo
- Publication number
- BR112021021200A2 BR112021021200A2 BR112021021200A BR112021021200A BR112021021200A2 BR 112021021200 A2 BR112021021200 A2 BR 112021021200A2 BR 112021021200 A BR112021021200 A BR 112021021200A BR 112021021200 A BR112021021200 A BR 112021021200A BR 112021021200 A2 BR112021021200 A2 BR 112021021200A2
- Authority
- BR
- Brazil
- Prior art keywords
- modified
- cells
- chimeric receptor
- locus
- methods
- Prior art date
Links
- 108700010039 chimeric receptor Proteins 0.000 title abstract 5
- 238000000034 method Methods 0.000 title abstract 3
- 102000040430 polynucleotide Human genes 0.000 title abstract 2
- 108091033319 polynucleotide Proteins 0.000 title abstract 2
- 239000002157 polynucleotide Substances 0.000 title abstract 2
- 210000004027 cell Anatomy 0.000 abstract 5
- 210000002865 immune cell Anatomy 0.000 abstract 3
- 210000001744 T-lymphocyte Anatomy 0.000 abstract 2
- 108700019146 Transgenes Proteins 0.000 abstract 1
- 238000002619 cancer immunotherapy Methods 0.000 abstract 1
- 238000002659 cell therapy Methods 0.000 abstract 1
- 230000001413 cellular effect Effects 0.000 abstract 1
- 230000010354 integration Effects 0.000 abstract 1
- 230000003834 intracellular effect Effects 0.000 abstract 1
- 238000004519 manufacturing process Methods 0.000 abstract 1
- 239000000203 mixture Substances 0.000 abstract 1
- 102000039446 nucleic acids Human genes 0.000 abstract 1
- 108020004707 nucleic acids Proteins 0.000 abstract 1
- 150000007523 nucleic acids Chemical class 0.000 abstract 1
- 230000011664 signaling Effects 0.000 abstract 1
- 230000008685 targeting Effects 0.000 abstract 1
Classifications
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
- C07K14/70503—Immunoglobulin superfamily
- C07K14/7051—T-cell receptor (TcR)-CD3 complex
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- 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
- A61K35/12—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
- A61K35/14—Blood; Artificial blood
- A61K35/17—Lymphocytes; B-cells; T-cells; Natural killer cells; Interferon-activated or cytokine-activated lymphocytes
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- C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
- C12N5/06—Animal cells or tissues; Human cells or tissues
- C12N5/0602—Vertebrate cells
- C12N5/0634—Cells from the blood or the immune system
- C12N5/0636—T lymphocytes
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K39/00—Medicinal preparations containing antigens or antibodies
- A61K39/46—Cellular immunotherapy
- A61K39/461—Cellular immunotherapy characterised by the cell type used
- A61K39/4611—T-cells, e.g. tumor infiltrating lymphocytes [TIL], lymphokine-activated killer cells [LAK] or regulatory T cells [Treg]
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K39/00—Medicinal preparations containing antigens or antibodies
- A61K39/46—Cellular immunotherapy
- A61K39/463—Cellular immunotherapy characterised by recombinant expression
- A61K39/4631—Chimeric Antigen Receptors [CAR]
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K39/00—Medicinal preparations containing antigens or antibodies
- A61K39/46—Cellular immunotherapy
- A61K39/464—Cellular immunotherapy characterised by the antigen targeted or presented
- A61K39/4643—Vertebrate antigens
- A61K39/4644—Cancer antigens
- A61K39/464402—Receptors, cell surface antigens or cell surface determinants
- A61K39/464416—Receptors for cytokines
- A61K39/464417—Receptors for tumor necrosis factors [TNF], e.g. lymphotoxin receptor [LTR], CD30
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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- A61K39/46—Cellular immunotherapy
- A61K39/464—Cellular immunotherapy characterised by the antigen targeted or presented
- A61K39/4643—Vertebrate antigens
- A61K39/4644—Cancer antigens
- A61K39/464402—Receptors, cell surface antigens or cell surface determinants
- A61K39/464429—Molecules with a "CD" designation not provided for elsewhere
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P35/00—Antineoplastic agents
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
- C07K14/70503—Immunoglobulin superfamily
- C07K14/70521—CD28, CD152
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
- C07K14/70578—NGF-receptor/TNF-receptor superfamily, e.g. CD27, CD30, CD40, CD95
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
- C07K14/715—Receptors; Cell surface antigens; Cell surface determinants for cytokines; for lymphokines; for interferons
- C07K14/7151—Receptors; Cell surface antigens; Cell surface determinants for cytokines; for lymphokines; for interferons for tumor necrosis factor [TNF], for lymphotoxin [LT]
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- 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
- C07K16/28—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants
- C07K16/2878—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants against the NGF-receptor/TNF-receptor superfamily, e.g. CD27, CD30, CD40, CD95
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- 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
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
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- 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
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/62—DNA sequences coding for fusion proteins
- C12N15/625—DNA sequences coding for fusion proteins containing a sequence coding for a signal sequence
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- 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
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- 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
- C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
- C12N15/90—Stable introduction of foreign DNA into chromosome
- C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
- C12N15/907—Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
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- 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)
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- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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- A61K2239/46—Indexing codes associated with cellular immunotherapy of group A61K39/46 characterised by the cancer treated
- A61K2239/48—Blood cells, e.g. leukemia or lymphoma
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- C07K2317/00—Immunoglobulins specific features
- C07K2317/60—Immunoglobulins specific features characterized by non-natural combinations of immunoglobulin fragments
- C07K2317/62—Immunoglobulins specific features characterized by non-natural combinations of immunoglobulin fragments comprising only variable region components
- C07K2317/622—Single chain antibody (scFv)
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- C07K2319/00—Fusion polypeptide
- C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
- C07K2319/02—Fusion polypeptide containing a localisation/targetting motif containing a signal sequence
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- C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
- C07K2319/03—Fusion polypeptide containing a localisation/targetting motif containing a transmembrane segment
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPRs]
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- C12N2501/00—Active agents used in cell culture processes, e.g. differentation
- C12N2501/50—Cell markers; Cell surface determinants
- C12N2501/51—B7 molecules, e.g. CD80, CD86, CD28 (ligand), CD152 (ligand)
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- C12N2501/515—CD3, T-cell receptor complex
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- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14141—Use of virus, viral particle or viral elements as a vector
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- C12N2800/00—Nucleic acids vectors
- C12N2800/80—Vectors containing sites for inducing double-stranded breaks, e.g. meganuclease restriction sites
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- Genetics & Genomics (AREA)
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Abstract
células expressando um receptor quimérico de um locus cd247 modificado, polinucleotídeos relacionados e métodos. são fornecidas aqui células imunes modificadas, por exemplo, células t, expressando um receptor quimérico que compreende uma região intracelular compreendendo um domínio de sinalização cd3zeta (cd3¿). em algumas modalidades, as células imunes modificadas contêm um locus cd247 modificado que codifica o receptor quimérico ou uma porção do mesmo. em algumas modalidades, pelo menos uma porção de uma cadeia cd3zeta codificada por um locus genômico cd247. são também fornecidas composições celulares contendo as células imunes modificadas, ácidos nucleicos para modificação de células e métodos, kits e artigos de fabricação para a produção de células modificadas, tal como por alvejamento de um transgene que codifica uma porção de um receptor quimérico para integração em uma região de um locus genômico cd247. em algumas modalidades, as células modificadas, por exemplo, células t, podem ser usadas em conexão com terapia celular, incluindo em conexão com a imunoterapia contra o câncer que compreende a transferência adotiva das células modificadas.
Applications Claiming Priority (2)
Application Number | Priority Date | Filing Date | Title |
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US201962841578P | 2019-05-01 | 2019-05-01 | |
PCT/US2020/030875 WO2020223571A1 (en) | 2019-05-01 | 2020-04-30 | Cells expressing a chimeric receptor from a modified cd247 locus, related polynucleotides and methods |
Publications (1)
Publication Number | Publication Date |
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BR112021021200A2 true BR112021021200A2 (pt) | 2021-12-21 |
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BR112021021200A BR112021021200A2 (pt) | 2019-05-01 | 2020-04-30 | Células expressando um receptor quimérico de um locus cd247 modificado, polinucleotídeos relacionados e métodos |
Country Status (12)
Country | Link |
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US (1) | US20220218750A1 (pt) |
EP (1) | EP3962519A1 (pt) |
JP (1) | JP2022531577A (pt) |
KR (1) | KR20220016474A (pt) |
CN (1) | CN114007640A (pt) |
AU (1) | AU2020265749A1 (pt) |
BR (1) | BR112021021200A2 (pt) |
CA (1) | CA3136742A1 (pt) |
IL (1) | IL287174A (pt) |
MX (1) | MX2021013223A (pt) |
SG (1) | SG11202111372VA (pt) |
WO (1) | WO2020223571A1 (pt) |
Families Citing this family (3)
Publication number | Priority date | Publication date | Assignee | Title |
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EP4019538A1 (en) * | 2020-12-22 | 2022-06-29 | Charité - Universitätsmedizin Berlin | Reprogramming immune cells by targeted integration of zeta-deficient chimeric antigen receptor transgenes |
WO2024081879A1 (en) * | 2022-10-14 | 2024-04-18 | Chroma Medicine, Inc. | Compositions and methods for epigenetic regulation of cd247 expression |
WO2024092026A1 (en) * | 2022-10-25 | 2024-05-02 | Kyverna Therapeutics, Inc. | Methods for treating lupus nephritis using anti-cd19 car-t cell therapies |
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IL287174A (en) | 2021-12-01 |
KR20220016474A (ko) | 2022-02-09 |
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AU2020265749A1 (en) | 2022-01-06 |
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US20220218750A1 (en) | 2022-07-14 |
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