BR112023001852A2 - Terapia gênica com placofilina-2 (pkp2) usando vetor de aav - Google Patents
Terapia gênica com placofilina-2 (pkp2) usando vetor de aavInfo
- Publication number
- BR112023001852A2 BR112023001852A2 BR112023001852A BR112023001852A BR112023001852A2 BR 112023001852 A2 BR112023001852 A2 BR 112023001852A2 BR 112023001852 A BR112023001852 A BR 112023001852A BR 112023001852 A BR112023001852 A BR 112023001852A BR 112023001852 A2 BR112023001852 A2 BR 112023001852A2
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- Prior art keywords
- pkp2
- gene therapy
- vector
- placophyllin
- promoter
- Prior art date
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- 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
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P9/00—Drugs for disorders of the cardiovascular system
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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
- C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
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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
- C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
- C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
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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
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
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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
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- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; CARE OF BIRDS, FISHES, INSECTS; 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
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- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; CARE OF BIRDS, FISHES, INSECTS; 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
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- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; CARE OF BIRDS, FISHES, INSECTS; 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
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- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; CARE OF BIRDS, FISHES, INSECTS; 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
- A01K2267/035—Animal model for multifactorial diseases
- A01K2267/0375—Animal model for cardiovascular diseases
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
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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
- 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
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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
- 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
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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
- 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
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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
- 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)
- Genetics & Genomics (AREA)
- Engineering & Computer Science (AREA)
- Organic Chemistry (AREA)
- General Health & Medical Sciences (AREA)
- Molecular Biology (AREA)
- Biotechnology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biomedical Technology (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- Medicinal Chemistry (AREA)
- Biophysics (AREA)
- Biochemistry (AREA)
- Public Health (AREA)
- Pharmacology & Pharmacy (AREA)
- Veterinary Medicine (AREA)
- Animal Behavior & Ethology (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Microbiology (AREA)
- Virology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Epidemiology (AREA)
- Gastroenterology & Hepatology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Cardiology (AREA)
- Toxicology (AREA)
- General Chemical & Material Sciences (AREA)
- Heart & Thoracic Surgery (AREA)
- Immunology (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
Abstract
TERAPIA GÊNICA COM PLACOFILINA-2 (PKP2) USANDO VETOR DE AAV. É fornecida neste documento uma terapia gênica para PKP2 (placofilina-2), por exemplo, usando um vetor de vírus adenoassociado (AAV). O promotor do vetor pode ser um promotor de MHCK7 ou um promotor de troponina cardíaca T (HTNNT2). O capsídeo pode ser um capsídeo de AAV9 ou AAVrh74 ou uma variante funcional deste. Outros promotores ou capsídeos podem ser usados. São fornecidos ainda métodos de tratamento, tais como por administração intravenosa, intracoronária, intracarotídea ou intracardíaca do vetor rAAV e outras composições e métodos.
Applications Claiming Priority (2)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US202063063032P | 2020-08-07 | 2020-08-07 | |
PCT/US2021/045220 WO2022032226A1 (en) | 2020-08-07 | 2021-08-09 | Plakophilin-2 (pkp2) gene therapy using aav vector |
Publications (1)
Publication Number | Publication Date |
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BR112023001852A2 true BR112023001852A2 (pt) | 2023-02-23 |
Family
ID=80118582
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
BR112023001852A BR112023001852A2 (pt) | 2020-08-07 | 2021-08-09 | Terapia gênica com placofilina-2 (pkp2) usando vetor de aav |
Country Status (11)
Country | Link |
---|---|
US (2) | US20220168447A1 (pt) |
EP (1) | EP4192487A1 (pt) |
JP (1) | JP2023538519A (pt) |
KR (1) | KR20230043869A (pt) |
CN (1) | CN116194154A (pt) |
AU (1) | AU2021320902A1 (pt) |
BR (1) | BR112023001852A2 (pt) |
CA (1) | CA3185267A1 (pt) |
IL (1) | IL300263A (pt) |
MX (1) | MX2023001615A (pt) |
WO (1) | WO2022032226A1 (pt) |
Families Citing this family (7)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
AU2021320902A1 (en) | 2020-08-07 | 2023-04-06 | Spacecraft Seven, Llc | Plakophilin-2 (PKP2) gene therapy using AAV vector |
US11781156B2 (en) * | 2020-10-09 | 2023-10-10 | Tenaya Therapeutics, Inc. | Plakophillin-2 gene therapy methods and compositions |
TW202300180A (zh) * | 2021-03-19 | 2023-01-01 | 英商Ucl商業有限責任公司 | 右心室心律失常性心肌病之基因療法組合物及治療 |
KR20240032971A (ko) * | 2021-07-08 | 2024-03-12 | 테나야 테라퓨틱스, 인코포레이티드 | 유전자 요법을 위한 최적화된 발현 카세트 |
WO2023200736A2 (en) * | 2022-04-11 | 2023-10-19 | Tenaya Therapeutics, Inc. | Plakophillin-2 gene therapy treatment methods |
TW202404992A (zh) * | 2022-04-11 | 2024-02-01 | 美商特納亞治療股份有限公司 | 用於斑菲素蛋白—2(plakophillin—2)基因療法之蛋白殼 |
WO2024064863A2 (en) * | 2022-09-22 | 2024-03-28 | Biomarin Pharmaceutical Inc. | Treatment of arrhythmogenic cardiomyopathy with aav gene therapy vectors |
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CA3109640A1 (en) | 2018-08-16 | 2020-02-20 | Spacecraft Seven, Llc | Production methods for viral vectors |
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US20220143215A1 (en) * | 2019-02-12 | 2022-05-12 | Spacecraft Seven, Llc | Gene therapy vectors for treatment of danon disease |
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CA3151036A1 (en) * | 2019-09-20 | 2021-03-25 | Thomas Voit | Gene therapy composition and treatment of right ventricular arrythmogenic cardiomyopathy |
WO2021163357A2 (en) | 2020-02-13 | 2021-08-19 | Tenaya Therapeutics, Inc. | Gene therapy vectors for treating heart disease |
JPWO2021187380A1 (pt) | 2020-03-16 | 2021-09-23 | ||
CA3180202A1 (en) | 2020-04-20 | 2021-10-28 | Tenaya Therapeutics, Inc. | Adeno-associated virus with engineered capsid |
EP4153614A2 (en) | 2020-05-20 | 2023-03-29 | Spacecraft Seven, LLC | Engineered parkin and uses thereof |
WO2022017630A1 (en) | 2020-07-23 | 2022-01-27 | Ucl Business Ltd | GENE THERAPY VECTOR FOR eEF1A2 AND USES THEREOF |
JP2023536618A (ja) | 2020-08-05 | 2023-08-28 | スペースクラフト セブン リミテッド ライアビリティ カンパニー | Csrp3(システイン及びグリシンリッチタンパク質3)遺伝子治療 |
AU2021321412A1 (en) | 2020-08-05 | 2023-04-06 | Spacecraft Seven, Llc | Adeno-associated viral vector for GLUT1 expression and uses thereof |
AU2021320902A1 (en) | 2020-08-07 | 2023-04-06 | Spacecraft Seven, Llc | Plakophilin-2 (PKP2) gene therapy using AAV vector |
KR20230084542A (ko) | 2020-10-09 | 2023-06-13 | 테나야 테라퓨틱스, 인코포레이티드 | 플라코필린 2 유전자 치료 방법 및 조성물 |
US20240033325A1 (en) | 2020-12-07 | 2024-02-01 | Spacecraft Seven, Llc | Treatment of danon disease |
TW202300180A (zh) | 2021-03-19 | 2023-01-01 | 英商Ucl商業有限責任公司 | 右心室心律失常性心肌病之基因療法組合物及治療 |
EP4323534A1 (en) | 2021-04-12 | 2024-02-21 | The Regents of the University of California | Gene therapy for arrhythmogenic right ventricular cardiomyopathy |
KR20240032971A (ko) | 2021-07-08 | 2024-03-12 | 테나야 테라퓨틱스, 인코포레이티드 | 유전자 요법을 위한 최적화된 발현 카세트 |
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2021
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- 2021-08-09 KR KR1020237003609A patent/KR20230043869A/ko unknown
- 2021-08-09 WO PCT/US2021/045220 patent/WO2022032226A1/en active Search and Examination
- 2021-08-09 JP JP2023507876A patent/JP2023538519A/ja active Pending
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- 2021-08-09 BR BR112023001852A patent/BR112023001852A2/pt unknown
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- 2021-08-09 MX MX2023001615A patent/MX2023001615A/es unknown
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US20220168447A1 (en) | 2022-06-02 |
US20220168446A1 (en) | 2022-06-02 |
US11883506B2 (en) | 2024-01-30 |
EP4192487A1 (en) | 2023-06-14 |
WO2022032226A1 (en) | 2022-02-10 |
CN116194154A (zh) | 2023-05-30 |
IL300263A (en) | 2023-03-01 |
KR20230043869A (ko) | 2023-03-31 |
AU2021320902A1 (en) | 2023-04-06 |
MX2023001615A (es) | 2023-03-08 |
JP2023538519A (ja) | 2023-09-08 |
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