RS65241B1 - Vektor adeno-asociranih virusa (aav) iz podgrupe f i njegove upotrebe - Google Patents
Vektor adeno-asociranih virusa (aav) iz podgrupe f i njegove upotrebeInfo
- Publication number
- RS65241B1 RS65241B1 RS20240217A RSP20240217A RS65241B1 RS 65241 B1 RS65241 B1 RS 65241B1 RS 20240217 A RS20240217 A RS 20240217A RS P20240217 A RSP20240217 A RS P20240217A RS 65241 B1 RS65241 B1 RS 65241B1
- Authority
- RS
- Serbia
- Prior art keywords
- hsa
- mir
- aavhu68
- seq
- proteins
- Prior art date
Links
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 That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
Applications Claiming Priority (5)
| 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 | |
| EP18711202.4A EP3589730B1 (en) | 2017-02-28 | 2018-02-27 | Adeno-associated virus (aav) clade f vector and uses therefor |
| PCT/US2018/019992 WO2018160582A1 (en) | 2017-02-28 | 2018-02-27 | Adeno-associated virus (aav) clade f vector and uses therefor |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| RS65241B1 true RS65241B1 (sr) | 2024-03-29 |
Family
ID=61628475
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| RS20240217A RS65241B1 (sr) | 2017-02-28 | 2018-02-27 | Vektor adeno-asociranih virusa (aav) iz podgrupe f i njegove upotrebe |
Country Status (29)
| Country | Link |
|---|---|
| US (2) | US11827906B2 (enExample) |
| EP (2) | EP3589730B1 (enExample) |
| JP (3) | JP7455579B2 (enExample) |
| KR (1) | KR102775350B1 (enExample) |
| CN (2) | CN110741082B (enExample) |
| AU (2) | AU2018227440B2 (enExample) |
| BR (1) | BR112019017839A2 (enExample) |
| CA (1) | CA3053399A1 (enExample) |
| CL (1) | CL2019002474A1 (enExample) |
| CO (1) | CO2019009525A2 (enExample) |
| DK (1) | DK3589730T5 (enExample) |
| ES (1) | ES2971872T3 (enExample) |
| FI (1) | FI3589730T3 (enExample) |
| HR (1) | HRP20240257T1 (enExample) |
| HU (1) | HUE065885T2 (enExample) |
| IL (1) | IL268644B2 (enExample) |
| LT (1) | LT3589730T (enExample) |
| MD (1) | MD3589730T2 (enExample) |
| MX (2) | MX2019010275A (enExample) |
| PE (1) | PE20191362A1 (enExample) |
| PL (1) | PL3589730T3 (enExample) |
| PT (1) | PT3589730T (enExample) |
| RS (1) | RS65241B1 (enExample) |
| SA (1) | SA519402565B1 (enExample) |
| SG (4) | SG10201912973XA (enExample) |
| SI (1) | SI3589730T1 (enExample) |
| SM (1) | SMT202400080T1 (enExample) |
| WO (1) | WO2018160582A1 (enExample) |
| ZA (1) | ZA201905407B (enExample) |
Families Citing this family (78)
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|---|---|---|---|---|
| CA2915124C (en) | 2001-11-13 | 2018-08-14 | The Trustees Of The University Of Pennsylvania | A method of detecting and/or identifying adeno-associated virus (aav) sequences and isolating novel sequences identified thereby |
| EP2298926A1 (en) | 2003-09-30 | 2011-03-23 | The Trustees of The University of Pennsylvania | Adeno-associated virus (AAV) clades, sequences, vectors containing same, and uses thereof |
| US10494645B2 (en) | 2013-04-18 | 2019-12-03 | Fondazione Telethon | 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 |
| CA3011529A1 (en) * | 2016-01-15 | 2017-07-20 | American Gene Technologies International Inc. | Methods and compositions for the activation of gamma-delta t-cells |
| JOP20190200A1 (ar) | 2017-02-28 | 2019-08-27 | Univ Pennsylvania | تركيبات نافعة في معالجة ضمور العضل النخاعي |
| US11827906B2 (en) | 2017-02-28 | 2023-11-28 | The Trustees Of The University Of Pennsylvania | Adeno-associated virus (AAV) clade f vector and uses therefor |
| RU2019139555A (ru) | 2017-05-11 | 2021-06-11 | Зе Трастис Оф Зе Юниверсити Оф Пеннсильвания | Генная терапия нейрональных цероидных липофусцинозов |
| CA3083761A1 (en) | 2017-11-30 | 2019-06-06 | The Trustees Of The University Of Pennsylvania | Gene therapy for mucopolysaccharidosis iiib |
| JP7389744B2 (ja) | 2017-11-30 | 2023-11-30 | ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア | ムコ多糖症ｉｉｉａ型のための遺伝子療法 |
| EP3758724A4 (en) * | 2018-02-27 | 2022-07-06 | The Trustees of The University of Pennsylvania | NOVEL VECTORS OF ADENO-ASSOCIATED VIRUS (AAV), AAV VECTORS WITH REDUCED CAPSIID DEAMIDATION AND USES THEREOF |
| 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. |
| EP3773743A1 (en) | 2018-04-03 | 2021-02-17 | Stridebio, Inc. | Virus vectors for targeting ophthalmic tissues |
| CN112543766A (zh) | 2018-04-03 | 2021-03-23 | 斯特里迪比奥公司 | 抗体逃避性病毒载体 |
| WO2019195449A1 (en) | 2018-04-03 | 2019-10-10 | Stridebio, Inc. | Antibody-evading virus vectors |
| KR20210071017A (ko) * | 2018-10-01 | 2021-06-15 | 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 | Gm1 강글리오시드증 치료에 유용한 조성물 |
| UY38407A (es) | 2018-10-15 | 2020-05-29 | Novartis Ag | Anticuerpos estabilizadores de trem2 |
| SG11202108614QA (en) | 2019-02-15 | 2021-09-29 | Sangamo Therapeutics Inc | Compositions and methods for producing recombinant aav |
| EP3927381A1 (en) | 2019-02-22 | 2021-12-29 | The Trustees of The University of Pennsylvania | Recombinant adeno-associated virus for treatment of grn-associated adult-onset neurodegeneration |
| MX2021010266A (es) * | 2019-02-26 | 2021-09-23 | Univ Pennsylvania | Composiciones utiles en el tratamiento de la enfermedad de krabbe. |
| CN113727992A (zh) | 2019-03-21 | 2021-11-30 | 斯特里迪比奥公司 | 重组腺相关病毒载体 |
| US20220202960A1 (en) * | 2019-04-24 | 2022-06-30 | The Trustees Of The University Of Pennsylvania | Compositions useful in treatment of rett syndrome |
| PH12021552608A1 (en) * | 2019-04-29 | 2022-06-20 | Univ Pennsylvania | Novel aav capsids and compositions containing same |
| WO2020227166A1 (en) * | 2019-05-03 | 2020-11-12 | The Trustees Of The University Of Pennsylvania | Compositions useful in treatment of metachromatic leukodystrophy |
| TW202128736A (zh) | 2019-10-17 | 2021-08-01 | 美商史崔德生物公司 | 用於治療ｃ型尼曼—匹克病之腺相關病毒載體 |
| EP4072572A4 (en) * | 2019-12-10 | 2024-01-03 | Homology Medicines, Inc. | ADENO-ASSOCIATED VIRUS COMPOSITIONS AND METHODS OF USE THEREOF |
| WO2021155337A1 (en) | 2020-02-02 | 2021-08-05 | The Trustees Of The University Of Pennsylvania | Compositions useful for treating gm1 gangliosidosis |
| CN115287350B (zh) * | 2020-03-30 | 2025-07-04 | 中国医学科学院肿瘤医院 | 包括外泌体miR-106b-3p、miR-10a-3p等在肺癌诊断中的应用 |
| IL297635A (en) * | 2020-04-27 | 2022-12-01 | Univ Pennsylvania | Compositions useful in treatment of cdkl5 deficiency disorder (cdd) |
| AU2021270447A1 (en) | 2020-05-12 | 2023-01-05 | The Trustees Of The University Of Pennsylvania | Compositions for DRG-specific reduction of transgene expression |
| TW202208622A (zh) * | 2020-05-12 | 2022-03-01 | 賓州大學委員會 | 用於治療克拉培氏病之組成物 |
| WO2021242909A1 (en) | 2020-05-26 | 2021-12-02 | Shape Therapeutics Inc. | High throughput engineering of functional aav capsids |
| TW202216244A (zh) | 2020-07-13 | 2022-05-01 | 賓州大學委員會 | 有用於治療夏馬杜三氏病之組成物 |
| JP2023536148A (ja) | 2020-07-30 | 2023-08-23 | シェイプ セラピューティクス インク． | Ｒａａｖウイルス粒子の誘導産生のための安定な細胞株 |
| TW202227632A (zh) | 2020-08-19 | 2022-07-16 | 美商史崔德生物公司 | 用於治療雷特症候群之腺相關病毒載體 |
| MX2023002293A (es) | 2020-08-24 | 2023-05-19 | Univ Pennsylvania | Vectores virales que codifican fusiones de agonistas del receptor de glp-1 y usos de estos en el tratamiento de enfermedades metabólica. |
| MX2023002364A (es) | 2020-08-26 | 2023-05-22 | Univ Pennsylvania | Virus adenoasociado recombinante para el tratamiento de la neurodegeneración iniciada en adultos asociada a grn. |
| KR20230118075A (ko) | 2020-10-09 | 2023-08-10 | 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 | 파브리병 치료를 위한 조성물 및 방법 |
| TW202229315A (zh) | 2020-10-18 | 2022-08-01 | 賓州大學委員會 | 改良的腺相關病毒(aav)載體及其用途 |
| CA3199234A1 (en) * | 2020-10-22 | 2022-04-28 | Scout Bio, Inc. | Method of suppressing immune response to vector-delivered therapeutic protein |
| WO2022094078A1 (en) | 2020-10-28 | 2022-05-05 | The Trustees Of The University Of Pennsylvania | Compositions useful in treatment of rett syndrome |
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| EP4256065A2 (en) | 2020-12-01 | 2023-10-11 | The Trustees of The University of Pennsylvania | Novel compositions with tissue-specific targeting motifs and compositions containing same |
| CA3200192A1 (en) | 2020-12-01 | 2022-06-09 | Justin PERCIVAL | Compositions and uses thereof for treatment of angelman syndrome |
| EP4284335A1 (en) | 2021-02-01 | 2023-12-06 | RegenxBio Inc. | Gene therapy for neuronal ceroid lipofuscinoses |
| US20240191258A1 (en) | 2021-04-12 | 2024-06-13 | The Trustees Of The University Of Pennsylvania | Compositions useful for treating spinal and bulbar muscular atrophy (sbma) |
| EP4334334A1 (en) | 2021-04-23 | 2024-03-13 | The Trustees of The University of Pennsylvania | Novel compositions with brain-specific targeting motifs and compositions containing same |
| AU2022274162A1 (en) | 2021-05-12 | 2023-11-30 | Fondazione Telethon | Vector system |
| IL308787A (en) * | 2021-06-08 | 2024-01-01 | Univ Pennsylvania | Recombinant adeno-associated viruses for Leish-Nihan disorders and their uses |
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| US20250295807A1 (en) | 2021-11-15 | 2025-09-25 | The Trustees Of The University Of Pennsylvania | Compositions for drg-specific reduction of transgene expression |
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| WO2023102517A1 (en) | 2021-12-02 | 2023-06-08 | The Trustees Of The University Of Pennsylvania | Compositions and methods for treatment of fabry disease |
| WO2023133584A1 (en) | 2022-01-10 | 2023-07-13 | The Trustees Of The University Of Pennsylvania | Compositions useful in treatment of metachromatic leukodystrophy |
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| WO2025102034A1 (en) | 2023-11-10 | 2025-05-15 | The Trustees Of The University Of Pennsylvania | Gene therapy for barth syndrome |
| WO2025106661A1 (en) | 2023-11-14 | 2025-05-22 | The Trustees Of The University Of Pennsylvania | Compositions with cardiac and skeletal musclespecific targeting motifs and uses thereof |
| WO2025129157A1 (en) | 2023-12-15 | 2025-06-19 | The Trustees Of The University Of Pennsylvania | Gene therapy for treatment of canavan disease |
| WO2025188625A1 (en) | 2024-03-03 | 2025-09-12 | Passage Bio, Inc. | Recombinant adeno-associated virus for treatment of neurodegenerative disorders |
| WO2025207948A1 (en) | 2024-03-27 | 2025-10-02 | Biogen Ma Inc. | Aav capsids for targeting human transferrin receptor |
| WO2025207932A1 (en) | 2024-03-27 | 2025-10-02 | Biogen Ma Inc. | Aav capsids comprising anti-transferrin antibody domains |
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2023
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