PE20190401A1 - Novedosas proteinas de la capside del virus adenoasociado - Google Patents
Novedosas proteinas de la capside del virus adenoasociadoInfo
- Publication number
- PE20190401A1 PE20190401A1 PE2019000266A PE2019000266A PE20190401A1 PE 20190401 A1 PE20190401 A1 PE 20190401A1 PE 2019000266 A PE2019000266 A PE 2019000266A PE 2019000266 A PE2019000266 A PE 2019000266A PE 20190401 A1 PE20190401 A1 PE 20190401A1
- Authority
- PE
- Peru
- Prior art keywords
- capsid proteins
- adeno
- associated virus
- seq
- region
- Prior art date
Links
- 108090000565 Capsid Proteins Proteins 0.000 title abstract 3
- 241000702421 Dependoparvovirus Species 0.000 title abstract 2
- 102100023321 Ceruloplasmin Human genes 0.000 abstract 2
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 abstract 2
- 108090000623 proteins and genes Proteins 0.000 abstract 2
- 239000013607 AAV vector Substances 0.000 abstract 1
- 206010013801 Duchenne Muscular Dystrophy Diseases 0.000 abstract 1
- 108700019146 Transgenes Proteins 0.000 abstract 1
- 125000003275 alpha amino acid group Chemical group 0.000 abstract 1
- 201000010099 disease Diseases 0.000 abstract 1
- 208000035475 disorder Diseases 0.000 abstract 1
- 230000001105 regulatory effect 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
- C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P21/00—Drugs for disorders of the muscular or neuromuscular system
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P21/00—Drugs for disorders of the muscular or neuromuscular system
- A61P21/04—Drugs for disorders of the muscular or neuromuscular system for myasthenia gravis
-
- 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
- C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
- C07K14/4707—Muscular dystrophy
-
- 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
- A61K38/00—Medicinal preparations containing peptides
-
- 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
-
- 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
-
- 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
- C12N2750/14144—Chimeric viral vector comprising heterologous viral elements for production of another viral 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
- C12N2750/14151—Methods of production or purification of viral material
- C12N2750/14152—Methods of production or purification of viral material relating to complementing cells and packaging systems for producing virus or viral particles
-
- 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
- C12N2799/00—Uses of viruses
- C12N2799/02—Uses of viruses as vector
- C12N2799/021—Uses of viruses as vector for the expression of a heterologous nucleic acid
- C12N2799/025—Uses of viruses as vector for the expression of a heterologous nucleic acid where the vector is derived from a parvovirus
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)
- Bioinformatics & Cheminformatics (AREA)
- Molecular Biology (AREA)
- Medicinal Chemistry (AREA)
- Biotechnology (AREA)
- Zoology (AREA)
- Biophysics (AREA)
- Biochemistry (AREA)
- Virology (AREA)
- Wood Science & Technology (AREA)
- Biomedical Technology (AREA)
- General Engineering & Computer Science (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Gastroenterology & Hepatology (AREA)
- Microbiology (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Physical Education & Sports Medicine (AREA)
- Neurology (AREA)
- Epidemiology (AREA)
- Toxicology (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Peptides Or Proteins (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Saccharide Compounds (AREA)
Applications Claiming Priority (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201662366838P | 2016-07-26 | 2016-07-26 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| PE20190401A1 true PE20190401A1 (es) | 2019-03-13 |
Family
ID=59558468
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| PE2019000266A PE20190401A1 (es) | 2016-07-26 | 2017-07-25 | Novedosas proteinas de la capside del virus adenoasociado |
Country Status (15)
| Country | Link |
|---|---|
| US (3) | US11584780B2 (https=) |
| EP (1) | EP3491008A2 (https=) |
| JP (4) | JP6994018B2 (https=) |
| KR (2) | KR102604510B1 (https=) |
| CN (1) | CN109715650B (https=) |
| AU (2) | AU2017301600B2 (https=) |
| BR (1) | BR112019001532A2 (https=) |
| CA (1) | CA3031026A1 (https=) |
| CL (6) | CL2019000167A1 (https=) |
| IL (2) | IL305149A (https=) |
| MX (1) | MX2019000962A (https=) |
| PE (1) | PE20190401A1 (https=) |
| PH (1) | PH12019500068A1 (https=) |
| SG (1) | SG11201811603WA (https=) |
| WO (1) | WO2018022608A2 (https=) |
Families Citing this family (53)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| GB201508026D0 (en) | 2015-05-11 | 2015-06-24 | Ucl Business Plc | Capsid |
| BR112018003665A2 (en) | 2015-09-28 | 2018-09-25 | The University Of North Carolina At Chapel Hill | methods and compositions for antibody evasion viral vectors |
| JP2019530737A (ja) | 2016-08-23 | 2019-10-24 | アコーオス インコーポレイテッド | ヒト対象において非加齢性聴力障害を治療するための組成物および方法 |
| 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. |
| CA3094465A1 (en) | 2018-04-03 | 2019-10-10 | Stridebio, Inc. | Virus vectors for targeting ophthalmic tissues |
| US20210363191A1 (en) | 2018-04-03 | 2021-11-25 | Stridebio, Inc. | Antibody-evading virus vectors |
| 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 |
| EP3775173A1 (en) * | 2018-04-05 | 2021-02-17 | Genethon | Hybrid recombinant adeno-associated virus serotype between aav9 and aavrh74 with reduced liver tropism |
| US12054724B2 (en) | 2018-04-10 | 2024-08-06 | President And Fellows Of Harvard College | AAV vectors encoding clarin-1 or GJB2 and uses thereof |
| BR112020022722A8 (pt) | 2018-05-09 | 2022-01-18 | Biomarin Pharm Inc | Genoma e seu uso, vetor, partícula, molécula de ácido nucleico isolada, ácido nucleico, composição, tratamento, método |
| IL278598B2 (en) | 2018-05-11 | 2025-11-01 | Massachusetts Eye & Ear Infirmary | Altering tissue tropism of adeno-associated viruses |
| TW202005978A (zh) * | 2018-05-14 | 2020-02-01 | 美商拜奧馬林製藥公司 | 新穎肝靶向腺相關病毒載體 |
| US11821009B2 (en) | 2018-05-15 | 2023-11-21 | Cornell University | Genetic modification of the AAV capsid resulting in altered tropism and enhanced vector delivery |
| EP3841109A4 (en) * | 2018-08-21 | 2022-09-21 | Massachusetts Eye and Ear Infirmary | COMPOSITIONS AND METHODS TO MODULATE THE TRANSDUCTION EFFICIENCY OF ADENO-ASSOCIATED VIRUSES |
| US12454701B2 (en) * | 2018-12-05 | 2025-10-28 | Abeona Therapeutics Inc. | Recombinant adeno-associated viral vector for gene delivery |
| US20220133909A1 (en) * | 2019-01-23 | 2022-05-05 | University Of Florida Research Foundation, Incorporated | Highly efficient transduction and lateral spread in the retina by a novel aav virus enhanced by rational design |
| BR112021018776A2 (pt) | 2019-03-21 | 2021-11-30 | Stridebio Inc | Vetores de vírus adenoassociado recombinante |
| MX2021011701A (es) * | 2019-03-28 | 2021-12-10 | Massachusetts Gen Hospital | Vectores adenoasociados fabricados por ingenieria (aav) para expresion transgenica. |
| AR118928A1 (es) | 2019-05-14 | 2021-11-10 | Biomarin Pharm Inc | Métodos de redosificación de vectores de terapia génica |
| US12570998B2 (en) | 2019-09-09 | 2026-03-10 | Massachusetts Eye & Ear Infirmary | Methods and compositions for modulating the interaction between adeno-associated virus (AAV) and the AAV receptor (AAVR) for altered bio-distribution of AAV |
| US20220403414A1 (en) * | 2019-10-16 | 2022-12-22 | Wuxi Apptec (Shanghai) Co., Ltd. | Novel aav variant |
| KR20220083714A (ko) | 2019-10-17 | 2022-06-20 | 스트라이드바이오 인코포레이티드 | 니만-피크 질환 c형의 치료를 위한 아데노-연관 바이러스 벡터 |
| CN114829391A (zh) | 2019-11-14 | 2022-07-29 | 生物马林药物股份有限公司 | 用肝特异性基因疗法载体治疗遗传性血管性水肿 |
| JP7807380B2 (ja) | 2020-02-21 | 2026-01-27 | アコーオス インコーポレイテッド | ヒト対象において非加齢関連聴力障害を処置するための組成物および方法 |
| TW202142554A (zh) * | 2020-02-25 | 2021-11-16 | 澳洲兒童醫學研究所 | 腺相關病毒蛋白殼多肽及載體 |
| WO2021183895A1 (en) | 2020-03-13 | 2021-09-16 | Biomarin Pharmaceutical Inc. | Treatment of fabry disease with aav gene therapy vectors |
| AR122409A1 (es) | 2020-04-03 | 2022-09-07 | Biomarin Pharm Inc | Tratamiento de la fenilcetonuria con aav y formulaciones terapéuticas |
| IL297391A (en) | 2020-04-20 | 2022-12-01 | Tenaya Therapeutics Inc | An adeno-associated virus with an engineered capsid |
| AU2021265103A1 (en) | 2020-04-29 | 2023-01-19 | Bristol-Myers Squibb Company | Miniaturized dystrophins having spectrin fusion domains and uses thereof |
| WO2021236908A2 (en) | 2020-05-20 | 2021-11-25 | Biomarin Pharmaceutical Inc. | Use of regulatory proteins for the production of adeno-associated virus |
| CN116209768A (zh) * | 2020-07-03 | 2023-06-02 | 吉尼松公司 | 用于通过高变区交换工程化新杂合aav衣壳的方法 |
| EP4200408A1 (en) | 2020-08-19 | 2023-06-28 | Sarepta Therapeutics, Inc. | Adeno-associated virus vectors for treatment of rett syndrome |
| PL4211231T3 (pl) * | 2020-09-10 | 2024-11-04 | Genethon | Kapsyd AAV modyfikowany peptydem |
| US12129287B2 (en) | 2020-09-14 | 2024-10-29 | President And Fellows Of Harvard College | Recombinant adeno associated virus encoding clarin-1 and uses thereof |
| IL302127A (en) | 2020-10-18 | 2023-06-01 | Univ Pennsylvania | An improved adeno-associated virus (AAV) vector and its uses |
| US20230407328A1 (en) | 2020-11-02 | 2023-12-21 | Biomarin Pharmaceutical Inc. | Process for enriching adeno-associated virus |
| WO2023034994A1 (en) | 2021-09-03 | 2023-03-09 | Biomarin Pharmaceutical Inc. | Aav capsid compositions and methods for delivery |
| WO2023034990A1 (en) | 2021-09-03 | 2023-03-09 | Biomarin Pharmaceutical Inc. | Aav capsid compositions and methods for delivery |
| EP4396203A1 (en) | 2021-09-03 | 2024-07-10 | BioMarin Pharmaceutical Inc. | Aav capsid compositions and methods for delivery |
| US20240376496A1 (en) | 2021-09-03 | 2024-11-14 | Biomarin Pharmaceutical Inc. | Aav capsid compositions and methods for delivery |
| WO2023034980A1 (en) | 2021-09-03 | 2023-03-09 | Bomarin Pharmaceutical Inc. | Aav capsid compositions and methods for delivery |
| US20240384242A1 (en) | 2021-09-03 | 2024-11-21 | Biomarin Pharmaceutical Inc. | Aav capsid compositions and methods for delivery |
| AR127217A1 (es) | 2021-10-01 | 2023-12-27 | Biomarin Pharm Inc | Tratamiento de angioedema hereditario con vectores de genoterapia de aav y formulaciones terapéuticas |
| CN114276419B (zh) * | 2021-12-30 | 2023-11-17 | 上海勉亦生物科技有限公司 | 肌肉高亲和性的新型腺相关病毒衣壳蛋白及其应用 |
| WO2023183623A1 (en) * | 2022-03-25 | 2023-09-28 | Regenxbio Inc. | Dominant-negative tumor necrosis factor alpha adeno-associated virus gene therapy |
| KR20250108100A (ko) | 2022-09-22 | 2025-07-15 | 디나코 아게 | Aav 유전자 치료법 벡터를 이용한 심근병증의 치료 |
| UY40442A (es) | 2022-09-22 | 2024-02-15 | Biomarin Pharm Inc | Tratamiento de la miocardiopatía arritmogénica con vectores de genoterapia con aav |
| CN115925819B (zh) * | 2022-12-30 | 2023-10-13 | 广州派真生物技术有限公司 | 腺相关病毒突变体及其应用 |
| WO2025051970A2 (en) | 2023-09-06 | 2025-03-13 | Dinaqor Ag | Methods of treatment of hypertrophic cardiomyopathy with aav gene therapy vectors and therapeutic formulations |
| WO2025054465A1 (en) * | 2023-09-08 | 2025-03-13 | California Institute Of Technology | Compositions for transport of therapeutic cargos using miniprotein binders targeting ca-iv |
| WO2025104321A1 (en) * | 2023-11-17 | 2025-05-22 | Genethon | Prediction of multiple aav properties by deep learning |
| WO2025214477A1 (en) | 2024-04-12 | 2025-10-16 | Skyline Therapeutics (Shanghai) Co., Ltd. | Treatment of genetic cardiomyopathies with aav gene therapy vectors |
| WO2025250683A1 (en) * | 2024-05-29 | 2025-12-04 | Tenaya Therapeutics, Inc. | Adeno-associated virus with engineered capsid |
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