MX2020002148A - Vectores adenoasociados recombinantes. - Google Patents
Vectores adenoasociados recombinantes.Info
- Publication number
- MX2020002148A MX2020002148A MX2020002148A MX2020002148A MX2020002148A MX 2020002148 A MX2020002148 A MX 2020002148A MX 2020002148 A MX2020002148 A MX 2020002148A MX 2020002148 A MX2020002148 A MX 2020002148A MX 2020002148 A MX2020002148 A MX 2020002148A
- Authority
- MX
- Mexico
- Prior art keywords
- vectors
- recombinant adeno
- aav
- associated vectors
- adeno
- Prior art date
Links
- 239000013598 vector Substances 0.000 title abstract 2
- 239000013607 AAV vector Substances 0.000 abstract 2
- 210000003169 central nervous system Anatomy 0.000 abstract 2
- 210000001519 tissue Anatomy 0.000 abstract 2
- 241000702421 Dependoparvovirus Species 0.000 abstract 1
- 210000000577 adipose tissue Anatomy 0.000 abstract 1
- 239000003085 diluting agent Substances 0.000 abstract 1
- 239000008194 pharmaceutical composition Substances 0.000 abstract 1
- 239000000546 pharmaceutical excipient Substances 0.000 abstract 1
- 108090000623 proteins and genes Proteins 0.000 abstract 1
- 230000010415 tropism Effects 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
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
-
- 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
- A61K48/0008—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
- A61K48/0025—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid
-
- 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
-
- 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
- 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
-
- 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
- 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)
- Engineering & Computer Science (AREA)
- Organic Chemistry (AREA)
- Biotechnology (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- General Health & Medical Sciences (AREA)
- Biomedical Technology (AREA)
- Molecular Biology (AREA)
- General Engineering & Computer Science (AREA)
- Biochemistry (AREA)
- Medicinal Chemistry (AREA)
- Virology (AREA)
- Microbiology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Public Health (AREA)
- Pharmacology & Pharmacy (AREA)
- Epidemiology (AREA)
- Animal Behavior & Ethology (AREA)
- Veterinary Medicine (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Gastroenterology & Hepatology (AREA)
- Immunology (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Medicinal Preparation (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Peptides Or Proteins (AREA)
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201762550458P | 2017-08-25 | 2017-08-25 | |
| PCT/US2018/047466 WO2019040586A1 (en) | 2017-08-25 | 2018-08-22 | RECOMBINANT ADENO-ASSOCIATE VECTORS |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| MX2020002148A true MX2020002148A (es) | 2020-07-20 |
Family
ID=65439655
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| MX2020002148A MX2020002148A (es) | 2017-08-25 | 2018-08-22 | Vectores adenoasociados recombinantes. |
Country Status (10)
| Country | Link |
|---|---|
| US (2) | US20190085358A1 (enExample) |
| EP (1) | EP3655041A4 (enExample) |
| JP (1) | JP2020533968A (enExample) |
| KR (1) | KR20200042935A (enExample) |
| CN (1) | CN111163811A (enExample) |
| AU (1) | AU2018320849A1 (enExample) |
| CA (1) | CA3073937A1 (enExample) |
| IL (1) | IL272655A (enExample) |
| MX (1) | MX2020002148A (enExample) |
| WO (1) | WO2019040586A1 (enExample) |
Families Citing this family (5)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| KR20210110585A (ko) | 2018-11-21 | 2021-09-08 | 썰테고 테라퓨틱스 아이엔씨. | 자살의 위험을 감소시키고 우울증의 신속한 완화를 위한 가복사돌 |
| US20230118087A1 (en) * | 2019-09-03 | 2023-04-20 | Krystal Biotech, Inc. | Compositions and methods for the treatment of congenital ichthyoses |
| IL298334A (en) | 2020-05-20 | 2023-01-01 | Certego Therapeutics Inc | Ring deuterated gaboxadol and its use for the treatment of psychiatric disorders |
| AU2021335601A1 (en) * | 2020-09-04 | 2023-04-06 | Ohio State Innovation Foundation | Novel engineered capsid serotype of recombinant adeno-associated viral vector with enhanced transduction efficiency and widespread distribution in the brain |
| WO2023044306A1 (en) * | 2021-09-14 | 2023-03-23 | California Institute Of Technology | Aav capsid variants |
Family Cites Families (31)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| DE69939169D1 (de) * | 1998-05-28 | 2008-09-04 | Us Gov Health & Human Serv | Aav5 vektoren und deren verwendung |
| JP4969002B2 (ja) * | 1999-06-08 | 2012-07-04 | ユニバーシテイ・オブ・アイオワ・リサーチ・フアウンデーシヨン | ｒＡＡＶ形質導入を増加するための化合物および方法 |
| EP1546369A4 (en) * | 2002-08-12 | 2007-01-17 | Univ Michigan | DIAGNOSIS AND TREATMENT OF DISEASES THROUGH DEFECTS IN THE TUBEROUS SKLEROSEPFAD |
| US7427396B2 (en) * | 2004-06-03 | 2008-09-23 | Genzyme Corporation | AAV vectors for gene delivery to the lung |
| US7838657B2 (en) * | 2004-12-03 | 2010-11-23 | University Of Massachusetts | Spinal muscular atrophy (SMA) treatment via targeting of SMN2 splice site inhibitory sequences |
| DE23193606T1 (de) * | 2005-04-07 | 2024-02-15 | The Trustees Of The University Of Pennsylvania | Verfahren zur erhöhung der funktion eines aav-vektors |
| US9415121B2 (en) * | 2008-12-19 | 2016-08-16 | Nationwide Children's Hospital | Delivery of MECP2 polynucleotide using recombinant AAV9 |
| EP2692868A1 (en) * | 2012-08-02 | 2014-02-05 | Universitat Autònoma De Barcelona | Adeno-associated viral (AAV) vectors useful for transducing adipose tissue |
| BR112015027336A2 (pt) * | 2013-05-01 | 2017-09-26 | Genzyme Corp | composições e processos para tratamento de atrofia muscular espinhal |
| NZ716102A (en) * | 2013-07-22 | 2021-12-24 | The Children’S Hospital Of Philadelphia | Variant aav and compositions, methods and uses for gene transfer to cells, organs and tissues |
| GB201403684D0 (en) * | 2014-03-03 | 2014-04-16 | King S College London | Vector |
| WO2015171547A1 (en) * | 2014-05-05 | 2015-11-12 | Ovid Therapeutics Inc. | Methods of treating cognitive impairment associated with neurodegenerative disorders |
| WO2015184262A1 (en) * | 2014-05-30 | 2015-12-03 | The Board Of Trustees Of The Leland Stanford Junior University | Compositions and methods of delivering treatments for latent viral infections |
| US10577627B2 (en) * | 2014-06-09 | 2020-03-03 | Voyager Therapeutics, Inc. | Chimeric capsids |
| WO2016054557A1 (en) * | 2014-10-03 | 2016-04-07 | University Of Massachusetts | Novel high efficiency library-identified aav vectors |
| AU2016366549B2 (en) * | 2015-12-11 | 2022-11-10 | California Institute Of Technology | Targeting peptides for directing adeno-associated viruses (AAVs) |
| AU2016370630B2 (en) * | 2015-12-14 | 2023-04-13 | The Trustees Of The University Of Pennsylvania | Adeno-associated viral vectors useful in treatment of spinal muscular atropy |
| WO2017197355A2 (en) * | 2016-05-13 | 2017-11-16 | 4D Molecular Therapeutics Inc. | Adeno-associated virus variant capsids and methods of use thereof |
| EP3583220A4 (en) * | 2017-02-15 | 2021-07-07 | The University of North Carolina at Chapel Hill | PROCEDURES AND COMPOSITIONS FOR GENERAL TRANSFER IN THE VASCULAR SYSTEM |
| CN110621318A (zh) * | 2017-03-15 | 2019-12-27 | 奥维德医疗公司 | 排他地由设计者药物激活的设计者受体在治疗癫痫发作紊乱中的用途 |
| CN111867595A (zh) * | 2017-12-20 | 2020-10-30 | 奥维德医疗公司 | hM4Di在治疗癫痫发作紊乱中的用途 |
| KR20210040358A (ko) * | 2018-06-14 | 2021-04-13 | 오비드 테라퓨틱스 인크. | 안젤만 증후군의 치료에서 mir-92a 또는 mir-145의 사용 |
| US11149256B2 (en) * | 2018-09-26 | 2021-10-19 | California Institute Of Technology | Adeno-associated virus compositions for targeted gene therapy |
| US10557149B1 (en) * | 2019-07-15 | 2020-02-11 | Vigene Biosciences, Inc. | Recombinantly-modified adeno-associated virus helper vectors and their use to improve the packaging efficiency of recombinantly-modified adeno-associated virus |
| WO2021102107A1 (en) * | 2019-11-19 | 2021-05-27 | Asklepios Biopharmaceutical, Inc. | Therapeutic adeno-associated virus comprising liver-specific promoters for treating pompe disease and lysosomal disorders |
| US11891616B2 (en) * | 2019-12-05 | 2024-02-06 | Board Of Regents Of The University Of Texas System | Transgene cassettes designed to express a human MECP2 gene |
| CA3116391A1 (en) * | 2020-09-14 | 2022-03-14 | President And Fellows Of Harvard College | Recombinant adeno associated virus encoding clarin-1 and uses thereof |
| KR20230152008A (ko) * | 2021-02-03 | 2023-11-02 | 더 유니버시티 오브 노쓰 캐롤라이나 엣 채플 힐 | 단백질 m 유사체 및 융합 단백질 및 이들의 항체 기능 억제 용도 |
| EP4334332A4 (en) * | 2021-05-04 | 2025-11-12 | California Inst Of Techn | VAA RECOMMENDANTS FOR ADMINISTRATION TO THE CENTRAL NERVOUS SYSTEM AND CEREBRAL VASCULATURE |
| CN114259502A (zh) * | 2021-12-24 | 2022-04-01 | 南京鼓楼医院 | 一种基于脂肪组织的中枢靶向递送siRNA的方法及应用 |
| CN116350801A (zh) * | 2022-11-22 | 2023-06-30 | 四川至善唯新生物科技有限公司 | 一种重组腺相关病毒载体的药物组合物及其用途 |
-
2018
- 2018-08-22 JP JP2020511199A patent/JP2020533968A/ja active Pending
- 2018-08-22 KR KR1020207008689A patent/KR20200042935A/ko not_active Withdrawn
- 2018-08-22 CA CA3073937A patent/CA3073937A1/en active Pending
- 2018-08-22 EP EP18849356.3A patent/EP3655041A4/en not_active Withdrawn
- 2018-08-22 US US16/108,393 patent/US20190085358A1/en not_active Abandoned
- 2018-08-22 WO PCT/US2018/047466 patent/WO2019040586A1/en not_active Ceased
- 2018-08-22 MX MX2020002148A patent/MX2020002148A/es unknown
- 2018-08-22 AU AU2018320849A patent/AU2018320849A1/en not_active Abandoned
- 2018-08-22 CN CN201880064381.6A patent/CN111163811A/zh active Pending
-
2020
- 2020-02-13 IL IL272655A patent/IL272655A/en unknown
-
2021
- 2021-08-12 US US17/400,768 patent/US20210371880A1/en not_active Abandoned
Also Published As
| Publication number | Publication date |
|---|---|
| US20210371880A1 (en) | 2021-12-02 |
| EP3655041A1 (en) | 2020-05-27 |
| JP2020533968A (ja) | 2020-11-26 |
| US20190085358A1 (en) | 2019-03-21 |
| EP3655041A4 (en) | 2021-04-21 |
| AU2018320849A1 (en) | 2020-03-05 |
| WO2019040586A1 (en) | 2019-02-28 |
| CA3073937A1 (en) | 2019-02-28 |
| CN111163811A (zh) | 2020-05-15 |
| KR20200042935A (ko) | 2020-04-24 |
| IL272655A (en) | 2020-03-31 |
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