AU2018234695B2 - Polyploid adeno-associated virus vectors and methods of making and using the same - Google Patents
Polyploid adeno-associated virus vectors and methods of making and using the same Download PDFInfo
- Publication number
- AU2018234695B2 AU2018234695B2 AU2018234695A AU2018234695A AU2018234695B2 AU 2018234695 B2 AU2018234695 B2 AU 2018234695B2 AU 2018234695 A AU2018234695 A AU 2018234695A AU 2018234695 A AU2018234695 A AU 2018234695A AU 2018234695 B2 AU2018234695 B2 AU 2018234695B2
- Authority
- AU
- Australia
- Prior art keywords
- aav
- capsid
- virus
- vector
- transduction
- Prior art date
- Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
- Active
Links
Classifications
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P3/00—Drugs for disorders of the metabolism
- A61P3/08—Drugs for disorders of the metabolism for glucose homeostasis
-
- 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
- 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
-
- 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
- C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
- C12N2710/00011—Details
- C12N2710/14011—Baculoviridae
- C12N2710/14111—Nucleopolyhedrovirus, e.g. autographa californica nucleopolyhedrovirus
- C12N2710/14141—Use of virus, viral particle or viral elements as a vector
- C12N2710/14144—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
- C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
- C12N2710/00011—Details
- C12N2710/16011—Herpesviridae
- C12N2710/16611—Simplexvirus, e.g. human herpesvirus 1, 2
- C12N2710/16641—Use of virus, viral particle or viral elements as a vector
- C12N2710/16644—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
- C12N2750/14122—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
- C12N2750/14123—Virus like particles [VLP]
-
- 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
- C12N2750/14142—Use of virus, viral particle or viral elements as a vector virus or viral particle as vehicle, e.g. encapsulating small organic molecule
-
- 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
- C12N2750/14151—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
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Genetics & Genomics (AREA)
- Organic Chemistry (AREA)
- General Health & Medical Sciences (AREA)
- Virology (AREA)
- Engineering & Computer Science (AREA)
- Medicinal Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Molecular Biology (AREA)
- Biophysics (AREA)
- Biochemistry (AREA)
- General Engineering & Computer Science (AREA)
- Biotechnology (AREA)
- Microbiology (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Biomedical Technology (AREA)
- Gastroenterology & Hepatology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Mycology (AREA)
- Epidemiology (AREA)
- Diabetes (AREA)
- Emergency Medicine (AREA)
- Endocrinology (AREA)
- Hematology (AREA)
- Obesity (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Peptides Or Proteins (AREA)
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| AU2024227303A AU2024227303A1 (en) | 2017-03-15 | 2024-10-14 | Polyploid adeno-associated virus vectors and methods of making and using the same |
Applications Claiming Priority (7)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201762471762P | 2017-03-15 | 2017-03-15 | |
| US62/471,762 | 2017-03-15 | ||
| US201762520901P | 2017-06-16 | 2017-06-16 | |
| US62/520,901 | 2017-06-16 | ||
| US201862630558P | 2018-02-14 | 2018-02-14 | |
| US62/630,558 | 2018-02-14 | ||
| PCT/US2018/022725 WO2018170310A1 (en) | 2017-03-15 | 2018-03-15 | Polyploid adeno-associated virus vectors and methods of making and using the same |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| AU2024227303A Division AU2024227303A1 (en) | 2017-03-15 | 2024-10-14 | Polyploid adeno-associated virus vectors and methods of making and using the same |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| AU2018234695A1 AU2018234695A1 (en) | 2019-09-12 |
| AU2018234695B2 true AU2018234695B2 (en) | 2024-10-03 |
Family
ID=63522576
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| AU2018234695A Active AU2018234695B2 (en) | 2017-03-15 | 2018-03-15 | Polyploid adeno-associated virus vectors and methods of making and using the same |
| AU2024227303A Pending AU2024227303A1 (en) | 2017-03-15 | 2024-10-14 | Polyploid adeno-associated virus vectors and methods of making and using the same |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| AU2024227303A Pending AU2024227303A1 (en) | 2017-03-15 | 2024-10-14 | Polyploid adeno-associated virus vectors and methods of making and using the same |
Country Status (9)
| Country | Link |
|---|---|
| EP (3) | EP4303225B1 (https=) |
| JP (1) | JP7132934B2 (https=) |
| CN (2) | CN110770346B (https=) |
| AU (2) | AU2018234695B2 (https=) |
| CA (1) | CA3054711A1 (https=) |
| ES (1) | ES2966692T3 (https=) |
| IL (3) | IL268891B2 (https=) |
| NZ (1) | NZ756587A (https=) |
| WO (1) | WO2018170310A1 (https=) |
Families Citing this family (26)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP3151866B1 (en) | 2014-06-09 | 2023-03-08 | Voyager Therapeutics, Inc. | Chimeric capsids |
| GB201508026D0 (en) | 2015-05-11 | 2015-06-24 | Ucl Business Plc | Capsid |
| ES2865487T3 (es) | 2015-09-28 | 2021-10-15 | Univ North Carolina Chapel Hill | Métodos y composiciones para vectores virales que evaden los anticuerpos |
| WO2017205739A1 (en) | 2016-05-26 | 2017-11-30 | University Of Iowa Research Foundation | cis AND trans REQUIREMENTS FOR TERMINAL RESOLUTION OF HUMAN BOCAVIRUS 1 |
| US10550405B2 (en) | 2017-03-15 | 2020-02-04 | The University Of North Carolina At Chapel Hill | Rational polyploid adeno-associated virus vectors and methods of making and using the same |
| EP3645021A4 (en) * | 2017-06-30 | 2021-04-21 | Intima Bioscience, Inc. | ADENO-ASSOCIATED VIRAL VECTORS FOR GENE THERAPY |
| EP3774852A1 (en) | 2018-04-03 | 2021-02-17 | Stridebio, Inc. | Antibody-evading virus vectors |
| AU2019247748A1 (en) | 2018-04-03 | 2020-10-08 | Ginkgo Bioworks, Inc. | Antibody-evading virus vectors |
| MX2020010465A (es) | 2018-04-03 | 2021-01-08 | Vectores de virus para direccionamiento a tejidos oftalmicos. | |
| AU2018422759B2 (en) * | 2018-05-07 | 2026-01-22 | The University Of North Carolina At Chapel Hill | Rational polyploid adeno-associated virus vectors and methods of making and using the same |
| TW202016298A (zh) * | 2018-05-14 | 2020-05-01 | 美商拜奧馬林製藥公司 | Aav 載體於幼年受試者中之穩定表現 |
| CA3119349A1 (en) * | 2018-11-16 | 2020-05-22 | Spark Therapeutics, Inc. | In vitro assay for detecting enhancers and inhibitors of adeno associated virus (aav) vector transduction and/or detecting or quantitating anti-aav binding antibodies |
| JP2022513618A (ja) * | 2018-11-20 | 2022-02-09 | 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 | 血液脳関門の標的化に有用な組成物および方法 |
| WO2020142236A1 (en) * | 2019-01-02 | 2020-07-09 | Intima Bioscience, Inc. | Modified adeno-associated viral vectors for use in genetic engineering |
| AR118465A1 (es) | 2019-03-21 | 2021-10-06 | Stridebio Inc | Vectores de virus adenoasociados recombinantes |
| BR112021020706A2 (pt) * | 2019-04-15 | 2022-03-15 | Spirovant Sciences Inc | Métodos e composições para expressão de transgene |
| SG11202111375RA (en) | 2019-04-29 | 2021-11-29 | Univ Pennsylvania | Novel aav capsids and compositions containing same |
| JP2022551739A (ja) | 2019-10-17 | 2022-12-13 | ストライドバイオ,インコーポレイテッド | ニーマン・ピック病c型の治療のためのアデノ随伴ウイルスベクター |
| US12611436B2 (en) | 2019-10-17 | 2026-04-28 | Sarepta Therapeutics, Inc. | AAV transfer cassette |
| CN111349148A (zh) * | 2020-03-13 | 2020-06-30 | 辉大(上海)生物科技有限公司 | 一种腺相关病毒载体及其用途 |
| CA3177869A1 (en) * | 2020-05-05 | 2021-11-11 | Duke University | Cross-species compatible adeno-associated virus compositions and methods of use thereof |
| CN111876432B (zh) * | 2020-07-29 | 2022-10-28 | 舒泰神(北京)生物制药股份有限公司 | 一组肝靶向新型腺相关病毒的获得及其应用 |
| AU2021328475A1 (en) | 2020-08-19 | 2023-03-16 | Sarepta Therapeutics, Inc. | Adeno-associated virus vectors for treatment of Rett syndrome |
| WO2022221529A1 (en) * | 2021-04-16 | 2022-10-20 | Asklepios Biopharmaceutical, Inc. | Rational polyploid aav virions that cross the blood brain barrier and elicit reduced humoral response |
| AU2022420596A1 (en) * | 2021-12-21 | 2024-07-11 | Research Institute At Nationwide Children's Hospital | Materials and methods for the treatment of limb girdle muscular dystrophy |
| EP4703468A1 (en) * | 2024-08-29 | 2026-03-04 | Sartorius Albumedix Limited | Methods and uses |
Family Cites Families (48)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US4501729A (en) | 1982-12-13 | 1985-02-26 | Research Corporation | Aerosolized amiloride treatment of retained pulmonary secretions |
| AU7966987A (en) | 1986-09-08 | 1988-04-07 | Applied Biotechnology, Inc. | Empty viral capsid vaccines |
| US4968603A (en) | 1986-12-31 | 1990-11-06 | The Regents Of The University Of California | Determination of status in neoplastic disease |
| EP0442926A1 (en) | 1988-11-10 | 1991-08-28 | Imperial Cancer Research Technology Limited | Polypeptides |
| US5916563A (en) | 1988-11-14 | 1999-06-29 | United States Of America | Parvovirus protein presenting capsids |
| US5399346A (en) | 1989-06-14 | 1995-03-21 | The United States Of America As Represented By The Department Of Health And Human Services | Gene therapy |
| ES2026826A6 (es) | 1991-03-26 | 1992-05-01 | Ercros Sa | Procedimiento para la produccion de una vacuna subunidad contra el parvovirus canino y otros virus relacionados. |
| US5478745A (en) | 1992-12-04 | 1995-12-26 | University Of Pittsburgh | Recombinant viral vector system |
| US5869248A (en) | 1994-03-07 | 1999-02-09 | Yale University | Targeted cleavage of RNA using ribonuclease P targeting and cleavage sequences |
| US6204059B1 (en) | 1994-06-30 | 2001-03-20 | University Of Pittsburgh | AAV capsid vehicles for molecular transfer |
| US5599706A (en) | 1994-09-23 | 1997-02-04 | Stinchcomb; Dan T. | Ribozymes targeted to apo(a) mRNA |
| ATE452981T1 (de) | 1995-06-07 | 2010-01-15 | Univ North Carolina | Die transduktion von myoblasten mittels vektoren aus adenoassoziierten viren |
| US6040183A (en) | 1995-06-07 | 2000-03-21 | University Of North Carloina At Chapel Hill | Helper virus-free AAV production |
| US6093570A (en) | 1995-06-07 | 2000-07-25 | The University Of North Carolina At Chapel Hill | Helper virus-free AAV production |
| ATE354957T1 (de) | 1995-12-15 | 2006-03-15 | Intronn Inc | Durch trans-spaltung erhaltene therapeutische molekule |
| US6083702A (en) | 1995-12-15 | 2000-07-04 | Intronn Holdings Llc | Methods and compositions for use in spliceosome mediated RNA trans-splicing |
| WO1998011244A2 (en) | 1996-09-11 | 1998-03-19 | The Government Of The United States Of America, Represented By The Secretary, Department Of Health And Human Services | Aav4 vector and uses thereof |
| US6156303A (en) | 1997-06-11 | 2000-12-05 | University Of Washington | Adeno-associated virus (AAV) isolates and AAV vectors derived therefrom |
| WO1999061601A2 (en) | 1998-05-28 | 1999-12-02 | The Government Of The United States Of America, As Represented By The Secretary, Department Of Health And Human Services | Aav5 vector and uses thereof |
| AU774706B2 (en) | 1998-09-22 | 2004-07-08 | Johns Hopkins University, The | Methods for large-scale production of recombinant AAV vectors |
| ATE362542T1 (de) | 1998-11-05 | 2007-06-15 | Univ Pennsylvania | Nukleinsäuresequenzen des adeno-assoziierten virus des serotyps i, und vektoren und wirtszellen, die diese enthalten |
| AU780231B2 (en) | 1998-11-10 | 2005-03-10 | University Of North Carolina At Chapel Hill, The | Virus vectors and methods of making and administering the same |
| US7314912B1 (en) | 1999-06-21 | 2008-01-01 | Medigene Aktiengesellschaft | AAv scleroprotein, production and use thereof |
| CA2373110A1 (en) * | 2000-03-14 | 2001-09-20 | Neurologix, Inc. | Production of chimeric capsid vectors |
| US7201898B2 (en) | 2000-06-01 | 2007-04-10 | The University Of North Carolina At Chapel Hill | Methods and compounds for controlled release of recombinant parvovirus vectors |
| ATE318923T1 (de) | 2000-06-01 | 2006-03-15 | Univ North Carolina | Doppelsträngige parvovirus-vektoren |
| US6623729B2 (en) | 2001-07-09 | 2003-09-23 | Korea Advanced Institute Of Science And Technology | Process for preparing sustained release micelle employing conjugate of anticancer drug and biodegradable polymer |
| US6648075B2 (en) * | 2001-07-13 | 2003-11-18 | Weatherford/Lamb, Inc. | Method and apparatus for expandable liner hanger with bypass |
| AU2003223766A1 (en) | 2002-04-30 | 2003-11-17 | University Of North Carolina At Chapel Hill | Secretion signal vectors |
| SI1496944T1 (sl) * | 2002-05-01 | 2009-02-28 | Univ Florida | Izboljšani sistemi ekspresije RAAV za genetsko modifikacijo specifičnih kapsidnih proteinov |
| ITRM20020253A1 (it) | 2002-05-08 | 2003-11-10 | Univ Roma | Molecole chimeriche di snrna con sequenze antisenso per le giunzioni di splicing del gene della distrofina e applicazioni terapeutiche. |
| FR2874384B1 (fr) | 2004-08-17 | 2010-07-30 | Genethon | Vecteur viral adeno-associe pour realiser du saut d'exons dans un gene codant une proteine a domaines dispensables |
| JP4346526B2 (ja) | 2004-08-31 | 2009-10-21 | 株式会社東芝 | 半導体集積回路装置 |
| CN101056539B (zh) | 2004-09-09 | 2012-12-12 | 综合医院公司 | 调节心脏细胞中的磷酸酶活性 |
| JP5136766B2 (ja) | 2004-12-15 | 2013-02-06 | ユニバーシティ オブ ノース カロライナ アット チャペル ヒル | キメラベクター |
| JP2008539698A (ja) | 2005-04-29 | 2008-11-20 | ザ・ユニヴァーシティ・オヴ・ノース・キャロライナ・アト・チャペル・ヒル | 転写後レベルでの核酸発現調節のための方法および組成物 |
| US7867484B2 (en) | 2006-01-27 | 2011-01-11 | University Of North Carolina At Chapel Hill | Heparin and heparan sulfate binding chimeric vectors |
| CA2638913A1 (en) | 2006-02-10 | 2007-09-07 | The University Of Cincinnati | Phosphatase inhibitor protein-1 as a regulator of cardiac function |
| WO2008027084A2 (en) * | 2006-04-28 | 2008-03-06 | The Trustees Of The University Of Pennsylvania | Modified aav vectors having reduced capsid immunogenicity and use thereof |
| CN101711164B (zh) | 2007-01-18 | 2014-06-04 | 密苏里-哥伦比亚大学 | 可修复肌纤维膜中的神经元型一氧化氮合酶的合成小/微-抗肌萎缩蛋白基因 |
| WO2009108274A2 (en) | 2008-02-26 | 2009-09-03 | The University Of North Carolina At Chapel Hill | Methods and compositions for adeno-associated virus (aav) with hi loop mutations |
| US20120093775A1 (en) * | 2009-03-27 | 2012-04-19 | Proyecto De Biomedicina Cima, S.L. | Methods and compositions for the treatment of cirrhosis and liver fibrosis |
| EP2675484B1 (en) | 2011-02-14 | 2018-05-30 | The Children's Hospital of Philadelphia | Improved aav8 vector with enhanced functional activity and methods of use thereof |
| CA2853482C (en) | 2011-10-28 | 2020-05-19 | Joshua Grieger | Cell line for production of adeno-associated virus |
| NZ701693A (en) | 2012-04-18 | 2017-02-24 | The Children’S Hospital Of Philadelphia | Composition and methods for highly efficient gene transfer using aav capsid variants |
| US9163259B2 (en) * | 2012-05-04 | 2015-10-20 | Novartis Ag | Viral vectors for the treatment of retinal dystrophy |
| IL297919A (en) * | 2013-07-22 | 2023-01-01 | Childrens Hospital Philadelphia | Variant aav and compositions, methods and uses for gene transfer to cells, organs and tissues |
| MX390848B (es) * | 2015-06-23 | 2025-03-21 | Childrens Hospital Philadelphia | Factor ix modificado, y composiciones, metodos y usos para la transferencia de genes a celulas, organos y tejidos. |
-
2018
- 2018-03-15 NZ NZ756587A patent/NZ756587A/en unknown
- 2018-03-15 WO PCT/US2018/022725 patent/WO2018170310A1/en not_active Ceased
- 2018-03-15 EP EP23195775.4A patent/EP4303225B1/en active Active
- 2018-03-15 JP JP2019545960A patent/JP7132934B2/ja active Active
- 2018-03-15 ES ES18768025T patent/ES2966692T3/es active Active
- 2018-03-15 IL IL268891A patent/IL268891B2/en unknown
- 2018-03-15 EP EP18768025.1A patent/EP3610023B1/en active Active
- 2018-03-15 IL IL312266A patent/IL312266B2/en unknown
- 2018-03-15 AU AU2018234695A patent/AU2018234695B2/en active Active
- 2018-03-15 CN CN201880031964.9A patent/CN110770346B/zh active Active
- 2018-03-15 CA CA3054711A patent/CA3054711A1/en active Pending
- 2018-03-15 IL IL316259A patent/IL316259A/en unknown
- 2018-03-15 CN CN202311805114.9A patent/CN117801075A/zh active Pending
- 2018-03-15 EP EP25211519.1A patent/EP4707292A3/en active Pending
-
2024
- 2024-10-14 AU AU2024227303A patent/AU2024227303A1/en active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| CN110770346B (zh) | 2024-01-12 |
| WO2018170310A1 (en) | 2018-09-20 |
| IL268891B1 (en) | 2024-05-01 |
| IL312266B2 (en) | 2025-03-01 |
| AU2024227303A1 (en) | 2024-10-31 |
| CN110770346A (zh) | 2020-02-07 |
| JP2020511127A (ja) | 2020-04-16 |
| IL312266A (en) | 2024-06-01 |
| IL316259A (en) | 2024-12-01 |
| IL312266B1 (en) | 2024-11-01 |
| EP3610023B1 (en) | 2023-10-04 |
| EP3610023A1 (en) | 2020-02-19 |
| EP4303225A2 (en) | 2024-01-10 |
| IL268891B2 (en) | 2024-09-01 |
| AU2018234695A1 (en) | 2019-09-12 |
| EP4303225B1 (en) | 2025-11-26 |
| EP4303225A3 (en) | 2024-04-10 |
| IL268891A (en) | 2019-10-31 |
| EP4707292A2 (en) | 2026-03-11 |
| EP4707292A3 (en) | 2026-05-06 |
| CN117801075A (zh) | 2024-04-02 |
| EP3610023A4 (en) | 2021-01-20 |
| CA3054711A1 (en) | 2018-09-20 |
| JP7132934B2 (ja) | 2022-09-07 |
| ES2966692T3 (es) | 2024-04-23 |
| NZ756587A (en) | 2025-12-19 |
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| AU2018234695B2 (en) | Polyploid adeno-associated virus vectors and methods of making and using the same | |
| US11702676B2 (en) | Rational polyploid adeno-associated virus vectors for the treatment of disease | |
| CA3085945A1 (en) | Methods and compositions for delivery of viral vectors across the blood-brain barrier | |
| AU2018422759B2 (en) | Rational polyploid adeno-associated virus vectors and methods of making and using the same | |
| JP7725514B2 (ja) | 結節性硬化症の遺伝子治療 | |
| CA3054600A1 (en) | Rational polyploid adeno-associated virus vectors and methods of making and using the same | |
| AU2023252508B2 (en) | Treatment of neuromuscular diseases via gene therapy that expresses klotho protein | |
| HK40098885A (en) | Polyploid adeno-associated virus vectors and methods of making and using the same | |
| HK40104382A (zh) | 多倍体腺相关病毒载体及其制备和使用方法 | |
| HK40022719B (zh) | 多倍体腺相关病毒载体及其制备和使用方法 | |
| HK40021301B (en) | Polyploid adeno-associated virus vectors and methods of making and using the same | |
| HK40021301A (en) | Polyploid adeno-associated virus vectors and methods of making and using the same |
Legal Events
| Date | Code | Title | Description |
|---|---|---|---|
| FGA | Letters patent sealed or granted (standard patent) |