PL3198018T3 - Warianty wektora wirusa związanego z adenowirusami dla wysokiej skuteczności edycji genomu i jego sposoby - Google Patents
Warianty wektora wirusa związanego z adenowirusami dla wysokiej skuteczności edycji genomu i jego sposobyInfo
- Publication number
- PL3198018T3 PL3198018T3 PL15775884T PL15775884T PL3198018T3 PL 3198018 T3 PL3198018 T3 PL 3198018T3 PL 15775884 T PL15775884 T PL 15775884T PL 15775884 T PL15775884 T PL 15775884T PL 3198018 T3 PL3198018 T3 PL 3198018T3
- Authority
- PL
- Poland
- Prior art keywords
- adeno
- methods
- high efficiency
- associated virus
- virus vector
- Prior art date
Links
Classifications
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- 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
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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
- A61K48/0008—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
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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
- A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P37/00—Drugs for immunological or allergic disorders
- A61P37/02—Immunomodulators
- A61P37/04—Immunostimulants
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P7/00—Drugs for disorders of the blood or the extracellular fluid
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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
- 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
- C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
- C12N15/102—Mutagenizing nucleic acids
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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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- 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
- C12N15/864—Parvoviral vectors, e.g. parvovirus, densovirus
- C12N15/8645—Adeno-associated virus
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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
- C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
- C12N15/90—Stable introduction of foreign DNA into chromosome
- C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
- C12N15/907—Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
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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
- C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
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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
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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
- 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
- C12N2710/16622—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
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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
- C12N2750/14121—Viruses as such, e.g. new isolates, mutants or their genomic sequences
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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
- C12N2750/14122—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
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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
- 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
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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
- C12N2750/14145—Special targeting system for viral vectors
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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
- C12N2750/14151—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
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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
- C12N2750/14171—Demonstrated in vivo effect
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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
- C12N2800/00—Nucleic acids vectors
- C12N2800/24—Vectors characterised by the absence of particular element, e.g. selectable marker, viral origin of replication
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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
- C12N2800/00—Nucleic acids vectors
- C12N2800/70—Vectors containing special elements for cloning, e.g. topoisomerase, adaptor sites
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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
- C12N2840/00—Vectors comprising a special translation-regulating system
- C12N2840/44—Vectors comprising a special translation-regulating system being a specific part of the splice mechanism, e.g. donor, acceptor
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Genetics & Genomics (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biotechnology (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- General Health & Medical Sciences (AREA)
- Biomedical Technology (AREA)
- General Engineering & Computer Science (AREA)
- Molecular Biology (AREA)
- Biochemistry (AREA)
- Medicinal Chemistry (AREA)
- Microbiology (AREA)
- Biophysics (AREA)
- Virology (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Veterinary Medicine (AREA)
- Pharmacology & Pharmacy (AREA)
- Immunology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Epidemiology (AREA)
- Mycology (AREA)
- Cell Biology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Gastroenterology & Hepatology (AREA)
- Crystallography & Structural Chemistry (AREA)
- Diabetes (AREA)
- Hematology (AREA)
- Communicable Diseases (AREA)
- Oncology (AREA)
- Obesity (AREA)
Applications Claiming Priority (5)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US201462054899P | 2014-09-24 | 2014-09-24 | |
US201462063587P | 2014-10-14 | 2014-10-14 | |
US201562209862P | 2015-08-25 | 2015-08-25 | |
EP15775884.8A EP3198018B1 (en) | 2014-09-24 | 2015-09-23 | Adeno-associated virus vector variants for high efficiency genome editing and methods thereof |
PCT/US2015/051785 WO2016049230A1 (en) | 2014-09-24 | 2015-09-23 | Adeno-associated virus vector variants for high efficiency genome editing and methods thereof |
Publications (1)
Publication Number | Publication Date |
---|---|
PL3198018T3 true PL3198018T3 (pl) | 2021-07-19 |
Family
ID=54261111
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
PL15775884T PL3198018T3 (pl) | 2014-09-24 | 2015-09-23 | Warianty wektora wirusa związanego z adenowirusami dla wysokiej skuteczności edycji genomu i jego sposoby |
Country Status (15)
Country | Link |
---|---|
US (5) | US9890396B2 (pl) |
EP (2) | EP3800260A1 (pl) |
JP (3) | JP6683691B2 (pl) |
KR (2) | KR102526711B1 (pl) |
CN (2) | CN107295802B (pl) |
AU (2) | AU2015320694B2 (pl) |
BR (1) | BR112017005892A2 (pl) |
CA (1) | CA2961555A1 (pl) |
DK (1) | DK3198018T3 (pl) |
ES (1) | ES2856090T3 (pl) |
MX (2) | MX2017003815A (pl) |
PL (1) | PL3198018T3 (pl) |
PT (1) | PT3198018T (pl) |
SG (3) | SG10201902574RA (pl) |
WO (1) | WO2016049230A1 (pl) |
Families Citing this family (165)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
JP5054975B2 (ja) | 2003-09-30 | 2012-10-24 | 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 | アデノ随伴ウイルス(aav)の同源系統群、配列、それらを含有するベクターおよびそれらの用途 |
ES2400235T3 (es) | 2006-04-28 | 2013-04-08 | The Trustees Of The University Of Pennsylvania | Método de producción escalable de AAV |
US9839696B2 (en) | 2010-04-30 | 2017-12-12 | City Of Hope | Recombinant adeno-associated vectors for targeted treatment |
CA2853829C (en) | 2011-07-22 | 2023-09-26 | President And Fellows Of Harvard College | Evaluation and improvement of nuclease cleavage specificity |
KR102346455B1 (ko) | 2013-03-15 | 2022-01-04 | 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 | Mpsi 치료를 위한 조성물 및 방법 |
US20150044192A1 (en) | 2013-08-09 | 2015-02-12 | President And Fellows Of Harvard College | Methods for identifying a target site of a cas9 nuclease |
US9359599B2 (en) | 2013-08-22 | 2016-06-07 | President And Fellows Of Harvard College | Engineered transcription activator-like effector (TALE) domains and uses thereof |
US9526784B2 (en) | 2013-09-06 | 2016-12-27 | President And Fellows Of Harvard College | Delivery system for functional nucleases |
US9388430B2 (en) | 2013-09-06 | 2016-07-12 | President And Fellows Of Harvard College | Cas9-recombinase fusion proteins and uses thereof |
US9340800B2 (en) | 2013-09-06 | 2016-05-17 | President And Fellows Of Harvard College | Extended DNA-sensing GRNAS |
US20150166982A1 (en) | 2013-12-12 | 2015-06-18 | President And Fellows Of Harvard College | Methods for correcting pi3k point mutations |
US10077453B2 (en) | 2014-07-30 | 2018-09-18 | President And Fellows Of Harvard College | CAS9 proteins including ligand-dependent inteins |
WO2016200543A2 (en) | 2015-05-13 | 2016-12-15 | The Trustees Of The University Of Pennsylvania | Aav-mediated expression of anti-inluenza antibodies and methods of use thereof |
US10966414B2 (en) | 2015-05-26 | 2021-04-06 | California Institute Of Technology | Population control using engineered translocations |
PT3356390T (pt) | 2015-09-28 | 2021-04-21 | Univ Florida | Métodos e composições para vetores virais que se evadem a anticorpos |
EP4269577A3 (en) | 2015-10-23 | 2024-01-17 | President and Fellows of Harvard College | Nucleobase editors and uses thereof |
WO2017075335A1 (en) | 2015-10-28 | 2017-05-04 | Voyager Therapeutics, Inc. | Regulatable expression using adeno-associated virus (aav) |
WO2017096164A1 (en) | 2015-12-02 | 2017-06-08 | The Board Of Trustees Of The Leland Stanford Junior University | Novel recombinant adeno-associated virus capsids with enhanced human skeletal muscle tropism |
US10406244B2 (en) | 2015-12-02 | 2019-09-10 | The Board Of Trustees Of The Leland Stanford Junior University | AAV vectors with expanded packaging capacity |
US11098286B2 (en) | 2015-12-11 | 2021-08-24 | The Trustees Of The University Of Pennsylvania | Scalable purification method for AAV9 |
US11028372B2 (en) | 2015-12-11 | 2021-06-08 | The Trustees Of The University Of Pennsylvania | Scalable purification method for AAVRH10 |
ES2934848T3 (es) | 2015-12-11 | 2023-02-27 | Univ Pennsylvania | Método de purificación escalable para AAV8 |
WO2017100674A1 (en) | 2015-12-11 | 2017-06-15 | The Trustees Of The University Of Pennsylvania | Scalable purification method for aav1 |
MA43968A (fr) | 2016-02-03 | 2018-12-12 | Univ Pennsylvania | Thérapie génique pour traiter la mucopolysaccharidose de type i |
CA3014637A1 (en) | 2016-02-16 | 2017-08-24 | Mark A. Kay | Novel recombinant adeno-associated virus capsids resistant to pre-existing human neutralizing antibodies |
IL305449A (en) | 2016-04-15 | 2023-10-01 | Univ Pennsylvania | Gene therapy for the treatment of type II mucositis |
WO2017192750A1 (en) * | 2016-05-04 | 2017-11-09 | Oregon Health & Science University | Recombinant adeno-associated viral vectors |
KR102392236B1 (ko) | 2016-05-18 | 2022-05-03 | 보이저 테라퓨틱스, 인크. | 조절성 폴리뉴클레오티드 |
IL305149A (en) | 2016-07-26 | 2023-10-01 | Biomarin Pharm Inc | Novel adeno-associated virus capsid proteins |
SG11201900907YA (en) | 2016-08-03 | 2019-02-27 | Harvard College | Adenosine nucleobase editors and uses thereof |
WO2018031683A1 (en) | 2016-08-09 | 2018-02-15 | President And Fellows Of Harvard College | Programmable cas9-recombinase fusion proteins and uses thereof |
US11542509B2 (en) | 2016-08-24 | 2023-01-03 | President And Fellows Of Harvard College | Incorporation of unnatural amino acids into proteins using base editing |
CN110214180A (zh) | 2016-10-14 | 2019-09-06 | 哈佛大学的校长及成员们 | 核碱基编辑器的aav递送 |
US20180245065A1 (en) | 2016-11-01 | 2018-08-30 | Novartis Ag | Methods and compositions for enhancing gene editing |
US10745677B2 (en) | 2016-12-23 | 2020-08-18 | President And Fellows Of Harvard College | Editing of CCR5 receptor gene to protect against HIV infection |
JOP20190200A1 (ar) | 2017-02-28 | 2019-08-27 | Univ Pennsylvania | تركيبات نافعة في معالجة ضمور العضل النخاعي |
SG11201907714UA (en) | 2017-02-28 | 2019-09-27 | Univ Pennsylvania | Adeno-associated virus (aav) clade f vector and uses therefor |
CA3052487A1 (en) | 2017-02-28 | 2018-09-07 | The Trustees Of The University Of Pennsylvania | Influenza vaccines based on aav vectors |
EP3592853A1 (en) | 2017-03-09 | 2020-01-15 | President and Fellows of Harvard College | Suppression of pain by gene editing |
EP3592777A1 (en) | 2017-03-10 | 2020-01-15 | President and Fellows of Harvard College | Cytosine to guanine base editor |
CN110914426A (zh) | 2017-03-23 | 2020-03-24 | 哈佛大学的校长及成员们 | 包含核酸可编程dna结合蛋白的核碱基编辑器 |
WO2018204722A1 (en) * | 2017-05-05 | 2018-11-08 | Califorinia Institute Of Technology | Dna sequence modification-based gene drive |
WO2018209205A1 (en) | 2017-05-11 | 2018-11-15 | The Trustees Of The University Of Pennsylvania | Gene therapy for neuronal ceroid lipofuscinoses |
WO2018209320A1 (en) | 2017-05-12 | 2018-11-15 | President And Fellows Of Harvard College | Aptazyme-embedded guide rnas for use with crispr-cas9 in genome editing and transcriptional activation |
CN107034233B (zh) * | 2017-05-18 | 2021-06-08 | 中国科学院重庆绿色智能技术研究院 | 一种内源性启动子驱动外源基因表达的方法 |
JP7123982B2 (ja) * | 2017-06-15 | 2022-08-23 | ツールゲン インコーポレイテッド | 肝臓において目的のタンパク質を発現するためのプラットフォーム |
CA3068328A1 (en) | 2017-07-06 | 2019-01-10 | The Trustees Of The University Of Pennsylvania | Aav9-mediated gene therapy for treating mucopolysaccharidosis type i |
WO2019023680A1 (en) | 2017-07-28 | 2019-01-31 | President And Fellows Of Harvard College | METHODS AND COMPOSITIONS FOR EVOLUTION OF BASIC EDITORS USING PHAGE-ASSISTED CONTINUOUS EVOLUTION (PACE) |
JP7221275B2 (ja) | 2017-08-03 | 2023-02-13 | ボイジャー セラピューティクス インコーポレイテッド | Aavを送達するための組成物および方法 |
US11319532B2 (en) | 2017-08-30 | 2022-05-03 | President And Fellows Of Harvard College | High efficiency base editors comprising Gam |
TWI835747B (zh) | 2017-09-22 | 2024-03-21 | 賓州大學委員會 | 用於治療黏多醣病 ii 型之基因治療 |
JP2021500036A (ja) | 2017-10-16 | 2021-01-07 | ザ ブロード インスティテュート, インコーポレーテッドThe Broad Institute, Inc. | アデノシン塩基編集因子の使用 |
CA3079437A1 (en) * | 2017-10-18 | 2019-04-25 | City Of Hope | Adeno-associated virus compositions for restoring hbb gene function and methods of use thereof |
CN107937441B (zh) * | 2017-11-14 | 2021-02-26 | 中国科学院苏州生物医学工程技术研究所 | AAVS1位点定点整合外源基因的人iPS细胞的构建方法 |
EP3717652A4 (en) | 2017-11-30 | 2021-11-24 | The Trustees of the University of Pennsylvania | GENE THERAPY FOR MUCOPOLY SACCHARIDOSIS IIIB |
KR20200104307A (ko) | 2017-11-30 | 2020-09-03 | 더 트러스티스 오브 더 유니버시티 오브 펜실바니아 | 뮤코다당류증 iiia형에 대한 유전자 요법 |
PE20210341A1 (es) | 2018-02-01 | 2021-02-23 | Homology Medicines Inc | Composiciones de virus adeno-asociadas para la transferencia de genes pah y metodos de uso de los mismos |
WO2019152843A1 (en) * | 2018-02-01 | 2019-08-08 | Homology Medicines, Inc. | Adeno-associated virus compositions for restoring pah gene function and methods of use thereof |
US10610606B2 (en) | 2018-02-01 | 2020-04-07 | Homology Medicines, Inc. | Adeno-associated virus compositions for PAH gene transfer and methods of use thereof |
EP3755795A4 (en) | 2018-02-19 | 2022-07-20 | Homology Medicines, Inc. | ADENO-ASSOCIATED VIRUS COMPOSITIONS FOR RECOVERING F8 GENE FUNCTION AND METHODS OF USE THEREOF |
KR20210006327A (ko) * | 2018-02-27 | 2021-01-18 | 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 | 캡시드 탈아미드화가 감소된 신규 아데노-연관 바이러스(aav) 벡터 및 이의 용도 |
BR112020020266A2 (pt) | 2018-04-03 | 2021-01-19 | Stridebio, Inc. | Vetores de vírus com evasão de anticorpos |
BR112020020223A2 (pt) | 2018-04-05 | 2021-01-19 | Genethon | Sorotipo de vírus adeno-associado híbrido recombinante entre aav9 e aavrh74 com tropismo pelo fígado reduzido |
WO2019212921A1 (en) | 2018-04-29 | 2019-11-07 | Regenxbio Inc. | Scalable clarification process for recombinant aav production |
WO2019212922A1 (en) | 2018-04-29 | 2019-11-07 | Regenxbio Inc. | Systems and methods of spectrophotometry for the determination of genome content, capsid content and full/empty ratios of adeno-associated virus particles |
CN113164762A (zh) | 2018-05-09 | 2021-07-23 | 生物马林药物股份有限公司 | 苯丙酮尿症的治疗方法 |
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 |
WO2019222328A1 (en) | 2018-05-15 | 2019-11-21 | Voyager Therapeutics, Inc. | Compositions and methods for the treatment of parkinson's disease |
TW202015742A (zh) | 2018-05-15 | 2020-05-01 | 美商航海家醫療公司 | 投遞腺相關病毒(aav)之組成物和方法 |
KR20210020100A (ko) | 2018-06-14 | 2021-02-23 | 리젠엑스바이오 인크. | 재조합 aav 생성을 위한 음이온 교환 크로마토그래피 |
US20210254103A1 (en) * | 2018-07-02 | 2021-08-19 | Voyager Therapeutics, Inc. | Treatment of amyotrophic lateral sclerosis and disorders associated with the spinal cord |
US20210163991A1 (en) | 2018-08-10 | 2021-06-03 | Regenxbio Inc. | Scalable method for recombinant aav production |
WO2020069461A1 (en) | 2018-09-28 | 2020-04-02 | Voyager Therapeutics, Inc. | Frataxin expression constructs having engineered promoters and methods of use thereof |
JP2022512589A (ja) | 2018-10-01 | 2022-02-07 | 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 | Gm1ガングリオシドーシスの治療に有用な組成物 |
WO2020077165A1 (en) | 2018-10-12 | 2020-04-16 | Voyager Therapeutics, Inc. | Compositions and methods for delivery of aav |
JP2022514112A (ja) | 2018-10-15 | 2022-02-09 | リジェネクスバイオ インコーポレイテッド | 複製欠損型ウイルスベクター及びウイルスの感染性を測定するための方法 |
US11965172B2 (en) | 2018-11-05 | 2024-04-23 | California Institute Of Technology | DNA sequence modification-based gene drive |
EP4269579A3 (en) | 2019-01-04 | 2024-01-24 | Ultragenyx Pharmaceutical Inc. | Gene therapy constructs for treating wilson disease |
EP3911410A1 (en) | 2019-01-18 | 2021-11-24 | Voyager Therapeutics, Inc. | Methods and systems for producing aav particles |
KR20210132095A (ko) | 2019-02-26 | 2021-11-03 | 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 | 크라베병의 치료에 유용한 조성물 |
BR112021018607A2 (pt) | 2019-03-19 | 2021-11-23 | Massachusetts Inst Technology | Métodos e composições para editar sequências de nucleotídeos |
CA3133453A1 (en) | 2019-03-21 | 2020-09-24 | Daniel Mccoy | Recombinant adeno-associated virus vectors |
TW202102526A (zh) | 2019-04-04 | 2021-01-16 | 美商銳進科斯生物股份有限公司 | 重組腺相關病毒及其用途 |
WO2020210600A1 (en) | 2019-04-11 | 2020-10-15 | Regenxbio Inc. | Methods of size exclusion chromatography for the characterization of recombinant adeno-associated virus compositions |
CA3136939A1 (en) | 2019-04-19 | 2020-10-22 | Regenxbio Inc. | Adeno-associated virus vector formulations and methods |
KR20220012231A (ko) | 2019-04-24 | 2022-02-03 | 리젠엑스바이오 인크. | 완전-인간 번역 후 변형된 항체 치료제 |
EP3966227A1 (en) | 2019-05-07 | 2022-03-16 | Voyager Therapeutics, Inc. | Compositions and methods for the vectored augmentation of protein destruction, expression and/or regulation |
US20220251567A1 (en) | 2019-07-10 | 2022-08-11 | Inserm (Institut National De La Santè Et De La Recherche Médicale) | Methods for the treatment of epilepsy |
CA3145112A1 (en) | 2019-07-26 | 2021-02-04 | Chunping Qiao | Engineered nucleic acid regulatory element and methods of uses thereof |
US20220333133A1 (en) | 2019-09-03 | 2022-10-20 | Voyager Therapeutics, Inc. | Vectorized editing of nucleic acids to correct overt mutations |
AU2020358863A1 (en) | 2019-10-03 | 2022-05-12 | Artisan Development Labs, Inc. | CRISPR systems with engineered dual guide nucleic acids |
WO2021067598A1 (en) | 2019-10-04 | 2021-04-08 | Ultragenyx Pharmaceutical Inc. | Methods for improved therapeutic use of recombinant aav |
BR112022006530A2 (pt) | 2019-10-17 | 2022-07-05 | Stridebio Inc | Vetores virais adenoassociados para o tratamento da doença de niemann-pick tipo c |
WO2021099394A1 (en) | 2019-11-19 | 2021-05-27 | INSERM (Institut National de la Santé et de la Recherche Médicale) | Antisense oligonucleotides and their use for the treatment of cancer |
EP4065596A2 (en) | 2019-11-28 | 2022-10-05 | RegenxBio Inc. | Microdystrophin gene therapy constructs and uses thereof |
CN113122657B (zh) * | 2019-12-31 | 2022-07-29 | 上海吉凯基因医学科技股份有限公司 | 一种多重aav病毒滴度的检测方法 |
TW202140791A (zh) | 2020-01-13 | 2021-11-01 | 美商霍蒙拉奇醫藥公司 | 治療苯酮尿症之方法 |
EP4096721A1 (en) | 2020-02-02 | 2022-12-07 | The Trustees of The University of Pennsylvania | Compositions useful for treating gm1 gangliosidosis |
CA3164714A1 (en) | 2020-02-14 | 2021-08-19 | Ultragenyx Pharmaceutical Inc. | Gene therapy for treating cdkl5 deficiency disorder |
EP4127189A1 (en) | 2020-03-31 | 2023-02-08 | Ultragenyx Pharmaceutical Inc. | Gene therapy for treating propionic acidemia |
EP4126910A1 (en) | 2020-04-01 | 2023-02-08 | Voyager Therapeutics, Inc. | Redirection of tropism of aav capsids |
BR112022020753A2 (pt) | 2020-04-15 | 2022-12-20 | Voyager Therapeutics Inc | Compostos de ligação a tau |
CN116096873A (zh) | 2020-05-08 | 2023-05-09 | 布罗德研究所股份有限公司 | 同时编辑靶标双链核苷酸序列的两条链的方法和组合物 |
AU2021271635A1 (en) | 2020-05-12 | 2023-01-05 | The Trustees Of The University Of Pennsylvania | Compositions useful in treatment of krabbe disease |
US20230304034A1 (en) | 2020-05-12 | 2023-09-28 | The Trustees Of The University Of Pennsylvania | Compositions for drg-specific reduction of transgene expression |
KR20230022175A (ko) | 2020-05-13 | 2023-02-14 | 보이저 테라퓨틱스, 인크. | Aav 캡시드의 향성 방향변경 |
WO2021247995A2 (en) | 2020-06-04 | 2021-12-09 | Voyager Therapeutics, Inc. | Compositions and methods of treating neuropathic pain |
EP4179091A1 (en) | 2020-07-10 | 2023-05-17 | Institut National De La Sante Et De La Recherche Medicale - Inserm | Methods and compositions for treating epilepsy |
WO2022026410A2 (en) | 2020-07-27 | 2022-02-03 | Voyager Therapeutics, Inc | Compositions and methods for the treatment of niemann-pick type c1 disease |
BR112023001456A2 (pt) | 2020-07-27 | 2023-04-11 | Voyager Therapeutics Inc | Composições e métodos para o tratamento de distúrbios neurológicos relacionados à deficiência de beta glicosilceramidase |
WO2022060916A1 (en) | 2020-09-15 | 2022-03-24 | Regenxbio Inc. | Vectorized antibodies for anti-viral therapy |
WO2022060915A1 (en) | 2020-09-15 | 2022-03-24 | Regenxbio Inc. | Vectorized lanadelumab and administration thereof |
WO2022061002A1 (en) | 2020-09-18 | 2022-03-24 | The United States Of America, As Represented By The Secretary, Department Of Health And Human Services | Novel adeno-associated viral (aav) vectors to treat hereditary methylmalonic acidemia (mma) caused by methylmalonyl-coa mutase (mmut) deficiency |
EP4225777A2 (en) | 2020-10-07 | 2023-08-16 | RegenxBio Inc. | Adeno-associated viruses for ocular delivery of gene therapy |
WO2022076750A2 (en) | 2020-10-07 | 2022-04-14 | Regenxbio Inc. | Recombinant adeno-associated viruses for cns or muscle delivery |
IL301643A (en) | 2020-10-07 | 2023-05-01 | Regenxbio Inc | Gene therapy for ocular manifestations of CLN2 disease |
US20230365955A1 (en) | 2020-10-09 | 2023-11-16 | The Trustees Of The University Of Pennsylvania | Compositions and methods for treatment of fabry disease |
AU2021371307A1 (en) | 2020-10-28 | 2023-06-01 | Regenxbio, Inc. | VECTORIZED ANTI-TNF-α ANTIBODIES FOR OCULAR INDICATIONS |
WO2022094078A1 (en) | 2020-10-28 | 2022-05-05 | The Trustees Of The University Of Pennsylvania | Compositions useful in treatment of rett syndrome |
WO2022094157A1 (en) | 2020-10-28 | 2022-05-05 | Regenxbio Inc. | Vectorized anti-cgrp and anti-cgrpr antibodies and administration thereof |
EP4236974A2 (en) | 2020-10-29 | 2023-09-06 | RegenxBio Inc. | Vectorized factor xii antibodies and administration thereof |
IL302282A (en) | 2020-10-29 | 2023-06-01 | Regenxbio Inc | Vectored TNF-alpha antagonists for ocular indications |
WO2022104344A2 (en) * | 2020-11-10 | 2022-05-19 | The Board Of Trustees Of The Leland Stanford Junior University | Knock-in of large dna for long-term high genomic expression |
US20240024507A1 (en) | 2020-12-01 | 2024-01-25 | The Trustees Of The University Of Pennsylvania | Novel compositions with tissue-specific targeting motifs and compositions containing same |
CA3201743A1 (en) | 2020-12-16 | 2022-06-23 | Robert STADELMAN | Method of producing a recombinant adeno-associated virus particle |
TW202241943A (zh) | 2020-12-29 | 2022-11-01 | 美商銳進科斯生物股份有限公司 | Tau特異性抗體基因療法組合物、方法及其用途 |
US20240084329A1 (en) | 2021-01-21 | 2024-03-14 | Regenxbio Inc. | Improved production of recombinant polypeptides and viruses |
CA3209779A1 (en) | 2021-02-01 | 2022-08-04 | Regenxbio Inc. | Gene therapy for neuronal ceroid lipofuscinoses |
CN115197967B (zh) * | 2021-04-08 | 2023-09-15 | 广州派真生物技术有限公司 | 制备重组腺相关病毒的辅助质粒及其应用 |
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 |
JP2024517143A (ja) | 2021-04-26 | 2024-04-19 | リジェネックスバイオ インコーポレイテッド | ジストロフィン異常症の治療のためのマイクロジストロフィン遺伝子療法投与 |
EP4330416A1 (en) * | 2021-04-26 | 2024-03-06 | University of Massachusetts | Direct raav-mediated in vivo gene editing of hematopoietic stem cells |
EP4334454A2 (en) | 2021-05-04 | 2024-03-13 | RegenxBio Inc. | Novel aav vectors and methods and uses thereof |
WO2022241030A1 (en) | 2021-05-11 | 2022-11-17 | Regenxbio Inc. | Treatment of duchenne muscular dystrophy and combinations thereof |
WO2022256448A2 (en) | 2021-06-01 | 2022-12-08 | Artisan Development Labs, Inc. | Compositions and methods for targeting, editing, or modifying genes |
EP4370676A2 (en) | 2021-06-18 | 2024-05-22 | Artisan Development Labs, Inc. | Compositions and methods for targeting, editing or modifying human genes |
AU2022299552A1 (en) | 2021-06-25 | 2024-01-04 | Oxford Biomedica (Us) Llc | Adeno-associated virus packaging systems |
WO2023028513A1 (en) | 2021-08-24 | 2023-03-02 | Oxford Biomedica Solutions Llc | Adeno-associated virus formulations |
CN118202060A (zh) | 2021-10-05 | 2024-06-14 | 再生生物股份有限公司 | 用于重组aav生产的组合物和方法 |
WO2023060113A1 (en) | 2021-10-05 | 2023-04-13 | Regenxbio Inc. | Compositions and methods for recombinant aav production |
WO2023060272A2 (en) | 2021-10-07 | 2023-04-13 | Regenxbio Inc. | Recombinant adeno-associated viruses for cns tropic delivery |
WO2023060269A1 (en) | 2021-10-07 | 2023-04-13 | Regenxbio Inc. | Recombinant adeno-associated viruses for targeted delivery |
WO2023077092A1 (en) | 2021-10-28 | 2023-05-04 | Regenxbio Inc. | Engineered nucleic acid regulatory elements and methods and uses thereof |
US20230183658A1 (en) | 2021-10-29 | 2023-06-15 | Oxford Biomedica Solutions Llc | Methods and compositions for the purification of adeno-associated virus |
WO2023087019A2 (en) | 2021-11-15 | 2023-05-19 | The Trustees Of The University Of Pennsylvania | Compositions for drg-specific reduction of transgene expression |
WO2023102517A1 (en) | 2021-12-02 | 2023-06-08 | The Trustees Of The University Of Pennsylvania | Compositions and methods for treatment of fabry disease |
US20230323395A1 (en) | 2021-12-15 | 2023-10-12 | Homology Medicines, Inc. | Methods and compositions for the production of adeno-associated virus |
TW202338086A (zh) | 2022-01-10 | 2023-10-01 | 賓州大學委員會 | 有用於治療異染性白質失養症之組成物 |
WO2023147304A1 (en) | 2022-01-25 | 2023-08-03 | The Trustees Of The University Of Pennsylvania | Aav capsids for improved heart transduction and detargeting of liver |
WO2023167882A1 (en) | 2022-03-01 | 2023-09-07 | Artisan Development Labs, Inc. | Composition and methods for transgene insertion |
WO2023178053A1 (en) | 2022-03-13 | 2023-09-21 | Regenxbio Inc. | Modified muscle-specific promoters |
WO2023183623A1 (en) | 2022-03-25 | 2023-09-28 | Regenxbio Inc. | Dominant-negative tumor necrosis factor alpha adeno-associated virus gene therapy |
WO2023201308A1 (en) | 2022-04-14 | 2023-10-19 | Regenxbio Inc. | Gene therapy for treating an ocular disease |
WO2023201277A1 (en) | 2022-04-14 | 2023-10-19 | Regenxbio Inc. | Recombinant adeno-associated viruses for cns tropic delivery |
WO2023215807A1 (en) | 2022-05-03 | 2023-11-09 | Regenxbio Inc. | VECTORIZED ANTI-TNF-α INHIBITORS FOR OCULAR INDICATIONS |
TW202400803A (zh) | 2022-05-03 | 2024-01-01 | 美商銳進科斯生物股份有限公司 | 載體化抗補體抗體與補體劑及其投與 |
WO2023225410A2 (en) | 2022-05-20 | 2023-11-23 | Artisan Development Labs, Inc. | Systems and methods for assessing risk of genome editing events |
WO2023239627A2 (en) | 2022-06-08 | 2023-12-14 | Regenxbio Inc. | Methods for recombinant aav production |
WO2024017990A1 (en) | 2022-07-21 | 2024-01-25 | Institut National de la Santé et de la Recherche Médicale | Methods and compositions for treating chronic pain disorders |
WO2024044725A2 (en) | 2022-08-24 | 2024-02-29 | Regenxbio Inc. | Recombinant adeno-associated viruses and uses thereof |
CN115948408A (zh) * | 2022-09-23 | 2023-04-11 | 上海信致医药科技有限公司 | 改进的人凝血因子viii基因表达盒及其应用 |
WO2024062138A1 (en) | 2022-09-23 | 2024-03-28 | Mnemo Therapeutics | Immune cells comprising a modified suv39h1 gene |
WO2024081746A2 (en) | 2022-10-11 | 2024-04-18 | Regenxbio Inc. | Engineered nucleic acid regulatory elements and methods and uses thereof |
CN115896135B (zh) * | 2022-11-02 | 2024-03-01 | 苏州诺洁贝生物技术有限公司 | 优化的pah基因和表达盒及其用途 |
CN116726198B (zh) * | 2023-03-13 | 2024-02-13 | 陕西师范大学 | 一种抗肿瘤药物及其在脑胶质瘤治疗中的应用 |
Family Cites Families (21)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
US4866042A (en) | 1987-11-18 | 1989-09-12 | Neuwelt Edward A | Method for the delivery of genetic material across the blood brain barrier |
AU7906691A (en) | 1990-05-23 | 1991-12-10 | United States of America, as represented by the Secretary, U.S. Department of Commerce, The | Adeno-associated virus (aav)-based eucaryotic vectors |
US5252479A (en) | 1991-11-08 | 1993-10-12 | Research Corporation Technologies, Inc. | Safe vector for gene therapy |
US6207457B1 (en) | 1995-09-08 | 2001-03-27 | Avigen, Inc. | Targeted nucleotide sequence delivery and integration system |
WO1998048005A1 (en) | 1997-04-24 | 1998-10-29 | University Of Washington | Targeted gene modification by parvoviral vectors |
US6534261B1 (en) | 1999-01-12 | 2003-03-18 | Sangamo Biosciences, Inc. | Regulation of endogenous gene expression in cells using zinc finger proteins |
CA2461290C (en) | 2001-09-24 | 2014-11-25 | Sangamo Biosciences, Inc. | Modulation of stem cells using zinc finger proteins |
US20060292117A1 (en) * | 2002-04-17 | 2006-12-28 | Loiler Scott A | Improved rAAv vectors |
NZ561656A (en) * | 2002-05-01 | 2009-03-31 | Univ Florida | Improved rAAV expression systems for genetic modification of specific capsid proteins |
GB0322402D0 (en) * | 2003-09-24 | 2003-10-22 | Oxxon Pharmaccines Ltd | HIV pharmaccines |
JP5054975B2 (ja) | 2003-09-30 | 2012-10-24 | 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 | アデノ随伴ウイルス(aav)の同源系統群、配列、それらを含有するベクターおよびそれらの用途 |
US20100310583A1 (en) | 2007-01-31 | 2010-12-09 | Immune Disease Institute | Let-7 microrna and mimetics thereof as therapeutics for cancer |
AU2010224029B2 (en) | 2009-03-11 | 2015-10-22 | Sapphire Energy, Inc. | Engineering salt tolerance in photosynthetic microorganisms |
CN102439157B (zh) * | 2009-04-30 | 2015-09-16 | 宾夕法尼亚大学托管会 | 包含腺伴随病毒构建体的靶向传导气道细胞组合物 |
US8628966B2 (en) * | 2010-04-30 | 2014-01-14 | City Of Hope | CD34-derived recombinant adeno-associated vectors for stem cell transduction and systemic therapeutic gene transfer |
US8927514B2 (en) | 2010-04-30 | 2015-01-06 | City Of Hope | Recombinant adeno-associated vectors for targeted treatment |
JP6018069B2 (ja) * | 2010-10-12 | 2016-11-02 | ザ・チルドレンズ・ホスピタル・オブ・フィラデルフィアThe Children’S Hospital Of Philadelphia | 血友病bを治療する方法及び組成物 |
EP2500434A1 (en) * | 2011-03-12 | 2012-09-19 | Association Institut de Myologie | Capsid-free AAV vectors, compositions, and methods for vector production and gene delivery |
US9873894B2 (en) | 2013-05-15 | 2018-01-23 | Sangamo Therapeutics, Inc. | Methods and compositions for treatment of a genetic condition |
CA2926078C (en) | 2013-10-17 | 2021-11-16 | Sangamo Biosciences, Inc. | Delivery methods and compositions for nuclease-mediated genome engineering in hematopoietic stem cells |
KR20160135729A (ko) | 2014-03-21 | 2016-11-28 | 더 보드 어브 트러스티스 어브 더 리랜드 스탠포드 주니어 유니버시티 | 뉴클레아제 없는 게놈 편집 |
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