[go: up one dir, main page]

SG10202009852PA - Novel aav8 mutant capsids and compositions containing same - Google Patents

Novel aav8 mutant capsids and compositions containing same

Info

Publication number
SG10202009852PA
SG10202009852PA SG10202009852PA SG10202009852PA SG10202009852PA SG 10202009852P A SG10202009852P A SG 10202009852PA SG 10202009852P A SG10202009852P A SG 10202009852PA SG 10202009852P A SG10202009852P A SG 10202009852PA SG 10202009852P A SG10202009852P A SG 10202009852PA
Authority
SG
Singapore
Prior art keywords
compositions containing
containing same
mutant capsids
novel
aav8 mutant
Prior art date
Application number
SG10202009852PA
Inventor
James Wilson
Qiang Wang
Original Assignee
Univ Pennsylvania
Priority date (The priority date 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 date listed.)
Filing date
Publication date
Application filed by Univ Pennsylvania filed Critical Univ Pennsylvania
Publication of SG10202009852PA publication Critical patent/SG10202009852PA/en

Links

Classifications

    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2710/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011Details
    • C12N2710/10011Adenoviridae
    • C12N2710/10032Use of virus as therapeutic agent, other than vaccine, e.g. as cytolytic agent
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2710/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011Details
    • C12N2710/10011Adenoviridae
    • C12N2710/10033Use of viral protein as therapeutic agent other than vaccine, e.g. apoptosis inducing or anti-inflammatory
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2710/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011Details
    • C12N2710/10011Adenoviridae
    • C12N2710/10041Use of virus, viral particle or viral elements as a vector
    • C12N2710/10045Special targeting system for viral vectors
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14122New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2810/00Vectors comprising a targeting moiety
    • C12N2810/50Vectors comprising as targeting moiety peptide derived from defined protein
    • C12N2810/60Vectors comprising as targeting moiety peptide derived from defined protein from viruses
    • C12N2810/6027Vectors comprising as targeting moiety peptide derived from defined protein from viruses ssDNA viruses

Landscapes

  • Life Sciences & Earth Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Virology (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Medicinal Chemistry (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Peptides Or Proteins (AREA)
  • Knitting Of Fabric (AREA)
SG10202009852PA 2016-04-15 2017-04-13 Novel aav8 mutant capsids and compositions containing same SG10202009852PA (en)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US201662323389P 2016-04-15 2016-04-15

Publications (1)

Publication Number Publication Date
SG10202009852PA true SG10202009852PA (en) 2020-11-27

Family

ID=60042790

Family Applications (2)

Application Number Title Priority Date Filing Date
SG10202009852PA SG10202009852PA (en) 2016-04-15 2017-04-13 Novel aav8 mutant capsids and compositions containing same
SG11201808812RA SG11201808812RA (en) 2016-04-15 2017-04-13 Novel aav8 mutant capsids and compositions containing same

Family Applications After (1)

Application Number Title Priority Date Filing Date
SG11201808812RA SG11201808812RA (en) 2016-04-15 2017-04-13 Novel aav8 mutant capsids and compositions containing same

Country Status (9)

Country Link
US (2) US11091776B2 (en)
EP (1) EP3443108A4 (en)
JP (2) JP7140683B2 (en)
CN (1) CN109661470A (en)
AU (2) AU2017248656B2 (en)
CA (1) CA3019423A1 (en)
IL (2) IL298604A (en)
SG (2) SG10202009852PA (en)
WO (1) WO2017180854A1 (en)

Families Citing this family (48)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2013163628A2 (en) 2012-04-27 2013-10-31 Duke University Genetic correction of mutated genes
US9828582B2 (en) 2013-03-19 2017-11-28 Duke University Compositions and methods for the induction and tuning of gene expression
US10577627B2 (en) 2014-06-09 2020-03-03 Voyager Therapeutics, Inc. Chimeric capsids
SG11201703148TA (en) 2014-11-05 2017-05-30 Voyager Therapeutics Inc Aadc polynucleotides for the treatment of parkinson's disease
MX2017006217A (en) 2014-11-14 2018-05-02 Voyager Therapeutics Inc Modulatory polynucleotides.
US10597660B2 (en) 2014-11-14 2020-03-24 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (ALS)
HK1245326A1 (en) 2014-12-12 2018-08-24 Voyager Therapeutics, Inc. Compositions and methods for the production of scaav
JP6929791B2 (en) 2015-02-09 2021-09-01 デューク ユニバーシティ Compositions and methods for epigenome editing
WO2017023803A1 (en) 2015-07-31 2017-02-09 Regents Of The University Of Minnesota Modified cells and methods of therapy
ES2865487T3 (en) 2015-09-28 2021-10-15 Univ North Carolina Chapel Hill Methods and compositions for viral vectors that evade antibodies
WO2017066497A2 (en) 2015-10-13 2017-04-20 Duke University Genome engineering with type i crispr systems in eukaryotic cells
WO2017095967A2 (en) 2015-11-30 2017-06-08 Duke University Therapeutic targets for the correction of the human dystrophin gene by gene editing and methods of use
WO2017096162A1 (en) 2015-12-02 2017-06-08 Voyager Therapeutics, Inc. Assays for the detection of aav neutralizing antibodies
US20190127713A1 (en) 2016-04-13 2019-05-02 Duke University Crispr/cas9-based repressors for silencing gene targets in vivo and methods of use
KR20240056729A (en) 2016-05-18 2024-04-30 보이저 테라퓨틱스, 인크. Modulatory polynucleotides
EP4275747A3 (en) 2016-07-19 2024-01-24 Duke University Therapeutic applications of cpf1-based genome editing
AU2018261790B2 (en) 2017-05-05 2024-10-03 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (ALS)
JOP20190269A1 (en) 2017-06-15 2019-11-20 Voyager Therapeutics Inc Aadc polynucleotides for the treatment of parkinson's disease
JP2020530307A (en) * 2017-06-30 2020-10-22 インティマ・バイオサイエンス,インコーポレーテッド Adeno-associated virus vector for gene therapy
EP3758724A4 (en) * 2018-02-27 2022-07-06 The Trustees of The University of Pennsylvania NOVEL ADENO-ASSOCIATED VIRUS (AAV) VECTORS, AAV VECTORS WITH REDUCED CAPSID DEAMIDATION AND USES THEREOF
US12416016B2 (en) * 2018-02-27 2025-09-16 The Trustees Of The University Of Pennsylvania Adeno-associated virus (AAV) vectors, AAV vectors having reduced capsid deamidation and uses therefor
KR102597383B1 (en) * 2018-03-20 2023-11-01 다이오 페이퍼 코퍼레이션 Tape type disposable diaper
JP2021519581A (en) 2018-04-03 2021-08-12 ストライドバイオ,インコーポレイテッド Viral vector to avoid antibodies
AU2019247191A1 (en) 2018-04-03 2020-10-15 Ginkgo Bioworks, Inc. Virus vectors for targeting ophthalmic tissues
SG11202009452WA (en) * 2018-04-03 2020-10-29 Stridebio Inc Antibody-evading virus vectors
US12460226B2 (en) 2018-04-16 2025-11-04 The Trustees Of The University Of Pennsylvania Compositions and methods for treating duchenne muscular dystrophy
TW202005978A (en) 2018-05-14 2020-02-01 美商拜奧馬林製藥公司 Novel liver targeting adeno-associated viral vectors
EP3794126A1 (en) 2018-05-15 2021-03-24 Voyager Therapeutics, Inc. Compositions and methods for the treatment of parkinson's disease
US20210292373A1 (en) * 2018-07-10 2021-09-23 University Of Florida Research Foundation, Incorporated Aav vp1u chimeras
AU2019346655B2 (en) 2018-09-28 2025-03-13 Voyager Therapeutics, Inc. Frataxin expression constructs having engineered promoters and methods of use thereof
WO2020072451A1 (en) 2018-10-01 2020-04-09 Ultragenyx Pharmaceutical Inc. Gene therapy for treating propionic acidemia
AU2019428629A1 (en) * 2019-02-06 2021-01-28 Sangamo Therapeutics, Inc. Method for the treatment of mucopolysaccharidosis type I
AR118465A1 (en) 2019-03-21 2021-10-06 Stridebio Inc RECOMBINANT ADENO-ASSOCIATED VIRUS VECTORS
EP3969061A1 (en) * 2019-05-14 2022-03-23 BioMarin Pharmaceutical Inc. Methods of redosing gene therapy vectors
US20220265853A1 (en) * 2019-07-12 2022-08-25 Gene Therapy Research Institution Co., Ltd. Adeno-associated virus virion for gene transfer to human liver
CN110423281B (en) * 2019-07-31 2021-04-30 成都金唯科生物科技有限公司 Fusion protein, viral vector and drug for treating age-related macular degeneration
WO2021030764A1 (en) * 2019-08-14 2021-02-18 University Of Florida Research Foundation, Incorporated Aav capsid variants for gene therapy
CA3157700A1 (en) 2019-10-17 2021-04-22 Stridebio, Inc. Adeno-associated viral vectors for treatment of niemann-pick disease type c
JP2023520402A (en) 2020-03-31 2023-05-17 ウルトラジェニックス ファーマシューティカル インコーポレイテッド Gene therapy to treat propionic acidemia
US20230348870A1 (en) * 2020-04-27 2023-11-02 Duke University Gene editing of satellite cells in vivo using aav vectors encoding muscle-specific promoters
US20230048732A1 (en) * 2020-05-26 2023-02-16 Shape Therapeutics Inc. High throughput engineering of functional aav capsids
EP4176064A1 (en) * 2020-07-03 2023-05-10 Genethon Method for engineering novel hybrid aav capsids through hypervariable regions swapping
CA3189878A1 (en) 2020-08-19 2022-02-24 Colin O'BANION Adeno-associated virus vectors for treatment of rett syndrome
BR112023015303A2 (en) 2021-02-01 2023-11-14 Regenxbio Inc METHOD TO TREAT CLN2 DISEASE DUE TO TPP1 DEFICIENCY IN A SUBJECT
GB202202842D0 (en) 2022-03-01 2022-04-13 Belgian Volition Srl Chimeric antigen receptor T-cell treatments targeted to chromatin fragments and extracellular traps
US20250108127A1 (en) * 2022-08-29 2025-04-03 Shanghai Ophthal-Bright Biomedicine Technology Modified vector, construction method, and application of modified aav-8 serotype for gene targeting and expression
WO2024143440A1 (en) * 2022-12-28 2024-07-04 富士フイルム株式会社 Method for producing adeno-associated virus, and vector set
CN118956906B (en) * 2024-10-17 2025-03-25 杭州嘉因生物科技有限公司 A virus packaging plasmid and its application

Family Cites Families (14)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6174666B1 (en) 1992-03-27 2001-01-16 The United States Of America As Represented By The Department Of Health And Human Services Method of eliminating inhibitory/instability regions from mRNA
US5478745A (en) 1992-12-04 1995-12-26 University Of Pittsburgh Recombinant viral vector system
ATE317916T1 (en) * 2001-11-13 2006-03-15 Univ Pennsylvania METHOD FOR IDENTIFYING ADENO-ASSOCIATED VIRUS (AAV) SEQUENCES AND KIT FOR IMPLEMENTING THE METHOD
EP1453547B1 (en) 2001-12-17 2016-09-21 The Trustees Of The University Of Pennsylvania Adeno-associated virus (aav) serotype 8 sequences, vectors containing same, and uses therefor
EP1486567A1 (en) 2003-06-11 2004-12-15 Deutsches Krebsforschungszentrum Stiftung des öffentlichen Rechts Improved adeno-associated virus (AAV) vector for gene therapy
ES2874298T3 (en) * 2003-09-30 2021-11-04 Univ Pennsylvania Adeno-associated virus (AAV) clades, sequences, vectors containing the same, and uses thereof
ES2428218T3 (en) * 2005-04-07 2013-11-06 The Trustees Of The University Of Pennsylvania AAV rh48 modified capsules, compositions containing them and uses thereof
US20090191597A1 (en) 2006-01-20 2009-07-30 Asklepios Biopharmaceutical, Inc. Enhanced production of infectious parvovirus vectors in insect cells
EP2287323A1 (en) 2009-07-31 2011-02-23 Association Institut de Myologie Widespread gene delivery to the retina using systemic administration of AAV vectors
WO2011038187A1 (en) 2009-09-25 2011-03-31 The Trustees Of The University Of Pennsylvania Controlled adeno-associated virus (aav) diversification and libraries prepared therefrom
US20130023033A1 (en) 2010-03-29 2013-01-24 The Trustees Of The University Of Pennsylvania Pharmacologically induced transgene ablation system
WO2013155222A2 (en) 2012-04-10 2013-10-17 The Regents Of The University Of California Brain-specific enhancers for cell-based therapy
JP6199965B2 (en) 2012-07-11 2017-09-20 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 AAV-mediated gene therapy for RPGRX-linked retinal degeneration
WO2014124282A1 (en) * 2013-02-08 2014-08-14 The Trustees Of The University Of Pennsylvania Enhanced aav-mediated gene transfer for retinal therapies

Also Published As

Publication number Publication date
SG11201808812RA (en) 2018-11-29
US20210340569A1 (en) 2021-11-04
AU2023204146A1 (en) 2023-09-07
AU2017248656B2 (en) 2023-07-27
CN109661470A (en) 2019-04-19
IL262214A (en) 2018-11-29
AU2017248656A1 (en) 2018-10-18
CA3019423A1 (en) 2017-10-19
JP7140683B2 (en) 2022-09-21
JP2019513401A (en) 2019-05-30
US20190078119A1 (en) 2019-03-14
WO2017180854A1 (en) 2017-10-19
IL298604A (en) 2023-01-01
US11091776B2 (en) 2021-08-17
IL262214B1 (en) 2023-01-01
EP3443108A1 (en) 2019-02-20
EP3443108A4 (en) 2019-11-20
IL262214B2 (en) 2023-05-01
JP2022116275A (en) 2022-08-09

Similar Documents

Publication Publication Date Title
IL262214A (en) Novel aav8 mutant capsids and compositions containing same
IL268970A (en) Novel compositions and methods
EP3528785A4 (en) Modified aav capsids and uses thereof
ZA201807668B (en) Compositions and methods for dust control and the manufacture of construction materials
EP3589277A4 (en) Modified aav capsids and uses thereof
IL264446A (en) Compounds and compositions and uses thereof
ZA201803451B (en) Probiotic compositions and uses thereof
PL3122448T3 (en) Dispersions containing encapsulated materials and compositions using same
GB2556619B (en) Suspension compositions
ZA202104969B (en) Compositions comprising 15-hepe and methods of using the same
ZA201807267B (en) Cosmetic material compositions
PL4245896T3 (en) New polyester and compositions containing it
GB202201861D0 (en) Novel methods and compositions
SG11201710212UA (en) Uv-blocking cosmetic composition
PL3576846T3 (en) Sunscreen composition
EP3193637A4 (en) Improved drink stabilizer composition and stabilized drink compositions
PL3463259T3 (en) Sunscreen compositions
GB201608762D0 (en) Compositions and uses thereof
GB201717826D0 (en) Sunscreen compositions
ZA201802565B (en) New adjuvant and vaccine composition containing the same
PL3568203T3 (en) Compounds and compositions
GB201603591D0 (en) Improved promotors and compositions
GB2570376B (en) Suspension compositions
IL269723A (en) Mutant alpha-1-antitrypsin compositions and use thereof
GB201602409D0 (en) Compositions and uses thereof