SG11202008182TA - Novel adeno-associated virus (aav) vectors, aav vectors having reduced capsid deamidation and uses therefor - Google Patents

Novel adeno-associated virus (aav) vectors, aav vectors having reduced capsid deamidation and uses therefor

Info

Publication number
SG11202008182TA
SG11202008182TA SG11202008182TA SG11202008182TA SG11202008182TA SG 11202008182T A SG11202008182T A SG 11202008182TA SG 11202008182T A SG11202008182T A SG 11202008182TA SG 11202008182T A SG11202008182T A SG 11202008182TA SG 11202008182T A SG11202008182T A SG 11202008182TA
Authority
SG
Singapore
Prior art keywords
aav
vectors
associated virus
uses therefor
novel adeno
Prior art date
Application number
SG11202008182TA
Inventor
James Wilson
April Tepe
Kevin Turner
Joshua Sims
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 SG11202008182TA publication Critical patent/SG11202008182TA/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
    • C12N7/00Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • 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
    • C12N2750/14121Viruses as such, e.g. new isolates, mutants or their genomic sequences
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Virology (AREA)
  • Biophysics (AREA)
  • Molecular Biology (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Medicinal Chemistry (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Immunology (AREA)
  • Peptides Or Proteins (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
SG11202008182TA 2018-02-27 2019-02-27 Novel adeno-associated virus (aav) vectors, aav vectors having reduced capsid deamidation and uses therefor SG11202008182TA (en)

Applications Claiming Priority (6)

Application Number Priority Date Filing Date Title
US201862635964P 2018-02-27 2018-02-27
US201862667585P 2018-05-29 2018-05-29
US201862677471P 2018-05-29 2018-05-29
US201862703670P 2018-07-26 2018-07-26
US201862722382P 2018-08-24 2018-08-24
PCT/US2019/019804 WO2019168961A1 (en) 2018-02-27 2019-02-27 Novel adeno-associated virus (aav) vectors, aav vectors having reduced capsid deamidation and uses therefor

Publications (1)

Publication Number Publication Date
SG11202008182TA true SG11202008182TA (en) 2020-09-29

Family

ID=67805569

Family Applications (1)

Application Number Title Priority Date Filing Date
SG11202008182TA SG11202008182TA (en) 2018-02-27 2019-02-27 Novel adeno-associated virus (aav) vectors, aav vectors having reduced capsid deamidation and uses therefor

Country Status (13)

Country Link
US (1) US20200407750A1 (en)
EP (1) EP3758724A4 (en)
JP (3) JP2021516046A (en)
KR (1) KR20210010434A (en)
CN (1) CN112352050A (en)
AU (1) AU2019227726A1 (en)
BR (1) BR112020017348A2 (en)
CA (1) CA3091806A1 (en)
CL (2) CL2020002200A1 (en)
IL (1) IL276859A (en)
MX (1) MX2020008933A (en)
SG (1) SG11202008182TA (en)
WO (1) WO2019168961A1 (en)

Families Citing this family (31)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2017058892A2 (en) 2015-09-28 2017-04-06 The University Of North Carolina At Chapel Hill Methods and compositions for antibody-evading virus vectors
BR112020020266A2 (en) 2018-04-03 2021-01-19 Stridebio, Inc. VIRUSES WITH ANTIBODY EVASION
EP3773743A1 (en) 2018-04-03 2021-02-17 Stridebio, Inc. Virus vectors for targeting ophthalmic tissues
MX2020010466A (en) 2018-04-03 2021-01-08 Antibody-evading virus vectors.
KR20220011616A (en) 2019-03-21 2022-01-28 스트라이드바이오 인코포레이티드 Recombinant adeno-associated viral vectors
JP2022550435A (en) 2019-10-04 2022-12-01 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 Methods for improved therapeutic use of recombinant AAV
AU2020367532A1 (en) 2019-10-17 2022-05-12 Ginkgo Bioworks, Inc. Adeno-associated viral vectors for treatment of Niemann-Pick disease type C
JP2022552892A (en) * 2019-10-21 2022-12-20 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 AAV3B mutants with improved production yields and liver tropism
CA3222852A1 (en) * 2020-01-03 2021-07-08 Sarepta Therapeutics, Inc. Methods for analyzing aav capsid proteins
CN115151648A (en) * 2020-02-14 2022-10-04 阿尔特拉吉尼克斯制药公司 Gene therapy for the treatment of CDKL5 deficient disorders
US20230129893A1 (en) 2020-03-31 2023-04-27 Ultragenyx Pharmaceutical Inc. Gene therapy for treating propionic acidemia
JP2023531451A (en) 2020-06-17 2023-07-24 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 Compositions and methods for treatment of gene therapy patients
MX2023000658A (en) 2020-07-13 2023-02-23 Univ Pennsylvania Compositions useful for treatment of charcot-marie-tooth disease.
KR20230068444A (en) 2020-08-19 2023-05-17 사렙타 쎄러퓨틱스 인코퍼레이티드 Adeno-associated viral vectors for the treatment of Rett syndrome
EP4229186A1 (en) * 2020-10-18 2023-08-23 The Trustees of The University of Pennsylvania Improved adeno-associated virus (aav) vector and uses therefor
AU2021369793A1 (en) * 2020-10-29 2023-06-08 The Trustees Of The University Of Pennsylvania Aav capsids and compositions containing same
TW202237850A (en) 2020-12-01 2022-10-01 賓州大學委員會 Novel compositions with tissue-specific targeting motifs and compositions containing same
AU2022214429A1 (en) 2021-02-01 2023-09-14 Regenxbio Inc. Gene therapy for neuronal ceroid lipofuscinoses
WO2022221276A1 (en) 2021-04-12 2022-10-20 The Trustees Of The University Of Pennsylvania Compositions useful for treating spinal and bulbar muscular atrophy (sbma)
US20240207452A1 (en) 2021-04-23 2024-06-27 The Trustees Of The University Of Pennsylvania Novel compositions with brain-specific targeting motifs and compositions containing same
IL313832A (en) * 2021-12-28 2024-08-01 Chengdu Origen Biotechnology Co Ltd Modified aav capsid protein and use thereof
TW202338086A (en) 2022-01-10 2023-10-01 賓州大學委員會 Compositions useful in treatment of metachromatic leukodystrophy
AR128239A1 (en) 2022-01-10 2024-04-10 Univ Pennsylvania COMPOSITIONS AND USEFUL METHODS FOR THE TREATMENT OF DISORDERS MEDIATED BY C9ORF72
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
TW202342740A (en) 2022-03-07 2023-11-01 美商奧崔基尼克斯製藥公司 Modified batch aav production systems and methods
WO2023196892A1 (en) 2022-04-06 2023-10-12 The Trustees Of The University Of Pennsylvania Passive immunization with anti- aav neutralizing antibodies to prevent off-target transduction of intrathecally delivered aav vectors
CN115011601B (en) * 2022-06-27 2023-07-21 山东大学齐鲁医院 shRNA (short hairpin ribonucleic acid) interfering with JUND expression, recombinant adeno-associated virus vector and application thereof
WO2024015966A2 (en) * 2022-07-15 2024-01-18 The Trustees Of The University Of Pennsylvania Recombinant aav having aav clade d and clade e capsids and compositions containing same
WO2024130070A2 (en) 2022-12-17 2024-06-20 The Trustees Of The University Of Pennsylvania Recombinant aav capsids with cardiac- and skeletal muscle- specific targeting motifs and uses thereof
WO2024130067A2 (en) 2022-12-17 2024-06-20 The Trustees Of The University Of Pennsylvania Recombinant aav mutant vectors with cardiac and skeletal muscle-specific targeting motifs and compositions containing same
WO2024138129A2 (en) * 2022-12-23 2024-06-27 Spark Therapeutics, Inc. Adeno-associated virus formulations

Family Cites Families (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
ES2648241T3 (en) * 2003-09-30 2017-12-29 The Trustees Of The University Of Pennsylvania Adeno-associated virus clades (AAV), sequences, vectors containing the same, and uses thereof
US8999678B2 (en) * 2005-04-07 2015-04-07 The Trustees Of The University Of Pennsylvania Method of increasing the function of an AAV vector
MY172457A (en) * 2012-04-18 2019-11-26 Childrens Hospital Philadelphia Composition and methods for highly efficient gene transfer using aav capsid variants
EP3024498B1 (en) * 2013-07-22 2019-12-04 The Children's Hospital of Philadelphia Variant aav and compositions, methods and uses for gene transfer to cells, organs and tissues
US11015173B2 (en) * 2015-12-11 2021-05-25 The Trustees Of The University Of Pennsylvania Scalable purification method for AAV1
WO2017180854A1 (en) * 2016-04-15 2017-10-19 The Trustees Of The University Of Pennsylvania Novel aav8 mutant capsids and compositions containing same
CN118688363A (en) * 2016-08-15 2024-09-24 建新公司 Methods of detecting AAV
LT3589730T (en) * 2017-02-28 2024-03-12 The Trustees Of The University Of Pennsylvania Adeno-associated virus (aav) clade f vector and uses therefor

Also Published As

Publication number Publication date
IL276859A (en) 2020-10-29
MX2020008933A (en) 2021-01-15
CL2020002200A1 (en) 2021-01-29
CL2022003757A1 (en) 2023-05-19
EP3758724A4 (en) 2022-07-06
JP2023159235A (en) 2023-10-31
JP2024041967A (en) 2024-03-27
AU2019227726A1 (en) 2020-09-10
JP2021516046A (en) 2021-07-01
BR112020017348A2 (en) 2020-12-29
WO2019168961A1 (en) 2019-09-06
US20200407750A1 (en) 2020-12-31
CN112352050A (en) 2021-02-09
KR20210010434A (en) 2021-01-27
EP3758724A1 (en) 2021-01-06
CA3091806A1 (en) 2019-09-06

Similar Documents

Publication Publication Date Title
IL276859A (en) Novel adeno-associated virus (aav) vectors, aav vectors having reduced capsid deamidation and uses therefor
IL276860A (en) Novel adeno-associated virus (aav) vectors, aav vectors having reduced capsid deamidation and uses therefor
IL286464A (en) Recombinant adeno-associated virus vectors
IL286911A (en) Recombinant adeno-associated viruses and uses thereof
ZA201905407B (en) Adeno-associated virus (aav) clade f vector and uses therefor
IL282885A (en) Aav viral vectors and uses thereof
IL263801A (en) Novel adeno-associated virus capsid proteins
ZA201801657B (en) Adeno-associated virus factor viii vectors, associated viral particles and therapeutic formulations comprising the same
IL286831A (en) Adeno-associated virus with engineered capsid
EP3575398A4 (en) Mutant of adeno-associated virus (aav) capsid protein
EP3943503A4 (en) Adeno-associated virus having variant capsid protein and use thereof
EP4061427A4 (en) Adeno-associated viral vector variants
IL287214A (en) Adeno-associated virus vector formulations and methods
IL286725A (en) Engineered adeno-associated (aav) vectors for transgene expression
EP3790979A4 (en) Adeno-associated virus (aav) delivery of anti-fam19a5 antibodies
EP4110792A4 (en) Adeno-associated virus capsid polypeptides and vectors
IL287614A (en) Modified adeno-associated virus (aav) particles for gene therapy
SG11201704919SA (en) Methods of purifying adeno-associated virus (aav) and/or recombinant adeno-associated virus (raav)
IL304688A (en) Adeno-associated virus (aav) production
SG11202006169PA (en) Adeno-associated virus (aav) vector having hybrid hgf gene introduced thereto
EP3810635A4 (en) Aav polynucleotides, polypeptides and virions
AU2019902377A0 (en) AAV capsid polypeptides and AAV vectors
EP3751000A4 (en) Improved adeno-associated virus vector
AU2020904687A0 (en) Adeno-associated virus capsids and vectors
AU2020900529A0 (en) Adeno-associated virus capsid polypeptides and vectors