KR102082032B1 - 아데노-관련 바이러스 벡터 - Google Patents

아데노-관련 바이러스 벡터 Download PDF

Info

Publication number
KR102082032B1
KR102082032B1 KR1020167022254A KR20167022254A KR102082032B1 KR 102082032 B1 KR102082032 B1 KR 102082032B1 KR 1020167022254 A KR1020167022254 A KR 1020167022254A KR 20167022254 A KR20167022254 A KR 20167022254A KR 102082032 B1 KR102082032 B1 KR 102082032B1
Authority
KR
South Korea
Prior art keywords
gly
pro
asn
ser
leu
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
Application number
KR1020167022254A
Other languages
English (en)
Korean (ko)
Other versions
KR20160130996A (ko
Inventor
랄프 마이클 린덴
Original Assignee
킹즈 컬리지 런던
이칸 스쿨 오브 메디슨 엣 마운트 시나이
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
Family has litigation
First worldwide family litigation filed litigation Critical https://patents.darts-ip.com/?family=50490674&utm_source=google_patent&utm_medium=platform_link&utm_campaign=public_patent_search&patent=KR102082032(B1) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Application filed by 킹즈 컬리지 런던, 이칸 스쿨 오브 메디슨 엣 마운트 시나이 filed Critical 킹즈 컬리지 런던
Publication of KR20160130996A publication Critical patent/KR20160130996A/ko
Application granted granted Critical
Publication of KR102082032B1 publication Critical patent/KR102082032B1/ko
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Images

Classifications

    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/02—Ophthalmic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/02—Ophthalmic agents
    • A61P27/06—Antiglaucoma agents or miotics
    • 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
    • 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
    • C12N15/861—Adenoviral vectors
    • 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
    • C12N2750/14132—Use of virus as therapeutic agent, other than vaccine, e.g. as cytolytic agent
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Biomedical Technology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Ophthalmology & Optometry (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Virology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Epidemiology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Peptides Or Proteins (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
KR1020167022254A 2014-02-17 2015-02-17 아데노-관련 바이러스 벡터 Active KR102082032B1 (ko)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201461940639P 2014-02-17 2014-02-17
US61/940,639 2014-02-17
GB1403684.2 2014-03-03
GBGB1403684.2A GB201403684D0 (en) 2014-03-03 2014-03-03 Vector
PCT/EP2015/053335 WO2015121501A1 (en) 2014-02-17 2015-02-17 Adeno-associated virus vector

Publications (2)

Publication Number Publication Date
KR20160130996A KR20160130996A (ko) 2016-11-15
KR102082032B1 true KR102082032B1 (ko) 2020-02-26

Family

ID=50490674

Family Applications (1)

Application Number Title Priority Date Filing Date
KR1020167022254A Active KR102082032B1 (ko) 2014-02-17 2015-02-17 아데노-관련 바이러스 벡터

Country Status (20)

Country Link
US (4) US20180135076A1 (enExample)
EP (2) EP3108000B1 (enExample)
JP (4) JP6602788B2 (enExample)
KR (1) KR102082032B1 (enExample)
CN (1) CN106062200B (enExample)
AU (3) AU2015216847B2 (enExample)
BR (1) BR112016018598A2 (enExample)
CA (2) CA3126061C (enExample)
DK (1) DK3108000T3 (enExample)
ES (1) ES2749235T3 (enExample)
GB (1) GB201403684D0 (enExample)
HU (1) HUE045801T2 (enExample)
IL (2) IL247332B (enExample)
MX (1) MX372992B (enExample)
PL (1) PL3108000T3 (enExample)
PT (1) PT3108000T (enExample)
RU (1) RU2743382C2 (enExample)
SG (1) SG11201606365TA (enExample)
SI (1) SI3108000T1 (enExample)
WO (1) WO2015121501A1 (enExample)

Families Citing this family (196)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US9217155B2 (en) 2008-05-28 2015-12-22 University Of Massachusetts Isolation of novel AAV'S and uses thereof
WO2010138263A2 (en) 2009-05-28 2010-12-02 University Of Massachusetts Novel aav 's and uses thereof
WO2011133874A1 (en) 2010-04-23 2011-10-27 University Of Massachusetts Multicistronic expression constructs
DK2826860T3 (en) 2010-04-23 2018-12-03 Univ Massachusetts CNS targeting AAV vectors and methods for their use
WO2014170480A1 (en) 2013-04-18 2014-10-23 Fondazione Telethon Effective delivery of large genes by dual aav vectors
CN105247044B (zh) 2013-05-31 2021-05-07 加利福尼亚大学董事会 腺相关病毒变体及其使用方法
EP3633041A3 (en) 2013-09-26 2020-07-29 University of Florida Research Foundation, Inc. Synthetic combinatorial aav capsid library for targeted gene therapy
GB201403684D0 (en) 2014-03-03 2014-04-16 King S College London Vector
WO2015127128A2 (en) 2014-02-19 2015-08-27 University Of Massachusetts Recombinant aavs having useful transcytosis properties
EP4410805A3 (en) 2014-03-18 2024-11-27 University of Massachusetts Raav-based compositions and methods for treating amyotrophic lateral sclerosis
WO2015164786A1 (en) 2014-04-25 2015-10-29 University Of Massachusetts Recombinant aav vectors useful for reducing immunity against transgene products
AU2015252797C1 (en) * 2014-05-02 2021-04-22 Genzyme Corporation AAV vectors for retinal and CNS gene therapy
WO2015187825A2 (en) 2014-06-03 2015-12-10 University Of Massachusetts Compositions and methods for modulating dysferlin expression
US10577627B2 (en) 2014-06-09 2020-03-03 Voyager Therapeutics, Inc. Chimeric capsids
WO2016054554A1 (en) 2014-10-03 2016-04-07 University Of Massachusetts Heterologous targeting peptide grafted aavs
JP6842410B2 (ja) 2014-10-03 2021-03-17 ユニバーシティ オブ マサチューセッツ 新規の高効率ライブラリーにより同定されるａａｖベクター
EP3209311B1 (en) 2014-10-21 2024-03-06 University of Massachusetts Recombinant aav variants and uses thereof
SG11201703148TA (en) 2014-11-05 2017-05-30 Voyager Therapeutics Inc Aadc polynucleotides for the treatment of parkinson's disease
US10597660B2 (en) 2014-11-14 2020-03-24 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (ALS)
MX2017006217A (es) 2014-11-14 2018-05-02 Voyager Therapeutics Inc Polinucleotidos moduladores.
EP3224362B1 (en) * 2014-11-26 2024-09-25 The Regents of The University of California Therapeutic compositions comprising transcription factors and methods of making and using the same
HK1245326A1 (zh) 2014-12-12 2018-08-24 Voyager Therapeutics, Inc. 用於生产scaav的组合物和方法
US10584321B2 (en) 2015-02-13 2020-03-10 University Of Massachusetts Compositions and methods for transient delivery of nucleases
EP3285780A4 (en) 2015-04-24 2018-12-19 University of Massachusetts Modified aav constructions and uses thereof
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
US11253576B2 (en) 2015-10-22 2022-02-22 University Of Massachusetts Methods and compositions for treating metabolic imbalance in neurodegenerative disease
EP3364996B1 (en) 2015-10-22 2021-08-25 University of Massachusetts Prostate-targeting adeno-associated virus serotype vectors
US12188037B2 (en) 2015-10-22 2025-01-07 University Of Florida Research Foundation, Incorporated Synthetic combinatorial AAV3 capsid library
GB201519086D0 (en) * 2015-10-28 2015-12-09 Syncona Partners Llp Gene Therapy
WO2017075335A1 (en) 2015-10-28 2017-05-04 Voyager Therapeutics, Inc. Regulatable expression using adeno-associated virus (aav)
PE20231949A1 (es) * 2015-10-30 2023-12-05 Spark Therapeutics Inc VARIANTES DEL FACTOR VIII REDUCIDO CON CpG, COMPOSICIONES Y METODOS Y USOS PARA EL TRATAMIENTO DE TRASTORNOS DE LA HEMOSTASIA
KR20210005994A (ko) 2015-11-05 2021-01-15 뱀부 테라퓨틱스 인코포레이티드 유전자 치료를 위한 변형된 프리드라이히 운동실조증 유전자 및 벡터
WO2017096162A1 (en) 2015-12-02 2017-06-08 Voyager Therapeutics, Inc. Assays for the detection of aav neutralizing antibodies
ES2869284T3 (es) * 2015-12-11 2021-10-25 California Inst Of Techn Péptidos de diana para dirigir virus adenoasociados (AAV)
EP3394270A1 (en) * 2015-12-14 2018-10-31 The Trustees Of The University Of Pennsylvania Adeno-associated viral vectors useful in treatment of spinal muscular atropy
WO2017136536A1 (en) 2016-02-02 2017-08-10 University Of Massachusetts Method to enhance the efficiency of systemic aav gene delivery to the central nervous system
EP4094780A3 (en) 2016-02-12 2023-02-08 University of Massachusetts Anti-angiogenic mirna therapeutics for inhibiting corneal neovascularization
US20190071681A1 (en) * 2016-02-26 2019-03-07 University Of Florida Research Foundation, Incorporated Aav heparin mutants that display significantly improved eye and brain transduction
US11207426B2 (en) 2016-04-05 2021-12-28 University Of Massachusetts Compositions and methods for selective inhibition of grainyhead-like protein expression
CN107287238B (zh) * 2016-04-11 2020-10-16 厦门继景生物技术有限责任公司 一种基因载体及其用于治疗雷柏氏先天性黑矇2型疾病的基因治疗药物
KR102574810B1 (ko) 2016-04-15 2023-09-08 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 습성 연령 관련 황반 변성의 치료를 위한 조성물
WO2017181105A1 (en) 2016-04-15 2017-10-19 University Of Massachusetts Methods and compositions for treating metabolic imbalance
US11326182B2 (en) 2016-04-29 2022-05-10 Voyager Therapeutics, Inc. Compositions for the treatment of disease
EP3448987A4 (en) 2016-04-29 2020-05-27 Voyager Therapeutics, Inc. COMPOSITIONS FOR TREATING A DISEASE
KR20240056729A (ko) 2016-05-18 2024-04-30 보이저 테라퓨틱스, 인크. 조절성 폴리뉴클레오티드
MX2018014152A (es) 2016-05-18 2019-03-28 Voyager Therapeutics Inc Composiciones y métodos para tratar la enfermedad de huntington.
US11882815B2 (en) 2016-06-15 2024-01-30 University Of Massachusetts Recombinant adeno-associated viruses for delivering gene editing molecules to embryonic cells
MA44546B1 (fr) 2016-06-15 2021-03-31 Univ California Virus adéno-associés variants et procédés d'utilisation
CA2971303A1 (en) 2016-06-21 2017-12-21 Bamboo Therapeutics, Inc. Optimized mini-dystrophin genes and expression cassettes and their use
WO2018002812A1 (en) 2016-06-29 2018-01-04 Crispr Therapeutics Ag Materials and methods for treatment of myotonic dystrophy type 1 (dm1) and other related disorders
EP3478313B1 (en) 2016-06-29 2022-05-04 CRISPR Therapeutics AG Materials and methods for treatment of amyotrophic lateral sclerosis (als) and other related disorders
US11564997B2 (en) 2016-06-29 2023-01-31 Crispr Therapeutics Ag Materials and methods for treatment of friedreich ataxia and other related disorders
CA3029132A1 (en) 2016-07-06 2018-01-11 Crispr Therapeutics Ag Materials and methods for treatment of pain related disorders
US11459587B2 (en) 2016-07-06 2022-10-04 Vertex Pharmaceuticals Incorporated Materials and methods for treatment of pain related disorders
EP3485005A1 (en) 2016-07-12 2019-05-22 The University Of Manchester Gene therapy
KR20230039779A (ko) 2016-07-29 2023-03-21 더 리젠츠 오브 더 유니버시티 오브 캘리포니아 변이체 캡시드를 갖는 아데노-관련된 바이러스 비리온 및 이의 사용 방법
US20190262373A1 (en) * 2016-08-16 2019-08-29 The University Of North Carolina At Chapel Hill Methods and compositions for targeted gene transfer
US11298041B2 (en) 2016-08-30 2022-04-12 The Regents Of The University Of California Methods for biomedical targeting and delivery and devices and systems for practicing the same
US10457940B2 (en) 2016-09-22 2019-10-29 University Of Massachusetts AAV treatment of Huntington's disease
US11078238B2 (en) 2016-09-29 2021-08-03 University Of Florida Research Foundation, Incorporated AAVrh.10 variants with host antibody escape capabilities and altered tissue targeting properties
AU2017341849B2 (en) * 2016-10-13 2024-03-21 University Of Massachusetts AAV capsid designs
CA3040179A1 (en) 2016-10-19 2018-04-26 Adverum Biotechnologies, Inc. Modified aav capsids and uses thereof
PL3534922T3 (pl) 2016-11-04 2024-08-19 Takeda Pharmaceutical Company Limited Preparaty wirusa związanego z adenowirusem
CA3114549A1 (en) 2017-02-21 2018-08-30 University Of Florida Research Foundation, Incorporated Modified aav capsid proteins and uses thereof
US20200216857A1 (en) 2017-02-22 2020-07-09 Crispr Therapeutics Ag Materials and methods for treatment of spinocerebellar ataxia type 2 (sca2) and other spinocerebellar ataxia type 2 protein (atxn2) gene related conditions or disorders
EP3585807A1 (en) 2017-02-22 2020-01-01 CRISPR Therapeutics AG Materials and methods for treatment of early onset parkinson's disease (park1) and other synuclein, alpha (snca) gene related conditions or disorders
US11407997B2 (en) 2017-02-22 2022-08-09 Crispr Therapeutics Ag Materials and methods for treatment of primary hyperoxaluria type 1 (PH1) and other alanine-glyoxylate aminotransferase (AGXT) gene related conditions or disorders
CN110662838B (zh) 2017-02-22 2024-05-28 克里斯珀医疗股份公司 用于基因编辑的组合物和方法
EP3585898A1 (en) 2017-02-22 2020-01-01 CRISPR Therapeutics AG Materials and methods for treatment of spinocerebellar ataxia type 1 (sca1) and other spinocerebellar ataxia type 1 protein (atxn1) gene related conditions or disorders
UY37679A (es) 2017-04-14 2018-11-30 Regenxbio Inc Tratamiento de mucopolisacaridosis ii con iduronato-2-sulfatasa (ids) humana recombinante producida por células neurales o gliales humanas
AU2018261003A1 (en) 2017-05-05 2019-11-14 Voyager Therapeutics, Inc. Compositions and methods of treating Huntington's Disease
AU2018261790B2 (en) 2017-05-05 2024-10-03 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (ALS)
EP3622073A4 (en) 2017-05-09 2021-01-06 University of Massachusetts Methods of treating amyotrophic lateral sclerosis (als)
CN110831611A (zh) * 2017-05-10 2020-02-21 马萨诸塞眼科耳科诊所 用于修饰病毒的组装活化蛋白(aap)依赖性的方法和组合物
AU2018265414B2 (en) 2017-05-10 2022-01-20 Massachusetts Eye And Ear Infirmary Methods and compositions for modifying assembly-activating protein (APP)-dependence of viruses
JOP20190269A1 (ar) 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
BR112019019015A2 (pt) 2017-06-30 2020-04-14 Univ California vírions de vírus adeno-associado com capsídeos variantes e seus métodos de uso
JP7229989B2 (ja) 2017-07-17 2023-02-28 ボイジャー セラピューティクス インコーポレイテッド 軌道アレイガイドシステム
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
JP2020533968A (ja) * 2017-08-25 2020-11-26 オービッド・セラピューティクス・インコーポレイテッドＯｖｉｄ Ｔｈｅｒａｐｅｕｔｉｃｓ Ｉｎｃ． 組換えアデノ随伴ベクター
KR20200039617A (ko) 2017-08-28 2020-04-16 더 리젠츠 오브 더 유니버시티 오브 캘리포니아 아데노-연관 바이러스 캡시드 변이체 및 이의 사용 방법
WO2019060686A1 (en) 2017-09-22 2019-03-28 University Of Massachusetts NEW DUAL EXPRESSION VECTORS OF SOD1 AND USES THEREOF
CA3075656A1 (en) 2017-09-29 2019-04-04 Voyager Therapeutics, Inc. Rescue of central and peripheral neurological phenotype of friedreich's ataxia by intravenous delivery
WO2019079240A1 (en) 2017-10-16 2019-04-25 Voyager Therapeutics, Inc. TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS)
WO2019079242A1 (en) 2017-10-16 2019-04-25 Voyager Therapeutics, Inc. TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS)
CA3079568A1 (en) * 2017-10-18 2019-04-25 Regenxbio Inc. Fully-human post-translationally modified antibody therapeutics
CA3084633A1 (en) 2017-12-21 2019-06-27 Crispr Therapeutics Ag Materials and methods for treatment of usher syndrome type 2a and/or non-syndromic autosomal recessive retinitis pigmentosa (arrp)
AU2018393050A1 (en) 2017-12-21 2020-06-18 Bayer Healthcare Llc Materials and methods for treatment of Usher Syndrome Type 2A
BR112020018354A2 (pt) * 2018-03-16 2020-12-29 Research Institute At Nationwide Children's Hospital Aumento de fornecimento gênico específico a tecido através de modificação de capsídeo
CN112218880A (zh) * 2018-03-29 2021-01-12 阿斯克肋匹奥生物制药公司 逃避中和的肝趋向性重组aav6载体
AU2019247191A1 (en) 2018-04-03 2020-10-15 Ginkgo Bioworks, Inc. Virus vectors for targeting ophthalmic tissues
JP2021519581A (ja) 2018-04-03 2021-08-12 ストライドバイオ，インコーポレイテッド 抗体を回避するウイルスベクター
SG11202009452WA (en) 2018-04-03 2020-10-29 Stridebio Inc Antibody-evading virus vectors
CN108660159B (zh) * 2018-04-12 2020-10-30 四川大学 重组蝙蝠腺相关病毒载体及其用途
KR102051393B1 (ko) 2018-05-04 2019-12-04 (주)지뉴인텍 아데노부속바이러스 기반 유전자 전달용 재조합 벡터 및 이를 이용한 암질환 예방 또는 치료용 조성물
US20210230632A1 (en) 2018-05-15 2021-07-29 Voyager Therapeutics, Inc. Compositions and methods for delivery of aav
EP3794126A1 (en) 2018-05-15 2021-03-24 Voyager Therapeutics, Inc. Compositions and methods for the treatment of parkinson's disease
JP2021522840A (ja) * 2018-05-15 2021-09-02 プレジデント アンド フェローズ オブ ハーバード カレッジ 改善された遺伝子送達特性を示すウイルスベクター
WO2019236995A1 (en) 2018-06-08 2019-12-12 University Of Massachusetts Antisense oligonucleotides to restore dysferlin protein expression in dysferlinopathy patient cells
BR112021001878A2 (pt) * 2018-07-31 2021-04-27 Cornell University métodos de terapia genética para o controle da função do órgão
AU2019346655B2 (en) 2018-09-28 2025-03-13 Voyager Therapeutics, Inc. Frataxin expression constructs having engineered promoters and methods of use thereof
CN119684417A (zh) 2018-10-02 2025-03-25 沃雅戈治疗公司 重新定向aav衣壳的嗜性
WO2020077165A1 (en) 2018-10-12 2020-04-16 Voyager Therapeutics, Inc. Compositions and methods for delivery of aav
UY38407A (es) 2018-10-15 2020-05-29 Novartis Ag Anticuerpos estabilizadores de trem2
EP3880823A4 (en) * 2018-11-16 2022-08-17 Asklepios Biopharmaceutical, Inc. Therapeutic adeno-associated virus for treating pompe disease
GB201900702D0 (en) * 2019-01-18 2019-03-06 Univ Bristol Therapy
CN113631225A (zh) 2019-01-18 2021-11-09 沃雅戈治疗公司 用于生产aav颗粒的方法和系统
AU2020223149B2 (en) * 2019-02-15 2025-05-22 Sangamo Therapeutics, Inc. Compositions and methods for producing recombinant AAV
AR118465A1 (es) 2019-03-21 2021-10-06 Stridebio Inc Vectores de virus adenoasociados recombinantes
JP7455138B2 (ja) * 2019-03-28 2024-03-25 ペキン シャオミ モバイル ソフトウェア カンパニー， リミテッド コアページング処理
WO2020205889A1 (en) 2019-04-01 2020-10-08 Tenaya Therapeutics, Inc. Adeno-associated virus with engineered capsid
CN114127088B (zh) * 2019-04-23 2024-05-14 国家医疗保健研究所 变体衣壳蛋白和腺相关病毒aav变体及其药物组合物
US12421526B2 (en) 2019-04-24 2025-09-23 Takara Bio Inc. AAV mutant having brain-targeting property
US20220194992A1 (en) * 2019-04-26 2022-06-23 The University Of North Carolina At Chapel Hill Methods and compositions for dual glycan binding aav2.5 vector
WO2020227515A1 (en) 2019-05-07 2020-11-12 Voyager Therapeutics, Inc. Compositions and methods for the vectored augmentation of protein destruction, expression and/or regulation
US20230241248A1 (en) 2019-06-27 2023-08-03 Pfizer Inc. Methods of treating duchenne muscular dystrophy using aav mini-dystrophin gene therapy
EP3990636A1 (en) 2019-06-28 2022-05-04 CRISPR Therapeutics AG Materials and methods for controlling gene editing
EP3999120A4 (en) * 2019-07-04 2024-02-21 Children's Medical Research Institute Methods and aav vectors for in vivo transduction
US20220265853A1 (en) * 2019-07-12 2022-08-25 Gene Therapy Research Institution Co., Ltd. Adeno-associated virus virion for gene transfer to human liver
SI3999119T1 (sl) * 2019-07-15 2025-03-31 Meiragtx Uk Ii Limited Modificirani aav kapsidni proteini za zdravljenje artritičnih bolezni
WO2021030764A1 (en) * 2019-08-14 2021-02-18 University Of Florida Research Foundation, Incorporated Aav capsid variants for gene therapy
US20220380413A1 (en) * 2019-08-29 2022-12-01 The Board Of Trustees Of The Leland Stanford Junior University Adeno-Associated Viral Vectors for Crossing the Human Blood Brain Barrier
WO2021046155A1 (en) 2019-09-03 2021-03-11 Voyager Therapeutics, Inc. Vectorized editing of nucleic acids to correct overt mutations
IL291944A (en) * 2019-10-10 2022-06-01 Solid Biosciences Inc Modified aav capsids and uses thereof
US20220403414A1 (en) * 2019-10-16 2022-12-22 Wuxi Apptec (Shanghai) Co., Ltd. Novel aav variant
CA3157700A1 (en) 2019-10-17 2021-04-22 Stridebio, Inc. Adeno-associated viral vectors for treatment of niemann-pick disease type c
CN115989234A (zh) * 2020-01-29 2023-04-18 建新公司 用于眼部基因疗法的经修饰的腺相关病毒衣壳蛋白及其使用方法
CN116249712A (zh) 2020-04-15 2023-06-09 沃雅戈治疗公司 Tau结合化合物
AU2021273447A1 (en) 2020-05-13 2022-12-08 Voyager Therapeutics, Inc. Redirection of tropism of AAV capsids
WO2021247995A2 (en) 2020-06-04 2021-12-09 Voyager Therapeutics, Inc. Compositions and methods of treating neuropathic pain
KR20230093241A (ko) 2020-07-27 2023-06-27 보이저 테라퓨틱스, 인크. 글루코실세라미데이스 베타 결핍증과 관련된 신경 장애의 치료를 위한 조성물 및 방법
WO2022026410A2 (en) 2020-07-27 2022-02-03 Voyager Therapeutics, Inc Compositions and methods for the treatment of niemann-pick type c1 disease
CN111825772B (zh) * 2020-07-30 2023-10-20 中国科学院精密测量科学与技术创新研究院 具有变异衣壳蛋白的腺相关病毒及其应用
JP2023536080A (ja) 2020-08-06 2023-08-23 ファンダシオン パラ ラ インベスティガシオン メディカ アプリカダ 遺伝子治療によるアルツハイマー病などのタウオパチーの治療に使用するためのウイルス粒子
MX2023001701A (es) 2020-08-12 2023-03-09 UCB Biopharma SRL Terapia genica utilizando constructos de acido nucleico que comprenden secuencias promotoras de la proteina de union a metil cpg 2 (mecp2).
CA3189878A1 (en) 2020-08-19 2022-02-24 Colin O'BANION Adeno-associated virus vectors for treatment of rett syndrome
AU2021331947A1 (en) * 2020-08-28 2023-05-04 Joint Stock Company "Biocad" Aav5-based vaccine against sars-cov-2
CN112194706B (zh) * 2020-09-30 2022-03-08 广州派真生物技术有限公司 腺相关病毒突变体及其应用
CA3193697A1 (en) * 2020-10-07 2022-04-14 Joseph Bruder Adeno-associated viruses for ocular delivery of gene therapy
JP2023544264A (ja) 2020-10-09 2023-10-23 ユーシービー バイオファルマ エスアールエル 核酸構築物、ウイルスベクター及びウイルス粒子
CA3196036A1 (en) 2020-10-28 2022-05-05 The University Of North Carolina At Chapel Hill Methods and compositions for dual glycan binding aav2.5 vector
WO2022093769A1 (en) * 2020-10-28 2022-05-05 The University Of North Carolina At Chapel Hill Methods and compositions for dual glycan binding aav2.5 vector
MX2023005218A (es) 2020-11-03 2023-05-16 Pfizer Metodos de purificacion de vectores de aav mediante cromatografia de intercambio anionico.
EP4244209A1 (en) 2020-11-11 2023-09-20 European Molecular Biology Laboratory Modified viral particles for gene therapy
US20240052322A1 (en) 2020-12-15 2024-02-15 Pfizer Inc. HILIC UPLC-MS Method For Separating and Analyzing Intact Adeno-Associated Virus Capsid Proteins
US20240011048A1 (en) 2020-12-21 2024-01-11 Pfizer Inc. Methods and compositions for inhibiting excess nucleic acid precipitation
JP2024503234A (ja) 2020-12-23 2024-01-25 ファイザー・インク 親和性クロマトグラフィーによるａａｖベクターの精製方法
US20240117328A1 (en) 2021-02-10 2024-04-11 Regenxbio Inc. Treatment of mucopolysaccharidosis ii with recombinant human iduronate-2-sulfatase (ids)
GB202101958D0 (en) 2021-02-12 2021-03-31 Ucl Business Ltd Gene therapy for dopamine transporter deficiency syndrome
CN112961219B (zh) * 2021-02-24 2022-08-12 中国农业科学院生物技术研究所 重组腺相关病毒、其突变体及其构建方法和应用
CN115044614B (zh) * 2021-03-09 2023-10-20 上海目镜生物医药科技有限公司 一种用于基因靶向与表达的aav-8型血清型的改造型载体及其构建方法及应用
WO2022208342A1 (en) 2021-04-01 2022-10-06 Pfizer Inc. Pharmaceutical compositions containing adeno-associated viral vector
EP4329820A1 (en) 2021-04-26 2024-03-06 Alexion Pharma International Operations Limited Adeno-associated viral vector capsids with improved tissue tropism
TW202309295A (zh) * 2021-05-07 2023-03-01 美商奧伊斯特普安生物製藥公司 編碼神經滋養因子之腺相關病毒病毒顆粒及其用途
KR20240005950A (ko) 2021-05-12 2024-01-12 폰다지오네 텔레톤 이티에스 벡터 시스템
KR20240023638A (ko) 2021-06-22 2024-02-22 화이자 인코포레이티드 곤충 세포에서의 아데노-연관 바이러스 벡터의 생산
WO2023283962A1 (en) 2021-07-16 2023-01-19 Huigene Therapeutics Co., Ltd. Modified aav capsid for gene therapy and methods thereof
CN118660710A (zh) 2021-10-20 2024-09-17 罗切斯特大学 用于年龄相关白质损失的竞争性治疗的分离神经胶质祖细胞
AU2022378524A1 (en) 2021-10-28 2024-05-02 UCB Biopharma SRL Nucleic acid constructs, viral vectors and viral particles
CA3235632A1 (en) 2021-11-02 2023-05-11 Voyager Therapeutics, Inc. Aav capsid variants and uses thereof
WO2023081633A1 (en) 2021-11-02 2023-05-11 University Of Rochester Tcf7l2 mediated remyelination in the brain
EP4442830A4 (en) 2021-11-29 2025-04-30 Shanghai Regenelead Therapies Co., Ltd. AADC/GDNF polynucleotide and use thereof in the treatment of Parkinson's disease
WO2023114816A1 (en) 2021-12-14 2023-06-22 Neurogene, Inc. Recombinant optimized galc constructs and methods for treating galc-associated disorders
AU2022423978A1 (en) * 2021-12-28 2024-07-18 Chengdu Origen Biotechnology Co., Ltd. Modified aav capsid protein and use thereof
GB202201242D0 (en) 2022-01-31 2022-03-16 Univ Edinburgh Recombinant optimized mecp2 cassettes and methods for treating rett syndrome and related disorders
CN118660913A (zh) 2022-02-21 2024-09-17 上海瑞宏迪医药有限公司 Vegf结合分子及其医药用途
CA3245130A1 (en) 2022-03-01 2023-09-07 Crispr Therapeutics Ag METHODS AND COMPOSITIONS FOR THE TREATMENT OF DISORDERS RELATED TO ANGIOPOIETIN-TYPE PROTEIN 3 (ANGPTL3)
AU2023250649A1 (en) 2022-04-04 2024-11-14 The Regents Of The University Of California Genetic complementation compositions and methods
GB202206336D0 (en) 2022-04-29 2022-06-15 Univ Edinburgh Recombinant therapeutic FMR1 constructs and methods of treating fragile X syndrome and related disorders
US20250353883A1 (en) 2022-05-06 2025-11-20 Novartis Ag Novel recombinant aav vp2 fusion polypeptides
WO2024003687A1 (en) 2022-06-28 2024-01-04 Pfizer Inc. Nucleic acids encoding acid alpha-glucosidase (gaa) and vectors for gene therapy
WO2024011112A1 (en) * 2022-07-06 2024-01-11 Voyager Therapeutics, Inc. Aav capsid variants and uses thereof
WO2024015877A2 (en) * 2022-07-12 2024-01-18 University Of Florida Research Foundation, Incorporated Novel aav3b capsid variants with enhanced hepatocyte tropism
KR20240014846A (ko) * 2022-07-26 2024-02-02 숙명여자대학교산학협력단 Aav2-anks1a 재조합 바이러스를 포함하는 알츠하이머 질병 치료용 조성물
WO2024038365A1 (en) 2022-08-16 2024-02-22 Pfizer Inc. Methods for purification of aav vectors by anion exchange chromatography
CN116063404B (zh) * 2022-09-27 2025-08-29 广州译码基因科技有限公司 可提高AAV包装能力的衣壳蛋白MutD及其应用
GB202216168D0 (en) 2022-10-31 2022-12-14 UCB Biopharma SRL Route of administration
CN116041443B (zh) * 2022-12-30 2023-09-22 广州派真生物技术有限公司 腺相关病毒突变体及其应用
CN115925819B (zh) * 2022-12-30 2023-10-13 广州派真生物技术有限公司 腺相关病毒突变体及其应用
CN120769917A (zh) * 2023-01-05 2025-10-10 恩沐辑医药有限责任公司 嵌合aav及其用途
AU2023427408A1 (en) 2023-02-02 2025-09-04 Voyager Therapeutics, Inc. Compositions and methods for the treatment of neurological disorders related to glucosylceramidase beta deficiency
EP4658294A2 (en) 2023-02-02 2025-12-10 University of Rochester Competitive replacement of glial cells
WO2024189094A1 (en) 2023-03-14 2024-09-19 UCB Biopharma SRL Gene therapy
GB202304767D0 (en) * 2023-03-30 2023-05-17 Ucl Business Ltd Synthetic AAV Capsid
WO2024238853A1 (en) * 2023-05-16 2024-11-21 Regenxbio Inc. Adeno-associated viruses for ocular delivery of gene therapy
WO2025022290A1 (en) 2023-07-21 2025-01-30 Crispr Therapeutics Ag Modulating expression of alas1 (5'-aminolevulinate synthase 1) gene
WO2025049917A1 (en) * 2023-08-31 2025-03-06 Dyno Therapeutics, Inc. Capsid polypeptides and methods of use thereof
WO2025090427A1 (en) 2023-10-23 2025-05-01 University Of Rochester Glial-targeted relief of hyperexcitability in neurodegenerative diseases
WO2025108407A2 (en) 2023-11-23 2025-05-30 Neuexcell Therapeutics (Suzhou) Co., Ltd. Gene therapy compositions and methods for treating glioma
WO2025113676A1 (en) 2023-11-29 2025-06-05 Neuexcell Therapeutics (Suzhou) Co., Ltd. Compositions and methods for treating stroke in primates
CN118459557B (zh) * 2024-01-16 2024-11-22 沈阳兴齐眼药股份有限公司 提高aav病毒视网膜穿透能力的衣壳蛋白突变体及其应用
WO2025186726A1 (en) 2024-03-05 2025-09-12 Crispr Therapeutics Ag Modulating expression of agt (angiotensinogen) gene
WO2025228976A1 (en) 2024-04-29 2025-11-06 UCB Biopharma SRL Gene therapy
GB202407038D0 (en) 2024-05-17 2024-07-03 Axovia Therapeutics Ltd Novel Gene Therapy
WO2025250457A1 (en) 2024-05-28 2025-12-04 University Of Rochester Enhanced brain transduction by gene therapeutics

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2005033321A2 (en) * 2003-09-30 2005-04-14 The Trustees Of The University Of Pennsylvania Adeno-associated virus (aav) clades, sequences, vectors containing same, and uses therefor
WO2006110689A2 (en) * 2005-04-07 2006-10-19 The Trustees Of The University Of Pennsylvania Method of increasing the function of an aav vector

Family Cites Families (54)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5639872A (en) 1993-07-27 1997-06-17 Hybridon, Inc. Human VEGF-specific oligonucleotides
AU1686997A (en) 1995-12-08 1997-06-27 Hybridon, Inc. Modified vegf antisense oligonucleotides for treatment of skin disorders
CA2265460A1 (en) 1996-09-11 1998-03-19 The Government Of The United States Of America, Represented By The Secre Tary, 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
EP1082413B1 (en) 1998-05-28 2008-07-23 THE GOVERNMENT OF THE UNITED STATES OF AMERICA, as represented by THE SECRETARY, DEPARTMENT OF HEALTH AND HUMAN SERVICES Aav5 vector and uses thereof
AU768729B2 (en) 1998-11-05 2004-01-08 Trustees Of The University Of Pennsylvania, The Adeno-associated virus serotype 1 nucleic acid sequences, vectors and host cells containing same
US6962815B2 (en) 2001-01-05 2005-11-08 Children's Hopital Inc. AAV2 vectors and methods
US7749492B2 (en) 2001-01-05 2010-07-06 Nationwide Children's Hospital, Inc. AAV vectors and methods
US20030144221A1 (en) 2001-07-17 2003-07-31 Isis Pharmaceuticals Inc. Antisense modulation of BCL2-associated X protein expression
EP1572893B1 (en) 2001-11-09 2009-01-07 THE GOVERNMENT OF THE UNITED STATES OF AMERICA as represented by THE SECRETARY of the DEPARTMENT OF HEALTH AND HUMAN SERVICES Production of adeno-associated virus in insect cells
ATE317916T1 (de) 2001-11-13 2006-03-15 Univ Pennsylvania Verfahren zur identifizierung von adeno- assoziiertem virus (aav) sequenzen sowie kit zur ausführung der methode
EP2573170B1 (en) 2001-12-17 2017-12-20 The Trustees Of The University Of Pennsylvania Adeno-associated virus (AAV) serotype 9 sequences, vectors containing same, and uses therefor
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
US20050106558A1 (en) 2001-12-21 2005-05-19 Luca Perabo Library of modified structural genes or capsid modified particles useful for the identification of viral clones with desired cell tropism
NZ536274A (en) 2002-05-01 2008-04-30 Univ Florida Improved rAAV expression systems for genetic modification of specific capsid proteins
US7148342B2 (en) 2002-07-24 2006-12-12 The Trustees Of The University Of Pennyslvania Compositions and methods for sirna inhibition of angiogenesis
EP1486567A1 (en) 2003-06-11 2004-12-15 Deutsches Krebsforschungszentrum Stiftung des öffentlichen Rechts Improved adeno-associated virus (AAV) vector for gene therapy
DK2357189T3 (en) 2003-06-19 2017-06-19 Genzyme Corp AAV virions with reduced immunoreactivity and applications thereof
US9441244B2 (en) 2003-06-30 2016-09-13 The Regents Of The University Of California Mutant adeno-associated virus virions and methods of use thereof
US9233131B2 (en) 2003-06-30 2016-01-12 The Regents Of The University Of California Mutant adeno-associated virus virions and methods of use thereof
EP2636739B1 (en) 2004-03-12 2014-12-10 Alnylam Pharmaceuticals Inc. iRNA agents targeting VEGF
EP1828390B1 (en) 2004-12-15 2012-06-13 The University Of North Carolina At Chapel Hill Chimeric vectors
US7543116B2 (en) 2006-01-30 2009-06-02 International Business Machines Corporation Data processing system, cache system and method for handling a flush operation in a data processing system having multiple coherency domains
US7588772B2 (en) 2006-03-30 2009-09-15 Board Of Trustees Of The Leland Stamford Junior University AAV capsid library and AAV capsid proteins
EP2018421B1 (en) 2006-04-28 2012-12-19 The Trustees of the University of Pennsylvania Scalable production method for aav
US20090317417A1 (en) 2006-04-28 2009-12-24 The Trustees Of The University Of Pennsylvania Modified AAV Vectors Having Reduced Capsid Immunogenicity and Use Thereof
ES2385679T3 (es) 2006-08-24 2012-07-30 Virovek, Inc. Expresión de células de insecto de genes con marcos de lectura abiertos superpuestos, métodos y composiciones de éstos
US7872118B2 (en) 2006-09-08 2011-01-18 Opko Ophthalmics, Llc siRNA and methods of manufacture
US9611302B2 (en) 2007-04-09 2017-04-04 University Of Florida Research Foundation, Inc. High-transduction-efficiency RAAV vectors, compositions, and methods of use
US9725485B2 (en) * 2012-05-15 2017-08-08 University Of Florida Research Foundation, Inc. AAV vectors with high transduction efficiency and uses thereof for gene therapy
EP2012122A1 (en) 2007-07-06 2009-01-07 Medigene AG Mutated parvovirus structural proteins as vaccines
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
US8632764B2 (en) 2008-04-30 2014-01-21 University Of North Carolina At Chapel Hill Directed evolution and in vivo panning of virus vectors
EP2396343B1 (en) * 2009-02-11 2017-05-17 The University of North Carolina At Chapel Hill Modified virus vectors and methods of making and using the same
DK2425000T3 (da) 2009-04-30 2019-05-13 Univ Pennsylvania Sammensætninger rettet mod ledende luftvejsceller omfattende konstrukter af adeno-associeret virus
WO2010138263A2 (en) 2009-05-28 2010-12-02 University Of Massachusetts Novel aav 's and uses thereof
US9133479B2 (en) 2009-06-03 2015-09-15 Cedars-Sinai Medical Center Effective vector platform for gene transfer and gene therapy
EP2457353A1 (en) 2009-07-22 2012-05-30 Aware, Inc. Improved packet detector
DK2826860T3 (en) 2010-04-23 2018-12-03 Univ Massachusetts CNS targeting AAV vectors and methods for their use
CN102071206A (zh) * 2010-10-22 2011-05-25 中山大学 一种腺相关病毒衣壳蛋白基因、相应的蛋白及其应用
SMT202400520T1 (it) * 2011-04-22 2025-01-14 Univ California Virioni di virus adeno-associato con capside variante e relativi metodi di utilizzo
US9169299B2 (en) 2011-08-24 2015-10-27 The Board Of Trustees Of The Leleand Stanford Junior University AAV capsid proteins for nucleic acid transfer
ES2862912T3 (es) * 2012-04-18 2021-10-08 Childrens Hospital Philadelphia Composición y procedimientos para la transferencia genética de alta eficiencia utilizando variantes de cápside de VAA
EP3470523A1 (en) 2012-05-09 2019-04-17 Oregon Health & Science University Adeno associated virus plasmids and vectors
TWI698240B (zh) 2012-05-15 2020-07-11 澳大利亞商艾佛蘭屈澳洲私營有限公司 使用腺相關病毒(aav)sflt-1治療老年性黃斑部退化(amd)
US10294281B2 (en) 2012-05-15 2019-05-21 University Of Florida Research Foundation, Incorporated High-transduction-efficiency rAAV vectors, compositions, and methods of use
US10006049B2 (en) 2013-05-16 2018-06-26 University Of Florida Research Foundation, Incorporated Hairpin mRNA elements and methods for the regulation of protein translation
GB201403684D0 (en) 2014-03-03 2014-04-16 King S College London Vector
US10308957B2 (en) 2014-03-04 2019-06-04 University Of Florida Research Foundation, Inc. rAAV vectors and methods for transduction of photoreceptors and RPE cells
WO2016133917A1 (en) 2015-02-16 2016-08-25 University Of Florida Research Foundation Raav vector compositions, methods for targeting vascular endothelial cells and use in treatment of type i diabetes
ES2865487T3 (es) 2015-09-28 2021-10-15 Univ North Carolina Chapel Hill Métodos y composiciones para vectores virales que evaden los anticuerpos
WO2017066764A2 (en) 2015-10-16 2017-04-20 William Marsh Rice University Modification of n-terminal region of capsid proteins for enhanced properties of adeno-associated viruses
US11326182B2 (en) 2016-04-29 2022-05-10 Voyager Therapeutics, Inc. Compositions for the treatment of disease
EP3485005A1 (en) 2016-07-12 2019-05-22 The University Of Manchester Gene therapy

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2005033321A2 (en) * 2003-09-30 2005-04-14 The Trustees Of The University Of Pennsylvania Adeno-associated virus (aav) clades, sequences, vectors containing same, and uses therefor
WO2006110689A2 (en) * 2005-04-07 2006-10-19 The Trustees Of The University Of Pennsylvania Method of increasing the function of an aav vector

Also Published As

Publication number Publication date
JP2023126885A (ja) 2023-09-12
CN106062200B (zh) 2020-06-23
US20210238631A1 (en) 2021-08-05
ES2749235T3 (es) 2020-03-19
DK3108000T3 (da) 2019-10-28
JP6942166B2 (ja) 2021-09-29
IL247332A0 (en) 2016-09-29
RU2016133623A (ru) 2018-03-22
JP6602788B2 (ja) 2019-11-06
US20220106612A1 (en) 2022-04-07
JP2021137016A (ja) 2021-09-16
US20240124894A1 (en) 2024-04-18
US20180135076A1 (en) 2018-05-17
MX372992B (es) 2020-05-12
CA3126061C (en) 2023-08-29
CA2939612C (en) 2021-10-12
PL3108000T3 (pl) 2020-05-18
EP3108000A1 (en) 2016-12-28
BR112016018598A2 (pt) 2017-10-17
CA2939612A1 (en) 2015-08-20
SI3108000T1 (sl) 2020-02-28
PT3108000T (pt) 2019-10-25
JP7612767B2 (ja) 2025-01-14
MX2016010649A (es) 2017-05-04
AU2023258448A1 (en) 2023-12-07
JP2017506521A (ja) 2017-03-09
AU2015216847B2 (en) 2021-05-20
RU2016133623A3 (enExample) 2018-11-16
CA3126061A1 (en) 2015-08-20
RU2743382C2 (ru) 2021-02-17
IL279154A (en) 2021-01-31
AU2021202538A1 (en) 2021-05-27
JP7309780B2 (ja) 2023-07-18
GB201403684D0 (en) 2014-04-16
RU2021102290A (ru) 2021-03-01
EP3108000B1 (en) 2019-08-28
EP3597760A2 (en) 2020-01-22
JP2020022476A (ja) 2020-02-13
IL279154B (en) 2022-06-01
HUE045801T2 (hu) 2020-01-28
KR20160130996A (ko) 2016-11-15
WO2015121501A1 (en) 2015-08-20
AU2021202538B2 (en) 2023-08-03
CN106062200A (zh) 2016-10-26
EP3597760A3 (en) 2020-03-11
AU2015216847A1 (en) 2016-08-11
US11802293B2 (en) 2023-10-31
SG11201606365TA (en) 2016-09-29
IL247332B (en) 2021-07-29

Similar Documents

Publication Publication Date Title
JP7612767B2 (ja) アデノ随伴ウイルスベクター
RU2611202C2 (ru) Вирионы аденоассоциированного вируса с вариантным капсидом и способы их использования
US20200231942A1 (en) Adeno-associated virus virions with variant capsid and methods of use thereof
KR20200022372A (ko) 변이체 캡시드를 보유한 아데노-부속 바이러스 비리온 및 이의 사용 방법
KR20220080019A (ko) 변이체 캡시드를 갖는 아데노-관련된 바이러스 비리온 및 이의 사용 방법
KR20230043794A (ko) 변이체 캡시드를 갖는 아데노-연관 바이러스 비리온 및 이의 사용 방법
RU2846967C2 (ru) Вектор аденоассоциированного вируса
HK40019369A (en) Adeno-associated virus vector
HK1226768B (zh) 腺相关病毒载体
HK1226768A1 (zh) 腺相關病毒載體
BR122021022437B1 (pt) Ácido nucleico que codifica uma variante da proteína do capsídeo de aav, uso de uma célula de inseto e método de produção de raav

Legal Events

Date Code Title Description
A201 Request for examination
PA0105 International application

Patent event date: 20160816

Patent event code: PA01051R01D

Comment text: International Patent Application

PA0201 Request for examination
PG1501 Laying open of application
E902 Notification of reason for refusal
PE0902 Notice of grounds for rejection

Comment text: Notification of reason for refusal

Patent event date: 20170911

Patent event code: PE09021S01D

AMND Amendment
E902 Notification of reason for refusal
PE0902 Notice of grounds for rejection

Comment text: Notification of reason for refusal

Patent event date: 20180827

Patent event code: PE09021S01D

AMND Amendment
E601 Decision to refuse application
PE0601 Decision on rejection of patent

Patent event date: 20190729

Comment text: Decision to Refuse Application

Patent event code: PE06012S01D

Patent event date: 20180827

Comment text: Notification of reason for refusal

Patent event code: PE06011S01I

Patent event date: 20170911

Comment text: Notification of reason for refusal

Patent event code: PE06011S01I

X091 Application refused [patent]
AMND Amendment
PX0901 Re-examination

Patent event code: PX09011S01I

Patent event date: 20190729

Comment text: Decision to Refuse Application

Patent event code: PX09012R01I

Patent event date: 20190227

Comment text: Amendment to Specification, etc.

Patent event code: PX09012R01I

Patent event date: 20180411

Comment text: Amendment to Specification, etc.

PX0701 Decision of registration after re-examination

Patent event date: 20191120

Comment text: Decision to Grant Registration

Patent event code: PX07013S01D

Patent event date: 20191028

Comment text: Amendment to Specification, etc.

Patent event code: PX07012R01I

Patent event date: 20190729

Comment text: Decision to Refuse Application

Patent event code: PX07011S01I

Patent event date: 20190227

Comment text: Amendment to Specification, etc.

Patent event code: PX07012R01I

Patent event date: 20180411

Comment text: Amendment to Specification, etc.

Patent event code: PX07012R01I

X701 Decision to grant (after re-examination)
GRNT Written decision to grant
PR0701 Registration of establishment

Comment text: Registration of Establishment

Patent event date: 20200220

Patent event code: PR07011E01D

Comment text: Amendment to Specification, etc.

Patent event date: 20191028

Patent event code: PX07012R01I

Comment text: Decision to Refuse Application

Patent event date: 20190729

Patent event code: PX07011S01I

Comment text: Amendment to Specification, etc.

Patent event date: 20190227

Patent event code: PX07012R01I

Comment text: Amendment to Specification, etc.

Patent event date: 20180411

Patent event code: PX07012R01I

PR1002 Payment of registration fee

Payment date: 20200220

End annual number: 3

Start annual number: 1

PG1601 Publication of registration
PR1001 Payment of annual fee

Payment date: 20230117

Start annual number: 4

End annual number: 4

PR1001 Payment of annual fee

Payment date: 20240109

Start annual number: 5

End annual number: 5

PR1001 Payment of annual fee

Payment date: 20250106

Start annual number: 6

End annual number: 6