RU2743382C2 - Вектор аденоассоциированного вируса - Google Patents

Вектор аденоассоциированного вируса Download PDF

Info

Publication number
RU2743382C2
RU2743382C2 RU2016133623A RU2016133623A RU2743382C2 RU 2743382 C2 RU2743382 C2 RU 2743382C2 RU 2016133623 A RU2016133623 A RU 2016133623A RU 2016133623 A RU2016133623 A RU 2016133623A RU 2743382 C2 RU2743382 C2 RU 2743382C2
Authority
RU
Russia
Prior art keywords
capsid protein
aav2
aav
variant
sequence
Prior art date
Application number
RU2016133623A
Other languages
English (en)
Russian (ru)
Other versions
RU2016133623A (ru
RU2016133623A3 (enExample
Inventor
Ральф Майкл ЛИНДЕН
Original Assignee
Кинг'З Колледж Лондон
Айкэн Скул Оф Медисин Эт Маунт Синай
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=RU2743382(C2) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Application filed by Кинг'З Колледж Лондон, Айкэн Скул Оф Медисин Эт Маунт Синай filed Critical Кинг'З Колледж Лондон
Publication of RU2016133623A publication Critical patent/RU2016133623A/ru
Publication of RU2016133623A3 publication Critical patent/RU2016133623A3/ru
Application granted granted Critical
Publication of RU2743382C2 publication Critical patent/RU2743382C2/ru

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)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Ophthalmology & Optometry (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Virology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (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)
RU2016133623A 2014-02-17 2015-02-17 Вектор аденоассоциированного вируса RU2743382C2 (ru)

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

Related Child Applications (1)

Application Number Title Priority Date Filing Date
RU2021102290A Division RU2846967C2 (ru) 2014-02-17 2021-02-02 Вектор аденоассоциированного вируса

Publications (3)

Publication Number Publication Date
RU2016133623A RU2016133623A (ru) 2018-03-22
RU2016133623A3 RU2016133623A3 (enExample) 2018-11-16
RU2743382C2 true RU2743382C2 (ru) 2021-02-17

Family

ID=50490674

Family Applications (1)

Application Number Title Priority Date Filing Date
RU2016133623A RU2743382C2 (ru) 2014-02-17 2015-02-17 Вектор аденоассоциированного вируса

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)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
RU2861004C2 (ru) * 2021-12-28 2026-04-28 Чэньду Ориген Биотекнолоджи Ко., Лтд. Модифицированный капсидный белок aav и его применение

Families Citing this family (213)

* 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
US8734809B2 (en) 2009-05-28 2014-05-27 University Of Massachusetts AAV's and uses thereof
DK2561073T3 (en) 2010-04-23 2016-12-12 Univ Massachusetts Aav vectors targeted to central nervous system and methods of use thereof
CA3050894C (en) 2010-04-23 2022-10-18 University Of Massachusetts Multicistronic expression constructs
DK2986635T3 (en) 2013-04-18 2019-01-28 Fond Telethon EFFECTIVE DELIVERY OF BIG GENES THROUGH DUAL-AAV VECTORS
US11136557B2 (en) 2013-05-31 2021-10-05 The Regents Of The University Of California Adeno-associated virus variants and methods of use thereof
EP3049527A4 (en) 2013-09-26 2017-08-16 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
JP6756700B2 (ja) 2014-03-18 2020-09-16 ユニバーシティ オブ マサチューセッツ 筋萎縮性側索硬化症を処置するためのｒＡＡＶベースの組成物および方法
WO2015164786A1 (en) 2014-04-25 2015-10-29 University Of Massachusetts Recombinant aav vectors useful for reducing immunity against transgene products
LT3137497T (lt) * 2014-05-02 2021-07-26 Genzyme Corporation Aav vektoriai, skirti tinklainės ir cns genų terapijai
US10689653B2 (en) 2014-06-03 2020-06-23 University Of Massachusetts Compositions and methods for modulating dysferlin expression
US10577627B2 (en) 2014-06-09 2020-03-03 Voyager Therapeutics, Inc. Chimeric capsids
US10370432B2 (en) 2014-10-03 2019-08-06 University Of Massachusetts Heterologous targeting peptide grafted AAVS
WO2016054557A1 (en) 2014-10-03 2016-04-07 University Of Massachusetts Novel high efficiency library-identified aav vectors
WO2016065001A1 (en) 2014-10-21 2016-04-28 University Of Massachusetts Recombinant aav variants and uses thereof
EP3215191B1 (en) 2014-11-05 2024-08-14 Voyager Therapeutics, Inc. Aadc polynucleotides for the treatment of parkinson's disease
KR20230145206A (ko) 2014-11-14 2023-10-17 보이저 테라퓨틱스, 인크. 조절성 폴리뉴클레오티드
CN114717264A (zh) 2014-11-14 2022-07-08 沃雅戈治疗公司 治疗肌萎缩性侧索硬化(als)的组合物和方法
US20180057839A1 (en) * 2014-11-26 2018-03-01 The Regents Of The University Of California Therapeutic compositions comprising transcription factors and methods of making and using the same
US11697825B2 (en) 2014-12-12 2023-07-11 Voyager Therapeutics, Inc. Compositions and methods for the production of scAAV
EP3256170B1 (en) 2015-02-13 2020-09-23 University of Massachusetts Compositions and methods for transient delivery of nucleases
US11046955B2 (en) 2015-04-24 2021-06-29 University Of Massachusetts Modified AAV constructs and uses thereof
GB201508026D0 (en) 2015-05-11 2015-06-24 Ucl Business Plc Capsid
IL314171A (en) * 2015-09-28 2024-09-01 Univ Florida Methods and compositions for antibody-evading virus vectors
WO2017070476A2 (en) 2015-10-22 2017-04-27 University Of Florida Research Foundation, Inc. Synthetic combinatorial aav3 capsid library
EP3364997B1 (en) 2015-10-22 2024-01-17 University of Massachusetts Aspartoacylase gene therapy in the treatment of canavan disease
WO2017070516A1 (en) 2015-10-22 2017-04-27 University Of Massachusetts Prostate-targeting adeno-associated virus serotype vectors
US20180230489A1 (en) 2015-10-28 2018-08-16 Voyager Therapeutics, Inc. Regulatable expression using adeno-associated virus (aav)
GB201519086D0 (en) * 2015-10-28 2015-12-09 Syncona Partners Llp Gene Therapy
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
SG10201912763QA (en) 2015-11-05 2020-02-27 Bamboo Therapeutics Inc Modified friedreich ataxia genes and vectors for gene therapy
AU2016362477A1 (en) 2015-12-02 2018-06-14 Voyager Therapeutics, Inc. Assays for the detection of AAV neutralizing antibodies
AU2016366549B2 (en) * 2015-12-11 2022-11-10 California Institute Of Technology Targeting peptides for directing adeno-associated viruses (AAVs)
AU2016370630B2 (en) * 2015-12-14 2023-04-13 The Trustees Of The University Of Pennsylvania Adeno-associated viral vectors useful in treatment of spinal muscular atropy
CA3011939A1 (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
CA3012344A1 (en) 2016-02-12 2017-08-17 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
EP3440210A4 (en) 2016-04-05 2019-11-27 University of Massachusetts COMPOSITIONS AND METHOD FOR SELECTIVELY INHIBITING THE EXPRESSION OF GRAINYHEAD LIKE PROTEIN
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
IL262207B2 (en) 2016-04-15 2026-01-01 Univ Pennsylvania Preparations for the treatment of wet age-related macular degeneration
US11413356B2 (en) 2016-04-15 2022-08-16 University Of Massachusetts Methods and compositions for treating metabolic imbalance
EP3448987A4 (en) 2016-04-29 2020-05-27 Voyager Therapeutics, Inc. COMPOSITIONS FOR TREATING A DISEASE
EP3448874A4 (en) 2016-04-29 2020-04-22 Voyager Therapeutics, Inc. COMPOSITIONS FOR TREATING A DISEASE
US10584337B2 (en) 2016-05-18 2020-03-10 Voyager Therapeutics, Inc. Modulatory polynucleotides
IL297576B2 (en) 2016-05-18 2024-02-01 Voyager Therapeutics Inc Compositions and methods of treating huntington's disease
MA44546B1 (fr) 2016-06-15 2021-03-31 Univ California Virus adéno-associés variants et procédés d'utilisation
US11882815B2 (en) 2016-06-15 2024-01-30 University Of Massachusetts Recombinant adeno-associated viruses for delivering gene editing molecules to embryonic cells
CA2971303C (en) 2016-06-21 2026-03-03 Bamboo Therapeutics, Inc. Optimized mini-dystrophin genes and expression cassettes and their use
US11427838B2 (en) 2016-06-29 2022-08-30 Vertex Pharmaceuticals Incorporated Materials and methods for treatment of myotonic dystrophy type 1 (DM1) and other related disorders
CA3029119A1 (en) 2016-06-29 2018-01-04 Crispr Therapeutics Ag Materials and methods for treatment of friedreich ataxia and other related disorders
WO2018002762A1 (en) 2016-06-29 2018-01-04 Crispr Therapeutics Ag Materials and methods for treatment of amyotrophic lateral sclerosis (als) and other related disorders
EP4650364A3 (en) 2016-07-06 2026-01-21 Vertex Pharmaceuticals Incorporated Materials and methods for treatment of pain related disorders
EP3481857B1 (en) 2016-07-06 2026-02-11 Vertex Pharmaceuticals Incorporated Materials and methods for treatment of pain related disorders
US11458211B2 (en) 2016-07-12 2022-10-04 The University Of Manchester Gene therapy
CN109640949B (zh) 2016-07-29 2022-11-25 加利福尼亚大学董事会 具有变异衣壳的腺相关病毒病毒体和其使用方法
CA3033125A1 (en) * 2016-08-16 2018-02-22 The University Of North Carolina At Chapel Hill Methods and compositions for targeted gene transfer
EP3831281B1 (en) 2016-08-30 2026-02-25 The Regents of The University of California Devices and systems for biomedical targeting and delivery
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
KR20190075964A (ko) 2016-10-13 2019-07-01 유니버시티 오브 매사추세츠 Aav 캡시드 설계
AU2017345470B2 (en) * 2016-10-19 2023-08-03 Adverum Biotechnologies, Inc. Modified AAV capsids and uses thereof
CN110300591A (zh) 2016-11-04 2019-10-01 百深公司 腺相关病毒制剂
JP2020508289A (ja) 2017-01-31 2020-03-19 レジェンクスバイオ インコーポレーテッド 完全ヒトグリコシル化ヒトα−Ｌ−イズロニダーゼ（ＩＤＵＡ）を用いたムコ多糖症Ｉ型の治療
EP3585883A4 (en) * 2017-02-21 2021-04-14 University of Florida Research Foundation, Incorporated MODIFIED AAV CAPSID PROTEINS AND USES THEREOF
EP3585899A1 (en) 2017-02-22 2020-01-01 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
WO2018154418A1 (en) 2017-02-22 2018-08-30 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
WO2018154462A2 (en) 2017-02-22 2018-08-30 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
WO2018154439A1 (en) 2017-02-22 2018-08-30 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
AU2018224387B2 (en) 2017-02-22 2024-08-08 Crispr Therapeutics Ag Compositions and methods for gene editing
KR20240093919A (ko) 2017-04-14 2024-06-24 리젠엑스바이오 인크. 인간 뉴런 또는 신경교 세포에 의해 생성된 재조합 인간 이두로네이트-2 설파타제(ids)를 이용한 뮤코다당증 ii형의 치료
MX2019013172A (es) 2017-05-05 2020-09-07 Voyager Therapeutics Inc Composiciones y metodos para tratar la enfermedad de huntington.
EP3618839A4 (en) 2017-05-05 2021-06-09 Voyager Therapeutics, Inc. COMPOSITIONS AND METHODS OF TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS)
AU2018264996A1 (en) 2017-05-09 2019-12-05 University Of Massachusetts Methods of treating Amyotrophic Lateral Sclerosis (ALS)
WO2018209154A1 (en) 2017-05-10 2018-11-15 Massachusetts Eye And Ear Infirmary Methods and compositions for modifying assembly-activating protein (app)-dependence of viruses
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
JOP20190269A1 (ar) 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
KR20250023600A (ko) * 2017-06-30 2025-02-18 더 리젠츠 오브 더 유니버시티 오브 캘리포니아 변이체 캡시드를 보유한 아데노-부속 바이러스 비리온 및 이의 사용 방법
CA3070087A1 (en) 2017-07-17 2019-01-24 Voyager Therapeutics, Inc. Trajectory array guide system
US11512327B2 (en) 2017-08-03 2022-11-29 Voyager Therapeutics, Inc. Compositions and methods for delivery of AAV
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
MX2020001997A (es) 2017-08-28 2020-07-20 Univ California Variantes de capside de virus adenoasociado y metodos de uso de estas.
CN118006607A (zh) 2017-09-22 2024-05-10 马萨诸塞大学 Sod1双表达载体及其用途
MX2020003042A (es) 2017-09-29 2020-11-18 Voyager Therapeutics Inc Rescate del fenotipo neurológico central y periférico de la ataxia de friedreich mediante administración intravenosa.
TW202413649A (zh) 2017-10-16 2024-04-01 美商航海家醫療公司 肌萎縮性脊髓側索硬化症(als)之治療
WO2019079242A1 (en) 2017-10-16 2019-04-25 Voyager Therapeutics, Inc. TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS)
KR20200067195A (ko) * 2017-10-18 2020-06-11 리젠엑스바이오 인크. 완전-인간 번역후 변형된 항체 치료제
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
US12558434B2 (en) 2018-02-20 2026-02-24 The Trustees Of The University Of Pennsylvania Compositions for treatment of wet age-related macular degeneration
IL277270B2 (en) * 2018-03-16 2025-05-01 Res Inst Nationwide Childrens Hospital Increasing tissue-specific gene delivery through capsid modification
EP3781187A4 (en) * 2018-03-29 2021-12-22 Asklepios Biopharmaceutical, Inc. TROPICAL RECOMBINANT AAV6 LIVER VECTORS TO Bypass NEUTRALIZATION
CA3095179A1 (en) 2018-04-03 2019-10-10 Stridebio, Inc. Antibody-evading virus vectors
BR112020020266A2 (pt) 2018-04-03 2021-01-19 Stridebio, Inc. Vetores de vírus com evasão de anticorpos
MX2020010465A (es) 2018-04-03 2021-01-08 Vectores de virus para direccionamiento a tejidos oftalmicos.
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
WO2019221992A1 (en) 2018-05-15 2019-11-21 President And Fellows Of Harvard College Viral vectors exhibiting improved gene delivery properties
WO2019222329A1 (en) 2018-05-15 2019-11-21 Voyager Therapeutics, Inc. Compositions and methods for delivery of aav
CN112424359A (zh) 2018-05-15 2021-02-26 沃雅戈治疗公司 用于治疗帕金森氏病的组合物和方法
WO2019236995A1 (en) 2018-06-08 2019-12-12 University Of Massachusetts Antisense oligonucleotides to restore dysferlin protein expression in dysferlinopathy patient cells
MX2020013953A (es) 2018-07-11 2021-03-09 Takeda Pharmaceuticals Co Composiciones de virus adenoasociado (aav).
BR112021001878A2 (pt) * 2018-07-31 2021-04-27 Cornell University métodos de terapia genética para o controle da função do órgão
WO2020033842A1 (en) 2018-08-10 2020-02-13 Regenxbio Inc. Scalable method for recombinant aav production
US12281321B2 (en) 2018-09-28 2025-04-22 Voyager Therapeutics, Inc. Frataxin expression constructs having engineered promoters and methods of use thereof
CN113166208B (zh) 2018-10-02 2024-11-22 沃雅戈治疗公司 重新定向aav衣壳的嗜性
US20210371470A1 (en) 2018-10-12 2021-12-02 Voyager Therapeutics, Inc. Compositions and methods for delivery of aav
UY38407A (es) 2018-10-15 2020-05-29 Novartis Ag Anticuerpos estabilizadores de trem2
WO2020102645A1 (en) * 2018-11-16 2020-05-22 Asklepios Biopharmaceutical, Inc. Therapeutic adeno-associated virus for treating pompe disease
CA3125770A1 (en) 2019-01-18 2020-07-23 Voyager Therapeutics, Inc. Methods and systems for producing aav particles
GB201900702D0 (en) * 2019-01-18 2019-03-06 Univ Bristol Therapy
KR20210126096A (ko) * 2019-02-15 2021-10-19 상가모 테라퓨틱스, 인코포레이티드 재조합 aav를 제조하기 위한 조성물 및 방법
CN113727992A (zh) 2019-03-21 2021-11-30 斯特里迪比奥公司 重组腺相关病毒载体
KR20210142725A (ko) * 2019-03-28 2021-11-25 오피노 엘엘씨 코어 페이징 처리
CN114450411A (zh) 2019-04-01 2022-05-06 特纳亚治疗股份有限公司 具有工程化衣壳的腺相关病毒
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
CA3132447A1 (en) 2019-04-24 2020-10-29 Takara Bio Inc. Aav mutant having brain-targeting property
AU2020263392A1 (en) * 2019-04-26 2021-10-28 The University Of North Carolina At Chapel Hill Methods and compositions for dual glycan binding AAV2.5 vector
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
WO2020261178A1 (en) 2019-06-27 2020-12-30 Pfizer Inc. Methods of treating duchenne muscular dystrophy using aav mini-dystrophin gene therapy
US12173290B2 (en) 2019-06-28 2024-12-24 Crispr Therapeutics Ag Materials and methods for controlling gene editing
AU2020299026A1 (en) * 2019-07-04 2022-01-20 Children's Medical Research Institute Methods and AAV vectors for in vivo transduction
JP7371954B2 (ja) * 2019-07-12 2023-10-31 株式会社遺伝子治療研究所 ヒト肝臓への遺伝子導入のためのアデノ随伴ウイルスビリオン
SMT202400486T1 (it) * 2019-07-15 2025-01-14 Meiragtx Uk Ii Ltd Proteine di capside avv modificate per il trattamento della malattia artritica
EP4010357A2 (en) 2019-08-09 2022-06-15 University of Florida Research Foundation, Incorporated Aav capsid variants for targeting human glioblastoma cells
JP7785352B2 (ja) * 2019-08-14 2025-12-15 ユニバーシティー オブ フロリダ リサーチ ファンデーション， インク． 遺伝子治療のためのａａｖカプシドバリアント
AU2020336332A1 (en) * 2019-08-29 2022-03-03 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
WO2021073568A1 (en) * 2019-10-16 2021-04-22 Wuxi Apptec (Shanghai) Co., Ltd. A novel aav variant
CN114787180A (zh) 2019-10-17 2022-07-22 斯特里迪比奥公司 用于治疗c型尼曼-匹克病的腺相关病毒载体
MX2022004353A (es) 2019-10-17 2022-07-19 Casete de transferencia de aav.
EP4055034A4 (en) 2019-11-08 2024-05-29 President And Fellows Of Harvard College VIRAL CAPSID POLYPEPTIDES
EP4055030A4 (en) 2019-11-08 2024-05-22 President And Fellows Of Harvard College VIRAL CAPSID POLYPEPTIDES
WO2021108467A1 (en) 2019-11-25 2021-06-03 University Of Florida Research Foundation, Incorporated Novel aav3b variants that target human hepatocytes and evade the humoral immune response
AU2020392243A1 (en) 2019-11-28 2022-06-16 Regenxbio Inc. Microdystrophin gene therapy constructs and uses thereof
WO2021155137A1 (en) * 2020-01-29 2021-08-05 Genzyme Corporation Modified adeno-associated viral capsid proteins for ocular gene therapy and methods of use thereof
CA3180222A1 (en) 2020-04-15 2021-10-21 Voyager Therapeutics, Inc. Tau binding compounds
US12630842B2 (en) 2020-05-13 2026-05-19 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
GB202010009D0 (en) * 2020-06-30 2020-08-12 Syncona Investment Man Ltd Vector
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
US20230285596A1 (en) 2020-07-27 2023-09-14 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
CA3181024A1 (en) 2020-08-06 2022-02-10 Fundacion Para La Investigacion Medica Aplicada Viral particles for use in treating tauopathies such as alzheimer's diseases by gene therapy
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).
JP2023542614A (ja) 2020-08-19 2023-10-11 サレプタ セラピューティクス， インコーポレイテッド レット症候群の治療のためのアデノ随伴ウイルスベクター
IL300738A (en) * 2020-08-28 2023-04-01 Biocad Joint Stock Co A vaccine for SARS-COV-2 based on AAV5
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
EP4225777A2 (en) * 2020-10-07 2023-08-16 RegenxBio Inc. Adeno-associated viruses for ocular delivery of gene therapy
JP7415042B2 (ja) 2020-10-09 2024-01-16 テナヤ セラピューティクス， インコーポレイテッド プラコフィリン２遺伝子治療の方法および組成物
IL301255A (en) 2020-10-09 2023-05-01 UCB Biopharma SRL Nucleic acid structures, viral vectors and viral particles
CN116490514A (zh) 2020-10-28 2023-07-25 北卡罗来纳-查佩尔山大学 用于双重聚糖结合aav2.5载体的方法和组合物
AU2021375404A1 (en) 2020-11-03 2023-06-08 Pfizer Inc. Methods for purification of aav vectors by anion exchange chromatography
MX2023005446A (es) 2020-11-11 2023-07-18 European Molecular Biology Laboratory Particulas virales modificadas para terapia genica.
WO2022130172A1 (en) 2020-12-15 2022-06-23 Pfizer Inc. Hilic uplc-ms method for separating and analyzing intact adeno-associated virus capsid proteins
CA3205588A1 (en) 2020-12-21 2022-06-30 Pfizer Inc. Methods and systems for improved cell transfection
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
AU2022221284A1 (en) 2021-02-10 2023-08-17 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
CA3216420A1 (en) 2021-04-26 2022-11-03 Seungil Han Adeno-associated viral vector capsids with improved tissue tropism
BR112023023249A2 (pt) * 2021-05-07 2024-01-23 Oyster Point Pharma Inc Fator neurotrófico de codificação de vírion aav e usos do mesmo
CN117377771A (zh) 2021-05-12 2024-01-09 特莱索恩基金会 载体系统
MX2023015329A (es) 2021-06-22 2024-01-23 Pfizer Produccion de vector de virus adeno-asociado en celulas de insecto.
WO2023283962A1 (en) 2021-07-16 2023-01-19 Huigene Therapeutics Co., Ltd. Modified aav capsid for gene therapy and methods thereof
US20230241118A1 (en) 2021-10-20 2023-08-03 University Of Rochester Rejuvenation treatment of age-related white matter loss
IL312340A (en) 2021-10-28 2024-06-01 UCB Biopharma SRL Nucleic acid constructs, viral vectors and viral particles
EP4426716A1 (en) 2021-11-02 2024-09-11 Voyager Therapeutics, Inc. Aav capsid variants and uses thereof
CA3236365A1 (en) 2021-11-02 2023-05-11 University Of Rochester Tcf7l2 mediated remyelination in the brain
CA3238867A1 (en) 2021-11-29 2023-06-01 Shanghai Regenelead Therapies Co., Ltd. Aadc/gdnf polynucleotide, and use thereof in treating parkinson's disease
WO2023114816A1 (en) 2021-12-14 2023-06-22 Neurogene, Inc. Recombinant optimized galc constructs and methods for treating galc-associated disorders
WO2023125481A1 (zh) * 2021-12-28 2023-07-06 成都弘基生物科技有限公司 经过修饰的aav衣壳蛋白及其用途
GB202201242D0 (en) 2022-01-31 2022-03-16 Univ Edinburgh Recombinant optimized mecp2 cassettes and methods for treating rett syndrome and related disorders
WO2023155918A1 (zh) 2022-02-21 2023-08-24 上海瑞宏迪医药有限公司 Vegf结合分子及其医药用途
WO2023166425A1 (en) 2022-03-01 2023-09-07 Crispr Therapeutics Ag Methods and compositions for treating angiopoietin-like 3 (angptl3) related conditions
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
CA3255627A1 (en) 2022-05-06 2023-11-09 Novartis Ag NEW VP2 FUSION POLYPEPTIDES OF RECOMBINANT AAV
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
US20260041792A1 (en) * 2022-07-12 2026-02-12 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
JP2026501732A (ja) * 2023-01-05 2026-01-16 エミュジェン セラピューティクス エルエルシー キメラａａｖおよびその使用
EP4658294A2 (en) 2023-02-02 2025-12-10 University of Rochester Competitive replacement of glial cells
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
JP2026509301A (ja) 2023-03-14 2026-03-17 ユーシービー バイオファルマ エスアールエル 遺伝子治療
GB202304767D0 (en) * 2023-03-30 2023-05-17 Ucl Business Ltd Synthetic AAV Capsid
CN121311595A (zh) 2023-04-17 2026-01-09 阿斯克生物公司 转基因在灵长类动物中可调节的受控表达
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
TW202508610A (zh) 2023-08-23 2025-03-01 大陸商上海瑞宏迪醫藥有限公司 醫藥組成物及其用途
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
AU2024386366A1 (en) 2023-11-23 2026-06-04 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
CN118440161B (zh) * 2024-01-16 2025-02-25 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
WO2025250454A1 (en) 2024-05-28 2025-12-04 University Of Rochester Adeno-associated viruses evolved to specifically target human glial progenitor cells in vivo
WO2025262203A1 (en) 2024-06-21 2025-12-26 UCB Biopharma SRL Nucleic acid sequences
WO2026017965A1 (en) 2024-07-19 2026-01-22 The University Court Of The University Of Edinburgh Recombinant therapeutic fxn constructs and methods of treating friedreich ataxia and related conditions
WO2026114267A1 (en) 2024-11-29 2026-06-04 Neuexcell Therapeutics (Suzhou) Co., Ltd. Compositions and methods for treating alzheimer's disease in primates
WO2026150302A1 (en) 2025-01-09 2026-07-16 Novartis Ag Novel aav rep/cap encoding constructs

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2006110689A2 (en) * 2005-04-07 2006-10-19 The Trustees Of The University Of Pennsylvania Method of increasing the function of an aav vector
WO2012145601A2 (en) * 2011-04-22 2012-10-26 The Regents Of The University Of California Adeno-associated virus virions with variant capsid and methods of use thereof

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
WO1997020925A1 (en) 1995-12-08 1997-06-12 Hybridon, Inc. Modified vegf antisense oligonucleotides for treatment of skin disorders
AU4645697A (en) 1996-09-11 1998-04-02 Government Of The United States Of America, As Represented By The Secretary Of The Department Of Health And Human Services, The 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
DE69939169D1 (de) 1998-05-28 2008-09-04 Us Gov Health & Human Serv Aav5 vektoren und deren verwendung
DE69936104T2 (de) 1998-11-05 2008-01-17 The Trustees Of The University Of Pennsylvania Nukleinsäuresequenzen des adeno-assoziierten virus des serotyps i, und vektoren und wirtszellen, die diese enthalten
WO2002053703A2 (en) 2001-01-05 2002-07-11 Children's Hospital, 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
CN103589692B (zh) 2001-11-13 2018-07-24 宾夕法尼亚大学托管会 检测和/或鉴定腺伴随病毒(aav)序列以及分离所鉴定的新型序列的方法
DK2359869T3 (en) 2001-12-17 2019-04-15 Univ Pennsylvania Sequences of adeno-associated virus (AAV) serotype 8, vectors containing these, and uses thereof
EP1463805B1 (en) 2001-12-17 2014-10-22 The Trustees of The University of Pennsylvania Adeno-associated virus (aav) serotype 9 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
SI1496944T1 (sl) 2002-05-01 2009-02-28 Univ Florida Izboljšani sistemi ekspresije RAAV za genetsko modifikacijo specifičnih kapsidnih proteinov
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
PL2657248T3 (pl) 2003-06-19 2017-09-29 Genzyme Corporation Wiriony AAV o zmniejszonej immunoreaktywności i ich zastosowanie
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
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
EP1668143B1 (en) * 2003-09-30 2013-03-20 The Trustees of The University of Pennsylvania Adeno-associated virus (aav) clades, sequences, vectors containing same, and uses therefor
ES2423060T3 (es) 2004-03-12 2013-09-17 Alnylam Pharmaceuticals, Inc. Agentes iRNA que tienen como diana al 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
JP2009535339A (ja) * 2006-04-28 2009-10-01 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 低下されたキャプシド免疫原性を有する改変ａａｖベクターおよびその使用
CN101528916B (zh) 2006-04-28 2013-09-04 宾夕法尼亚大学托管会 规模可调的aav生产方法
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
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
US9611302B2 (en) 2007-04-09 2017-04-04 University Of Florida Research Foundation, Inc. High-transduction-efficiency RAAV vectors, compositions, and methods of use
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
US8734809B2 (en) 2009-05-28 2014-05-27 University Of Massachusetts 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
WO2011011572A1 (en) 2009-07-22 2011-01-27 Aware, Inc. Improved packet detector
DK2561073T3 (en) 2010-04-23 2016-12-12 Univ Massachusetts Aav vectors targeted to central nervous system and methods of use thereof
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
EP3795581A3 (en) 2011-08-24 2021-06-09 The Board of Trustees of the Leland Stanford Junior University New avv capsid proteins for nucleic acid transfer
JP6342886B2 (ja) * 2012-04-18 2018-06-13 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 Ａａｖカプシドバリアントを使用した高効率な遺伝子導入のための組成物及び方法
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
WO2014186746A1 (en) 2013-05-16 2014-11-20 University Of Florida Research Foundation, Inc. HAIRPIN mRNA ELEMENTS AND METHODS FOR THE REGULATION OF PROTEIN TRANSLATION
GB201403684D0 (en) 2014-03-03 2014-04-16 King S College London Vector
EP3113787B1 (en) 2014-03-04 2019-12-04 University of Florida Research Foundation, Inc. Improved raav vectors and methods for transduction of photoreceptors and rpe cells
US10648000B2 (en) 2015-02-16 2020-05-12 University Of Florida Research Foundation, Incorporated rAAV vector compositions, methods for targeting vascular endothelial cells and use in treatment of type I diabetes
IL314171A (en) 2015-09-28 2024-09-01 Univ Florida Methods and compositions for antibody-evading virus vectors
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
EP3448874A4 (en) 2016-04-29 2020-04-22 Voyager Therapeutics, Inc. COMPOSITIONS FOR TREATING A DISEASE
US11458211B2 (en) 2016-07-12 2022-10-04 The University Of Manchester Gene therapy

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2006110689A2 (en) * 2005-04-07 2006-10-19 The Trustees Of The University Of Pennsylvania Method of increasing the function of an aav vector
WO2012145601A2 (en) * 2011-04-22 2012-10-26 The Regents Of The University Of California Adeno-associated virus virions with variant capsid and methods of use thereof

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
Супотницкий М.В. Генотерапевтические векторные системы на основе вирусов// Биопрепараты. - 2011. -N3. - с. 15-26. *

Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
RU2861270C2 (ru) * 2021-05-07 2026-04-30 Ойстер Поинт Фарма, Инк. Совместная терапия вектором и никотиновым агонистом
RU2861004C2 (ru) * 2021-12-28 2026-04-28 Чэньду Ориген Биотекнолоджи Ко., Лтд. Модифицированный капсидный белок aav и его применение

Also Published As

Publication number Publication date
JP2017506521A (ja) 2017-03-09
CA3126061A1 (en) 2015-08-20
EP3597760A3 (en) 2020-03-11
AU2023258448B2 (en) 2026-03-26
CN106062200B (zh) 2020-06-23
US20180135076A1 (en) 2018-05-17
AU2015216847A1 (en) 2016-08-11
CA2939612A1 (en) 2015-08-20
AU2021202538B2 (en) 2023-08-03
US20210238631A1 (en) 2021-08-05
WO2015121501A1 (en) 2015-08-20
JP7612767B2 (ja) 2025-01-14
IL247332A0 (en) 2016-09-29
IL279154B (en) 2022-06-01
IL247332B (en) 2021-07-29
JP6942166B2 (ja) 2021-09-29
PL3108000T3 (pl) 2020-05-18
RU2016133623A (ru) 2018-03-22
MX372992B (es) 2020-05-12
DK3108000T3 (da) 2019-10-28
SG11201606365TA (en) 2016-09-29
AU2023258448A1 (en) 2023-12-07
CA2939612C (en) 2021-10-12
CA3126061C (en) 2023-08-29
RU2016133623A3 (enExample) 2018-11-16
JP6602788B2 (ja) 2019-11-06
BR112016018598A2 (pt) 2017-10-17
US20240124894A1 (en) 2024-04-18
JP2023126885A (ja) 2023-09-12
IL279154A (en) 2021-01-31
KR20160130996A (ko) 2016-11-15
EP3108000A1 (en) 2016-12-28
SI3108000T1 (sl) 2020-02-28
JP2021137016A (ja) 2021-09-16
ES2749235T3 (es) 2020-03-19
AU2015216847B2 (en) 2021-05-20
US11802293B2 (en) 2023-10-31
GB201403684D0 (en) 2014-04-16
US20220106612A1 (en) 2022-04-07
RU2021102290A (ru) 2021-03-01
MX2016010649A (es) 2017-05-04
KR102082032B1 (ko) 2020-02-26
EP3108000B1 (en) 2019-08-28
EP3597760A2 (en) 2020-01-22
JP7309780B2 (ja) 2023-07-18
AU2021202538A1 (en) 2021-05-27
CN106062200A (zh) 2016-10-26
HUE045801T2 (hu) 2020-01-28
PT3108000T (pt) 2019-10-25
JP2020022476A (ja) 2020-02-13

Similar Documents

Publication Publication Date Title
JP7612767B2 (ja) アデノ随伴ウイルスベクター
KR102766238B1 (ko) 변이체 캡시드를 보유한 아데노-부속 바이러스 비리온 및 이의 사용 방법
RU2611202C2 (ru) Вирионы аденоассоциированного вируса с вариантным капсидом и способы их использования
KR20230043794A (ko) 변이체 캡시드를 갖는 아데노-연관 바이러스 비리온 및 이의 사용 방법
RU2846967C2 (ru) Вектор аденоассоциированного вируса
HK40019369A (en) Adeno-associated virus vector
HK1226768A1 (en) Adeno-associated virus vector
HK1226768B (en) Adeno-associated virus vector
CA3299376A1 (en) Brain-tropic aav mutant
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