ATE318923T1 - Doppelsträngige parvovirus-vektoren - Google Patents

Doppelsträngige parvovirus-vektoren

Info

Publication number
ATE318923T1
ATE318923T1 AT01948252T AT01948252T ATE318923T1 AT E318923 T1 ATE318923 T1 AT E318923T1 AT 01948252 T AT01948252 T AT 01948252T AT 01948252 T AT01948252 T AT 01948252T AT E318923 T1 ATE318923 T1 AT E318923T1
Authority
AT
Austria
Prior art keywords
duplexed
parvovirus
double stranded
methods
vector
Prior art date
Application number
AT01948252T
Other languages
English (en)
Inventor
Richard Jude Samulski
Douglas M Mccarty
Original Assignee
Univ North Carolina
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Univ North Carolina filed Critical Univ North Carolina
Application granted granted Critical
Publication of ATE318923T1 publication Critical patent/ATE318923T1/de

Links

Classifications

    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • C12N15/864Parvoviral vectors, e.g. parvovirus, densovirus
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N7/00Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12Antivirals
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P33/00Antiparasitic agents
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00Antineoplastic agents
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00Drugs for immunological or allergic disorders
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14121Viruses as such, e.g. new isolates, mutants or their genomic sequences
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • Medicinal Chemistry (AREA)
  • Virology (AREA)
  • Veterinary Medicine (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Biophysics (AREA)
  • Molecular Biology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Immunology (AREA)
  • Communicable Diseases (AREA)
  • Oncology (AREA)
  • Tropical Medicine & Parasitology (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Separation By Low-Temperature Treatments (AREA)
  • Surface Acoustic Wave Elements And Circuit Networks Thereof (AREA)
  • Control Of Combustion (AREA)
AT01948252T 2000-06-01 2001-05-31 Doppelsträngige parvovirus-vektoren ATE318923T1 (de)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US20860400P 2000-06-01 2000-06-01

Publications (1)

Publication Number Publication Date
ATE318923T1 true ATE318923T1 (de) 2006-03-15

Family

ID=22775222

Family Applications (1)

Application Number Title Priority Date Filing Date
AT01948252T ATE318923T1 (de) 2000-06-01 2001-05-31 Doppelsträngige parvovirus-vektoren

Country Status (10)

Country Link
US (4) US7465583B2 (de)
EP (1) EP1290205B1 (de)
JP (1) JP4860886B2 (de)
AT (1) ATE318923T1 (de)
AU (2) AU6972301A (de)
CA (1) CA2410828C (de)
DE (1) DE60117550T2 (de)
ES (2) ES2505700T3 (de)
NZ (1) NZ522840A (de)
WO (1) WO2001092551A2 (de)

Families Citing this family (279)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2001011034A2 (en) * 1999-08-09 2001-02-15 Targeted Genetics Corporation Enhancement of expression of a single-stranded, heterologous nucleotide sequence from recombinant viral vectors by designing the sequence such that it forms intrastrand base pairs
US8241622B2 (en) 2001-07-13 2012-08-14 University Of Iowa Research Foundation Adeno-associated virus vectors with intravector heterologous terminal palindromic sequences
ATE549037T1 (de) 2004-09-22 2012-03-15 St Jude Childrens Res Hospital Verbesserte expression von faktor-ix in gentherapie-vektoren
MX2008000058A (es) * 2005-07-07 2008-03-19 Genzyme Corp Vectores aav que codifican superoxido dismutasa.
US7943374B2 (en) * 2005-08-21 2011-05-17 Markus Hildinger Super-size adeno-associated viral vector harboring a recombinant genome larger than 5.7 kb
EP1981548A4 (de) * 2006-01-20 2010-03-24 Univ North Carolina Verstärkte produktion von infektiösen parvovirus-vektoren in insektenzellen
EP1979485A2 (de) 2006-01-31 2008-10-15 The Board Of Trustees Of The Leland Stanford Junior University Selbstkomplementäre parvovirale vektoren und verfahren zur herstellung und verwendung davon
US7989606B2 (en) 2006-02-10 2011-08-02 The University Of Cincinnati Phosphatase inhibitor protein-1 as a regulator of cardiac function
EP2007795B1 (de) 2006-03-30 2016-11-16 The Board Of Trustees Of The Leland Stanford Junior University Aav-capsid-proteine
JP2009534394A (ja) * 2006-04-21 2009-09-24 ザ・ユニヴァーシティ・オヴ・ノース・キャロライナ・アト・チャペル・ヒル 結合組織障害の治療
US20070280906A1 (en) * 2006-06-03 2007-12-06 Ognjen Petras Method to generate mirrored adenoassociated viral vectors
CN104087591A (zh) 2006-06-19 2014-10-08 阿斯克肋匹奥生物制药公司 用于基因治疗的修饰的因子viii和因子ix基因和载体
US20100111873A1 (en) * 2007-02-20 2010-05-06 Russell Stephen J Treating cancer with viral nucleic acid
EP2019143A1 (de) * 2007-07-23 2009-01-28 Genethon Gentherapie des ZNS mit peripher verabreichten AAV-Vektoren
CA2694091A1 (en) * 2007-10-01 2009-04-09 Alcon Research, Ltd. Self complementary aav-mediated delivery of interfering rna molecules to treat or prevent ocular disorders
EP2058401A1 (de) 2007-10-05 2009-05-13 Genethon Weitverbreitete Genverabreichung an Motorneuronen unter Verwendung einer peripheren Injektion von AAV-Vektoren
CN101173294B (zh) * 2007-10-12 2011-01-26 中山大学 一种基因微链载体及其构建方法和应用
WO2010093784A2 (en) 2009-02-11 2010-08-19 The University Of North Carolina At Chapel Hill Modified virus vectors and methods of making and using the same
JP2012521750A (ja) 2009-03-27 2012-09-20 プロイェクト、デ、ビオメディシナ、シーマ、ソシエダッド、リミターダ 肝硬変及び肝線維症の治療のための方法及び組成物
MX356669B (es) 2009-05-02 2018-06-08 Genzyme Corp Terapia genica para trastornos neurodegenerativos.
EP2266588A1 (de) * 2009-06-04 2010-12-29 Deutsches Krebsforschungszentrum, Stiftung des öffentlichen Rechts Verfahren zur Krebstherapie basierend auf der gleichzeitigen Verabreichung eines Parvovirus und eines Zytokins
WO2011088081A1 (en) 2010-01-12 2011-07-21 The University Of North Carolina At Chapel Hill Restrictive inverted terminal repeats for viral vectors
WO2011122950A1 (en) 2010-04-01 2011-10-06 Amsterdam Molecular Therapeutics (Amt) Ip B.V. Monomeric duplex aav vectors
EP2591097A1 (de) 2010-07-06 2013-05-15 Novartis AG Immunogene zusammensetzungen aus norovirus und verfahren
DK2673289T3 (da) 2011-02-10 2023-07-24 Univ North Carolina Chapel Hill Virusvektorer med modificerede transduktionsprofiler og fremgangsmåder til fremstilling og anvendelse deraf
EA027259B1 (ru) 2011-04-29 2017-07-31 Селекта Байосайенсиз, Инк. Наноносители, вырабатывающие иммунную толерантность, для антиген-специфического удаления т-эффекторных клеток
US9163259B2 (en) 2012-05-04 2015-10-20 Novartis Ag Viral vectors for the treatment of retinal dystrophy
DK2900686T3 (da) 2012-09-28 2020-08-17 Univ North Carolina Chapel Hill Aav-vektorer targeteret til oligodendrocytter
WO2014071282A1 (en) 2012-11-05 2014-05-08 Genzyme Corporation Compositions and methods for treating proteinopathies
TW201427989A (zh) 2012-12-18 2014-07-16 Novartis Ag 長效性蛋白質之組合物及方法
AU2014243712B2 (en) 2013-03-13 2018-09-13 Genzyme Corporation Fusion proteins comprising PDGF and VEGF binding portions and methods of using thereof
US10385112B2 (en) 2013-03-14 2019-08-20 Asklepios Biopharmaceutical, Inc. Modified soluble VEGF receptor-1 genes and vectors for gene therapy
WO2014143932A1 (en) 2013-03-15 2014-09-18 The University Of North Carolina At Chapel Hill Synthetic adeno-associated virus inverted terminal repeats
IL293294B2 (en) 2013-03-15 2023-04-01 Univ North Carolina Chapel Hill Methods and compounds for ligation of paired aav glycan vectors
US9828582B2 (en) 2013-03-19 2017-11-28 Duke University Compositions and methods for the induction and tuning of gene expression
WO2014172560A1 (en) 2013-04-17 2014-10-23 Genzyme Corporation Compositions and methods for treating and preventing macular degeneration
WO2014178863A1 (en) 2013-05-01 2014-11-06 Genzyme Corporation Compositions and methods for treating spinal muscular atrophy
CN105263478A (zh) 2013-05-03 2016-01-20 西莱克塔生物科技公司 用于诱导免疫耐受的具有特定药效学有效持续期之免疫抑制剂与抗原的递送
DK3013855T3 (da) 2013-06-24 2021-01-11 Xiao Weidong Mutante faktor VIII-sammensætninger og fremgangsmåder
KR102445330B1 (ko) * 2013-07-12 2022-09-19 더 칠드런스 호스피탈 오브 필라델피아 Aav 벡터 및 항―aav (아데노-관련 바이러스) 중화 항체에 대한 검정
US9585971B2 (en) 2013-09-13 2017-03-07 California Institute Of Technology Recombinant AAV capsid protein
US20160243260A1 (en) 2013-10-24 2016-08-25 Uniqure Ip B.V. Treatment of neurological diseases using adeno-associated virus (AAV) comprising AAV-5 capsid proteins
TWI687225B (zh) 2014-02-06 2020-03-11 美商健臻公司 用於治療及預防黃斑部病變的組成物及方法
EP3628334B1 (de) 2014-03-21 2023-06-28 Genzyme Corporation Gentherapie für retinitis pigmentosa
JP6983511B2 (ja) 2014-04-25 2021-12-17 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 脳中の転移乳癌および他の癌を処置するための方法および組成物
KR102863726B1 (ko) 2014-05-02 2025-09-25 젠자임 코포레이션 망막 및 cns 유전자 요법을 위한 aav 벡터
EP3142750B1 (de) 2014-05-13 2020-07-01 The Trustees Of The University Of Pennsylvania Zusammensetzungen mit aav-expression von zwei antikörperkonstrukten und verwendungen davon
EP3154560B1 (de) * 2014-06-13 2019-03-13 University of Pittsburgh - Of the Commonwealth System of Higher Education Verfahren und materialien zur erhöhung der viralen vektorinfektiosität
US20170290876A1 (en) 2014-06-25 2017-10-12 Novartis Ag Compositions and methods for long acting proteins
EP3160991A2 (de) 2014-06-25 2017-05-03 Novartis AG Zusammensetzungen und verfahren für proteine mit langer wirkung
EA201790534A1 (ru) 2014-09-07 2017-07-31 Селекта Байосайенсиз, Инк. Способы и композиции для ослабления иммунных ответов против вирусного вектора для переноса, предназначенного для генной терапии
WO2016037931A1 (en) 2014-09-11 2016-03-17 Fundación Para La Investigación Médica Aplicada Nucleic acid constructs and expression vectors for gene therapy of acute porphyrias and other diseases
EP4012035B1 (de) 2014-09-16 2024-11-06 Genzyme Corporation Adeno-assoziierte virale vektoren zur behandlung von myocilin (myoc)-glaukom
PT4012035T (pt) 2014-09-16 2025-02-11 Genzyme Corp Vetores virais adenoassociados para tratar o glaucoma de miocilina (mioc)
CN107109407A (zh) 2014-11-14 2017-08-29 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
CN114480440A (zh) 2014-11-21 2022-05-13 北卡罗来纳-查佩尔山大学 靶向中枢神经系统的aav载体
WO2016094783A1 (en) 2014-12-12 2016-06-16 Voyager Therapeutics, Inc. Compositions and methods for the production of scaav
EP3245220B1 (de) 2015-01-14 2023-09-20 The University of North Carolina at Chapel Hill Verfahren und zusammensetzungen für gezielten gentransfer
AU2016209471B2 (en) 2015-01-20 2022-03-31 Genzyme Corporation Analytical ultracentrifugation for characterization of recombinant viral particles
EP3256487A4 (de) 2015-02-09 2018-07-18 Duke University Zusammensetzungen und verfahren zur epigenombearbeitung
CA2976082A1 (en) 2015-02-10 2016-08-18 Genzyme Corporation Enhanced delivery of viral particles to the striatum and cortex
MX2017010369A (es) 2015-02-10 2017-12-14 Genzyme Corp Arni variante.
TWI707951B (zh) 2015-04-08 2020-10-21 美商健臻公司 過大腺相關載體之製造
US10016514B2 (en) 2015-05-15 2018-07-10 New Hope Research Foundation Polynucleotides, vectors and methods for insertion and expression of transgenes
WO2016186772A2 (en) 2015-05-16 2016-11-24 Genzyme Corporation Gene editing of deep intronic mutations
ES2865487T3 (es) 2015-09-28 2021-10-15 Univ North Carolina Chapel Hill Métodos y composiciones para vectores virales que evaden los anticuerpos
BR112018007453A2 (pt) 2015-11-05 2018-10-23 Bamboo Therapeutics Inc genes modificados de ataxia de friedreich e vetores para a terapia gênica
EA201891317A3 (ru) 2015-11-30 2019-04-30 Дьюк Юниверсити Терапевтические мишени для коррекции гена дистрофина человека с помощью редактирования генов и способы их применения
IL259842B (en) 2015-12-11 2022-08-01 California Inst Of Techn Targeted peptides for the targeting of adeno-associated viruses -aavs-
EP3390429B1 (de) 2015-12-14 2025-06-18 The University of North Carolina at Chapel Hill Modifizierte kapsidproteine zur verbesserten abgabe von parvovirus-vektoren
FI3411484T3 (fi) 2016-02-05 2023-11-15 Univ Emory Yksisäikeisen tai itsekomplementaarisen adenoassosioidun viruksen 9 injektio serebrospinaaliseen fluidiin
WO2017139381A1 (en) 2016-02-08 2017-08-17 University Of Iowa Research Foundation Methods to produce chimeric adeno-associated virus/bocavirus parvovirus
BR112018017125A2 (pt) 2016-02-22 2018-12-26 The University Of North Carolina At Chapel Hill vetor aav-idua para tratamento de cegueira associada a mps i
IL261642B2 (en) 2016-03-07 2026-01-01 Univ Iowa Res Found AAV-mediated expression using a synthetic promoter and enhancer
US20190127713A1 (en) 2016-04-13 2019-05-02 Duke University Crispr/cas9-based repressors for silencing gene targets in vivo and methods of use
JP7125351B2 (ja) 2016-04-14 2022-08-24 2セブンティ バイオ インコーポレイテッド サルベージキメラ抗原受容体システム
US10585103B2 (en) 2016-04-28 2020-03-10 The Trustees Of Indiana University Methods and compositions for resolving components of a virus preparation
US12173305B2 (en) 2016-05-26 2024-12-24 University Of Iowa Research Foundation cis and trans requirements for terminal resolution of human bocavirus 1
JP2019517274A (ja) 2016-06-13 2019-06-24 ザ ユニバーシティ オブ ノース カロライナ アット チャペル ヒルThe University Of North Carolina At Chapel Hill 最適化されたcln1遺伝子および発現カセットおよびそれらの使用
WO2017221145A1 (en) 2016-06-21 2017-12-28 Bamboo Therapeutics, Inc. Optimized mini-dystrophin genes and expression cassettes and their use
WO2018017754A1 (en) 2016-07-19 2018-01-25 Duke University Therapeutic applications of cpf1-based genome editing
BR112019001344A2 (pt) 2016-07-26 2019-04-30 The University Of North Carolina At Chapel Hill tolerância imunológica mediada por vetor no olho
KR102425289B1 (ko) 2016-08-15 2022-07-27 젠자임 코포레이션 Aav의 검출 방법
CA3034094A1 (en) 2016-08-16 2018-02-22 Bluebird Bio, Inc. Il-10 receptor alpha homing endonuclease variants, compositions, and methods of use
WO2018035503A1 (en) 2016-08-18 2018-02-22 The Regents Of The University Of California Crispr-cas genome engineering via a modular aav delivery system
EP3500278B1 (de) 2016-08-19 2024-05-22 University of Florida Research Foundation, Incorporated Zusammensetzungen zur behandlung von erkrankungen mit rekombinantem selbstkomplementärem adeno-assoziiertem virus
MA46059A (fr) 2016-08-23 2019-07-03 Bluebird Bio Inc Variants de l'endonucléase homing tim3, compositions et procédés d'utilisation
WO2018049226A1 (en) 2016-09-08 2018-03-15 Bluebird Bio, Inc. Pd-1 homing endonuclease variants, compositions, and methods of use
US11530395B2 (en) 2016-10-17 2022-12-20 2Seventy Bio, Inc. TGFBetaR2 endonuclease variants, compositions, and methods of use
WO2018094244A1 (en) 2016-11-17 2018-05-24 Bluebird Bio, Inc. TGFβ SIGNAL CONVERTOR
CA3046347A1 (en) 2016-12-07 2018-06-14 University Of Florida Research Foundation, Incorporated Il-1ra cdnas
CA3045551A1 (en) 2016-12-16 2018-06-21 Uniqure Ip B.V. Immunoadsorption
KR20190104194A (ko) 2017-01-07 2019-09-06 셀렉타 바이오사이언시즈, 인크. 합성 나노담체에 커플링된 면역억제제의 패턴화된 투여
WO2018132747A1 (en) 2017-01-13 2018-07-19 University Of Iowa Research Foundation Bocaparvovirus small noncoding rna and uses thereof
DK3583203T5 (da) 2017-02-15 2024-09-02 2Seventy Bio Inc Donorreparationstemplates multiplex-genomeditering
US11426451B2 (en) 2017-03-11 2022-08-30 Selecta Biosciences, Inc. Methods and compositions related to combined treatment with antiinflammatories and synthetic nanocarriers comprising an immunosuppressant
CN110770346B (zh) 2017-03-15 2024-01-12 北卡罗来纳-查佩尔山大学 多倍体腺相关病毒载体及其制备和使用方法
DK3601367T3 (da) 2017-03-30 2025-01-13 Univ Queensland Kimære molekyler og anvendelse deraf
CA3058970A1 (en) * 2017-04-18 2018-10-25 Glaxosmithkline Intellectual Property Development Limited Methods for adeno-associated viral vector production
AU2018261790B2 (en) 2017-05-05 2024-10-03 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (ALS)
IL313037A (en) 2017-05-25 2024-07-01 2Seventy Bio Inc Cblb endonuclease variants, compositions, and methods of use
US12076369B2 (en) 2017-09-01 2024-09-03 The Frances Crick Institute Limited Immunoregulatory molecules and uses therefor
WO2019060538A1 (en) 2017-09-20 2019-03-28 The Trustees Of Indiana University METHODS FOR LIPOPROTEIN RESOLUTION BY MASS SPECTROMETRY
AU2018335410B2 (en) 2017-09-22 2025-04-03 Genzyme Corporation Variant RNAi
US11779654B2 (en) 2017-10-04 2023-10-10 2Seventy Bio, Inc. PCSK9 endonuclease variants, compositions, and methods of use
MX2020003838A (es) 2017-10-13 2020-08-06 Selecta Biosciences Inc Metodos y composiciones para atenuar las respuestas de inmunoglobulina m del vector de transferencia antiviral.
WO2019079240A1 (en) 2017-10-16 2019-04-25 Voyager Therapeutics, Inc. TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS)
BR112020008835A2 (pt) 2017-11-07 2020-10-20 The University Of North Carolina At Chapel Hill genes de aga otimizados e cassetes de expressão e seu uso
US11232941B2 (en) 2018-01-12 2022-01-25 The Trustees Of Indiana University Electrostatic linear ion trap design for charge detection mass spectrometry
EP3740580A4 (de) 2018-01-19 2021-10-20 Duke University Genom-engineering mit crispr-cas-systemen bei eukaryonten
BR112020015798A2 (pt) 2018-02-01 2021-03-09 Homology Medicines, Inc. Composições de vírus adeno-associado para restaurar função de gene da pah e métodos de uso das mesmas
US10610606B2 (en) 2018-02-01 2020-04-07 Homology Medicines, Inc. Adeno-associated virus compositions for PAH gene transfer and methods of use thereof
WO2019155833A1 (ja) * 2018-02-07 2019-08-15 学校法人日本医科大学 改良型アデノ随伴ウイルスベクター
MX2020008470A (es) * 2018-02-14 2020-09-25 Generation Bio Co Vectores de adn no virales y usos de estos para la produccion de anticuerpos y proteinas de fusion.
EP3755795A4 (de) 2018-02-19 2022-07-20 Homology Medicines, Inc. Adeno-assoziierte viruszusammensetzungen zur wiederherstellung der f8-genfunktion und verfahren zur verwendung davon
AU2019225251A1 (en) 2018-02-26 2020-10-15 AnTolRx, Inc. Tolerogenic liposomes and methods of use thereof
BR112020016699A2 (pt) 2018-02-28 2020-12-15 The University Of North Carolina At Chapel Hill Métodos e composições para vetores de vírus de evasão de anticorpos
SG11202009450SA (en) 2018-04-03 2020-10-29 Stridebio Inc Virus vectors for targeting ophthalmic tissues
SG11202009451VA (en) 2018-04-03 2020-10-29 Stridebio Inc Antibody-evading virus vectors
CN112272672A (zh) 2018-04-03 2021-01-26 斯特里迪比奥公司 抗体逃避性病毒载体
WO2019236143A1 (en) 2018-06-04 2019-12-12 The Trustees Of Indiana University Apparatus and method for calibrating or resetting a charge detector
WO2019236139A1 (en) 2018-06-04 2019-12-12 The Trustees Of Indiana University Interface for transporting ions from an atmospheric pressure environment to a low pressure environment
US11315780B2 (en) 2018-06-04 2022-04-26 The Trustees Of Indiana University Charge detection mass spectrometry with real time analysis and signal optimization
AU2019281255B2 (en) 2018-06-04 2023-01-12 The Trustees Of Indiana University Ion trap array for high throughput charge detection mass spectrometry
CN119242659A (zh) 2018-06-12 2025-01-03 北卡罗来纳大学教堂山分校 合成嗜肝性腺相关病毒衣壳及其用途
KR102887853B1 (ko) 2018-06-14 2025-11-19 리제너론 파마슈티칼스 인코포레이티드 Cd79a 키메라 항원 수용체
US20210269828A1 (en) 2018-06-22 2021-09-02 Asklepios Biopharmaceutical, Inc. Vectors for gene delivery that persist within cells
FI3814513T3 (fi) 2018-06-28 2025-05-28 Univ North Carolina Chapel Hill Optimoidut cln5-geenit ja -ilmentymiskasetit sekä niiden käyttö
JP2021530571A (ja) 2018-07-16 2021-11-11 セレクタ バイオサイエンシーズ インコーポレーテッドSelecta Biosciences, Inc. Mmaコンストラクトおよびベクターの方法および組成物
KR20210032438A (ko) 2018-07-16 2021-03-24 셀렉타 바이오사이언시즈, 인크. Otc 구축물 및 벡터의 방법 및 조성물
AU2019314529B2 (en) 2018-08-03 2025-09-11 Genzyme Corporation Variant RNAi against alpha-synuclein
IL280300B2 (en) 2018-08-10 2025-06-01 Univ North Carolina Chapel Hill Optimized cln7 genes and expression cassettes and their use
SG11202102058UA (en) 2018-08-30 2021-03-30 Tenaya Therapeutics Inc Cardiac cell reprogramming with myocardin and ascl1
CA3114199A1 (en) 2018-09-26 2020-04-02 California Institute Of Technology Adeno-associated virus compositions for targeted gene therapy
CN113383010B (zh) 2018-09-28 2025-09-09 沃雅戈治疗公司 具有经工程化改造的启动子的共济蛋白表达构建体及其使用方法
WO2020072059A1 (en) 2018-10-04 2020-04-09 Bluebird Bio, Inc. Cblb endonuclease variants, compositions, and methods of use
AU2019357039B2 (en) 2018-10-12 2025-10-09 Genzyme Corporation Generation of improved human pah for treatment of severe PKU by liver-directed gene replacement therapy
JP7578590B2 (ja) 2018-10-18 2024-11-06 インテリア セラピューティクス,インコーポレーテッド 第ix因子を発現するための組成物及び方法
CN113195001B (zh) * 2018-11-02 2025-04-29 耐克基因有限责任公司 重组细小病毒载体及其制备方法和用途
BR112021006074A2 (pt) 2018-11-05 2021-07-20 The University Of North Carolina At Chapel Hill genes e cassetes de expressão fig4 otimizados e seu uso
EP3884510A1 (de) 2018-11-20 2021-09-29 The Trustees of Indiana University Orbitrap für einzelteilchen-massenspektrometrie
WO2020112853A1 (en) 2018-11-28 2020-06-04 Genascence Corporation Methods and compositions for treating osteoarthritis
AU2019392058B2 (en) 2018-12-03 2024-12-05 The Trustees Of Indiana University Apparatus and method for simultaneously analyzing multiple ions with an electrostatic linear ion trap
US12577547B2 (en) 2018-12-10 2026-03-17 Regeneron Pharmaceuticals, Inc. PDCD-1 homing endonuclease variants
AU2019397364B8 (en) 2018-12-10 2025-08-07 Novo Nordisk A/S Homing endonuclease variants
EP3895052A4 (de) 2018-12-14 2022-10-26 2seventy bio, Inc. Dimerisierungsmittelregulierte immunorezeptorkomplexe
WO2020132385A1 (en) 2018-12-21 2020-06-25 The University Of North Carolina At Chapel Hill Optimized galc genes and expression cassettes and their use
WO2020142714A1 (en) 2019-01-04 2020-07-09 Exonics Therapeutics, Inc. Aav expression cassette and aav vectors comprising the same
WO2020174369A2 (en) 2019-02-25 2020-09-03 Novartis Ag Compositions and methods to treat bietti crystalline dystrophy
CN113677801A (zh) 2019-02-25 2021-11-19 诺华股份有限公司 治疗bietti晶体营养不良的组合物和方法
SG11202110146VA (en) 2019-03-21 2021-10-28 Stridebio Inc Recombinant adeno-associated virus vectors
JP7630176B2 (ja) 2019-04-01 2025-02-17 テナヤ セラピューティクス, インコーポレイテッド 操作されたカプシドを有するアデノ随伴ウイルス
WO2020219527A1 (en) 2019-04-23 2020-10-29 The Trustees Of Indiana University Identification of sample subspecies based on particle charge behavior under structural change-inducing sample conditions
WO2020219656A1 (en) 2019-04-26 2020-10-29 The University Of North Carolina At Chapel Hill Methods and compositions for dual glycan binding aav2.5 vector
WO2020223205A1 (en) 2019-04-28 2020-11-05 Selecta Biosciences, Inc. Methods for treatment of subjects with preexisting immunity to viral transfer vectors
CA3144654A1 (en) 2019-04-29 2020-11-05 The University Of North Carolina At Chapel Hill Optimized sumf1 genes and expression cassettes and their use
WO2020237130A1 (en) 2019-05-22 2020-11-26 The University Of North Carolina At Chapel Hill Ube3a genes and expression cassettes and their use
CA3141863A1 (en) 2019-05-28 2020-12-03 Selecta Biosciences, Inc. Methods and compositions for attenuated anti-viral transfer vector immune response
CN114207440A (zh) 2019-06-04 2022-03-18 西莱克塔生物科技公司 聚乙二醇化尿酸酶的制剂和剂量
CA3144864A1 (en) 2019-06-27 2020-12-30 Pfizer Inc. Methods of treating duchenne muscular dystrophy using aav mini-dystrophin gene therapy
WO2021007515A1 (en) 2019-07-11 2021-01-14 Tenaya Therapeutics, Inc. Cardiac cell reprogramming with micrornas and other factors
CN120109003A (zh) 2019-09-25 2025-06-06 印地安纳大学理事会 用于脉冲模式电荷检测质谱法的设备和方法
US20240060989A1 (en) 2019-10-04 2024-02-22 Novartis Ag Methods for measuring cralbp activity
JP7659325B2 (ja) 2019-10-10 2025-04-09 ザ・トラスティーズ・オブ・インディアナ・ユニバーシティー 粒子を識別、選択、および純化するためのシステムおよび方法
US20230392163A1 (en) 2019-10-17 2023-12-07 Ginkgo Bioworks Inc. Adeno-associated viral vectors for treatment of niemann-pick disease type c
US12521451B2 (en) 2019-11-08 2026-01-13 Regeneron Pharmaceuticals, Inc. CRISPR and AAV strategies for x-linked juvenile retinoschisis therapy
CN115038789A (zh) 2019-12-02 2022-09-09 塑造治疗公司 治疗性编辑
JP2023508121A (ja) * 2019-12-17 2023-03-01 ナイキジェン,リミテッド パルボウイルスベクターならびにその作製および使用方法
WO2021126972A1 (en) 2019-12-18 2021-06-24 The Trustees Of Indiana University Mass spectrometer with charge measurement arrangement
TW202140791A (zh) 2020-01-13 2021-11-01 美商霍蒙拉奇醫藥公司 治療苯酮尿症之方法
JP7607355B2 (ja) 2020-02-03 2024-12-27 ザ・トラスティーズ・オブ・インディアナ・ユニバーシティー 電荷検出質量分析用信号の時間ドメイン分析
EP4100532A4 (de) 2020-03-23 2024-03-27 The University of North Carolina at Chapel Hill Aav-naglu-vektoren zur behandlung von mukopolysaccharidose iiib
EP4132955A2 (de) 2020-04-10 2023-02-15 SOLA Biosciences LLC Zusammensetzungen und verfahren zur behandlung von proteinaggregationsstörungen
WO2021221956A1 (en) * 2020-04-27 2021-11-04 The Regents Of The University Of California Compositions and methods for production of recombinant adeno-associated virus
MX2022013288A (es) 2020-04-28 2023-02-22 Sola Biosciences Llc Composiciones y metodos para el tratamiento de proteinopatias por la proteina 43 de union a acido desoxirribonucleico (adn) de respuesta transactiva (tdp-43).
EP4125977A4 (de) 2020-05-05 2024-09-25 Duke University Speziesüberkreuzende kompatible adeno-assoziierte viruszusammensetzungen und verfahren zur verwendung davon
MX2022013819A (es) 2020-05-05 2023-01-30 Univ North Carolina Chapel Hill Capsides de virus adeno-asociado 5 modificado y usos del mismo.
TW202208632A (zh) 2020-05-27 2022-03-01 美商同源醫藥公司 用於恢復pah基因功能的腺相關病毒組成物及其使用方法
EP4161953A1 (de) 2020-06-05 2023-04-12 SOLA Biosciences LLC Zusammensetzungen und verfahren zur behandlung von synukleinopathien
CN116323664A (zh) 2020-07-16 2023-06-23 诺华股份有限公司 抗β细胞素抗体、其片段和多特异性结合分子
ES3057565T3 (en) 2020-07-23 2026-03-03 Univ North Carolina Chapel Hill Optimized slc13a5 genes and expression cassettes and their use
US20230285596A1 (en) 2020-07-27 2023-09-14 Voyager Therapeutics, Inc Compositions and methods for the treatment of niemann-pick type c1 disease
EP4189095A1 (de) 2020-07-27 2023-06-07 Voyager Therapeutics, Inc. Zusammensetzungen und verfahren zur behandlung von neurologischen erkrankungen im zusammenhang mit glucosylceramidase-beta-mangel
CN116745409A (zh) 2020-08-19 2023-09-12 萨雷普塔治疗公司 用于治疗雷特综合征的腺相关病毒载体
EP4222251A1 (de) 2020-10-01 2023-08-09 Genzyme Corporation Menschliche pah-expressionskassette zur behandlung von pku durch lebergerichtete genersatztherapie
US20230374542A1 (en) 2020-10-07 2023-11-23 Asklepios Biopharmaceutical, Inc. Therapeutic adeno-associated virus delivery of fukutin related protein (fkrp) for treating dystroglycanopathy. disorders including limb girdle 21 (lgmd21)
WO2022076648A1 (en) 2020-10-09 2022-04-14 Tenaya Therapeutics, Inc. Plakophillin-2 gene therapy methods and compositions
US11781156B2 (en) 2020-10-09 2023-10-10 Tenaya Therapeutics, Inc. Plakophillin-2 gene therapy methods and compositions
AU2021367952A1 (en) 2020-10-26 2023-06-08 Sola Biosciences Llc Compositions and methods for the treatment of alzheimer's disease
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
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
EP4240861A1 (de) 2020-11-03 2023-09-13 Pfizer Inc. Verfahren zur reinigung von aav-vektoren durch anionenaustauschchromatografie
MX2023007612A (es) 2020-12-23 2023-07-12 Pfizer Metodos para purificacion de vectores aav por cromatografia de afinidad.
CA3207247A1 (en) 2021-01-05 2022-07-14 Selecta Biosciences, Inc. Viral vector dosing protocols
TW202242124A (zh) 2021-01-14 2022-11-01 美商史崔德生物公司 靶向t細胞之aav載體
US11760788B2 (en) 2021-03-02 2023-09-19 Pathways Neuro Pharma, Inc. Neuroreceptor compositions and methods of use
JP2024511409A (ja) 2021-03-22 2024-03-13 ジェンザイム・コーポレーション 空および完全aavキャプシドのサイズ排除クロマトグラフィー解析
CN117157316A (zh) 2021-03-30 2023-12-01 艾诺健基因治疗股份有限公司 修饰的血浆凝血因子viii及其使用方法
US11795207B2 (en) 2021-03-30 2023-10-24 AAVnerGene Inc. Modified plasma clotting factor VIII and method of use thereof
JP2024515626A (ja) 2021-04-16 2024-04-10 アスクレピオス バイオファーマシューティカル, インコーポレイテッド 血液脳関門を横断し低減された液性応答を惹起する合理的ポリプロイドaavビリオン
CN117242183A (zh) 2021-04-27 2023-12-15 4D分子治疗有限公司 用于治疗与血管生成相关的眼部疾病的组合物和方法
EP4359547A1 (de) 2021-06-22 2024-05-01 Pfizer Inc. Herstellung eines adeno-assoziierten virusvektors in insektenzellen
WO2022272296A2 (en) 2021-06-25 2022-12-29 Homology Medicines, Inc. Adeno-associated virus packaging systems
US20240342215A1 (en) 2021-07-14 2024-10-17 Regeneron Pharmaceuticals, Inc. Engineered t cell receptors fused to binding domains from antibodies
CA3227105A1 (en) 2021-07-30 2023-02-02 Tune Therapeutics, Inc. Compositions and methods for modulating expression of methyl-cpg binding protein 2 (mecp2)
EP4377459A2 (de) 2021-07-30 2024-06-05 Tune Therapeutics, Inc. Zusammensetzungen und verfahren zur modulation der expression von frataxin (fxn)
US20230076072A1 (en) 2021-08-24 2023-03-09 Oxford Biomedica Solutions Llc Adeno-associated virus formulations
JP2024537178A (ja) 2021-10-08 2024-10-10 ソラ・バイオサイエンシズ・エルエルシー p53媒介性癌の処置のための組成物および方法
US20250042958A1 (en) 2021-10-08 2025-02-06 Sola Biosciences Llc Compositions and methods for the treatment of proteopathies
US20230141563A1 (en) 2021-10-12 2023-05-11 Selecta Biosciences, Inc. Methods and compositions for attenuating anti-viral transfer vector igm responses
WO2023064350A1 (en) 2021-10-12 2023-04-20 Selecta Biosciences, Inc. Viral vector dosing protocols
AU2022368907A1 (en) 2021-10-20 2024-05-02 University Of Copenhagen Rejuvenation treatment of age-related white matter loss
US20230183659A1 (en) 2021-10-29 2023-06-15 Homology Medicines, Inc. Methods and compositions for the purification of adeno-associated virus
JP2024542015A (ja) 2021-11-02 2024-11-13 ユニバーシティ オブ ロチェスター 脳内でのtcf7l2媒介性髄鞘再生
CA3237037A1 (en) 2021-11-14 2023-05-19 Cartesian Therapeutics, Inc. Multiple dosing with viral vectors
EP4433490A2 (de) 2021-11-17 2024-09-25 Voyager Therapeutics, Inc. Zusammensetzungen und verfahren zur behandlung von neurologischen erkrankungen im zusammenhang mit glucosylceramidase-beta-mangel
WO2023114816A1 (en) 2021-12-14 2023-06-22 Neurogene, Inc. Recombinant optimized galc constructs and methods for treating galc-associated disorders
US20230279433A1 (en) 2021-12-15 2023-09-07 Oxford Biomedica Solutions Llc Methods and compositions for the production of adeno-associated virus
CA3248631A1 (en) 2022-01-21 2023-07-27 AstraZeneca Ireland Limited GENE THERAPY FOR GAUCHER'S DISEASE
GB202201242D0 (en) 2022-01-31 2022-03-16 Univ Edinburgh Recombinant optimized mecp2 cassettes and methods for treating rett syndrome and related disorders
WO2023150687A1 (en) 2022-02-04 2023-08-10 Ginkgo Bioworks, Inc. Recombinant adeno-associated virus vectors, and methods of use thereof
US20230357437A1 (en) 2022-03-09 2023-11-09 Selecta Biosciences, Inc. Immunosuppressants in combination with anti-igm agents and related dosing
CN119836431A (zh) 2022-04-04 2025-04-15 维克多瑞私人有限公司 重组aav衣壳蛋白
EP4504936A1 (de) 2022-04-06 2025-02-12 Genzyme Corporation Gezielte gentherapie für dm-1-myotone dystrophie
JP2025513801A (ja) 2022-04-08 2025-04-30 リジェネロン・ファーマシューティカルズ・インコーポレイテッド 多部位受容体及びシグナル伝達複合体
EP4508062A1 (de) 2022-04-11 2025-02-19 Tenaya Therapeutics, Inc. Adeno-assoziiertes virus mit manipuliertem kapsid
JP2025513850A (ja) 2022-04-12 2025-04-30 ジェンザイム・コーポレーション 遺伝子治療のためのirak4モジュレーターの使用
AU2023254837A1 (en) 2022-04-12 2024-11-28 Genzyme Corporation Dendritic cell assay for innate immunogenicity to gene therapy agents
WO2023201274A1 (en) 2022-04-12 2023-10-19 Genzyme Corporation Use of an irak4 modulator for gene therapy
GB202206336D0 (en) 2022-04-29 2022-06-15 Univ Edinburgh Recombinant therapeutic FMR1 constructs and methods of treating fragile X syndrome and related disorders
JP2025521111A (ja) 2022-05-16 2025-07-08 ジェンザイム・コーポレーション 異染性白質ジストロフィーを治療する方法
CN119585438A (zh) 2022-05-26 2025-03-07 再生元制药公司 用于维持慢病毒载体的组合物及其用途
WO2023240236A1 (en) 2022-06-10 2023-12-14 Voyager Therapeutics, Inc. Compositions and methods for the treatment of spinal muscular atrophy related disorders
CN119630689A (zh) 2022-06-14 2025-03-14 维克多瑞私人有限公司 重组aav衣壳蛋白
WO2023248008A2 (en) 2022-06-20 2023-12-28 Vectory B.V. Degron fusion constructs
WO2024003687A1 (en) 2022-06-28 2024-01-04 Pfizer Inc. Nucleic acids encoding acid alpha-glucosidase (gaa) and vectors for gene therapy
EP4554967A2 (de) 2022-07-12 2025-05-21 Tune Therapeutics, Inc. Zusammensetzungen, systeme und verfahren zur gezielten transkriptionsaktivierung
US20240148841A1 (en) 2022-08-11 2024-05-09 Selecta Biosciences Inc. Compositions and methods related to immunoglobulin proteases and fusions thereof
WO2024038365A1 (en) 2022-08-16 2024-02-22 Pfizer Inc. Methods for purification of aav vectors by anion exchange chromatography
WO2024075012A1 (en) 2022-10-06 2024-04-11 Pfizer Inc. Improved host cells for aav vector production
EP4615518A1 (de) 2022-11-13 2025-09-17 Alexion Pharma International Operations Limited Gentherapie für frontotemporale demenz
WO2024124019A2 (en) 2022-12-07 2024-06-13 Ginkgo Bioworks, Inc. Aav vectors targeting hematopoietic stem cells
WO2024163678A2 (en) 2023-02-01 2024-08-08 Tune Therapeutics, Inc. Fusion proteins and systems for targeted activation of frataxin (fxn) and related methods
WO2024163683A2 (en) 2023-02-01 2024-08-08 Tune Therapeutics, Inc. Systems, compositions, and methods for modulating expression of methyl-cpg binding protein 2 (mecp2) and x-inactive specific transcript (xist)
EP4658294A2 (de) 2023-02-02 2025-12-10 University of Rochester Kompetitiver ersatz von gliazellen
WO2024163012A1 (en) 2023-02-02 2024-08-08 Voyager Therapeutics, Inc. Compositions and methods for the treatment of neurological disorders related to glucosylceramidase beta deficiency
WO2024200573A1 (en) 2023-03-27 2024-10-03 LAVA Therapeutics N.V. Nectin-4 binding agents and methods of use
CN121002180A (zh) 2023-04-05 2025-11-21 建新公司 Dm-1肌强直性营养不良的靶向基因疗法
WO2024215653A1 (en) 2023-04-10 2024-10-17 Tenaya Therapeutics, Inc. Guide rnas, vectors, and virions for targeting mutations in the pln gene
WO2024215655A1 (en) 2023-04-10 2024-10-17 Tenaya Therapeutics, Inc. Cardioprotective bag3 therapies
WO2024229173A2 (en) 2023-05-03 2024-11-07 Voyager Therapeutics, Inc. Compositions and methods for the treatment of disorders related to ataxin-2
PE20260124A1 (es) 2023-05-03 2026-01-16 Voyager Therapeutics Inc Composiciones y metodos para el tratamiento de trastornos relacionados con la deficiencia de cdkl5
WO2024229164A2 (en) 2023-05-03 2024-11-07 Voyager Therapeutics, Inc. Compositions and methods for the treatment of disorders related to dystrophia myotonica protein kinase
WO2025012118A2 (en) 2023-07-07 2025-01-16 LAVA Therapeutics N.V. 5t4 binding agents and methods of use
WO2025038796A1 (en) 2023-08-16 2025-02-20 Voyager Therapeutics, Inc. Compositions and methods for the treatment of disorders related to cdkl5 deficiency
WO2025038795A1 (en) 2023-08-16 2025-02-20 Voyager Therapeutics, Inc. Compositions and methods for the treatment of disorders related to dystrophia myotonica protein kinase
WO2025038802A1 (en) 2023-08-16 2025-02-20 Voyager Therapeutics, Inc. Compositions and methods for the treatment of disorders related to ataxin-2
WO2025038800A1 (en) 2023-08-16 2025-02-20 Voyager Therapeutics, Inc. Compositions and methods for the treatment of disorders related to frataxin deficiency
WO2025038805A1 (en) 2023-08-16 2025-02-20 Voyager Therapeutics, Inc. Compositions and methods for the treatment of disorders related to glucosylceramidase beta 1 deficiency
AU2024332181A1 (en) 2023-08-31 2026-02-12 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
TW202540434A (zh) 2023-11-15 2025-10-16 美商健臻公司 治療神經退化性疾病之方法
TW202540435A (zh) 2023-11-15 2025-10-16 美商健臻公司 治療高雪氏症和gba-帕金森氏症的方法
WO2025122548A1 (en) 2023-12-05 2025-06-12 Voyager Therapeutics, Inc. Compositions and methods for the treatment of disorders related to cdkl5 deficiency
WO2025122543A1 (en) 2023-12-05 2025-06-12 Voyager Therapeutics, Inc. Compositions and methods for the treatment of disorders related to syntaxin-binding protein 1 deficiency
WO2025122530A1 (en) 2023-12-05 2025-06-12 Voyager Therapeutics, Inc. Compositions and methods for the treatment of disorders related to glucosylceramidase beta 1 deficiency
AR134556A1 (es) 2023-12-05 2026-01-28 Voyager Therapeutics Inc Composiciones y métodos para el tratamiento de trastornos relacionados con la proteína quinasa de la distrofia miotónica
WO2025122532A1 (en) 2023-12-05 2025-06-12 Voyager Therapeutics, Inc. Compositions and methods for the treatment of disorders related to ataxin-2
WO2025128817A1 (en) 2023-12-15 2025-06-19 Genzyme Corporation Artificial micrornas targeting tau
WO2025137219A1 (en) 2023-12-21 2025-06-26 Voyager Therapeutics, Inc. Compositions and methods for the treatment of disorders related to dystrophia myotonica protein kinase
WO2025153530A1 (en) 2024-01-16 2025-07-24 Novo Nordisk A/S Albumin-targeted endonucleases, compositions, and methods of use
WO2025158385A1 (en) 2024-01-25 2025-07-31 Genzyme Corporation Pegylated il-2 for suppressing adaptive immune response to gene therapy
WO2025160452A1 (en) 2024-01-25 2025-07-31 Genzyme Corporation Methods for fus-based delivery of viral particles to the brain
WO2025160429A1 (en) 2024-01-26 2025-07-31 Genzyme Corporation Artificial micrornas targeting snca
WO2025160434A1 (en) 2024-01-26 2025-07-31 Genzyme Corporation Artificial micrornas targeting huntington's disease
WO2025235643A1 (en) 2024-05-08 2025-11-13 Genzyme Corporation Profiling of gene therapy agents
WO2025250457A1 (en) 2024-05-28 2025-12-04 University Of Rochester Enhanced brain transduction by gene therapeutics
WO2025250909A1 (en) 2024-05-31 2025-12-04 Sarepta Therapeutics, Inc. Muscle-tropic recombinant aav
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

Family Cites Families (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5869305A (en) 1992-12-04 1999-02-09 The University Of Pittsburgh Recombinant viral vector system
US6281010B1 (en) 1995-06-05 2001-08-28 The Trustees Of The University Of Pennsylvania Adenovirus gene therapy vehicle and cell line
JP2001522244A (ja) 1997-04-24 2001-11-13 ユニバーシティ オブ ワシントン パルボウイルスベクターによる標的化した遺伝子改変
US6156303A (en) 1997-06-11 2000-12-05 University Of Washington Adeno-associated virus (AAV) isolates and AAV vectors derived therefrom
IL121676A0 (en) 1997-09-01 1998-02-22 Yeda Res & Dev Parvovirus vectors
WO2000024917A1 (en) 1998-10-28 2000-05-04 University Of Washington Targeted gene modification by parvoviral vectors
CA2309313A1 (en) 1999-08-05 2001-02-05 Board Of Trustees Of The University Of Arkansas Stimulation of the immune response by human dendritic cell manipulation
WO2001011034A2 (en) * 1999-08-09 2001-02-15 Targeted Genetics Corporation Enhancement of expression of a single-stranded, heterologous nucleotide sequence from recombinant viral vectors by designing the sequence such that it forms intrastrand base pairs

Also Published As

Publication number Publication date
AU2001269723B2 (en) 2006-06-29
WO2001092551A2 (en) 2001-12-06
DE60117550T2 (de) 2006-12-07
CA2410828C (en) 2012-01-24
US7790154B2 (en) 2010-09-07
JP2004500858A (ja) 2004-01-15
AU2001269723B9 (en) 2006-11-16
US20040029106A1 (en) 2004-02-12
US20130252325A1 (en) 2013-09-26
US20100310516A1 (en) 2010-12-09
AU6972301A (en) 2001-12-11
EP1290205A2 (de) 2003-03-12
US8784799B2 (en) 2014-07-22
US7465583B2 (en) 2008-12-16
US20070110724A1 (en) 2007-05-17
WO2001092551A3 (en) 2002-08-01
DE60117550D1 (de) 2006-04-27
ES2505700T3 (es) 2014-10-10
JP4860886B2 (ja) 2012-01-25
US8361457B2 (en) 2013-01-29
ES2256265T3 (es) 2006-07-16
NZ522840A (en) 2004-12-24
CA2410828A1 (en) 2001-12-06
EP1290205B1 (de) 2006-03-01

Similar Documents

Publication Publication Date Title
ATE318923T1 (de) Doppelsträngige parvovirus-vektoren
WO2003023015A3 (en) Method for expression of small antiviral rna molecules within a cell
WO2002002624A3 (en) B7-like molecules and uses thereof
DE60042969D1 (de) Zubereitungen und methoden zur verwendung in rekombinatorischem klonen von nukleinsäuren
WO2002024891A3 (en) B7-like molecules and uses thereof
ATE513843T1 (de) Abstellen von genen in vivo durch chemischmodifizierte und stabile sirna
ATE389016T2 (de) Sirna knockout prüfverfahren und konstrukte
SI2105449T1 (sl) Antagonistična selektivna vezavna sredstva osteoprotegerin-vezavnega proteina
WO2005033321A3 (en) Adeno-associated virus (aav) clades, sequences, vectors containing same, and uses therefor
ATE362542T1 (de) Nukleinsäuresequenzen des adeno-assoziierten virus des serotyps i, und vektoren und wirtszellen, die diese enthalten
BR0106682A (pt) Molécula de anticorpo, variante de molécula de anticorpo, anticorpo, composto, sequência de dna, vetor de clonagem ou de expressão, vetor de expressão de e. coli, célula hospedeira, processo para a produção da molécula de anticorpo, composição terapêutica ou diagnóstica, uso da molécula de anticorpo, vetorpdnabeng-g1, vetorptto(cdp870), e, polipeptìdeo
DE60138715D1 (de) Tnf-rezeptor-ähnliche moleküle und deren anwendungen
WO2002086144A3 (en) Compositions and methods for recombinational cloning of nucleic acid molecules
WO2002074807A3 (en) Chimpanzee erythropoietin (chepo) - immunoadhesins
WO2002027007A3 (en) Production of viral vectors
EP1149917A3 (de) Hepatitis B Vektoren für Gentherapie
MXPA03010728A (es) Moleculas de proteina-2 relacionadas con b7 y uso de las mismas.
EP1626091A3 (de) Doppelsträngige Parvovirus-Vektoren
EP1407007A4 (de) Attenuierter circovirus
PL374550A1 (en) Il-17 like molecules and uses thereof
Ghidini et al. Clamping of RNA with PNA enables targeting of microRNA
DE69733948D1 (de) In vivo herstellung von replicativen molekülen
IL169256A0 (en) Metalloprotease proteins
WO2002022807A3 (en) Dermatophagoides nucleic acid molecules, proteins and uses thereof
WO2003091393A3 (en) Nucleic “acid molecules encoding human proteins, and uses thereof”

Legal Events

Date Code Title Description
RER Ceased as to paragraph 5 lit. 3 law introducing patent treaties