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BR112022025586A2 - Liberação de vetor de vírus adenoassociado para distrofias musculares - Google Patents

Liberação de vetor de vírus adenoassociado para distrofias musculares

Info

Publication number
BR112022025586A2
BR112022025586A2 BR112022025586A BR112022025586A BR112022025586A2 BR 112022025586 A2 BR112022025586 A2 BR 112022025586A2 BR 112022025586 A BR112022025586 A BR 112022025586A BR 112022025586 A BR112022025586 A BR 112022025586A BR 112022025586 A2 BR112022025586 A2 BR 112022025586A2
Authority
BR
Brazil
Prior art keywords
adeno
associated virus
muscular dystrophies
virus vector
release
Prior art date
Application number
BR112022025586A
Other languages
English (en)
Inventor
Rodino-Klapac Louise
R Mendell Jerry
Peterson Ellyn
Potter Rachael
Griffin Danielle
Original Assignee
Res Inst Nationwide Childrens Hospital
Sarepta Therapeutics Inc
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 Res Inst Nationwide Childrens Hospital, Sarepta Therapeutics Inc filed Critical Res Inst Nationwide Childrens Hospital
Publication of BR112022025586A2 publication Critical patent/BR112022025586A2/pt

Links

Classifications

    • 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
    • A61K48/005—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 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/66—Microorganisms or materials therefrom
    • A61K35/76—Viruses; Subviral particles; Bacteriophages
    • A61K35/761—Adenovirus
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K16/00—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies
    • C07K16/18—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans
    • C07K16/28—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants
    • C07K16/2887—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants against CD20
    • 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
    • C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Engineering & Computer Science (AREA)
  • Public Health (AREA)
  • Genetics & Genomics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Epidemiology (AREA)
  • Immunology (AREA)
  • Virology (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Neurology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Biomedical Technology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • General Engineering & Computer Science (AREA)
  • Biophysics (AREA)
  • Mycology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
  • Peptides Or Proteins (AREA)
  • Ultra Sonic Daignosis Equipment (AREA)

Abstract

LIBERAÇÃO DE VETOR DE VÍRUS ADENOASSOCIADO PARA DISTROFIAS MUSCULARES. A presente invenção refere-se a um método de tratamento de distrofia muscular em um indivíduo em necessidade, compreendendo a administração de um vetor de terapia gênica, tal como vetor do vírus adenoassociado (AAV), expressando um gene de microdistrofina humana miniaturizada em combinação com uma etapa de supressão do sistema imunológico do indivíduo.
BR112022025586A 2020-06-15 2021-06-15 Liberação de vetor de vírus adenoassociado para distrofias musculares BR112022025586A2 (pt)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US202063039252P 2020-06-15 2020-06-15
US202063083953P 2020-09-27 2020-09-27
US202163160376P 2021-03-12 2021-03-12
US202163188266P 2021-05-13 2021-05-13
PCT/US2021/037470 WO2021257595A1 (en) 2020-06-15 2021-06-15 Adeno-associated virus vector delivery for muscular dystrophies

Publications (1)

Publication Number Publication Date
BR112022025586A2 true BR112022025586A2 (pt) 2023-03-07

Family

ID=77051123

Family Applications (1)

Application Number Title Priority Date Filing Date
BR112022025586A BR112022025586A2 (pt) 2020-06-15 2021-06-15 Liberação de vetor de vírus adenoassociado para distrofias musculares

Country Status (13)

Country Link
US (1) US20210393801A1 (pt)
EP (1) EP4164668A1 (pt)
JP (1) JP2023530974A (pt)
KR (1) KR20230035043A (pt)
CN (1) CN116348149A (pt)
AU (1) AU2021293197A1 (pt)
BR (1) BR112022025586A2 (pt)
CA (1) CA3187353A1 (pt)
CO (1) CO2023000156A2 (pt)
IL (1) IL299094A (pt)
MX (1) MX2022016332A (pt)
TW (1) TW202208630A (pt)
WO (1) WO2021257595A1 (pt)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
DK3442600T5 (da) * 2016-04-15 2024-09-16 Res Inst Nationwide Childrens Hospital ADENOASSOCIERET VIRUSVEKTORFREMFØRING AF ß-SARCOGLYCAN OG MIKRORNA-29 OG BEHANDLING AF MUSKELDYSTROFI
WO2024006855A1 (en) * 2022-06-30 2024-01-04 Sarepta Therapeutics, Inc. Methods of treating muscular dystrophy
WO2024138158A2 (en) 2022-12-23 2024-06-27 Ultragenyx Pharmaceutical Inc. Method of producing soluble microdystrophin protein and uses of the same
WO2024216080A1 (en) * 2023-04-14 2024-10-17 Biogen Ma Inc. Immunosuppressive regimens and methods thereof

Family Cites Families (82)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5173414A (en) 1990-10-30 1992-12-22 Applied Immune Sciences, Inc. Production of recombinant adeno-associated virus vectors
JPH09509564A (ja) 1993-11-09 1997-09-30 ターゲテッド ジェネティックス コーポレイション 高力価組換えａａｖベクターの生成
CA2176215C (en) 1993-11-09 2007-06-26 James P. Trempe Stable cell lines capable of expressing the adeno-associated virus replication gene
US5658785A (en) 1994-06-06 1997-08-19 Children's Hospital, Inc. Adeno-associated virus materials and methods
US5856152A (en) 1994-10-28 1999-01-05 The Trustees Of The University Of Pennsylvania Hybrid adenovirus-AAV vector and methods of use therefor
WO1996017947A1 (en) 1994-12-06 1996-06-13 Targeted Genetics Corporation Packaging cell lines for generation of high titers of recombinant aav vectors
FR2737730B1 (fr) 1995-08-10 1997-09-05 Pasteur Merieux Serums Vacc Procede de purification de virus par chromatographie
US6143548A (en) 1995-08-30 2000-11-07 Genzyme Corporation Chromatographic purification of adeno-associated virus (AAV)
EP0850313B8 (en) 1995-09-08 2009-07-29 Genzyme Corporation Improved aav vectors for gene therapy
US5910434A (en) 1995-12-15 1999-06-08 Systemix, Inc. Method for obtaining retroviral packaging cell lines producing high transducing efficiency retroviral supernatant
EP0932418B1 (en) 1996-09-06 2007-12-05 The Trustees Of The University Of Pennsylvania Method for recombinant adeno-associated virus-directed gene therapy
US5965542A (en) 1997-03-18 1999-10-12 Inex Pharmaceuticals Corp. Use of temperature to control the size of cationic liposome/plasmid DNA complexes
CA2682108C (en) 1997-09-05 2013-12-24 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of recombinant aav vectors
US6566118B1 (en) 1997-09-05 2003-05-20 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
EP1053023B2 (en) 1998-02-03 2011-03-30 Protiva Biotherapeutics Inc. Systemic delivery of serum stable plasmid lipid particles for cancer therapy
US6410328B1 (en) 1998-02-03 2002-06-25 Protiva Biotherapeutics Inc. Sensitizing cells to compounds using lipid-mediated gene and compound delivery
US6258595B1 (en) 1999-03-18 2001-07-10 The Trustees Of The University Of Pennsylvania Compositions and methods for helper-free production of recombinant adeno-associated viruses
US6211140B1 (en) 1999-07-26 2001-04-03 The Procter & Gamble Company Cationic charge boosting systems
CA2406743A1 (en) 2000-04-28 2001-11-08 The Trustees Of The University Of Pennsylvania Recombinant aav vectors with aav5 capsids and aav5 vectors pseudotyped in heterologous capsids
US6962815B2 (en) 2001-01-05 2005-11-08 Children's Hopital Inc. AAV2 vectors and methods
ES2717377T3 (es) 2001-12-17 2019-06-20 Univ Pennsylvania Secuencias de serotipo 8 de virus adeno-asociado (AAV), vectores que las contienen y usos de las mismas
EP2338478B1 (en) 2002-06-28 2014-07-23 Protiva Biotherapeutics Inc. Method for producing liposomes
SG190613A1 (en) 2003-07-16 2013-06-28 Protiva Biotherapeutics Inc Lipid encapsulated interfering rna
US6927663B2 (en) 2003-07-23 2005-08-09 Cardiac Pacemakers, Inc. Flyback transformer wire attach method to printed circuit board
EP1664316B1 (en) 2003-09-15 2012-08-29 Protiva Biotherapeutics Inc. Polyethyleneglycol-modified lipid compounds and uses thereof
US20070053845A1 (en) 2004-03-02 2007-03-08 Shiladitya Sengupta Nanocell drug delivery system
CA2569664C (en) 2004-06-07 2013-07-16 Protiva Biotherapeutics, Inc. Lipid encapsulated interfering rna
WO2005120152A2 (en) 2004-06-07 2005-12-22 Protiva Biotherapeutics, Inc. Cationic lipids and methods of use
EP1937213B1 (en) 2005-07-27 2017-10-25 Protiva Biotherapeutics Inc. Systems and methods for manufacturing liposomes
US9149564B2 (en) 2006-06-23 2015-10-06 The Regents Of The University Of California Articles comprising large-surface-area bio-compatible materials and methods for making and using them
CA2709875C (en) 2008-01-02 2019-07-16 Tekmira Pharmaceuticals Corporation Improved compositions and methods for the delivery of nucleic acids
AU2009238175C1 (en) 2008-04-15 2023-11-30 Arbutus Biopharma Corporation Novel lipid formulations for nucleic acid delivery
CN102361650B (zh) 2008-11-10 2016-04-20 阿尔尼拉姆医药品有限公司 用于递送治疗剂的脂质和组合物
WO2011000106A1 (en) 2009-07-01 2011-01-06 Protiva Biotherapeutics, Inc. Improved cationic lipids and methods for the delivery of therapeutic agents
CA2767127A1 (en) 2009-07-01 2011-01-06 Protiva Biotherapeutics, Inc. Novel lipid formulations for delivery of therapeutic agents to solid tumors
WO2011004395A1 (en) 2009-07-06 2011-01-13 Intas Biopharmaceuticals Limited Microemulsion formulation for biologicals
WO2011066651A1 (en) 2009-12-01 2011-06-09 Protiva Biotherapeutics, Inc. Snalp formulations containing antioxidants
CA3044884A1 (en) 2009-12-07 2011-06-16 Arbutus Biopharma Corporation Compositions for nucleic acid delivery
WO2011075656A1 (en) 2009-12-18 2011-06-23 The University Of British Columbia Methods and compositions for delivery of nucleic acids
CN103002891B (zh) 2010-05-10 2016-10-26 加利福尼亚大学董事会 比例计量型组合药物递送
EP2569276B1 (en) 2010-05-12 2021-02-24 Arbutus Biopharma Corporation Novel cationic lipids and methods of use thereof
CA2800401C (en) 2010-06-03 2020-09-15 Alnylam Pharmaceuticals, Inc. Biodegradable lipids for the delivery of active agents
US20130323269A1 (en) 2010-07-30 2013-12-05 Muthiah Manoharan Methods and compositions for delivery of active agents
US8466122B2 (en) 2010-09-17 2013-06-18 Protiva Biotherapeutics, Inc. Trialkyl cationic lipids and methods of use thereof
DK2663548T3 (en) 2011-01-11 2017-07-24 Alnylam Pharmaceuticals Inc PEGYLED LIPIDS AND THEIR USE FOR PHARMACEUTICAL SUPPLY
US8691750B2 (en) 2011-05-17 2014-04-08 Axolabs Gmbh Lipids and compositions for intracellular delivery of biologically active compounds
WO2013016058A1 (en) 2011-07-22 2013-01-31 Merck Sharp & Dohme Corp. Novel bis-nitrogen containing cationic lipids for oligonucleotide delivery
US9763891B2 (en) 2011-07-22 2017-09-19 The General Hospital Corporation Therapeutic nanoparticles and methods of use thereof
CA3106285A1 (en) 2011-07-25 2013-01-31 Nationwide Children's Hospital, Inc. Recombinant virus products and methods for inhibition of expression of dux4
EP2760477B1 (en) 2011-09-27 2018-08-08 Alnylam Pharmaceuticals, Inc. Di-aliphatic substituted pegylated lipids
US8762704B2 (en) 2011-09-29 2014-06-24 Apple Inc. Customized content for electronic devices
CA3170051A1 (en) 2011-12-07 2013-06-13 Alnylam Pharmaceuticals, Inc. Branched alkyl and cycloalkyl terminated biodegradable lipids for the delivery of active agents
US20140308304A1 (en) 2011-12-07 2014-10-16 Alnylam Pharmaceuticals, Inc. Lipids for the delivery of active agents
EP3988537A1 (en) 2011-12-07 2022-04-27 Alnylam Pharmaceuticals, Inc. Biodegradable lipids for the delivery of active agents
ES2923757T3 (es) 2011-12-16 2022-09-30 Modernatx Inc Composiciones de ARNm modificado
DE102012007232B4 (de) 2012-04-07 2014-03-13 Susanne Weller Verfahren zur Herstellung von rotierenden elektrischen Maschinen
US9415109B2 (en) 2012-07-06 2016-08-16 Alnylam Pharmaceuticals, Inc. Stable non-aggregating nucleic acid lipid particle formulations
KR101890951B1 (ko) 2012-12-20 2018-08-22 에스케이이노베이션 주식회사 합성 가스 및 고품위 석탄의 동시 생산을 위한 건조 및 가스화 통합 공정
JP2015092462A (ja) 2013-09-30 2015-05-14 Tdk株式会社 正極及びそれを用いたリチウムイオン二次電池
JP6202701B2 (ja) 2014-03-21 2017-09-27 株式会社日立国際電気 基板処理装置、半導体装置の製造方法及びプログラム
SI3766916T1 (sl) 2014-06-25 2023-01-31 Acuitas Therapeutics Inc. Formulacije novih lipidov in lipidnih nanodelcev za dostavo nukleinskih kislin
JP6197169B2 (ja) 2014-09-29 2017-09-20 東芝メモリ株式会社 半導体装置の製造方法
CN107250364A (zh) 2015-01-16 2017-10-13 华盛顿大学 新的微小肌养蛋白及相关使用方法
ES2984981T3 (es) 2015-06-29 2024-10-31 Acuitas Therapeutics Inc Lípidos y formulaciones de nanopartículas lipídicas para el suministro de ácidos nucleicos
US10166298B2 (en) 2015-10-28 2019-01-01 Acuitas Therapeutics, Inc. Lipids and lipid nanoparticle formulations for delivery of nucleic acids
BR112018009657A2 (pt) 2015-11-12 2018-11-13 Research Institute At Nationwide Children's Hospital métodos de tratamento de distrofia muscular
EP3397613A1 (en) 2015-12-30 2018-11-07 Acuitas Therapeutics Inc. Lipids and lipid nanoparticle formulations for delivery of nucleic acids
MA45477A (fr) * 2016-04-15 2019-02-20 Res Inst Nationwide Childrens Hospital Administration à vecteurs de virus adéno-associé de microarn-29 et micro-dystrophine pour traiter la dystrophie musculaire
CA2971303A1 (en) * 2016-06-21 2017-12-21 Bamboo Therapeutics, Inc. Optimized mini-dystrophin genes and expression cassettes and their use
CN107369008A (zh) 2017-07-17 2017-11-21 北京京东金融科技控股有限公司 用于提高票据交易安全性的管理方法、装置及系统
EP3697915A4 (en) * 2017-10-18 2021-12-08 Research Institute at Nationwide Children's Hospital ADENO-ASSOCIATED VIRUS VECTOR ADMINISTRATION SPECIFIC TO MUSCLES TO TREAT MUSCLE DYSTROPHY
WO2019089828A1 (en) 2017-10-31 2019-05-09 Acuitas Therapeutics, Inc. Lamellar lipid nanoparticles
TW201936201A (zh) 2017-12-14 2019-09-16 美商堅固生物科技公司 基因之非病毒生產及遞送
US20200360534A1 (en) * 2018-01-31 2020-11-19 Research Institute At Nationwide Children's Hospital Gene therapy for limb-girdle muscular dystrophy type 2c
WO2019177550A1 (en) 2018-03-10 2019-09-19 Koc Universitesi Therapeutic nanoparticles containing argonaute for microrna delivery and compositions and methods using same
EP3773605A4 (en) 2018-04-03 2022-01-05 The Curators Of The University Of Missouri MODIFIED HINGE-REGION 1 AND / OR 4 DYSTROPHINS FOR DYSTROPHINOPATHY THERAPY
WO2019209777A1 (en) 2018-04-23 2019-10-31 The Curators Of The University Of Missouri Improved crispr therapy
WO2019245973A1 (en) * 2018-06-18 2019-12-26 Research Institute At Nationwide Children's Hospital Adeno-associated virus vector delivery of muscle specific micro-dystrophin to treat muscular dystrophy
AU2019295779A1 (en) 2018-06-29 2021-01-28 Research Institute At Nationwide Children's Hospital Recombinant adeno-associated virus products and methods for treating limb girdle muscular dystrophy 2A
WO2020081938A1 (en) 2018-10-18 2020-04-23 Acuitas Therapeutics, Inc. Lipids for lipid nanoparticle delivery of active agents
CN113646004A (zh) 2018-12-12 2021-11-12 坚固生物科技公司 用于治疗肌营养不良的组合疗法
TW202045531A (zh) 2019-02-26 2020-12-16 美國全美兒童醫院之研究學會 Β-肌聚糖之腺相關病毒載體遞送及肌肉營養不良症之治療

Also Published As

Publication number Publication date
CA3187353A1 (en) 2021-12-23
WO2021257595A1 (en) 2021-12-23
KR20230035043A (ko) 2023-03-10
CO2023000156A2 (es) 2023-03-27
AU2021293197A1 (en) 2023-02-09
CN116348149A (zh) 2023-06-27
TW202208630A (zh) 2022-03-01
IL299094A (en) 2023-02-01
MX2022016332A (es) 2023-04-03
JP2023530974A (ja) 2023-07-20
US20210393801A1 (en) 2021-12-23
EP4164668A1 (en) 2023-04-19

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