AU2017313844B2 - Methods and compositions for treating conditions using recombinant self-complementary adeno-associated virus - Google Patents

Methods and compositions for treating conditions using recombinant self-complementary adeno-associated virus Download PDF

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AU2017313844B2
AU2017313844B2 AU2017313844A AU2017313844A AU2017313844B2 AU 2017313844 B2 AU2017313844 B2 AU 2017313844B2 AU 2017313844 A AU2017313844 A AU 2017313844A AU 2017313844 A AU2017313844 A AU 2017313844A AU 2017313844 B2 AU2017313844 B2 AU 2017313844B2
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AU2017313844A1 (en
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Jeffrey S. Bartlett
Steven C. Ghivizzani
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University of Florida Research Foundation Inc
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    • 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
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/19—Cytokines; Lymphokines; Interferons
    • A61K38/20—Interleukins [IL]
    • A61K38/2006—IL-1
    • 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
    • A61K35/12—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
    • A61K35/32—Bones; Osteocytes; Osteoblasts; Tendons; Tenocytes; Teeth; Odontoblasts; Cartilage; Chondrocytes; Synovial membrane
    • 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
    • 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
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P19/00—Drugs for skeletal disorders
    • A61P19/02—Drugs for skeletal disorders for joint disorders, e.g. arthritis, arthrosis
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P29/00—Non-central analgesic, antipyretic or antiinflammatory agents, e.g. antirheumatic agents; Non-steroidal antiinflammatory drugs [NSAID]
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/52—Cytokines; Lymphokines; Interferons
    • C07K14/54—Interleukins [IL]
    • C07K14/545—IL-1
    • 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
    • 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

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Epidemiology (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Immunology (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • Genetics & Genomics (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Rheumatology (AREA)
  • Biotechnology (AREA)
  • Virology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Biophysics (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Biochemistry (AREA)
  • Toxicology (AREA)
  • Dermatology (AREA)
  • Developmental Biology & Embryology (AREA)
  • Pain & Pain Management (AREA)
  • Cell Biology (AREA)
  • Biomedical Technology (AREA)
  • Microbiology (AREA)
  • Mycology (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)
AU2017313844A 2016-08-19 2017-08-18 Methods and compositions for treating conditions using recombinant self-complementary adeno-associated virus Active AU2017313844B2 (en)

Applications Claiming Priority (3)

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US201662377297P 2016-08-19 2016-08-19
US62/377,297 2016-08-19
PCT/US2017/047589 WO2018035451A1 (en) 2016-08-19 2017-08-18 Methods and compositions for treating conditions using recombinant self-complementary adeno-associated virus

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AU2017313844B2 true AU2017313844B2 (en) 2023-10-12

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EP (1) EP3500278B1 (cg-RX-API-DMAC7.html)
JP (2) JP7165410B2 (cg-RX-API-DMAC7.html)
KR (2) KR20230130155A (cg-RX-API-DMAC7.html)
CN (1) CN109843306A (cg-RX-API-DMAC7.html)
AU (1) AU2017313844B2 (cg-RX-API-DMAC7.html)
CA (1) CA3073137A1 (cg-RX-API-DMAC7.html)
ES (1) ES2982489T3 (cg-RX-API-DMAC7.html)
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Families Citing this family (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US11207382B2 (en) 2016-08-19 2021-12-28 University Of Florida Research Foundation, Incorporated Compositions for treating conditions using recombinant self-complementary adeno-associated virus
AU2017371043B2 (en) * 2016-12-07 2022-12-15 University Of Florida Research Foundation, Incorporated IL-1Ra cDNAs
DK3740222T5 (da) 2018-01-17 2024-08-26 Meiragtx Uk Ltd Modificeret RAAV-capsidprotein til genterapi
US12370240B2 (en) 2018-11-28 2025-07-29 Genascence Corporation Methods and compositions for treating osteoarthritis
KR20210126096A (ko) 2019-02-15 2021-10-19 상가모 테라퓨틱스, 인코포레이티드 재조합 aav를 제조하기 위한 조성물 및 방법
CA3146791A1 (en) * 2019-07-15 2021-01-21 Meiragtx Uk Ii Limited Modified aav capsid proteins for treatment of arthritic disease
WO2021138559A1 (en) * 2019-12-31 2021-07-08 Swanbio Therapeutics Limited Improved aav-abcd1 constructs and use for treatment or prevention of adrenoleukodystrophy (ald) and/or adrenomyeloneuropathy (amn)
WO2025049993A1 (en) 2023-08-31 2025-03-06 Dyno Therapeutics, Inc. Capsid polypeptides and methods of use thereof

Family Cites Families (63)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6156304A (en) 1990-12-20 2000-12-05 University Of Pittsburgh Of The Commonwealth System Of Higher Education Gene transfer for studying and treating a connective tissue of a mammalian host
US5747072A (en) 1993-07-30 1998-05-05 University Of Michigan Adenoviral-mediated gene transfer to synovial cells in vivo
WO1995016353A1 (en) * 1993-12-14 1995-06-22 University Of Pittsburgh Of The Commonwealth System Of Higher Education Systemic gene treatment of connective tissue diseases
US5756283A (en) 1995-06-05 1998-05-26 The Trustees Of The University Of Pennsylvania Method for improved production of recombinant adeno-associated viruses for gene therapy
US6093570A (en) 1995-06-07 2000-07-25 The University Of North Carolina At Chapel Hill Helper virus-free AAV production
US6040183A (en) 1995-06-07 2000-03-21 University Of North Carloina At Chapel Hill Helper virus-free AAV production
US6083716A (en) 1996-09-06 2000-07-04 The Trustees Of The University Of Pennsylvania Chimpanzee adenovirus vectors
AU728220B2 (en) 1997-04-14 2001-01-04 Cell Genesys, Inc. Methods for increasing the efficiency of recombinant AAV product
AU9319198A (en) 1997-09-19 1999-04-05 Trustees Of The University Of Pennsylvania, The Methods and vector constructs useful for production of recombinant aav
WO1999049029A1 (en) 1998-03-20 1999-09-30 Benitec Australia Ltd Control of gene expression
WO2000028004A1 (en) 1998-11-10 2000-05-18 The University Of North Carolina At Chapel Hill Virus vectors and methods of making and administering the same
US6943153B1 (en) 1999-03-15 2005-09-13 The Regents Of The University Of California Use of recombinant gene delivery vectors for treating or preventing diseases of the eye
ES2312344T3 (es) 1999-05-28 2009-03-01 Targeted Genetics Corporation Metodos y composiciones para disminuir el nivel de factor de necrosis tumoral (tnf) en trastornos asociados con tnf.
AU3459701A (en) 2000-01-26 2001-08-07 Chiron Corporation Recombinant aav packaging systems
CA2410828C (en) 2000-06-01 2012-01-24 University Of North Carolina At Chapel Hill Duplexed parvovirus vectors
DE10056210A1 (de) 2000-11-13 2002-05-29 Arimedes Biotechnology Gmbh Virales Expressionssystem
WO2002062375A1 (en) * 2001-02-06 2002-08-15 Merck Patent Gmbh Modified interleukin-1 receptor antagonist (il-1ra) with reduced immunogenicity
KR100472297B1 (ko) 2001-09-27 2005-03-07 주식회사 두산 리소인지질의 수용성 조성물
KR100471942B1 (ko) 2002-03-19 2005-03-09 주식회사 에스원 복합형 카드와 카드정보 등록 제어시스템
DE10318048A1 (de) 2003-04-17 2004-11-04 Axaron Bioscience Ag Nicht fluoreszierende, durch Proteolyse zur Fluoreszenz aktivierbare Reporterproteine und ihre Verwendung zur Detektion Protease-abhängiger Ereignisse
NZ545656A (en) 2003-09-01 2009-02-28 Amc Amsterdam AAV vectors for in vivo gene therapy of rheumatoid arthritis
US20070009899A1 (en) 2003-10-02 2007-01-11 Mounts William M Nucleic acid arrays for detecting gene expression in animal models of inflammatory diseases
US7196112B2 (en) 2004-07-16 2007-03-27 Biogen Idec Ma Inc. Cell adhesion inhibitors
EP1828390B1 (en) 2004-12-15 2012-06-13 The University Of North Carolina At Chapel Hill Chimeric vectors
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
FR2891544A1 (fr) 2005-09-30 2007-04-06 Genethon Ass Loi De 1901 Substrat proteique pour la detection de l'activite calpaine 3
WO2007112001A2 (en) 2006-03-23 2007-10-04 Caritas St. Elizabeth Medical Center Of Boston, Inc. Compositions and methods for treating myocardial infarction
EP2037943A4 (en) 2006-04-21 2010-03-31 Univ North Carolina TREATMENT OF BIND WEB DISEASES
US20080187576A1 (en) * 2006-06-16 2008-08-07 University Of Florida Research Foundation, Inc. Methods for treating articular disease or dysfunction using self-complimentary adeno-associated viral vectors
US20080166762A1 (en) 2006-09-27 2008-07-10 Reliance Life Science Pvt.Ltd. HETEROLOGOUS PRODUCTION OF INTERLEUKIN 1 RECEPTOR ANTAGONIST (IL-1Ra) IN PICHIA PASTORIS
US7452696B2 (en) 2006-10-27 2008-11-18 National Taiwan University Recombinant plasmid and method for expressing hepatitis B viral antigens and virions in vivo
JP5575486B2 (ja) 2007-01-18 2014-08-20 ユニヴァーシティ オブ ミズーリー−コロンビア 筋鞘にｎＮＯＳを回復させる合成ミニ／ミクロジストロフィン遺伝子
GB0708376D0 (en) 2007-05-01 2007-06-06 Alligator Bioscience Ab Novel polypeptides and uses thereof
KR101667069B1 (ko) * 2008-11-17 2016-10-17 이노비오 파마수티컬즈, 인크. 플라비바이러스에 대한 면역반응을 유발하는 항원 및 이를 사용하는 방법
US9610363B2 (en) 2009-04-16 2017-04-04 President And Fellows Of Harvard College Methods for inhibiting starvation of a cell
BR112012007114A2 (pt) 2009-09-29 2016-07-05 Gilead Biologics Inc métodos e composições para tratamento da fibrose ocular
DE202009017772U1 (de) 2009-12-10 2011-04-21 Orthogen Ag Kombinationspräparate mit Cytokin-Antagonist und Corticosteroid
EP2394667A1 (en) 2010-06-10 2011-12-14 Laboratorios Del Dr. Esteve, S.A. Vectors and sequences for the treatment of diseases
WO2012047093A1 (en) 2010-10-05 2012-04-12 Academisch Ziekenhuis Bij De Universiteit Van Amsterdam Treatment of the sjögren's syndrome
US8883134B2 (en) * 2010-10-20 2014-11-11 Handok Pharmaceuticals, Inc. Human interleukin-1 receptor antagonist—hybrid Fc fusion protein
US8736207B2 (en) 2011-01-03 2014-05-27 General Electric Company Method and system for power conversion
CN103596591B (zh) 2011-02-08 2016-08-24 Abbvie公司 骨关节炎和疼痛的治疗
US20130021786A1 (en) 2011-07-21 2013-01-24 Noble Matthew D Utility bag lighting
ES2534782T3 (es) 2012-02-02 2015-04-28 Baylor College Of Medicine Sistema biológico de administración y expresión basado en adenovirus para uso en el tratamiento de la osteoartritis
WO2013151663A1 (en) 2012-04-02 2013-10-10 modeRNA Therapeutics Modified polynucleotides for the production of membrane proteins
US9254311B2 (en) * 2012-04-02 2016-02-09 Moderna Therapeutics, Inc. Modified polynucleotides for the production of proteins
EP2660325A3 (en) 2012-05-02 2014-02-12 Christian Medical College AAV vectors and corresponding nucleotide sequences and methods
CN103509100B (zh) 2012-06-15 2017-10-27 上海百迈博制药有限公司 一种白细胞介素‑1受体拮抗剂突变体
US9624282B2 (en) 2012-11-26 2017-04-18 The Curators Of The University Of Missouri Microdystrophin peptides and methods for treating muscular dystrophy using the same
ES2802878T3 (es) 2013-01-25 2021-01-21 Baylor College Medicine Un sistema de administración y expresión de terapia génica adenoviral dependiente de linfocitos T colaboradores
EP2792742A1 (en) 2013-04-17 2014-10-22 Universitätsklinikum Hamburg-Eppendorf (UKE) Gene-therapy vectors for treating cardiomyopathy
CN105682674A (zh) 2013-04-17 2016-06-15 建新公司 用于治疗和预防黄斑变性的组合物和方法
DE102013215817A1 (de) 2013-08-09 2015-02-12 Universitätsklinikum Hamburg-Eppendorf Neue peptide mit spezifität für die lunge
ES2936220T3 (es) 2013-08-27 2023-03-15 Res Inst Nationwide Childrens Hospital Productos y métodos para el tratamiento de la esclerosis lateral amiotrófica
EP3068889B1 (en) 2013-09-26 2019-04-17 Universitat Autònoma De Barcelona Gene therapy compositions for use in the prevention and/or treatment of non-alcoholic fatty liver disease
DE102013220859B4 (de) 2013-10-15 2016-09-08 Plasmidfactory Gmbh & Co. Kg Minicircles mit viralen Expressionskassetten und ihre Verwendung zur Transformation von Zellen zur Erzeugung rekombinanter Viren oder viraler Genvektoren
WO2015160893A1 (en) 2014-04-15 2015-10-22 Applied Genetic Technologies Corporation Codon optimized nucleic acid encoding a retinitis pigmentosa gtpase regulator (rpgr)
DE102014207498A1 (de) 2014-04-17 2015-10-22 Universitätsklinikum Hamburg-Eppendorf Viraler Vektor für den zielgerichteten Gentransfer in Gehirn und Rückenmark
IL248102B (en) 2014-05-02 2022-07-01 Genzyme Corp aav vectors for gene therapy of the central nervous system and retina
US11207382B2 (en) 2016-08-19 2021-12-28 University Of Florida Research Foundation, Incorporated Compositions for treating conditions using recombinant self-complementary adeno-associated virus
WO2018035457A1 (en) 2016-08-19 2018-02-22 Calimmune, Inc. Methods and compositions for treating canine conditions using recombinant self-complementary adeno-associated virus
US20200318080A1 (en) 2016-08-19 2020-10-08 Colorado State University Research Foundation Methods and compositions for treating equine conditions using recombinant self-complementary adeno-associated virus
AU2017371043B2 (en) 2016-12-07 2022-12-15 University Of Florida Research Foundation, Incorporated IL-1Ra cDNAs

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
Goodrich L. R., et al., 'scAAVIL-1ra dosing trial in a large animal model and validation of long-term expression with repeat administration for osteoarthritis therapy', 2015. Gene Therapy, vol. 22, no. 7, pages 536-545 *

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US20190381140A1 (en) 2019-12-19
KR20230130155A (ko) 2023-09-11
US11207382B2 (en) 2021-12-28
JP7165410B2 (ja) 2022-11-04
JP7653150B2 (ja) 2025-03-28
EP3500278C0 (en) 2024-05-22
CA3073137A1 (en) 2018-02-22
EP3500278A1 (en) 2019-06-26
KR102759317B1 (ko) 2025-01-31
JP2022130745A (ja) 2022-09-06
WO2018035451A1 (en) 2018-02-22
EP3500278B1 (en) 2024-05-22
US20220175887A1 (en) 2022-06-09
EP3500278A4 (en) 2020-04-01
AU2017313844A1 (en) 2019-04-11
KR20190055086A (ko) 2019-05-22
JP2019524890A (ja) 2019-09-05
CN109843306A (zh) 2019-06-04
ES2982489T3 (es) 2024-10-16

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