CA3073137A1 - 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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Publication number
CA3073137A1
CA3073137A1 CA3073137A CA3073137A CA3073137A1 CA 3073137 A1 CA3073137 A1 CA 3073137A1 CA 3073137 A CA3073137 A CA 3073137A CA 3073137 A CA3073137 A CA 3073137A CA 3073137 A1 CA3073137 A1 CA 3073137A1
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Canada
Prior art keywords
vector
raav
modified
capsid
location
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Pending
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CA3073137A
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English (en)
French (fr)
Inventor
Jeffrey S. Bartlett
Steven C Ghivizzani
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University of Florida Research Foundation Inc
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University of Florida Research Foundation Inc
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Publication of CA3073137A1 publication Critical patent/CA3073137A1/en
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    • 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
    • A61K48/005Medicinal 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/19Cytokines; Lymphokines; Interferons
    • A61K38/20Interleukins [IL]
    • A61K38/2006IL-1
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/12Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
    • A61K35/32Bones; Osteocytes; Osteoblasts; Tendons; Tenocytes; Teeth; Odontoblasts; Cartilage; Chondrocytes; Synovial membrane
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/66Microorganisms or materials therefrom
    • A61K35/76Viruses; Subviral particles; Bacteriophages
    • A61K35/761Adenovirus
    • 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
    • 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
    • A61K48/0008Medicinal 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/0012Galenical forms characterised by the site of application
    • A61K9/0019Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P19/00Drugs for skeletal disorders
    • A61P19/02Drugs for skeletal disorders for joint disorders, e.g. arthritis, arthrosis
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P29/00Non-central analgesic, antipyretic or antiinflammatory agents, e.g. antirheumatic agents; Non-steroidal antiinflammatory drugs [NSAID]
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/52Cytokines; Lymphokines; Interferons
    • C07K14/54Interleukins [IL]
    • C07K14/545IL-1
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • 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

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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)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Epidemiology (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Immunology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Genetics & Genomics (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Rheumatology (AREA)
  • Virology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Toxicology (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Dermatology (AREA)
  • Cell Biology (AREA)
  • Biomedical Technology (AREA)
  • Microbiology (AREA)
  • Developmental Biology & Embryology (AREA)
  • Mycology (AREA)
  • Pain & Pain Management (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)
CA3073137A 2016-08-19 2017-08-18 Methods and compositions for treating conditions using recombinant self-complementary adeno-associated virus Pending CA3073137A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
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

Publications (1)

Publication Number Publication Date
CA3073137A1 true CA3073137A1 (en) 2018-02-22

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CA3073137A Pending CA3073137A1 (en) 2016-08-19 2017-08-18 Methods and compositions for treating conditions using recombinant self-complementary adeno-associated virus

Country Status (9)

Country Link
US (2) US11207382B2 (enExample)
EP (1) EP3500278B1 (enExample)
JP (2) JP7165410B2 (enExample)
KR (2) KR102759317B1 (enExample)
CN (1) CN109843306A (enExample)
AU (1) AU2017313844B2 (enExample)
CA (1) CA3073137A1 (enExample)
ES (1) ES2982489T3 (enExample)
WO (1) WO2018035451A1 (enExample)

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JP7165410B2 (ja) 2016-08-19 2022-11-04 ユニバーシティ オブ フロリダ リサーチ ファウンデーション,インコーポレイティド 組換え自己相補的アデノ随伴ウイルスを使用して疾患を治療するための方法及び組成物
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BR112020014625A2 (pt) 2018-01-17 2020-12-08 Meiragtx Uk Ii Limited Proteína de capsídeo de raav modificada para terapia gênica
US12370240B2 (en) 2018-11-28 2025-07-29 Genascence Corporation Methods and compositions for treating osteoarthritis
JP2022520647A (ja) 2019-02-15 2022-03-31 サンガモ セラピューティクス, インコーポレイテッド 組み換えaav生産のための組成物および方法
RS66255B1 (sr) * 2019-07-15 2024-12-31 Meiragtx Uk Ii Ltd Modifikovani proteini aav kapsida za lečenje artritične bolesti
JP2023509443A (ja) * 2019-12-31 2023-03-08 スワンバイオ セラピューティクス リミテッド 改善されたaav-abcd1コンストラクトならびに副腎白質ジストロフィー(ald)および/または副腎脊髄ニューロパチー(amn)の処置または予防のための使用
TW202525832A (zh) 2023-08-31 2025-07-01 美商戴諾治療公司 衣殼多肽及其使用方法(二)

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Also Published As

Publication number Publication date
EP3500278C0 (en) 2024-05-22
KR102759317B1 (ko) 2025-01-31
KR20230130155A (ko) 2023-09-11
US11207382B2 (en) 2021-12-28
JP7653150B2 (ja) 2025-03-28
JP2022130745A (ja) 2022-09-06
AU2017313844A1 (en) 2019-04-11
CN109843306A (zh) 2019-06-04
EP3500278A4 (en) 2020-04-01
EP3500278B1 (en) 2024-05-22
JP7165410B2 (ja) 2022-11-04
US20220175887A1 (en) 2022-06-09
US20190381140A1 (en) 2019-12-19
EP3500278A1 (en) 2019-06-26
ES2982489T3 (es) 2024-10-16
KR20190055086A (ko) 2019-05-22
WO2018035451A1 (en) 2018-02-22
JP2019524890A (ja) 2019-09-05
AU2017313844B2 (en) 2023-10-12

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