CA3140594A1 - Methods of dosing circular polyribonucleotides - Google Patents

Methods of dosing circular polyribonucleotides Download PDF

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Publication number
CA3140594A1
CA3140594A1 CA3140594A CA3140594A CA3140594A1 CA 3140594 A1 CA3140594 A1 CA 3140594A1 CA 3140594 A CA3140594 A CA 3140594A CA 3140594 A CA3140594 A CA 3140594A CA 3140594 A1 CA3140594 A1 CA 3140594A1
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Prior art keywords
composition
days
level
providing
circular polyribonucleotide
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CA3140594A
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English (en)
French (fr)
Inventor
Avak Kahvejian
Alexandra Sophie DE BOER
Nicholas McCartney PLUGIS
Erica Gabrielle Weinstein
Catherine CIFUENTES-ROJAS
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Flagship Pioneering Innovations VI 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
    • A61K48/0083—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 administration regime
    • 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
    • A61K48/0016—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 wherein the nucleic acid is delivered as a 'naked' nucleic acid, i.e. not combined with an entity such as a cationic lipid
    • 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
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
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    • A61P19/00—Drugs for skeletal disorders
    • A61P19/08—Drugs for skeletal disorders for bone diseases, e.g. rachitism, Paget's disease
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    • A61P37/00—Drugs for immunological or allergic disorders
    • A61P37/02—Immunomodulators
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    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
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    • A61P9/14—Vasoprotectives; Antihaemorrhoidals; Drugs for varicose therapy; Capillary stabilisers
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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/67—General methods for enhancing the expression
    • C—CHEMISTRY; METALLURGY
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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12P—FERMENTATION OR ENZYME-USING PROCESSES TO SYNTHESISE A DESIRED CHEMICAL COMPOUND OR COMPOSITION OR TO SEPARATE OPTICAL ISOMERS FROM A RACEMIC MIXTURE
    • C12P19/00—Preparation of compounds containing saccharide radicals
    • C12P19/26—Preparation of nitrogen-containing carbohydrates
    • C12P19/28—N-glycosides
    • C12P19/30—Nucleotides
    • C12P19/34—Polynucleotides, e.g. nucleic acids, oligoribonucleotides

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  • Health & Medical Sciences (AREA)
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  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
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  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Molecular Biology (AREA)
  • Wood Science & Technology (AREA)
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  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Biochemistry (AREA)
  • Epidemiology (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Cardiology (AREA)
  • Rheumatology (AREA)
  • Neurology (AREA)
  • Immunology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Pulmonology (AREA)
  • Communicable Diseases (AREA)
  • Oncology (AREA)
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CA3140594A 2019-06-19 2020-06-19 Methods of dosing circular polyribonucleotides Pending CA3140594A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962863725P 2019-06-19 2019-06-19
US62/863,725 2019-06-19
PCT/US2020/038835 WO2020257727A1 (en) 2019-06-19 2020-06-19 Methods of dosing circular polyribonucleotides

Publications (1)

Publication Number Publication Date
CA3140594A1 true CA3140594A1 (en) 2020-12-24

Family

ID=71842849

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3140594A Pending CA3140594A1 (en) 2019-06-19 2020-06-19 Methods of dosing circular polyribonucleotides

Country Status (10)

Country Link
US (2) US20220296729A1 (https=)
EP (1) EP3987038A1 (https=)
JP (2) JP2022536951A (https=)
KR (1) KR20220024647A (https=)
CN (1) CN114096674A (https=)
AU (1) AU2020296190A1 (https=)
CA (1) CA3140594A1 (https=)
IL (1) IL288983A (https=)
MA (1) MA56538A (https=)
WO (1) WO2020257727A1 (https=)

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WO2020198403A2 (en) 2019-03-25 2020-10-01 Flagship Pioneering Innovations Vi, Llc Compositions comprising modified circular polyribonucleotides and uses thereof
CN114981409A (zh) 2019-09-03 2022-08-30 美洛德生物医药公司 用于基因组整合的方法和组合物
US20230203192A1 (en) * 2020-05-20 2023-06-29 Flagship Pioneering, Inc. Compositions and methods for producing human polyclonal antibodies
BR112023023642A2 (pt) 2021-05-11 2024-01-30 Myeloid Therapeutics Inc Métodos e composições para integração genômica
MX2024003262A (es) 2021-09-17 2024-06-19 Flagship Pioneering Innovations Vi Llc Composiciones y metodos para producir poliribonucleotidos circulares.
KR20240118174A (ko) 2021-12-17 2024-08-02 플래그쉽 파이어니어링 이노베이션스 브이아이, 엘엘씨 변성 조건 하에서의 원형 rna의 풍부화를 위한 방법
CN121057577A (zh) 2023-01-27 2025-12-02 赛欧生物医药股份有限公司 经修饰的脂质组合物及其用途
CN116870004B (zh) * 2023-02-24 2025-10-21 中南大学 以brd7为靶点在防治自身免疫性疾病药物中的应用
CN117070564B (zh) * 2023-03-30 2024-05-10 安可来(重庆)生物医药科技有限公司 一种用于合成环形rna的质粒及其构建方法与一种环形rna及其体外合成方法
CN120241980A (zh) * 2023-12-29 2025-07-04 复旦大学 一种基于环状rna的现货型体内原位car与肿瘤疫苗联用的免疫治疗技术及其应用

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Publication number Publication date
MA56538A (fr) 2022-04-27
JP2022536951A (ja) 2022-08-22
US20220296729A1 (en) 2022-09-22
AU2020296190A1 (en) 2022-01-06
IL288983A (en) 2022-02-01
US20230061936A1 (en) 2023-03-02
WO2020257727A1 (en) 2020-12-24
JP2026040556A (ja) 2026-03-09
KR20220024647A (ko) 2022-03-03
CN114096674A (zh) 2022-02-25
EP3987038A1 (en) 2022-04-27

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