CA3107683A1 - Compositions comprising circular polyribonucleotides and uses thereof - Google Patents

Compositions comprising circular polyribonucleotides and uses thereof Download PDF

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Publication number
CA3107683A1
CA3107683A1 CA3107683A CA3107683A CA3107683A1 CA 3107683 A1 CA3107683 A1 CA 3107683A1 CA 3107683 A CA3107683 A CA 3107683A CA 3107683 A CA3107683 A CA 3107683A CA 3107683 A1 CA3107683 A1 CA 3107683A1
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Canada
Prior art keywords
circular polyribonucleotide
target
cell
binding
sequence
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Pending
Application number
CA3107683A
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English (en)
French (fr)
Inventor
Avak Kahvejian
Nicholas McCartney PLUGIS
Alexandra Sophie DE BOER
Morag Helen STEWART
Catherine CIFUENTES-ROJAS
Ki Young PAEK
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Flagship Pioneering Innovations VI Inc
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Flagship Pioneering Innovations VI Inc
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Publication of CA3107683A1 publication Critical patent/CA3107683A1/en
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    • 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
    • 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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/115—Aptamers, i.e. nucleic acids binding a target molecule specifically and with high affinity without hybridising therewith ; Nucleic acids binding to non-nucleic acids, e.g. aptamers
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/111—General methods applicable to biologically active non-coding nucleic acids
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7028—Compounds having saccharide radicals attached to non-saccharide compounds by glycosidic linkages
    • A61K31/7034—Compounds having saccharide radicals attached to non-saccharide compounds by glycosidic linkages attached to a carbocyclic compound, e.g. phloridzin
    • A61K31/704—Compounds having saccharide radicals attached to non-saccharide compounds by glycosidic linkages attached to a carbocyclic compound, e.g. phloridzin attached to a condensed carbocyclic ring system, e.g. sennosides, thiocolchicosides, escin, daunorubicin
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C12N15/1131—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against viruses
    • 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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C12N15/1138—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against receptors or cell surface proteins
    • 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
    • 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
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90—Stable introduction of foreign DNA into chromosome
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/12—Type of nucleic acid catalytic nucleic acids, e.g. ribozymes
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/13—Decoys
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/15—Nucleic acids forming more than 2 strands, e.g. TFOs
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/16—Aptamers
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/50—Physical structure
    • C12N2310/51—Physical structure in polymeric form, e.g. multimers, concatemers
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/50—Physical structure
    • C12N2310/53—Physical structure partially self-complementary or closed
    • C12N2310/532—Closed or circular

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Mycology (AREA)
  • Virology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Medicinal Preparation (AREA)
CA3107683A 2018-07-24 2019-07-24 Compositions comprising circular polyribonucleotides and uses thereof Pending CA3107683A1 (en)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US201862702714P 2018-07-24 2018-07-24
US62/702,714 2018-07-24
US201962823569P 2019-03-25 2019-03-25
US62/823,569 2019-03-25
US201962863670P 2019-06-19 2019-06-19
US62/863,670 2019-06-19
PCT/US2019/043272 WO2020023655A1 (en) 2018-07-24 2019-07-24 Compositions comprising circular polyribonucleotides and uses thereof

Publications (1)

Publication Number Publication Date
CA3107683A1 true CA3107683A1 (en) 2020-01-30

Family

ID=69182387

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3107683A Pending CA3107683A1 (en) 2018-07-24 2019-07-24 Compositions comprising circular polyribonucleotides and uses thereof

Country Status (12)

Country Link
US (1) US20210292761A1 (enExample)
EP (1) EP3827084A4 (enExample)
JP (2) JP2021531022A (enExample)
KR (1) KR102950624B1 (enExample)
CN (1) CN112567038A (enExample)
AU (2) AU2019312269A1 (enExample)
BR (1) BR112021001343A2 (enExample)
CA (1) CA3107683A1 (enExample)
IL (1) IL280354A (enExample)
MX (1) MX2021000965A (enExample)
SG (1) SG11202100750XA (enExample)
WO (1) WO2020023655A1 (enExample)

Families Citing this family (25)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US11814627B2 (en) 2016-06-20 2023-11-14 The Board Of The Leland Stanford Junior University Circular RNAs and their use in immunomodulation
CN118416088A (zh) 2017-03-03 2024-08-02 加利福尼亚大学董事会 经由抑制性tRNAs和脱氨酶对突变进行RNA靶向
CA3084824A1 (en) 2017-12-15 2019-06-20 Flagship Pioneering Innovations Vi, Llc Compositions comprising circular polyribonucleotides and uses thereof
EP3946466A2 (en) * 2019-03-25 2022-02-09 Flagship Pioneering Innovations VI, LLC Compositions comprising modified circular polyribonucleotides and uses thereof
EP4022069A4 (en) * 2019-08-28 2023-12-20 The Board of Trustees of the Leland Stanford Junior University MODIFIED CIRCULAR RNAS AND METHOD OF USE THEREOF
AU2020395113A1 (en) 2019-12-02 2022-06-09 Shape Therapeutics Inc. Therapeutic editing
WO2021155175A1 (en) 2020-01-29 2021-08-05 Flagship Pioneering Innovations Vi, Llc Compositions for translation and methods of use thereof
CN111500681A (zh) * 2020-03-20 2020-08-07 青岛大学 用于检测piRNA与mRNA结合的可视化方法及其应用
EP4153152A1 (en) * 2020-05-20 2023-03-29 Flagship Pioneering Innovations VI, LLC Compositions and methods for producing human polyclonal antibodies
KR20230028375A (ko) 2020-06-25 2023-02-28 더 보드 어브 트러스티스 어브 더 리랜드 스탠포드 주니어 유니버시티 원형 rna 번역을 구동하는 유전 요소와 이를 사용하는 방법
CA3174356A1 (en) * 2020-07-01 2022-01-06 Santosh NARAYAN Compositions and methods for cellular reprogramming using circular rna
CN113249811B (zh) * 2021-05-13 2022-11-08 太原理工大学 一种中空纳米纤维内部固定化生物酶的制备方法
EP4341423A1 (en) 2021-05-18 2024-03-27 Flagship Pioneering Innovations VI, LLC Methods of enriching for circular polyribonucleotides
CN117425735A (zh) 2021-05-18 2024-01-19 旗舰创业创新第六有限责任公司 富集环状多核糖核苷酸的方法
WO2022256578A2 (en) * 2021-06-02 2022-12-08 Beam Therapeutics Inc. Circular guide rnas for crispr/cas editing systems
CA3219570A1 (en) * 2021-06-25 2022-12-29 The Board Of Trustees Of The Leland Stanford Junior University Compositions and methods for improved protein translation from recombinant circular rnas
CN113264842B (zh) 2021-07-21 2022-03-01 苏州科锐迈德生物医药科技有限公司 一种脂质化合物及包含其的脂质载体、核酸脂质纳米粒组合物和药物制剂
US20250002898A1 (en) 2021-07-27 2025-01-02 Flagship Pioneering Innovations Vi, Llc Devices, systems and methods for processing
CN114591986A (zh) * 2021-07-29 2022-06-07 苏州科锐迈德生物医药科技有限公司 环状rna分子及其在目标蛋白的靶向降解中的应用
DK4271818T3 (da) 2021-09-17 2024-11-04 Flagship Pioneering Innovations Vi Llc Sammensætninger og fremgangsmåder til fremstilling af cirkulære polyribonukleotider
CN116103284B (zh) * 2021-11-09 2025-09-23 中国科学院天津工业生物技术研究所 环形gRNA及其相关生物材料与应用
MX2024007307A (es) 2021-12-17 2024-08-06 Flagship Pioneering Innovations Vi Llc Métodos de enriquecimiento de arn circular en condiciones desnaturalizantes.
KR20250153793A (ko) 2023-01-27 2025-10-27 세일 바이오메디슨스, 인크. 변형된 지질 조성물 및 이의 용도
KR20250092117A (ko) * 2023-12-14 2025-06-23 한국과학기술원 Circular RNA를 이용한 표적 mRNA 발현 저해
WO2026096919A1 (en) 2024-11-01 2026-05-07 Sail Biomedicines, Inc. Novel dipepetide core lipids, lipid carriers, and methods of using thereof

Family Cites Families (7)

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Publication number Priority date Publication date Assignee Title
AUPR604101A0 (en) * 2001-06-29 2001-07-26 Unisearch Limited Aptamers
CN101292033B (zh) * 2005-08-01 2013-04-03 普渡研究基金会 用于活性试剂向细胞的递送的多价rna纳粒
US20110065086A1 (en) 2008-02-21 2011-03-17 Otc Biotechnologies, Llc Methods of producing homogeneous plastic-adherent aptamer-magnetic bead-fluorophore and other sandwich assays
LT3401400T (lt) * 2012-05-25 2019-06-10 The Regents Of The University Of California Būdai ir kompozicijos, skirtos rnr molekulės nukreipiamai tikslinės dnr modifikacijai ir rnr molekulės nukreipiamam transkripcijos moduliavimui
CA3221516A1 (en) 2013-08-22 2015-02-26 E. I. Du Pont De Nemours And Company Plant genome modification using guide rna/cas endonuclease systems and methods of use
US11299739B2 (en) 2016-06-07 2022-04-12 Cepheid Thermostable polymerase inhibitor compositions and methods
EP3986469A1 (en) * 2019-06-19 2022-04-27 Flagship Pioneering Innovations VI, LLC Compositions comprising circular polyribonucleotides for protein modulation and uses thereof

Also Published As

Publication number Publication date
JP2024169484A (ja) 2024-12-05
EP3827084A4 (en) 2023-03-15
AU2026201251A1 (en) 2026-03-12
BR112021001343A2 (pt) 2021-05-04
US20210292761A1 (en) 2021-09-23
MX2021000965A (es) 2021-06-15
IL280354A (en) 2021-03-25
EP3827084A1 (en) 2021-06-02
CN112567038A (zh) 2021-03-26
AU2019312269A1 (en) 2021-03-04
WO2020023655A1 (en) 2020-01-30
KR20210039401A (ko) 2021-04-09
JP2021531022A (ja) 2021-11-18
SG11202100750XA (en) 2021-02-25
KR102950624B1 (ko) 2026-04-10

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