AU2022273530A1 - Modified mrna, modified non-coding rna, and uses thereof - Google Patents

Modified mrna, modified non-coding rna, and uses thereof Download PDF

Info

Publication number
AU2022273530A1
AU2022273530A1 AU2022273530A AU2022273530A AU2022273530A1 AU 2022273530 A1 AU2022273530 A1 AU 2022273530A1 AU 2022273530 A AU2022273530 A AU 2022273530A AU 2022273530 A AU2022273530 A AU 2022273530A AU 2022273530 A1 AU2022273530 A1 AU 2022273530A1
Authority
AU
Australia
Prior art keywords
modified
nucleotides
mrna
nucleotide
sequence
Prior art date
Legal status (The legal status 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 status listed.)
Pending
Application number
AU2022273530A
Other languages
English (en)
Other versions
AU2022273530A9 (en
AU2022273530A2 (en
Inventor
Abhishek ADITHAM
Hailing SHI
Xiao Wang
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Massachusetts Institute of Technology
Broad Institute Inc
Original Assignee
Massachusetts Institute of Technology
Broad Institute 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 Massachusetts Institute of Technology, Broad Institute Inc filed Critical Massachusetts Institute of Technology
Publication of AU2022273530A1 publication Critical patent/AU2022273530A1/en
Publication of AU2022273530A9 publication Critical patent/AU2022273530A9/en
Publication of AU2022273530A2 publication Critical patent/AU2022273530A2/en
Pending legal-status Critical Current

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
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7125—Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
    • 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
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/53—Ligases (6)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00—Drugs for immunological or allergic disorders
    • A61P37/02—Immunomodulators
    • A61P37/04—Immunostimulants
    • 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
    • C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/1003—Extracting or separating nucleic acids from biological samples, e.g. pure separation or isolation methods; Conditions, buffers or apparatuses therefor
    • C12N15/1006—Extracting or separating nucleic acids from biological samples, e.g. pure separation or isolation methods; Conditions, buffers or apparatuses therefor by means of a solid support carrier, e.g. particles, polymers
    • C12N15/1013—Extracting or separating nucleic acids from biological samples, e.g. pure separation or isolation methods; Conditions, buffers or apparatuses therefor by means of a solid support carrier, e.g. particles, polymers by using magnetic beads
    • 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
    • C12N15/63—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
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y605/00—Ligases forming phosphoric ester bonds (6.5)
    • C12Y605/01—Ligases forming phosphoric ester bonds (6.5) forming phosphoric ester bonds (6.5.1)
    • C12Y605/01003—RNA ligase (ATP) (6.5.1.3)
    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/313—Phosphorodithioates
    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/322—2'-R Modification
    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/33—Chemical structure of the base
    • C12N2310/336—Modified G
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/50—Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Immunology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Epidemiology (AREA)
  • Physics & Mathematics (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Analytical Chemistry (AREA)
  • Saccharide Compounds (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
AU2022273530A 2021-05-12 2022-05-11 Modified mrna, modified non-coding rna, and uses thereof Pending AU2022273530A1 (en)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US202163187752P 2021-05-12 2021-05-12
US63/187,752 2021-05-12
US202163288522P 2021-12-10 2021-12-10
US63/288,522 2021-12-10
PCT/US2022/028849 WO2022241045A1 (en) 2021-05-12 2022-05-11 Modified mrna, modified non-coding rna, and uses thereof

Publications (3)

Publication Number Publication Date
AU2022273530A1 true AU2022273530A1 (en) 2023-11-23
AU2022273530A9 AU2022273530A9 (en) 2023-11-30
AU2022273530A2 AU2022273530A2 (en) 2023-12-07

Family

ID=82163587

Family Applications (1)

Application Number Title Priority Date Filing Date
AU2022273530A Pending AU2022273530A1 (en) 2021-05-12 2022-05-11 Modified mrna, modified non-coding rna, and uses thereof

Country Status (7)

Country Link
US (1) US20240277872A1 (https=)
EP (1) EP4337331A1 (https=)
JP (1) JP2024518546A (https=)
KR (1) KR20240021170A (https=)
AU (1) AU2022273530A1 (https=)
CA (1) CA3218778A1 (https=)
WO (1) WO2022241045A1 (https=)

Families Citing this family (15)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2023141474A1 (en) * 2022-01-18 2023-07-27 The Broad Institute, Inc. Poly-tailed and poly-capped mrna and uses thereof
CN114685586B (zh) * 2022-03-05 2025-07-18 武汉瑞佶生物科技有限公司 一种mRNA-脂肪酸靶向化合物及其制备方法和应用
JP2025519910A (ja) * 2022-06-24 2025-06-26 ザ・ブロード・インスティテュート・インコーポレイテッド キャップされた環状ｒｎａ分子を調製するための組成物及び方法
EP4655398A1 (en) * 2023-01-27 2025-12-03 Eleven Therapeutics Ltd Artificial polynucleotide molecules for enhanced stability and translation
KR20250166303A (ko) * 2023-03-29 2025-11-27 센젠 센신 바이오테크놀로지 컴퍼니 리미티드 폴리(a)를 포함하는 폴리뉴클레오타이드 분자 및 이의 용도
WO2025017029A1 (en) * 2023-07-17 2025-01-23 Eleven Therapeutics Ltd Non-natural building blocks for enhanced translation and stability of mrna
JP2025032058A (ja) * 2023-08-06 2025-03-11 シー‐ラン リン 新規なオキソ－ｒｎａ組成物及びその関連用途
WO2025111430A1 (en) * 2023-11-21 2025-05-30 Prime Medicine, Inc. CHEMICAL MODIFICATIONS IN mRNA POLY(A) TAIL
WO2025147660A2 (en) * 2024-01-04 2025-07-10 Trilink Biotechnologies, Llc Modified rna for increasing protein expression
WO2025175176A1 (en) * 2024-02-14 2025-08-21 Modernatx, Inc. Synthetic nucleic acids and methods of using the same for improved polypeptide production
WO2025217126A1 (en) * 2024-04-09 2025-10-16 Convergence Bio, Inc. Polynucleotides, 3' chain terminating tails, ligation and uses thereof
WO2025217135A1 (en) * 2024-04-09 2025-10-16 Convergence Bio, Inc. Rna ligation methods and products
WO2025227104A1 (en) * 2024-04-25 2025-10-30 Parcel Biosciences, Inc. Mrna complexes, their manufacturing, and their use for treatment
WO2026010855A1 (en) * 2024-07-02 2026-01-08 Trilink Biotechnologies, Llc Modified rna for increasing protein expression
WO2026011417A1 (en) * 2024-07-12 2026-01-15 The Hong Kong University Of Science And Technology Compositions and methods involving artificial poly (a) sequences

Family Cites Families (18)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2012019630A1 (en) * 2010-08-13 2012-02-16 Curevac Gmbh Nucleic acid comprising or coding for a histone stem-loop and a poly(a) sequence or a polyadenylation signal for increasing the expression of an encoded protein
CA3018046A1 (en) 2011-12-16 2013-06-20 Moderna Therapeutics, Inc. Modified nucleoside, nucleotide, and nucleic acid compositions
WO2013103659A1 (en) * 2012-01-04 2013-07-11 Board Of Supervisors Of Louisiana State University And Agricultural And Mechanical College Stabilizing rna by incorporating chain-terminating nucleosides at the 3'-terminus
JP6586075B2 (ja) 2013-03-14 2019-10-02 トランスレイト バイオ， インコーポレイテッド メッセンジャーｒｎａの精製方法
US9950065B2 (en) 2013-09-26 2018-04-24 Biontech Rna Pharmaceuticals Gmbh Particles comprising a shell with RNA
WO2015048744A2 (en) * 2013-09-30 2015-04-02 Moderna Therapeutics, Inc. Polynucleotides encoding immune modulating polypeptides
ES3032935T3 (en) 2013-10-22 2025-07-29 Translate Bio Inc Lipid formulations for delivery of messenger rna
AU2014348212C1 (en) 2013-11-18 2018-11-29 Arcturus Therapeutics, Inc. Ionizable cationic lipid for RNA delivery
US9365610B2 (en) 2013-11-18 2016-06-14 Arcturus Therapeutics, Inc. Asymmetric ionizable cationic lipid for RNA delivery
US9850269B2 (en) 2014-04-25 2017-12-26 Translate Bio, Inc. Methods for purification of messenger RNA
AU2015338923B2 (en) * 2014-11-02 2021-10-21 Arcturus Therapeutics, Inc. Messenger UNA molecules and uses thereof
WO2016155809A1 (en) 2015-03-31 2016-10-06 Biontech Rna Pharmaceuticals Gmbh Lipid particle formulations for delivery of rna and water-soluble therapeutically effective compounds to a target cell
US10808242B2 (en) 2015-08-28 2020-10-20 Biontech Rna Pharmaceuticals Gmbh Method for reducing immunogenicity of RNA
EP3585892B8 (en) 2017-02-27 2022-07-13 Translate Bio, Inc. Methods for purification of messenger rna
MA47824A (fr) 2017-03-07 2020-01-15 Translate Bio Inc Administration polyanionique d'acides nucléiques
MA53669A (fr) * 2018-09-20 2021-07-28 Modernatx Inc Compositions et procédés d'administration d'acides nucléiques
BR112021023411A2 (pt) 2019-05-22 2022-02-01 Massachusetts Inst Technology Composições e métodos de rna circular
JP2022552371A (ja) * 2019-10-15 2022-12-15 モデルナティエックス インコーポレイテッド 免疫調節ポリペプチドをコードするｍＲＮＡ及びその使用

Also Published As

Publication number Publication date
KR20240021170A (ko) 2024-02-16
CA3218778A1 (en) 2022-11-17
AU2022273530A9 (en) 2023-11-30
EP4337331A1 (en) 2024-03-20
JP2024518546A (ja) 2024-05-01
AU2022273530A2 (en) 2023-12-07
WO2022241045A1 (en) 2022-11-17
US20240277872A1 (en) 2024-08-22

Similar Documents

Publication Publication Date Title
US20240277872A1 (en) Modified mrna, modified non-coding rna, and uses thereof
CN113661242A (zh) 包含经修饰的环状多核糖核苷酸的组合物及其用途
EP4737574A2 (en) Poly-tailed and poly-capped mrna and uses thereof
EP4219723B1 (en) Circular rna platforms, uses thereof, and their manufacturing processes from engineered dna
WO2023222114A1 (en) Methods of making circular rna
KR20240055811A (ko) 프라임 편집을 위한 화학적 변형을 갖는 가이드 rna
TW202405168A (zh) 製造含有寡核苷酸之聚核苷酸的新穎方法
AU2022285744A1 (en) Circular guide rnas for crispr/cas editing systems
JP2024521304A (ja) 環状ポリリボヌクレオチドを濃縮する方法
WO2023250528A1 (en) Compositions and methods for preparing capped circular rna molecules
CN117616133A (zh) 富集环状多核糖核苷酸的方法
CN118076741A (zh) 修饰的mRNA、修饰的非编码RNA及其用途
Wei et al. Functional XNA and Biomedical Application
CN120051576A (zh) 生产合成环状核酸的无细胞方法
US20240060107A1 (en) Compositions and methods for preparing capped mrna
WO2024260432A1 (zh) 线性rna的环化组件及其应用
Aditham Site-specific chemical and topological modifications to augment mRNA therapeutic potential
WO2025170909A1 (en) Nucleic acids with region-specific modifications and methods of making the same
EP4713333A1 (en) Circular rna analogs, preparation and application thereof
KR20260052931A (ko) 캡핑된 mrna 및 이의 제조방법
CN119464283A (zh) 加帽的mRNA及其制备方法

Legal Events

Date Code Title Description
SREP Specification republished
DA3 Amendments made section 104

Free format text: THE NATURE OF THE AMENDMENT IS AS SHOWN IN THE STATEMENT(S) FILED 09 NOV 2023 AND 23 NOV 2023