CA3172423A1 - Circular rna compositions and methods - Google Patents

Circular rna compositions and methods Download PDF

Info

Publication number
CA3172423A1
CA3172423A1 CA3172423A CA3172423A CA3172423A1 CA 3172423 A1 CA3172423 A1 CA 3172423A1 CA 3172423 A CA3172423 A CA 3172423A CA 3172423 A CA3172423 A CA 3172423A CA 3172423 A1 CA3172423 A1 CA 3172423A1
Authority
CA
Canada
Prior art keywords
expression sequence
circular rna
rna polynucleotide
sequence encodes
virus
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
CA3172423A
Other languages
English (en)
French (fr)
Inventor
Alex WESSELHOEFT
Brian Goodman
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.)
Orna Therapeutics Inc
Original Assignee
Individual
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 Individual filed Critical Individual
Publication of CA3172423A1 publication Critical patent/CA3172423A1/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
    • A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
    • 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
    • C12N15/113—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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K40/00—Cellular immunotherapy
    • A61K40/10—Cellular immunotherapy characterised by the cell type used
    • A61K40/11—T-cells, e.g. tumour infiltrating lymphocytes [TIL] or regulatory T [Treg] cells; Lymphokine-activated killer [LAK] cells
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K40/00—Cellular immunotherapy
    • A61K40/30—Cellular immunotherapy characterised by the recombinant expression of specific molecules in the cells of the immune system
    • A61K40/31—Chimeric antigen receptors [CAR]
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K40/00—Cellular immunotherapy
    • A61K40/40—Cellular immunotherapy characterised by antigens that are targeted or presented by cells of the immune system
    • A61K40/41—Vertebrate antigens
    • A61K40/42—Cancer antigens
    • A61K40/4202—Receptors, cell surface antigens or cell surface determinants
    • A61K40/421—Immunoglobulin superfamily
    • A61K40/4211—CD19 or B4
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/69—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the conjugate being characterised by physical or galenical forms, e.g. emulsion, particle, inclusion complex, stent or kit
    • A61K47/6921—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the conjugate being characterised by physical or galenical forms, e.g. emulsion, particle, inclusion complex, stent or kit the form being a particulate, a powder, an adsorbate, a bead or a sphere
    • A61K47/6927—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the conjugate being characterised by physical or galenical forms, e.g. emulsion, particle, inclusion complex, stent or kit the form being a particulate, a powder, an adsorbate, a bead or a sphere the form being a solid microparticle having no hollow or gas-filled cores
    • A61K47/6929—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the conjugate being characterised by physical or galenical forms, e.g. emulsion, particle, inclusion complex, stent or kit the form being a particulate, a powder, an adsorbate, a bead or a sphere the form being a solid microparticle having no hollow or gas-filled cores the form being a nanoparticle, e.g. an immuno-nanoparticle
    • 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
    • A61K48/0025—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 non-active part clearly interacts with the delivered nucleic acid
    • 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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • 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
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4748—Tumour specific antigens; Tumour rejection antigen precursors [TRAP], e.g. MAGE
    • 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
    • C07K14/521—Chemokines
    • 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]
    • 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
    • C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
    • C07K14/70503—Immunoglobulin superfamily
    • C07K14/7051—T-cell receptor (TcR)-CD3 complex
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K16/00—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies
    • 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
    • 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
    • C12N15/79—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
    • 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
    • C12N15/88—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation using microencapsulation, e.g. using amphiphile liposome vesicle
    • 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
    • C12N2840/00—Vectors comprising a special translation-regulating system
    • C12N2840/20—Vectors comprising a special translation-regulating system translation of more than one cistron
    • C12N2840/203—Vectors comprising a special translation-regulating system translation of more than one cistron having an IRES
    • 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
    • C12N2840/00—Vectors comprising a special translation-regulating system
    • C12N2840/60—Vectors comprising a special translation-regulating system from viruses

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Biophysics (AREA)
  • Medicinal Chemistry (AREA)
  • Immunology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Plant Pathology (AREA)
  • Veterinary Medicine (AREA)
  • Microbiology (AREA)
  • Public Health (AREA)
  • Physics & Mathematics (AREA)
  • Epidemiology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Toxicology (AREA)
  • Cell Biology (AREA)
  • Nanotechnology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicinal Preparation (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
CA3172423A 2020-03-20 2021-03-22 Circular rna compositions and methods Pending CA3172423A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202062992518P 2020-03-20 2020-03-20
US62/992,518 2020-03-20
PCT/US2021/023540 WO2021189059A2 (en) 2020-03-20 2021-03-22 Circular rna compositions and methods

Publications (1)

Publication Number Publication Date
CA3172423A1 true CA3172423A1 (en) 2021-03-22

Family

ID=75539941

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3172423A Pending CA3172423A1 (en) 2020-03-20 2021-03-22 Circular rna compositions and methods

Country Status (10)

Country Link
US (1) US20240245805A1 (https=)
EP (1) EP4121453A2 (https=)
JP (1) JP7851254B2 (https=)
KR (1) KR20230069042A (https=)
CN (1) CN116034114A (https=)
AU (1) AU2021237738A1 (https=)
BR (1) BR112022018854A2 (https=)
CA (1) CA3172423A1 (https=)
MX (1) MX2022011677A (https=)
WO (1) WO2021189059A2 (https=)

Cited By (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN115702940A (zh) * 2021-08-05 2023-02-17 苏州科锐迈德生物医药科技有限公司 包含环状rna分子的制剂在制备治疗肿瘤的药物中的应用
CN116284006A (zh) * 2023-05-10 2023-06-23 北京因诺惟康医药科技有限公司 可电离脂质化合物、包含其的脂质载体及应用
CN119015401A (zh) * 2023-06-21 2024-11-26 浙江大学 一种基于rna的肿瘤疫苗、制备方法、免疫佐剂及应用

Families Citing this family (42)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN112399860B (zh) 2018-06-06 2024-08-16 麻省理工学院 用于在真核细胞中翻译的环状rna
AU2022234607B2 (en) 2021-03-10 2025-01-23 Rznomics Inc. Self-circularized RNA structure
CN113999852B (zh) * 2021-11-30 2024-04-02 杭州市富阳区第一人民医院 circ_0001772作为结直肠癌诊断和治疗标志物的应用
EP4444361A4 (en) * 2021-12-09 2025-12-10 Carisma Therapeutics Inc IN VIVO ADMINISTRATION TO IMMUNE CELLS
CN114574483B (zh) 2022-03-02 2024-05-10 苏州科锐迈德生物医药科技有限公司 基于翻译起始元件点突变的重组核酸分子及其在制备环状rna中的应用
CN117965543A (zh) * 2022-03-02 2024-05-03 苏州科锐迈德生物医药科技有限公司 一种重组核酸分子及其在制备环状rna中的应用
WO2023182948A1 (en) * 2022-03-21 2023-09-28 Bio Adventure Co., Ltd. Internal ribosome entry site (ires), plasmid vector and circular mrna for enhancing protein expression
CN119768517A (zh) * 2022-07-08 2025-04-04 上海环码生物医药有限公司 用于纯化环状核酸的方法和系统
CA3263810A1 (en) * 2022-08-02 2024-02-08 Baylor College Of Medicine COMPOSITIONS AND PROCESSES TO FACILITATE HEART AND LUNG REPAIR
EP4608871A1 (en) * 2022-10-26 2025-09-03 Memorial Sloan-Kettering Cancer Center Cd33 antibody compositions for treating alzheimer's disease
WO2024102677A1 (en) 2022-11-08 2024-05-16 Orna Therapeutics, Inc. Circular rna compositions
WO2024112717A1 (en) * 2022-11-21 2024-05-30 Avstera Therapeutics Corp. Mrna composition for preventing and treating cancer
AU2023398825A1 (en) 2022-12-15 2025-06-26 Orna Therapeutics, Inc. Circular rna compositions and methods
US20250382341A1 (en) * 2022-12-30 2025-12-18 The Regents Of The University Of California Methods and compositions of in vivo engineering of t-cells to anti-inflammatory cells and therapeutic applications there of
WO2024151583A2 (en) 2023-01-09 2024-07-18 Flagship Pioneering Innovations Vii, Llc Vaccines and related methods
AU2024211148A1 (en) * 2023-01-27 2025-08-28 The Methodist Hospital Methods of making and isolating circular rnas and circular rna compositions
WO2024167885A1 (en) 2023-02-06 2024-08-15 Flagship Pioneering Innovations Vii, Llc Immunomodulatory compositions and related methods
CN116411023B (zh) * 2023-02-24 2025-08-12 北京大学人民医院 一种新型环状rna成环序列构建car-t细胞及在治疗小细胞肺癌中的应用
CN121358494A (zh) * 2023-03-15 2026-01-16 旗舰创业创新第六有限责任公司 免疫原性组合物及其用途
WO2024193634A1 (en) * 2023-03-21 2024-09-26 Ribox Therapeutics Hk Limited Circular rna and preparation method thereof
WO2024220625A1 (en) 2023-04-19 2024-10-24 Sail Biomedicines, Inc. Delivery of polynucleotides from lipid nanoparticles comprising rna and ionizable lipids
EP4705481A2 (en) 2023-05-05 2026-03-11 Orna Therapeutics, Inc. Circular rna compositions and methods
AU2024279278A1 (en) 2023-05-31 2025-12-18 Capstan Therapeutics, Inc. Lipid nanoparticle formulations and compositions
WO2025007148A1 (en) 2023-06-30 2025-01-02 Orna Therapeutics, Inc. Polymer lipid nanoparticle compositions for delivering circular polynucleotides
WO2025011502A1 (zh) * 2023-07-07 2025-01-16 仁景(苏州)生物科技有限公司 制备环状rna方法及用于所述方法的核酸序列
WO2025049690A1 (en) 2023-08-29 2025-03-06 Orna Therapeutics, Inc. Circular polyethylene glycol lipids
AU2024335327A1 (en) 2023-09-01 2026-03-26 Renagade Therapeutics Management Inc. Gene editing systems, compositions, and methods for treatment of vexas syndrome
WO2025101501A1 (en) 2023-11-07 2025-05-15 Orna Therapeutics, Inc. Circular rna compositions
WO2025105906A1 (en) * 2023-11-17 2025-05-22 Green Cross Corporation Novel rna construct and a method of preparing circular rna using the same
WO2025117969A1 (en) 2023-12-01 2025-06-05 Orna Therapeutics, Inc. Process for manufacturing lipid nanoparticles
WO2025128871A2 (en) 2023-12-13 2025-06-19 Renagade Therapeutics Management Inc. Lipid nanoparticles comprising coding rna molecules for use in gene editing and as vaccines and therapeutic agents
CN117919450A (zh) * 2023-12-31 2024-04-26 上海市东方医院（同济大学附属东方医院） 一种以吞噬细胞为载体的环状rna药物及其制备和应用
CN117866948B (zh) * 2024-01-10 2024-12-13 上海环码生物医药有限公司 用于纯化环状核酸的阴选方法和系统
WO2025155753A2 (en) 2024-01-17 2025-07-24 Renagade Therapeutics Management Inc. Improved gene editing system, guides, and methods
TW202545974A (zh) 2024-01-26 2025-12-01 美商旗艦先鋒創新有限責任（Ｖｉｉ）公司 免疫受體抑制蛋白及相關方法
US20250353881A1 (en) 2024-05-16 2025-11-20 Flagship Pioneering Innovations Vii, Llc Immunoreceptor inhibitory proteins and related methods
WO2025245111A1 (en) 2024-05-22 2025-11-27 Flagship Pioneering Innovations Vii, Llc Immunoreceptor targeting proteins and related methods
CN118222638B (zh) * 2024-05-22 2024-08-13 中国农业大学 一种干扰蚯蚓目的基因表达的方法
WO2025250751A1 (en) 2024-05-31 2025-12-04 Orna Therapeutics, Inc. Circular rna compositions and methods
WO2026003582A2 (en) 2024-06-27 2026-01-02 Axelyf ehf. Lipids and lipid nanoparticles
WO2026006593A1 (en) 2024-06-28 2026-01-02 Orna Therapeutics, Inc. Synthetic internal ribosome entry sites
WO2026072709A1 (en) 2024-09-25 2026-04-02 Orna Therapeutics, Inc. Chimeric antigen receptors targeting bcma

Family Cites Families (30)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6319494B1 (en) 1990-12-14 2001-11-20 Cell Genesys, Inc. Chimeric chains for receptor-associated signal transduction pathways
IL104570A0 (en) 1992-03-18 1993-05-13 Yeda Res & Dev Chimeric genes and cells transformed therewith
IL135578A0 (en) 1997-10-29 2001-05-20 Genzyme Corp Compositions and methods for treating lysosomal storage disease
US5948902A (en) 1997-11-20 1999-09-07 South Alabama Medical Science Foundation Antisense oligonucleotides to human serine/threonine protein phosphatase genes
WO2001060414A2 (en) 2000-02-17 2001-08-23 Genzyme Corporation Genetic modification of the lung as a portal for gene delivery
MXPA05000511A (es) 2001-07-12 2005-09-30 Jefferson Foote Anticuepros super humanizados.
US20040014194A1 (en) 2002-03-27 2004-01-22 Schering Corporation Beta-secretase crystals and methods for preparing and using the same
CN101899114A (zh) 2002-12-23 2010-12-01 惠氏公司 抗pd-1抗体及其用途
ATE475669T1 (de) 2004-06-29 2010-08-15 Immunocore Ltd Einen modifizierten t-zellen-rezeptor exprimierende zellen
DK2439273T3 (da) 2005-05-09 2019-06-03 Ono Pharmaceutical Co Humane monoklonale antistoffer til programmeret død-1(pd-1) og fremgangsmåder til behandling af cancer ved anvendelse af anti-pd-1- antistoffer alene eller i kombination med andre immunterapeutika
CN101970499B (zh) 2008-02-11 2014-12-31 治疗科技公司 用于肿瘤治疗的单克隆抗体
EP2262837A4 (en) 2008-03-12 2011-04-06 Merck Sharp & Dohme PD-1 BINDING PROTEINS
AU2009288730B2 (en) 2008-08-25 2013-06-20 Amplimmune, Inc. Compositions of PD-1 antagonists and methods of use
JP2012500855A (ja) 2008-08-25 2012-01-12 アンプリミューン、インコーポレーテッド Ｐｄ−１アンタゴニストおよび感染性疾患を処置するための方法
WO2011066342A2 (en) 2009-11-24 2011-06-03 Amplimmune, Inc. Simultaneous inhibition of pd-l1/pd-l2
HUE042382T2 (hu) * 2013-09-03 2019-06-28 Medimmune Ltd Legyengített Newcastle betegség vírust tartalmazó készítmények és azok alkalmazása neoplázia kezelési eljárásában történõ alkalmazásra
EP3083556B1 (en) 2013-12-19 2019-12-25 Novartis AG Lipids and lipid compositions for the delivery of active agents
US20190135894A1 (en) * 2015-06-25 2019-05-09 iCell Gene Therapeuticics LLC COMPOUND CHIMERIC ANTIGEN RECEPTOR (cCAR) TARGETING MULTIPLE ANTIGENS, COMPOSITIONS AND METHODS OF USE THEREOF
JP6948313B6 (ja) 2015-09-17 2022-01-14 モデルナティエックス インコーポレイテッド 治療剤の細胞内送達のための化合物および組成物
HRP20230209T1 (hr) 2015-10-28 2023-04-14 Acuitas Therapeutics Inc. Novi lipidi i lipidne formulacije nanočestica za isporuku nukleinskih kiselina
EP4628587A3 (en) * 2016-04-15 2026-02-25 Memorial Sloan Kettering Cancer Center Transgenic t cell and chimeric antigen receptor t cell compositions and related methods
AU2018251187B2 (en) * 2017-04-14 2024-03-28 Dana-Farber Cancer Institute, Inc. Compositions and methods for transient gene therapy with enhanced stability
CN111819185A (zh) * 2017-12-15 2020-10-23 旗舰创业创新第六有限责任公司 包含环状多核糖核苷酸的组合物及其用途
US20190314291A1 (en) * 2018-01-30 2019-10-17 Modernatx, Inc. Compositions and methods for delivery of agents to immune cells
MX2020007944A (es) 2018-02-01 2020-11-24 Tufts College Nanocomplejos similares a los lípidos y sus usos.
WO2019191780A1 (en) 2018-03-30 2019-10-03 Arcturus Therapeutics, Inc. Lipid particles for nucleic acid delivery
CN112055695A (zh) * 2018-05-01 2020-12-08 弗莱德哈钦森癌症研究中心 用于基因表达的纳米粒子和其用途
WO2019222275A2 (en) * 2018-05-14 2019-11-21 TCR2 Therapeutics Inc. Compositions and methods for tcr reprogramming using inducible fusion proteins
AU2019310448A1 (en) * 2018-07-24 2021-03-11 Mayo Foundation For Medical Education And Research Circularized engineered rna and methods
BR112021023411A2 (pt) * 2019-05-22 2022-02-01 Massachusetts Inst Technology Composições e métodos de rna circular

Cited By (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN115702940A (zh) * 2021-08-05 2023-02-17 苏州科锐迈德生物医药科技有限公司 包含环状rna分子的制剂在制备治疗肿瘤的药物中的应用
CN116284006A (zh) * 2023-05-10 2023-06-23 北京因诺惟康医药科技有限公司 可电离脂质化合物、包含其的脂质载体及应用
CN116284006B (zh) * 2023-05-10 2023-08-25 北京因诺惟康医药科技有限公司 可电离脂质化合物、包含其的脂质载体及应用
CN119015401A (zh) * 2023-06-21 2024-11-26 浙江大学 一种基于rna的肿瘤疫苗、制备方法、免疫佐剂及应用

Also Published As

Publication number Publication date
JP7851254B2 (ja) 2026-04-24
EP4121453A2 (en) 2023-01-25
WO2021189059A2 (en) 2021-09-23
AU2021237738A1 (en) 2022-11-10
JP2023518295A (ja) 2023-04-28
WO2021189059A3 (en) 2021-11-11
BR112022018854A2 (pt) 2023-03-07
CN116034114A (zh) 2023-04-28
KR20230069042A (ko) 2023-05-18
MX2022011677A (es) 2023-01-11
US20240245805A1 (en) 2024-07-25

Similar Documents

Publication Publication Date Title
CA3172423A1 (en) Circular rna compositions and methods
US11603396B2 (en) Circular RNA compositions and methods
US12180138B2 (en) Lipid nanoparticle compositions for delivering circular polynucleotides
US20250283073A1 (en) Circular RNA Compositions and Methods
CA3233243A1 (en) Lipid nanoparticle compositions for delivering circular polynucleotides
CA3178111A1 (en) Circular rna compositions and methods
EP3920976A2 (en) Circular rna compositions and methods
WO2024205657A2 (en) Lipids and lipid nanoparticle compositions for delivering polynucleotides
EA049788B1 (ru) Композиции кольцевой рнк и способы

Legal Events

Date Code Title Description
D11 Substantive examination requested

Free format text: ST27 STATUS EVENT CODE: A-1-1-D10-D11-D117 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: REQUEST FOR EXAMINATION RECEIVED

Effective date: 20250210

P11 Amendment of application requested

Free format text: ST27 STATUS EVENT CODE: A-1-1-P10-P11-P101 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: AMENDMENT RECEIVED - VOLUNTARY AMENDMENT

Effective date: 20250210

W00 Other event occurred

Free format text: ST27 STATUS EVENT CODE: A-1-1-W10-W00-W111 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: CORRESPONDENT DETERMINED COMPLIANT

Effective date: 20250210

W00 Other event occurred

Free format text: ST27 STATUS EVENT CODE: A-1-1-W10-W00-W111 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: CORRESPONDENT DETERMINED COMPLIANT

Effective date: 20250211

D00 Search and/or examination requested or commenced

Free format text: ST27 STATUS EVENT CODE: A-1-1-D10-D00-D118 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: REQUEST FOR EXAMINATION REQUIREMENTS DETERMINED COMPLIANT

Effective date: 20250303

P11 Amendment of application requested

Free format text: ST27 STATUS EVENT CODE: A-1-1-P10-P11-P102 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: AMENDMENT DETERMINED COMPLIANT

Effective date: 20250303

P13 Application amended

Free format text: ST27 STATUS EVENT CODE: A-1-1-P10-P13-X000 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: APPLICATION AMENDED

Effective date: 20250303

MFA Maintenance fee for application paid

Free format text: FEE DESCRIPTION TEXT: MF (APPLICATION, 4TH ANNIV.) - STANDARD

Year of fee payment: 4

U00 Fee paid

Free format text: ST27 STATUS EVENT CODE: A-1-1-U10-U00-U101 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE REQUEST RECEIVED

Effective date: 20250305

U11 Full renewal or maintenance fee paid

Free format text: ST27 STATUS EVENT CODE: A-1-1-U10-U11-U102 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE FEE PAYMENT DETERMINED COMPLIANT

Effective date: 20250305

Free format text: ST27 STATUS EVENT CODE: A-1-1-U10-U11-U102 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE FEE PAYMENT PAID IN FULL

Effective date: 20250305

D11 Substantive examination requested

Free format text: ST27 STATUS EVENT CODE: A-1-2-D10-D11-D155 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: ALL REQUIREMENTS FOR EXAMINATION DETERMINED COMPLIANT

Effective date: 20250507

W00 Other event occurred

Free format text: ST27 STATUS EVENT CODE: A-2-2-W10-W00-W100 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: LETTER SENT

Effective date: 20250508

MFA Maintenance fee for application paid

Free format text: FEE DESCRIPTION TEXT: MF (APPLICATION, 5TH ANNIV.) - STANDARD

Year of fee payment: 5

U00 Fee paid

Free format text: ST27 STATUS EVENT CODE: A-2-2-U10-U00-U101 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE REQUEST RECEIVED

Effective date: 20260226

U11 Full renewal or maintenance fee paid

Free format text: ST27 STATUS EVENT CODE: A-2-2-U10-U11-U102 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE FEE PAYMENT PAID IN FULL

Effective date: 20260226