BR112023025515A2 - Composições e métodos de rna circular - Google Patents

Composições e métodos de rna circular

Info

Publication number
BR112023025515A2
BR112023025515A2 BR112023025515A BR112023025515A BR112023025515A2 BR 112023025515 A2 BR112023025515 A2 BR 112023025515A2 BR 112023025515 A BR112023025515 A BR 112023025515A BR 112023025515 A BR112023025515 A BR 112023025515A BR 112023025515 A2 BR112023025515 A2 BR 112023025515A2
Authority
BR
Brazil
Prior art keywords
methods
circular rna
rna
compositions
circularization
Prior art date
Application number
BR112023025515A
Other languages
English (en)
Portuguese (pt)
Inventor
T Horhota Allen
M Becker Amy
Brian Goodman
Gregory Motz
Huan Shu
Kristen Ott
Alexander Wesselhoeft Robert
Thomas Barnes
Varun Shivashankar
Original Assignee
Orna Therapeutics 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 Orna Therapeutics Inc filed Critical Orna Therapeutics Inc
Publication of BR112023025515A2 publication Critical patent/BR112023025515A2/pt

Links

Classifications

    • 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
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K39/12—Viral antigens
    • 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
    • A61K40/4203—Receptors for growth factors
    • A61K40/4205—Her-2/neu/ErbB2, Her-3/ErbB3 or Her 4/ ErbB4
    • 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
    • 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
    • A61K40/4214—Receptors for cytokines
    • A61K40/4215—Receptors for tumor necrosis factors [TNF], e.g. lymphotoxin receptor [LTR], CD30
    • 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
    • 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
    • A61K48/0008—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
    • A61K48/0041—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 the non-active part being polymeric
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12—Antivirals
    • A61P31/14—Antivirals for RNA viruses
    • 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
    • C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
    • C07K14/70503—Immunoglobulin superfamily
    • C07K14/7051—T-cell receptor (TcR)-CD3 complex
    • 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
    • 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
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K2039/51—Medicinal preparations containing antigens or antibodies comprising whole cells, viruses or DNA/RNA
    • A61K2039/53—DNA (RNA) vaccination
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/03—Fusion polypeptide containing a localisation/targetting motif containing a transmembrane segment
    • 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
    • C12N2310/53—Physical structure partially self-complementary or closed
    • C12N2310/532—Closed or circular
    • 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
    • C12N2770/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssRNA viruses positive-sense
    • C12N2770/00011—Details
    • C12N2770/20011—Coronaviridae
    • C12N2770/20034—Use of virus or viral component as vaccine, e.g. live-attenuated or inactivated virus, VLP, viral protein
    • 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
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Zoology (AREA)
  • Epidemiology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Immunology (AREA)
  • Virology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Oncology (AREA)
  • Cell Biology (AREA)
  • Toxicology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Communicable Diseases (AREA)
  • Mycology (AREA)
  • Analytical Chemistry (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
BR112023025515A 2021-06-10 2022-06-10 Composições e métodos de rna circular BR112023025515A2 (pt)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202163209271P 2021-06-10 2021-06-10
US202263311923P 2022-02-18 2022-02-18
PCT/US2022/033091 WO2022261490A2 (en) 2021-06-10 2022-06-10 Circular rna compositions and methods

Publications (1)

Publication Number Publication Date
BR112023025515A2 true BR112023025515A2 (pt) 2024-02-27

Family

ID=83081188

Family Applications (1)

Application Number Title Priority Date Filing Date
BR112023025515A BR112023025515A2 (pt) 2021-06-10 2022-06-10 Composições e métodos de rna circular

Country Status (11)

Country Link
US (1) US20250283073A1 (enExample)
EP (1) EP4352234A2 (enExample)
JP (1) JP2024521444A (enExample)
KR (1) KR20240019791A (enExample)
AU (1) AU2022288705A1 (enExample)
BR (1) BR112023025515A2 (enExample)
CA (1) CA3219932A1 (enExample)
IL (1) IL308746A (enExample)
MX (1) MX2023014702A (enExample)
TW (1) TW202317767A (enExample)
WO (1) WO2022261490A2 (enExample)

Families Citing this family (51)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2022332372B2 (en) 2021-08-27 2025-12-04 Peking University Constructs and methods for preparing circular rna
AR127073A1 (es) 2021-09-17 2023-12-13 Flagship Pioneering Innovations Vi Llc Composiciones y métodos para producir polirribonucleótidos circulares
CN114214237B (zh) * 2021-12-23 2023-06-16 南京大学 一种洗脱细胞表面吸附纳米颗粒的方法
TW202334412A (zh) * 2022-01-11 2023-09-01 北京大學 針對 sars-cov-2 變體的環狀 rna 疫苗及其使用方法
EP4466029A1 (en) * 2022-01-21 2024-11-27 Orna Therapeutics, Inc. Systemic administration of circular rna polynucleotides encoding muscle proteins or protein complexes
AU2023240827A1 (en) * 2022-03-21 2024-07-18 Bio Adventure Co., Ltd. Internal ribosome entry site (ires), plasmid vector and circular mrna for enhancing protein expression
US12297285B2 (en) * 2022-06-24 2025-05-13 Orna Therapeutics, Inc. Circular RNA encoding chimeric antigen receptors targeting BCMA
CN116064531B (zh) * 2022-09-07 2024-04-02 昆明理工大学 一种抑制cvb5病毒复制的长链非编码rna及应用
TW202428289A (zh) 2022-11-08 2024-07-16 美商歐納醫療公司 環狀rna組合物
EP4634390A2 (en) 2022-12-15 2025-10-22 Orna Therapeutics, Inc. Circular rna compositions and methods
EP4680750A1 (en) 2023-03-15 2026-01-21 Flagship Pioneering Innovations VI, LLC Compositions comprising polyribonucleotides and uses thereof
EP4680276A1 (en) 2023-03-15 2026-01-21 Flagship Pioneering Innovations VI, LLC Immunogenic compositions and uses thereof
KR20240159310A (ko) * 2023-04-28 2024-11-05 주식회사 메디치바이오 신규한 이온화지질을 이용한 지질나노입자 조성물
WO2024205657A2 (en) * 2023-03-29 2024-10-03 Orna Therapeutics, Inc. Lipids and lipid nanoparticle compositions for delivering polynucleotides
WO2024200820A1 (en) 2023-03-30 2024-10-03 Ose Immunotherapeutics Method of synthesis of targeted lipid nanoparticle and uses thereof
JP2026512425A (ja) 2023-03-30 2026-04-16 オーエスイー・イミュノセラピューティクス 免疫細胞増強分子の発現のための活性化免疫細胞に標的化された脂質ベースのナノ粒子及びその使用
WO2024220712A2 (en) 2023-04-19 2024-10-24 Sail Biomedicines, Inc. Vaccine compositions
WO2024220752A2 (en) 2023-04-19 2024-10-24 Sail Biomedicines, Inc. Rna therapeutic compositions
EP4698150A1 (en) 2023-04-19 2026-02-25 Sail Biomedicines, Inc. Delivery of polynucleotides from lipid nanoparticles comprising rna and ionizable lipids
WO2024233308A2 (en) 2023-05-05 2024-11-14 Orna Therapeutics, Inc. Circular rna compositions and methods
EP4719352A1 (en) 2023-05-31 2026-04-08 Capstan Therapeutics, Inc. Lipid nanoparticle formulations and compositions
WO2025006613A1 (en) * 2023-06-27 2025-01-02 Hopewell Therapeutics, Inc. Lipid nanoparticles for cancer immunotherapy
WO2025007148A1 (en) 2023-06-30 2025-01-02 Orna Therapeutics, Inc. Polymer lipid nanoparticle compositions for delivering circular polynucleotides
CN122270557A (zh) * 2023-08-17 2026-06-23 武汉大学 稳定性与表达量增强的环状rna
WO2025049690A1 (en) 2023-08-29 2025-03-06 Orna Therapeutics, Inc. Circular polyethylene glycol lipids
EP4771154A2 (en) 2023-09-01 2026-07-08 Renagade Therapeutics Management Inc. Gene editing systems, compositions, and methods for treatment of vexas syndrome
CN122497525A (zh) 2023-11-07 2026-07-31 欧纳医疗公司 环状rna组合物
WO2025106930A1 (en) 2023-11-17 2025-05-22 Sail Biomedicines, Inc. Circular polyribonucleotides encoding glucagon-like peptide 2 (glp-2) and uses thereof
WO2025106915A1 (en) 2023-11-17 2025-05-22 Sail Biomedicines, Inc. Circular polyribonucleotides encoding glucagon-like peptide 1 (glp-1) and uses thereof
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
WO2025133115A1 (en) 2023-12-21 2025-06-26 Ose Immunotherapeutics Lipid-based nanoparticles comprising il-35
CN120290557A (zh) * 2024-01-11 2025-07-11 深圳近邻生物科技有限公司 一种通用的且无“scar”的RNA环化方法
WO2025155753A2 (en) 2024-01-17 2025-07-24 Renagade Therapeutics Management Inc. Improved gene editing system, guides, and methods
WO2025166238A1 (en) 2024-01-31 2025-08-07 Orna Therapeutics, Inc. Fast-shedding polyethylene glycol lipids
WO2025179198A1 (en) 2024-02-23 2025-08-28 Sail Biomedicines, Inc. Circular polyribonucleotides and unmodified linear rnas with reduced immunogenicity
WO2025218812A1 (en) * 2024-04-19 2025-10-23 Shanghai Circode Biomed Co., Ltd. Novel internal ribosome entry sites and uses thereof
WO2025250751A1 (en) 2024-05-31 2025-12-04 Orna Therapeutics, Inc. Circular rna compositions and methods
CN118703488A (zh) * 2024-06-20 2024-09-27 毕昇(北京)生物科技有限公司 一种环状rna的纯化方法
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
WO2026068705A1 (en) 2024-09-26 2026-04-02 Ose Immunotherapeutics Lipid-based nanoparticles comprising non-glycosylated fc domains and uses thereof
GB202415784D0 (en) * 2024-10-25 2024-12-11 Rnavate Ltd Methods for purifying circular ribonucleic acid (RNA)
WO2026093414A1 (en) 2024-10-30 2026-05-07 Ose Immunotherapeutics Lipid-based nanoparticles comprising membrane il-35
WO2026096738A1 (en) 2024-10-31 2026-05-07 Orna Therapeutics, Inc. Chimeric antigen receptors targeting cd19
US20260115324A1 (en) * 2024-10-31 2026-04-30 Orna Therapeutics, Inc. Chimeric Antigen Receptors Targeting CD19
WO2026122418A1 (en) 2024-12-02 2026-06-11 Orna Therapeutics, Inc. Circular rna compositions and methods
WO2026122685A1 (en) 2024-12-03 2026-06-11 Orna Therapeutics, Inc. Lipid nanoparticles comprising coding rna molecules for use in gene editing and as vaccines and therapeutic agents
WO2026165530A1 (en) 2025-02-03 2026-08-06 Renagade Therapeutics Management Inc. Hydrophilic polymer lipids for use in lipid nanoparticles
CN119876152B (zh) * 2025-02-10 2026-07-24 毕昇(北京)生物科技有限公司 一种环状rna、嵌合抗原受体t细胞和构建方法及应用

Family Cites Families (18)

* 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
EP1027069B1 (en) 1997-10-29 2006-07-26 Genzyme Corporation Gene therapy for gaucher disease
US5948902A (en) 1997-11-20 1999-09-07 South Alabama Medical Science Foundation Antisense oligonucleotides to human serine/threonine protein phosphatase genes
EP1261379A2 (en) 2000-02-17 2002-12-04 Genzyme Corporation Genetic modification of the lung as a portal for gene delivery
AU2002368077B2 (en) 2001-07-12 2010-03-04 Jefferson Foote Super humanized antibodies
US20040014194A1 (en) 2002-03-27 2004-01-22 Schering Corporation Beta-secretase crystals and methods for preparing and using the same
EP1791865B1 (en) 2004-06-29 2010-07-28 Immunocore Ltd. Cells expressing a modified t cell receptor
US10059655B2 (en) 2013-12-19 2018-08-28 Novartis Ag Lipids and lipid compositions for the delivery of active agents
HRP20220156T1 (hr) 2015-09-17 2022-04-15 Modernatx, Inc. Spojevi i pripravci za unutarstaničnu isporuku terapeutskih sredstava
CN108368028B (zh) 2015-10-28 2021-09-03 爱康泰生治疗公司 用于递送核酸的新型脂质和脂质纳米颗粒制剂
WO2017222911A1 (en) * 2016-06-20 2017-12-28 The Board Of Trustees Of The Leland Stanford Junior University Circular rnas and their use in immunomodulation
WO2019081383A1 (en) 2017-10-25 2019-05-02 Universität Zürich EUCARYOTE 4 INITIATION FACTOR RECRUITMENT APTAMERS FOR ENHANCED TRANSLATION
US20190314291A1 (en) 2018-01-30 2019-10-17 Modernatx, Inc. Compositions and methods for delivery of agents to immune cells
IL276322B2 (en) 2018-02-01 2024-12-01 Tufts College Lipid-like nanocomplexes and uses thereof
WO2019191780A1 (en) 2018-03-30 2019-10-03 Arcturus Therapeutics, Inc. Lipid particles for nucleic acid delivery
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
JP7686574B2 (ja) * 2019-05-22 2025-06-02 マサチューセッツ インスティテュート オブ テクノロジー 環状ｒｎａ組成物及び方法

Also Published As

Publication number Publication date
US20250283073A1 (en) 2025-09-11
WO2022261490A3 (en) 2023-02-09
MX2023014702A (es) 2024-03-14
AU2022288705A9 (en) 2023-12-14
CA3219932A1 (en) 2022-12-15
AU2022288705A1 (en) 2023-12-07
TW202317767A (zh) 2023-05-01
IL308746A (en) 2024-03-01
KR20240019791A (ko) 2024-02-14
JP2024521444A (ja) 2024-05-31
EP4352234A2 (en) 2024-04-17
WO2022261490A2 (en) 2022-12-15

Similar Documents

Publication Publication Date Title
BR112022023554A2 (pt) Composições e métodos de rna circular
WO2022261490A3 (en) Circular rna compositions and methods
BR112022018854A2 (pt) Composições e métodos de rna circular
BR112021023411A2 (pt) Composições e métodos de rna circular
WO2024129982A3 (en) Circular rna compositions and methods
WO2021113777A3 (en) Circular rna compositions and methods
WO2021226597A3 (en) Circular rna compositions and methods
WO2022271965A3 (en) Compositions and methods for improved protein translation from recombinant circular rnas
MX2022009429A (es) Composiciones para la inmunizacion contra virus respiratorios.
CL2019003561A1 (es) Agentes potenciadores para la transfección celular mejorada y/o la producción del vector raav.
CY1126720T1 (el) Ανοσογονικη συνθεση
WO2023283642A3 (en) Pan-human coronavirus concatemeric vaccines
ZA202208795B (en) Compositions and methods for organ-protective expression and modulation of coding ribonucleic acids
CR20110629A (es) Anticuerpos contra el factor de crecimiento nervioso (ngf) con estabilidad in vivo mejorada
WO2019186274A3 (en) Micro rna expression constructs and uses thereof
MX2018009085A (es) Conjugados de anticuerpos anti-cd74, composiciones que comprenden conjugados de anticuerpos anti-cd74 y metodos de uso de congujados de anticuerpos anti-cd74.
PE20142406A1 (es) Antigenos asociados a prostata y regimenes de inmunoterapia basados en vacuna
CL2023002099A1 (es) Moléculas de unión al antígeno multiespecíficas dirigidas a claudina-6 y sus usos
BR112015019603A2 (pt) Moléculas de ácido nucleico isoladas, molécula de polipeptídeo isolada, molécula isolada de car, domínio de ligação anti-egfrviii, vetor, célula e uso de uma quantidade eficaz da mesma e métodos para produção de uma célula e para a produção de uma população de células modificadas por rna
BR112022010321A2 (pt) Composições e métodos para tratamento de neuropatia óptica hereditária de leber com proteínas nadh desidrogenase
WO2023104114A3 (en) Rna formulations and lipids
WO2023097003A3 (en) Immunogenic compositions and their uses
MX2022000055A (es) Enzimas de polimerización para reacciones de secuenciación.
CO2020000231A2 (es) Composiciones inmunogénicas que comprenden cea muc1 y tert
MX2025000087A (es) Edulcorantes proteicos