TW202317767A - 環狀rna組合物及方法 - Google Patents

環狀rna組合物及方法 Download PDF

Info

Publication number
TW202317767A
TW202317767A TW111121745A TW111121745A TW202317767A TW 202317767 A TW202317767 A TW 202317767A TW 111121745 A TW111121745 A TW 111121745A TW 111121745 A TW111121745 A TW 111121745A TW 202317767 A TW202317767 A TW 202317767A
Authority
TW
Taiwan
Prior art keywords
sequence
rna polynucleotide
precursor rna
polynucleotide according
fragment
Prior art date
Application number
TW111121745A
Other languages
English (en)
Chinese (zh)
Inventor
羅伯特 亞歷山大 魏索霍夫特
克莉絲汀 奧特
托馬斯 巴恩斯
格雷戈里 莫茲
艾咪 M 貝克
艾倫 T 霍哈塔
布萊恩 古德曼
舒緩
瓦倫 席凡夏卡
Original Assignee
美商歐納醫療公司
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 美商歐納醫療公司 filed Critical 美商歐納醫療公司
Publication of TW202317767A publication Critical patent/TW202317767A/zh

Links

Images

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)
TW111121745A 2021-06-10 2022-06-10 環狀rna組合物及方法 TW202317767A (zh)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US202163209271P 2021-06-10 2021-06-10
US63/209,271 2021-06-10
US202263311923P 2022-02-18 2022-02-18
US63/311,923 2022-02-18

Publications (1)

Publication Number Publication Date
TW202317767A true TW202317767A (zh) 2023-05-01

Family

ID=83081188

Family Applications (1)

Application Number Title Priority Date Filing Date
TW111121745A TW202317767A (zh) 2021-06-10 2022-06-10 環狀rna組合物及方法

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
IL308746A (en) 2024-03-01
KR20240019791A (ko) 2024-02-14
JP2024521444A (ja) 2024-05-31
EP4352234A2 (en) 2024-04-17
BR112023025515A2 (pt) 2024-02-27
WO2022261490A2 (en) 2022-12-15

Similar Documents

Publication Publication Date Title
KR20240019791A (ko) 원형 rna 조성물 및 방법
AU2020204196B2 (en) Optimal maize loci
AU2020203872B2 (en) Optimal maize loci
AU2020203837B2 (en) Isolated polynucleotides and polypeptides, and methods of using same for increasing plant yield and/or agricultural characteristics
AU2020202369B2 (en) Isolated polynucleotides and polypeptides, and methods of using same for increasing plant yield and/or agricultural characteristics
AU2023214237A1 (en) Modified polynucleotides for the production of biologics and proteins associated with human disease
KR102630357B1 (ko) 단백질 발현이 어려운 다중-부위 ssi 세포
AU2016381496B2 (en) Plant traits conferred by isolated polynucleotides and polypeptides
KR102719587B1 (ko) 아데노-관련 바이러스 생산을 위한 포유동물 세포
AU2017235661B2 (en) Oligonucleotide probes and uses thereof
AU2023251441A1 (en) RNA containing composition for treatment of tumor diseases
KR102880582B1 (ko) 다중화 게놈 편집
KR102523805B1 (ko) 면역 조정
TW202321448A (zh) 利用重組環狀rna改善蛋白質轉譯的組合物及方法
AU2019316556B2 (en) Methods for assessing the risk of developing progressive multifocal leukoencephalopathy caused by john cunningham virus by genetic testing
AU2021202753A1 (en) Isolated polynucleotides and polypeptides and methods of using same for increasing plant yield, biomass, growth rate, vigor, oil content, abiotic stress tolerance of plants and nitrogen use efficiency
AU2016376191A1 (en) Materials and methods for treatment of amyotrophic lateral sclerosis and/or frontal temporal lobular degeneration
AU2016364667A1 (en) Materials and methods for treatment of Alpha-1 antitrypsin deficiency
AU2017289886A1 (en) Engineered parasites for delivering protein to the central nervous system (CNS)
WO1995014772A1 (en) Gene signature
JP2003135075A (ja) 新規な全長cDNA
WO2000058473A2 (en) Nucleic acids including open reading frames encoding polypeptides; 'orfx'
JP2025011135A (ja) 核酸の標的化組込み
KR20110110030A (ko) 뇌종양의 재발 가능성 및 생존 예후 예측용 조성물 및 이를 포함하는 키트
KR101978399B1 (ko) 자궁내막증에 대한 진단 마커로서의 돌연변이 유전자 및 이의 용도