KR20240019791A - 원형 rna 조성물 및 방법 - Google Patents

원형 rna 조성물 및 방법 Download PDF

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Publication number
KR20240019791A
KR20240019791A KR1020247000046A KR20247000046A KR20240019791A KR 20240019791 A KR20240019791 A KR 20240019791A KR 1020247000046 A KR1020247000046 A KR 1020247000046A KR 20247000046 A KR20247000046 A KR 20247000046A KR 20240019791 A KR20240019791 A KR 20240019791A
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South Korea
Prior art keywords
sequence
rna
polypeptide
fragment
precursor rna
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Pending
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KR1020247000046A
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Korean (ko)
Inventor
로버트 알렉산더 베셀회프트
크리스텐 오트
토마스 반스
그레고리 모츠
에이미 엠. 베커
알렌 티. 호르호타
브라이언 굿맨
환 슈
바룬 시바샨카르
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오나 테라퓨틱스, 인코포레이티드
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Publication of KR20240019791A publication Critical patent/KR20240019791A/ko
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    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
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    • 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
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    • A61K40/31—Chimeric antigen receptors [CAR]
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61K40/41—Vertebrate antigens
    • A61K40/42—Cancer antigens
    • A61K40/4202—Receptors, cell surface antigens or cell surface determinants
    • A61K40/4203—Receptors for growth factors
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    • 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
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    • 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
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    • 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
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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/10—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
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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
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    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
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    • 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
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    • C12N15/09—Recombinant DNA-technology
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
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    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
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    • C07K2319/03—Fusion polypeptide containing a localisation/targetting motif containing a transmembrane segment
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  • Health & Medical Sciences (AREA)
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  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
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  • Biomedical Technology (AREA)
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  • 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)
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  • 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)
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  • Analytical Chemistry (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
KR1020247000046A 2021-06-10 2022-06-10 원형 rna 조성물 및 방법 Pending KR20240019791A (ko)

Applications Claiming Priority (5)

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
PCT/US2022/033091 WO2022261490A2 (en) 2021-06-10 2022-06-10 Circular rna compositions and methods

Publications (1)

Publication Number Publication Date
KR20240019791A true KR20240019791A (ko) 2024-02-14

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KR1020247000046A Pending KR20240019791A (ko) 2021-06-10 2022-06-10 원형 rna 조성물 및 방법

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

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CN119110847A (zh) * 2022-01-11 2024-12-10 北京大学 针对sars-cov-2变体的环状rna疫苗及其使用方法
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AU2023398825A1 (en) 2022-12-15 2025-06-26 Orna Therapeutics, Inc. Circular rna compositions and methods
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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
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