KR102475301B1 - 다량체 코딩 핵산 및 그 용도 - Google Patents

다량체 코딩 핵산 및 그 용도 Download PDF

Info

Publication number
KR102475301B1
KR102475301B1 KR1020227006425A KR20227006425A KR102475301B1 KR 102475301 B1 KR102475301 B1 KR 102475301B1 KR 1020227006425 A KR1020227006425 A KR 1020227006425A KR 20227006425 A KR20227006425 A KR 20227006425A KR 102475301 B1 KR102475301 B1 KR 102475301B1
Authority
KR
South Korea
Prior art keywords
mcna
delete delete
encoding
nucleic acid
multimeric
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.)
Active
Application number
KR1020227006425A
Other languages
English (en)
Korean (ko)
Other versions
KR20220031741A (ko
Inventor
프랑크 데로사
마이클 하트레인
다니엘 크로포드
스리랑 카르베
Original Assignee
트랜슬레이트 바이오 인코포레이티드
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 트랜슬레이트 바이오 인코포레이티드 filed Critical 트랜슬레이트 바이오 인코포레이티드
Publication of KR20220031741A publication Critical patent/KR20220031741A/ko
Application granted granted Critical
Publication of KR102475301B1 publication Critical patent/KR102475301B1/ko
Active legal-status Critical Current
Anticipated expiration legal-status Critical

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
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7115—Nucleic acids or oligonucleotides having modified bases, i.e. other than adenine, guanine, cytosine, uracil or thymine
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7125—Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/177—Receptors; Cell surface antigens; Cell surface determinants
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/18—Growth factors; Growth regulators
    • A61K38/1816—Erythropoietin [EPO]
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/44—Oxidoreductases (1)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/45—Transferases (2)
    • 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
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C07H21/02—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with ribosyl as saccharide radical
    • 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
    • C07K14/475—Growth factors; Growth regulators
    • C07K14/505—Erythropoietin [EPO]
    • 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
    • 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
    • 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
    • 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
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/0004—Oxidoreductases (1.)
    • C12N9/0071—Oxidoreductases (1.) acting on paired donors with incorporation of molecular oxygen (1.14)
    • 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
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/10—Transferases (2.)
    • C12N9/1003—Transferases (2.) transferring one-carbon groups (2.1)
    • C12N9/1018—Carboxy- and carbamoyl transferases (2.1.3)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12P—FERMENTATION OR ENZYME-USING PROCESSES TO SYNTHESISE A DESIRED CHEMICAL COMPOUND OR COMPOSITION OR TO SEPARATE OPTICAL ISOMERS FROM A RACEMIC MIXTURE
    • C12P21/00—Preparation of peptides or proteins
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y114/00—Oxidoreductases acting on paired donors, with incorporation or reduction of molecular oxygen (1.14)
    • C12Y114/16—Oxidoreductases acting on paired donors, with incorporation or reduction of molecular oxygen (1.14) with reduced pteridine as one donor, and incorporation of one atom of oxygen (1.14.16)
    • C12Y114/16001—Phenylalanine 4-monooxygenase (1.14.16.1)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y201/00—Transferases transferring one-carbon groups (2.1)
    • C12Y201/03—Carboxy- and carbamoyltransferases (2.1.3)
    • C12Y201/03003—Ornithine carbamoyltransferase (2.1.3.3)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/50—Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Biochemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Epidemiology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Immunology (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Cell Biology (AREA)
  • Toxicology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Saccharide Compounds (AREA)
KR1020227006425A 2016-04-08 2017-04-07 다량체 코딩 핵산 및 그 용도 Active KR102475301B1 (ko)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201662320073P 2016-04-08 2016-04-08
US62/320,073 2016-04-08
PCT/US2017/026660 WO2017177169A1 (en) 2016-04-08 2017-04-07 Multimeric coding nucleic acid and uses thereof
KR1020187028692A KR102369898B1 (ko) 2016-04-08 2017-04-07 다량체 코딩 핵산 및 그 용도

Related Parent Applications (1)

Application Number Title Priority Date Filing Date
KR1020187028692A Division KR102369898B1 (ko) 2016-04-08 2017-04-07 다량체 코딩 핵산 및 그 용도

Publications (2)

Publication Number Publication Date
KR20220031741A KR20220031741A (ko) 2022-03-11
KR102475301B1 true KR102475301B1 (ko) 2022-12-09

Family

ID=58671902

Family Applications (2)

Application Number Title Priority Date Filing Date
KR1020227006425A Active KR102475301B1 (ko) 2016-04-08 2017-04-07 다량체 코딩 핵산 및 그 용도
KR1020187028692A Active KR102369898B1 (ko) 2016-04-08 2017-04-07 다량체 코딩 핵산 및 그 용도

Family Applications After (1)

Application Number Title Priority Date Filing Date
KR1020187028692A Active KR102369898B1 (ko) 2016-04-08 2017-04-07 다량체 코딩 핵산 및 그 용도

Country Status (10)

Country Link
US (4) US10266843B2 (enExample)
EP (2) EP3825400B1 (enExample)
JP (1) JP7150608B6 (enExample)
KR (2) KR102475301B1 (enExample)
CN (1) CN109072223B (enExample)
AU (3) AU2017248189B2 (enExample)
CA (1) CA3020343A1 (enExample)
DK (1) DK3440206T3 (enExample)
ES (2) ES2844180T3 (enExample)
WO (1) WO2017177169A1 (enExample)

Families Citing this family (22)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP4074693A1 (en) 2011-06-08 2022-10-19 Translate Bio, Inc. Cleavable lipids
AU2017248189B2 (en) * 2016-04-08 2021-04-29 Translate Bio, Inc. Multimeric coding nucleic acid and uses thereof
WO2018132768A1 (en) * 2017-01-13 2018-07-19 Sanna Pietro P Methods and compositions for treating hpa hyperactivity
MX2019010155A (es) * 2017-02-27 2020-12-10 Translate Bio Inc Arnm de cftr optimizado por codón novedoso.
US11820999B2 (en) 2017-04-03 2023-11-21 American Gene Technologies International Inc. Compositions and methods for treating phenylketonuria
EP3714048B1 (en) 2017-11-22 2025-04-09 ModernaTX, Inc. Polynucleotides encoding ornithine transcarbamylase for the treatment of urea cycle disorders
US11939601B2 (en) 2017-11-22 2024-03-26 Modernatx, Inc. Polynucleotides encoding phenylalanine hydroxylase for the treatment of phenylketonuria
EP3793689A1 (en) * 2018-05-15 2021-03-24 Translate Bio, Inc. Subcutaneous delivery of messenger rna
WO2020023533A1 (en) * 2018-07-23 2020-01-30 Translate Bio, Inc. Dry power formulations for messenger rna
MY207814A (en) * 2019-03-07 2025-03-20 Reti Mark Co Ltd Combination of biomarkers for diagnosing of diabetic retinopathy and use thereof
WO2020180147A2 (ko) * 2019-03-07 2020-09-10 (주)레티마크 나이관련 황반변성 진단용 복합 마커 및 이의 용도
BR112021024124A2 (pt) * 2019-05-31 2022-01-11 American Gene Tech Int Inc Expressão otimizada de fenilalanina hidroxilase
CA3155003A1 (en) 2019-09-20 2021-03-25 Translate Bio, Inc. Mrna encoding engineered cftr
CA3170319A1 (en) 2020-02-10 2021-08-19 Translate Bio, Inc. Methods and compositions for messenger rna purification
CN115427021A (zh) 2020-02-25 2022-12-02 翻译生物公司 制备负载mrna的脂质纳米颗粒的改进方法
MX2023004371A (es) 2020-10-14 2023-07-26 George Mason Res Foundation Inc Lípidos ionizables, y métodos de fabricación y uso de los mismos.
CN113875706B (zh) * 2021-11-17 2024-11-01 贵州省烟草公司遵义市公司凤冈县分公司 一种便于统计野生蠋蝽量的循化装置的使用方法
US12083189B2 (en) * 2022-04-29 2024-09-10 Tiba Biotech, Llc Tail-conjugated RNAs
WO2024218715A1 (en) 2023-04-19 2024-10-24 Btl Healthcare Technologies A.S. Culture media based on protein hydrolysate and a process for preparing thereof
US12668774B2 (en) 2023-10-12 2026-06-30 Btl Healthcare Technologies A.S. Product, system and method of cell cultivation
CN117987436B (zh) * 2024-04-03 2024-06-25 南京鸿明生物科技有限公司 一种双链靶dna序列的制备方法
WO2026052111A1 (zh) * 2024-09-06 2026-03-12 图维生物医药科技(苏州)有限公司 一种多聚mRNA

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2013040429A1 (en) 2011-09-14 2013-03-21 Rana Therapeutics Inc. Multimeric oligonucleotide compounds
WO2015023975A1 (en) 2013-08-16 2015-02-19 Rana Therapeutics, Inc. Compositions and methods for modulating rna

Family Cites Families (158)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4500707A (en) 1980-02-29 1985-02-19 University Patents, Inc. Nucleosides useful in the preparation of polynucleotides
US4458066A (en) 1980-02-29 1984-07-03 University Patents, Inc. Process for preparing polynucleotides
US5132418A (en) 1980-02-29 1992-07-21 University Patents, Inc. Process for preparing polynucleotides
US4415732A (en) 1981-03-27 1983-11-15 University Patents, Inc. Phosphoramidite compounds and processes
US4973679A (en) 1981-03-27 1990-11-27 University Patents, Inc. Process for oligonucleo tide synthesis using phosphormidite intermediates
US4668777A (en) 1981-03-27 1987-05-26 University Patents, Inc. Phosphoramidite nucleoside compounds
US4401796A (en) 1981-04-30 1983-08-30 City Of Hope Research Institute Solid-phase synthesis of polynucleotides
US4373071A (en) 1981-04-30 1983-02-08 City Of Hope Research Institute Solid-phase synthesis of polynucleotides
US4897355A (en) 1985-01-07 1990-01-30 Syntex (U.S.A.) Inc. N[ω,(ω-1)-dialkyloxy]- and N-[ω,(ω-1)-dialkenyloxy]-alk-1-yl-N,N,N-tetrasubstituted ammonium lipids and uses therefor
US4737323A (en) 1986-02-13 1988-04-12 Liposome Technology, Inc. Liposome extrusion method
US5153319A (en) 1986-03-31 1992-10-06 University Patents, Inc. Process for preparing polynucleotides
US5262530A (en) 1988-12-21 1993-11-16 Applied Biosystems, Inc. Automated system for polynucleotide synthesis and purification
US5047524A (en) 1988-12-21 1991-09-10 Applied Biosystems, Inc. Automated system for polynucleotide synthesis and purification
FR2645866B1 (fr) 1989-04-17 1991-07-05 Centre Nat Rech Scient Nouvelles lipopolyamines, leur preparation et leur emploi
WO1996040703A1 (en) * 1995-06-07 1996-12-19 Nexstar Pharmaceuticals, Inc. High affinity nucleic acid ligands to lectins
US6280932B1 (en) * 1990-06-11 2001-08-28 Gilead Sciences, Inc. High affinity nucleic acid ligands to lectins
EP0464638B1 (de) * 1990-07-02 1997-04-02 Hoechst Aktiengesellschaft Oligonucleotid-analoge mit terminalen 3'-3'-bzw. 5'-5'-Internucleotidverknüpfungen
US5334761A (en) 1992-08-28 1994-08-02 Life Technologies, Inc. Cationic lipids
DE69514351T2 (de) * 1994-06-01 2000-08-10 Hybridon, Inc. Verzweigte Oligonukleotide als pathogenhemmende Mittel
US5885613A (en) 1994-09-30 1999-03-23 The University Of British Columbia Bilayer stabilizing components and their use in forming programmable fusogenic liposomes
US5700642A (en) 1995-05-22 1997-12-23 Sri International Oligonucleotide sizing using immobilized cleavable primers
JP4335310B2 (ja) 1995-06-07 2009-09-30 ザ ユニバーシティ オブ ブリティッシュ コロンビア 疎水性脂質−核酸複合中間体を通して調製される脂質−核酸粒子、及び遺伝子移送のための使用
US7422902B1 (en) 1995-06-07 2008-09-09 The University Of British Columbia Lipid-nucleic acid particles prepared via a hydrophobic lipid-nucleic acid complex intermediate and use for gene transfer
US5981501A (en) 1995-06-07 1999-11-09 Inex Pharmaceuticals Corp. Methods for encapsulating plasmids in lipid bilayers
US5705385A (en) 1995-06-07 1998-01-06 Inex Pharmaceuticals Corporation Lipid-nucleic acid particles prepared via a hydrophobic lipid-nucleic acid complex intermediate and use for gene transfer
US5744335A (en) 1995-09-19 1998-04-28 Mirus Corporation Process of transfecting a cell with a polynucleotide mixed with an amphipathic compound and a DNA-binding protein
DE10162480A1 (de) 2001-12-19 2003-08-07 Ingmar Hoerr Die Applikation von mRNA für den Einsatz als Therapeutikum gegen Tumorerkrankungen
EP1519714B1 (en) 2002-06-28 2010-10-20 Protiva Biotherapeutics Inc. Method and apparatus for producing liposomes
CN1882693B (zh) 2003-09-15 2012-08-15 普洛体维生物治疗公司 聚乙二醇修饰的脂质化合物及其应用
CA2569664C (en) 2004-06-07 2013-07-16 Protiva Biotherapeutics, Inc. Lipid encapsulated interfering rna
US7745651B2 (en) 2004-06-07 2010-06-29 Protiva Biotherapeutics, Inc. Cationic lipids and methods of use
DE102004042546A1 (de) 2004-09-02 2006-03-09 Curevac Gmbh Kombinationstherapie zur Immunstimulation
BRPI0606260A2 (pt) * 2005-01-07 2009-11-10 De Ruiter Seeds R & D Bv vìrus, ácido nucleico isolado ou recombinante ou fragmento especìfico do mesmo para totv, vetor de expressão, polinucleotìdeo, antìgeno, anticorpo, métodos para produzir um anticorpo contra totv, para identificar um isolado viral como um vìrus totv, para detectar a presença de totv em uma amostra, e para indentificar e produzir uma planta resistente a totv, planta resistente a totv, ou parte da mesma, kit diagnóstico, uso de um vìrus, composição diagnóstica, uso de um vìrus, ou seu genoma, ou partes do mesmo, e, uso de uma forma atenuada de um vìrus, ou seu genoma, ou partes do mesmo
EP1948674A4 (en) 2005-11-02 2009-02-04 Protiva Biotherapeutics Inc MODIFIED SIRNA MOLECULES AND APPLICATIONS THEREOF
EP2049665A2 (en) 2006-07-28 2009-04-22 Applera Corporation Dinucleotide mrna cap analogs
WO2009030254A1 (en) 2007-09-04 2009-03-12 Curevac Gmbh Complexes of rna and cationic peptides for transfection and for immunostimulation
WO2009046739A1 (en) 2007-10-09 2009-04-16 Curevac Gmbh Composition for treating prostate cancer (pca)
WO2009058911A2 (en) 2007-10-31 2009-05-07 Applied Biosystems Inc. Preparation and isolation of 5' capped mrna
EP2229186A2 (en) 2007-12-04 2010-09-22 Alnylam Pharmaceuticals Inc. Carbohydrate conjugates as delivery agents for oligonucleotides
US9006191B2 (en) 2007-12-27 2015-04-14 Protiva Biotherapeutics, Inc. Silencing of polo-like kinase expression using interfering RNA
US20110097720A1 (en) 2008-01-02 2011-04-28 Alnylam Pharmaceuticals, Inc. Screening method for selected amino lipid-containing compositions
US8575123B2 (en) 2008-04-11 2013-11-05 Tekmira Pharmaceuticals Corporation Site-specific delivery of nucleic acids by combining targeting ligands with endosomolytic components
EP2279254B1 (en) 2008-04-15 2017-07-05 Protiva Biotherapeutics Inc. Novel lipid formulations for nucleic acid delivery
WO2009127230A1 (en) * 2008-04-16 2009-10-22 Curevac Gmbh MODIFIED (m)RNA FOR SUPPRESSING OR AVOIDING AN IMMUNOSTIMULATORY RESPONSE AND IMMUNOSUPPRESSIVE COMPOSITION
WO2010037408A1 (en) 2008-09-30 2010-04-08 Curevac Gmbh Composition comprising a complexed (m)rna and a naked mrna for providing or enhancing an immunostimulatory response in a mammal and uses thereof
AU2009303345B2 (en) 2008-10-09 2015-08-20 Arbutus Biopharma Corporation Improved amino lipids and methods for the delivery of nucleic acids
WO2010053572A2 (en) 2008-11-07 2010-05-14 Massachusetts Institute Of Technology Aminoalcohol lipidoids and uses thereof
EP3699172A3 (en) 2008-11-10 2020-11-18 Arbutus Biopharma Corporation Novel lipids and compositions for the delivery of therapeutics
EP2405921A4 (en) 2009-01-26 2013-05-22 Protiva Biotherapeutics Inc COMPOSITIONS AND METHODS FOR VAPORING APOLIPOPROTIEN C-III EXPRESSION
CA3042927C (en) 2009-05-05 2022-05-17 Arbutus Biopharma Corporation Lipid compositions for the delivery of therapeutic agents
KR20220038506A (ko) 2009-06-10 2022-03-28 알닐람 파마슈티칼스 인코포레이티드 향상된 지질 조성물
US9051567B2 (en) 2009-06-15 2015-06-09 Tekmira Pharmaceuticals Corporation Methods for increasing efficacy of lipid formulated siRNA
US8569256B2 (en) 2009-07-01 2013-10-29 Protiva Biotherapeutics, Inc. Cationic lipids and methods for the delivery of therapeutic agents
EP2449106B1 (en) 2009-07-01 2015-04-08 Protiva Biotherapeutics Inc. Compositions and methods for silencing apolipoprotein b
WO2011011447A1 (en) 2009-07-20 2011-01-27 Protiva Biotherapeutics, Inc. Compositions and methods for silencing ebola virus gene expression
SI2506857T1 (en) 2009-12-01 2018-08-31 Translate Bio, Inc. Delivery of mRNA for the enrichment of proteins and enzymes in human genetic diseases
CA3044884A1 (en) 2009-12-07 2011-06-16 Arbutus Biopharma Corporation Compositions for nucleic acid delivery
WO2011069529A1 (en) 2009-12-09 2011-06-16 Curevac Gmbh Mannose-containing solution for lyophilization, transfection and/or injection of nucleic acids
US20130017223A1 (en) 2009-12-18 2013-01-17 The University Of British Columbia Methods and compositions for delivery of nucleic acids
US20130123338A1 (en) 2010-05-12 2013-05-16 Protiva Biotherapeutics, Inc. Novel cationic lipids and methods of use thereof
US9006417B2 (en) 2010-06-30 2015-04-14 Protiva Biotherapeutics, Inc. Non-liposomal systems for nucleic acid delivery
WO2012016184A2 (en) 2010-07-30 2012-02-02 Alnylam Pharmaceuticals, Inc. Methods and compositions for delivery of active agents
CA2807552A1 (en) 2010-08-06 2012-02-09 Moderna Therapeutics, Inc. Engineered nucleic acids and methods of use thereof
EP2857499A1 (en) 2010-10-01 2015-04-08 Moderna Therapeutics, Inc. Engineered nucleic acids and methods of use thereof
US8853377B2 (en) * 2010-11-30 2014-10-07 Shire Human Genetic Therapies, Inc. mRNA for use in treatment of human genetic diseases
WO2012116715A1 (en) 2011-03-02 2012-09-07 Curevac Gmbh Vaccination in newborns and infants
WO2012113413A1 (en) 2011-02-21 2012-08-30 Curevac Gmbh Vaccine composition comprising complexed immunostimulatory nucleic acids and antigens packaged with disulfide-linked polyethyleneglycol/peptide conjugates
WO2012116714A1 (en) 2011-03-02 2012-09-07 Curevac Gmbh Vaccination in elderly patients
CA2831613A1 (en) 2011-03-31 2012-10-04 Moderna Therapeutics, Inc. Delivery and formulation of engineered nucleic acids
CN103687957A (zh) 2011-05-17 2014-03-26 现代治疗公司 工程化核酸及其用于非人类脊椎动物的方法
KR20220025112A (ko) 2011-06-08 2022-03-03 샤이어 휴먼 지네틱 테라피즈 인크. Mrna 전달을 위한 지질 나노입자 조성물 및 방법
EP4074693A1 (en) 2011-06-08 2022-10-19 Translate Bio, Inc. Cleavable lipids
EP3384938A1 (en) 2011-09-12 2018-10-10 Moderna Therapeutics, Inc. Engineered nucleic acids and methods of use thereof
WO2013039857A1 (en) 2011-09-12 2013-03-21 modeRNA Therapeutics Engineered nucleic acids and methods of use thereof
US9464124B2 (en) 2011-09-12 2016-10-11 Moderna Therapeutics, Inc. Engineered nucleic acids and methods of use thereof
PT3682905T (pt) 2011-10-03 2022-04-07 Modernatx Inc Nucleósidos, nucleótidos e ácidos nucleicos modificados e respetivas utilizações
US9512073B2 (en) 2011-10-27 2016-12-06 Massachusetts Institute Of Technology Amino acid-, peptide-and polypeptide-lipids, isomers, compositions, and uses thereof
EP2791159A4 (en) 2011-12-14 2015-10-14 Moderna Therapeutics Inc MODIFIED NUCLEIC ACIDS AND ACUTE TREATMENT USES THEREOF
WO2013130161A1 (en) 2011-12-14 2013-09-06 modeRNA Therapeutics Methods of responding to a biothreat
ES2923757T3 (es) 2011-12-16 2022-09-30 Modernatx Inc Composiciones de ARNm modificado
AU2012358384A1 (en) 2011-12-21 2014-07-31 Moderna Therapeutics, Inc. Methods of increasing the viability or longevity of an organ or organ explant
US20140371302A1 (en) 2011-12-29 2014-12-18 Modema Therapeutics, Inc. Modified mrnas encoding cell-penetrating polypeptides
WO2013120499A1 (en) 2012-02-15 2013-08-22 Curevac Gmbh Nucleic acid comprising or coding for a histone stem-loop and a poly (a) sequence or a polyadenylation signal for increasing the expression of an encoded pathogenic antigen
WO2013120497A1 (en) 2012-02-15 2013-08-22 Curevac Gmbh Nucleic acid comprising or coding for a histone stem-loop and a poly(a) sequence or a polyadenylation signal for increasing the expression of an encoded therapeutic protein
US9352042B2 (en) 2012-02-24 2016-05-31 Protiva Biotherapeutics, Inc. Trialkyl cationic lipids and methods of use thereof
WO2013151664A1 (en) 2012-04-02 2013-10-10 modeRNA Therapeutics Modified polynucleotides for the production of proteins
WO2013143698A1 (en) * 2012-03-27 2013-10-03 Curevac Gmbh Artificial nucleic acid molecules
RU2658490C2 (ru) 2012-03-27 2018-06-21 Кьюрвак Аг Искусственные молекулы нуклеиновых кислот для улучшенной экспрессии белков или пептидов
US10501512B2 (en) 2012-04-02 2019-12-10 Modernatx, Inc. Modified polynucleotides
US9283287B2 (en) 2012-04-02 2016-03-15 Moderna Therapeutics, Inc. Modified polynucleotides for the production of nuclear proteins
CN104411338A (zh) 2012-04-02 2015-03-11 现代治疗公司 用于产生与人类疾病相关的生物制剂和蛋白质的修饰多核苷酸
US9572897B2 (en) 2012-04-02 2017-02-21 Modernatx, Inc. Modified polynucleotides for the production of cytoplasmic and cytoskeletal proteins
US20150050354A1 (en) 2012-04-02 2015-02-19 Moderna Therapeutics, Inc. Modified polynucleotides for the treatment of otic diseases and conditions
US20140275229A1 (en) 2012-04-02 2014-09-18 Moderna Therapeutics, Inc. Modified polynucleotides encoding udp glucuronosyltransferase 1 family, polypeptide a1
WO2014093924A1 (en) 2012-12-13 2014-06-19 Moderna Therapeutics, Inc. Modified nucleic acid molecules and uses thereof
CA2889044A1 (en) * 2012-11-15 2014-05-22 Roche Innovation Center Copenhagen A/S Anti apob antisense conjugate compounds
PT2922554T (pt) 2012-11-26 2022-06-28 Modernatx Inc Arn modificado nas porções terminais
US9636301B2 (en) 2012-12-04 2017-05-02 Arbutus Biopharma Corporation In vitro release assay for liposome encapsulated vincristine
US20150366997A1 (en) 2012-12-07 2015-12-24 Shire Human Genetics Therapies, Inc. COMPOSITIONS AND METHODS FOR mRNA DELIVERY
WO2014093574A1 (en) 2012-12-13 2014-06-19 Moderna Therapeutics, Inc. Modified polynucleotides for altering cell phenotype
EP3434774A1 (en) 2013-01-17 2019-01-30 ModernaTX, Inc. Signal-sensor polynucleotides for the alteration of cellular phenotypes
US20160022774A1 (en) 2013-03-12 2016-01-28 Moderna Therapeutics, Inc. Diagnosis and treatment of fibrosis
US20160024181A1 (en) 2013-03-13 2016-01-28 Moderna Therapeutics, Inc. Long-lived polynucleotide molecules
KR102311614B1 (ko) 2013-03-14 2021-10-08 샤이어 휴먼 지네틱 테라피즈 인크. Cftr mrna 조성물 및 관련 방법 및 사용
US10258698B2 (en) 2013-03-14 2019-04-16 Modernatx, Inc. Formulation and delivery of modified nucleoside, nucleotide, and nucleic acid compositions
US10266559B2 (en) * 2013-03-14 2019-04-23 Translate Bio, Inc. Ribonucleic acids with 4′-thio-modified nucleotides and related methods
US8980864B2 (en) 2013-03-15 2015-03-17 Moderna Therapeutics, Inc. Compositions and methods of altering cholesterol levels
WO2014144711A1 (en) 2013-03-15 2014-09-18 Moderna Therapeutics, Inc. Analysis of mrna heterogeneity and stability
US10077439B2 (en) 2013-03-15 2018-09-18 Modernatx, Inc. Removal of DNA fragments in mRNA production process
EP2971033B8 (en) 2013-03-15 2019-07-10 ModernaTX, Inc. Manufacturing methods for production of rna transcripts
EP4279610A3 (en) 2013-03-15 2024-01-03 ModernaTX, Inc. Ribonucleic acid purification
US20160032273A1 (en) 2013-03-15 2016-02-04 Moderna Therapeutics, Inc. Characterization of mrna molecules
WO2014144767A1 (en) 2013-03-15 2014-09-18 Moderna Therapeutics, Inc. Ion exchange purification of mrna
PL3019619T3 (pl) 2013-07-11 2022-01-10 Modernatx, Inc. Kompozycje zawierające syntetyczne polinkleotydy kodujące białka powiązane z crispr i syntetyczne sgrna oraz sposoby ich stosowania
WO2015011633A1 (en) 2013-07-23 2015-01-29 Protiva Biotherapeutics, Inc. Compositions and methods for delivering messenger rna
BR112016003361A2 (pt) 2013-08-21 2017-11-21 Curevac Ag vacina do vírus sincicial respiratório (rsv)
WO2015034925A1 (en) 2013-09-03 2015-03-12 Moderna Therapeutics, Inc. Circular polynucleotides
CA2923029A1 (en) 2013-09-03 2015-03-12 Moderna Therapeutics, Inc. Chimeric polynucleotides
US10023626B2 (en) 2013-09-30 2018-07-17 Modernatx, Inc. Polynucleotides encoding immune modulating polypeptides
WO2015051173A2 (en) 2013-10-02 2015-04-09 Moderna Therapeutics, Inc Polynucleotide molecules and uses thereof
EP3052511A4 (en) 2013-10-02 2017-05-31 Moderna Therapeutics, Inc. Polynucleotide molecules and uses thereof
US20160243221A1 (en) 2013-10-18 2016-08-25 Moderna Therapeutics, Inc. Compositions and methods for tolerizing cellular systems
EP3574923A1 (en) * 2013-10-22 2019-12-04 Translate Bio, Inc. Mrna therapy for phenylketonuria
CN110003066B (zh) 2013-11-18 2021-09-03 阿克丘勒斯治疗公司 用于rna递送的可电离的阳离子脂质
US20170173128A1 (en) 2013-12-06 2017-06-22 Moderna TX, Inc. Targeted adaptive vaccines
US20150167017A1 (en) 2013-12-13 2015-06-18 Moderna Therapeutics, Inc. Alternative nucleic acid molecules and uses thereof
AU2014375402B2 (en) 2013-12-30 2020-10-01 CureVac SE Artificial nucleic acid molecules
US20170002060A1 (en) 2014-01-08 2017-01-05 Moderna Therapeutics, Inc. Polynucleotides for the in vivo production of antibodies
CA2935878C (en) 2014-03-12 2023-05-02 Curevac Ag Combination of vaccination and ox40 agonists
HRP20220070T1 (hr) 2014-04-23 2022-04-01 Modernatx, Inc. Cjepiva nukleinske kiseline
SG11201608605QA (en) 2014-06-10 2016-12-29 Curevac Ag Methods and means for enhancing rna production
US20170136132A1 (en) 2014-06-19 2017-05-18 Moderna Therapeutics, Inc. Alternative nucleic acid molecules and uses thereof
WO2015196130A2 (en) 2014-06-19 2015-12-23 Moderna Therapeutics, Inc. Alternative nucleic acid molecules and uses thereof
US10286086B2 (en) 2014-06-19 2019-05-14 Modernatx, Inc. Alternative nucleic acid molecules and uses thereof
CA2953341C (en) 2014-06-25 2023-01-24 Acuitas Therapeutics Inc. Lipids and lipid nanoparticle formulations for delivery of nucleic acids
WO2016005004A1 (en) 2014-07-11 2016-01-14 Biontech Rna Pharmaceuticals Gmbh Stabilization of poly(a) sequence encoding dna sequences
US10415037B2 (en) 2014-10-02 2019-09-17 Arbutus Biopharma Corporation Compositions and methods for silencing hepatitis B virus gene expression
WO2016071857A1 (en) 2014-11-07 2016-05-12 Protiva Biotherapeutics, Inc. Compositions and methods for silencing ebola virus expression
EP4324473A3 (en) 2014-11-10 2024-05-29 ModernaTX, Inc. Multiparametric nucleic acid optimization
EP3461904A1 (en) 2014-11-10 2019-04-03 ModernaTX, Inc. Alternative nucleic acid molecules containing reduced uracil content and uses thereof
WO2016118724A1 (en) 2015-01-21 2016-07-28 Moderna Therapeutics, Inc. Lipid nanoparticle compositions
EP3247398A4 (en) 2015-01-23 2018-09-26 Moderna Therapeutics, Inc. Lipid nanoparticle compositions
CA2979998A1 (en) 2015-03-20 2016-09-29 Protiva Biotherapeutics, Inc. Compositions and methods for treating hypertriglyceridemia
WO2016164762A1 (en) 2015-04-08 2016-10-13 Moderna Therapeutics, Inc. Polynucleotides encoding low density lipoprotein receptor egf-a and intracellular domain mutants and methods of using the same
WO2016183366A2 (en) 2015-05-12 2016-11-17 Protiva Biotherapeutics, Inc. Compositions and methods for silencing expression of hepatitis d virus rna
WO2016197133A1 (en) 2015-06-04 2016-12-08 Protiva Biotherapeutics, Inc. Delivering crispr therapeutics with lipid nanoparticles
WO2016197132A1 (en) 2015-06-04 2016-12-08 Protiva Biotherapeutics Inc. Treating hepatitis b virus infection using crispr
WO2016201377A1 (en) 2015-06-10 2016-12-15 Moderna Therapeutics, Inc. Targeted adaptive vaccines
CN108350455A (zh) 2015-07-29 2018-07-31 阿布特斯生物制药公司 用于使b型肝炎病毒基因表达沉默的组合物和方法
EP4101930A1 (en) 2015-09-17 2022-12-14 ModernaTX, Inc. Polynucleotides containing a stabilizing tail region
WO2017049286A1 (en) 2015-09-17 2017-03-23 Moderna Therapeutics, Inc. Polynucleotides containing a morpholino linker
US20190054112A1 (en) 2015-09-18 2019-02-21 Moderna Therapeutics, Inc. Polynucleotide formulations for use in the treatment of renal diseases
WO2017102010A1 (en) 2015-12-17 2017-06-22 Biontech Rna Pharmaceuticals Gmbh Novel cytokine fusion proteins
WO2017108087A1 (en) 2015-12-21 2017-06-29 Curevac Ag Inlay for a culture plate and corresponding method for preparing a culture plate system with such inlay
WO2017109134A1 (en) 2015-12-22 2017-06-29 Curevac Ag Method for producing rna molecule compositions
EP3394280A1 (en) 2015-12-23 2018-10-31 CureVac AG Method of rna in vitro transcription using a buffer containing a dicarboxylic acid or tricarboxylic acid or a salt thereof
AU2017248189B2 (en) * 2016-04-08 2021-04-29 Translate Bio, Inc. Multimeric coding nucleic acid and uses thereof
USD787703S1 (en) 2016-04-26 2017-05-23 Curevac Ag Inlay for a culture plate

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2013040429A1 (en) 2011-09-14 2013-03-21 Rana Therapeutics Inc. Multimeric oligonucleotide compounds
WO2015023975A1 (en) 2013-08-16 2015-02-19 Rana Therapeutics, Inc. Compositions and methods for modulating rna

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
J Nucleic Acids.,doi: 10.1155/2012/371379(2012.9.23.)
Nat Struct Mol Biol.,20(10):1214-1220(2013.9.12.)

Also Published As

Publication number Publication date
US10266843B2 (en) 2019-04-23
AU2024202844A1 (en) 2024-05-23
KR20190027353A (ko) 2019-03-14
AU2017248189B2 (en) 2021-04-29
US20190249191A1 (en) 2019-08-15
US20220073944A1 (en) 2022-03-10
US20170314041A1 (en) 2017-11-02
ES2844180T3 (es) 2021-07-21
EP3440206B1 (en) 2020-10-28
CA3020343A1 (en) 2017-10-12
WO2017177169A1 (en) 2017-10-12
JP7150608B6 (ja) 2022-11-11
AU2021206908A1 (en) 2021-08-19
DK3440206T3 (da) 2020-11-23
EP3825400C0 (en) 2024-12-25
AU2017248189A1 (en) 2018-10-18
JP2019513372A (ja) 2019-05-30
KR102369898B1 (ko) 2022-03-03
AU2021206908B2 (en) 2024-02-08
KR20220031741A (ko) 2022-03-11
EP3825400A1 (en) 2021-05-26
CN109072223B (zh) 2022-10-21
EP3440206A1 (en) 2019-02-13
ES3009710T3 (en) 2025-03-31
EP3825400B1 (en) 2024-12-25
US11124804B2 (en) 2021-09-21
US20200172923A1 (en) 2020-06-04
CN109072223A (zh) 2018-12-21
JP7150608B2 (ja) 2022-10-11
US10428349B2 (en) 2019-10-01

Similar Documents

Publication Publication Date Title
KR102369898B1 (ko) 다량체 코딩 핵산 및 그 용도
AU2024202731B2 (en) Mrna therapy for pompe disease
AU2022200099B2 (en) Messenger rna therapy for treatment of articular disease
JP6506749B2 (ja) フェニルケトン尿症のためのｍＲＮＡ療法
EP3122878B1 (en) Mrna therapy for the treatment of ocular diseases
KR20220078557A (ko) 개선된 mrna 로딩된 지질 나노입자 및 이의 제조 방법
JP2019522047A (ja) オルニチントランスカルバミラーゼ欠損症治療のためのメッセンジャーｒｎａ療法
US20250057765A1 (en) Compositions, methods and uses of messenger rna
HK40061848A (en) Messenger rna therapy for treatment of articular disease

Legal Events

Date Code Title Description
A107 Divisional application of patent
PA0104 Divisional application for international application

St.27 status event code: A-0-1-A10-A18-div-PA0104

St.27 status event code: A-0-1-A10-A16-div-PA0104

A201 Request for examination
E13-X000 Pre-grant limitation requested

St.27 status event code: A-2-3-E10-E13-lim-X000

P11-X000 Amendment of application requested

St.27 status event code: A-2-2-P10-P11-nap-X000

P13-X000 Application amended

St.27 status event code: A-2-2-P10-P13-nap-X000

PA0201 Request for examination

St.27 status event code: A-1-2-D10-D11-exm-PA0201

PG1501 Laying open of application

St.27 status event code: A-1-1-Q10-Q12-nap-PG1501

E902 Notification of reason for refusal
PE0902 Notice of grounds for rejection

St.27 status event code: A-1-2-D10-D21-exm-PE0902

P11-X000 Amendment of application requested

St.27 status event code: A-2-2-P10-P11-nap-X000

P13-X000 Application amended

St.27 status event code: A-2-2-P10-P13-nap-X000

E701 Decision to grant or registration of patent right
PE0701 Decision of registration

St.27 status event code: A-1-2-D10-D22-exm-PE0701

R18-X000 Changes to party contact information recorded

St.27 status event code: A-3-3-R10-R18-oth-X000

GRNT Written decision to grant
PR0701 Registration of establishment

St.27 status event code: A-2-4-F10-F11-exm-PR0701

PR1002 Payment of registration fee

St.27 status event code: A-2-2-U10-U12-oth-PR1002

Fee payment year number: 1

PG1601 Publication of registration

St.27 status event code: A-4-4-Q10-Q13-nap-PG1601

PR1001 Payment of annual fee

St.27 status event code: A-4-4-U10-U11-oth-PR1001

Fee payment year number: 4

U11 Full renewal or maintenance fee paid

Free format text: ST27 STATUS EVENT CODE: A-4-4-U10-U11-OTH-PR1001 (AS PROVIDED BY THE NATIONAL OFFICE)

Year of fee payment: 4