DK3071696T3 - C/ebp alfa kort aktiverings-rna-sammensætninger og fremgangsmåder til anvendelse - Google Patents

C/ebp alfa kort aktiverings-rna-sammensætninger og fremgangsmåder til anvendelse Download PDF

Info

Publication number
DK3071696T3
DK3071696T3 DK14843226.3T DK14843226T DK3071696T3 DK 3071696 T3 DK3071696 T3 DK 3071696T3 DK 14843226 T DK14843226 T DK 14843226T DK 3071696 T3 DK3071696 T3 DK 3071696T3
Authority
DK
Denmark
Prior art keywords
ebp
alfa
methods
short activation
rna compositions
Prior art date
Application number
DK14843226.3T
Other languages
English (en)
Inventor
Pal Saertrom
Original Assignee
Mina Therapeutics Ltd
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 Mina Therapeutics Ltd filed Critical Mina Therapeutics Ltd
Application granted granted Critical
Publication of DK3071696T3 publication Critical patent/DK3071696T3/da

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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • 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
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/54—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound
    • A61K47/549—Sugars, nucleosides, nucleotides or nucleic acids
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/62—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being a protein, peptide or polyamino acid
    • A61K47/64—Drug-peptide, drug-protein or drug-polyamino acid conjugates, i.e. the modifying agent being a peptide, protein or polyamino acid which is covalently bonded or complexed to a therapeutically active agent
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P29/00—Non-central analgesic, antipyretic or antiinflammatory agents, e.g. antirheumatic agents; Non-steroidal antiinflammatory drugs [NSAID]
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/04—Anorexiants; Antiobesity agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/06—Antihyperlipidemics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/08—Drugs for disorders of the metabolism for glucose homeostasis
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/08—Drugs for disorders of the metabolism for glucose homeostasis
    • A61P3/10—Drugs for disorders of the metabolism for glucose homeostasis for hyperglycaemia, e.g. antidiabetics
    • 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
    • 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
    • A61P31/04—Antibacterial agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • A61P35/02—Antineoplastic agents specific for leukemia
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00—Drugs for immunological or allergic disorders
    • A61P37/02—Immunomodulators
    • A61P37/04—Immunostimulants
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P5/00—Drugs for disorders of the endocrine system
    • A61P5/48—Drugs for disorders of the endocrine system of the pancreatic hormones
    • A61P5/50—Drugs for disorders of the endocrine system of the pancreatic hormones for increasing or potentiating the activity of insulin
    • 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
    • C12N15/1135—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 against oncogenes or tumor suppressor genes
    • 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
    • C12N2310/11—Antisense
    • 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
    • C12N2310/11—Antisense
    • C12N2310/113—Antisense targeting other non-coding nucleic acids, e.g. antagomirs
    • 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
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • C12N2310/141—MicroRNAs, miRNAs

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Genetics & Genomics (AREA)
  • Molecular Biology (AREA)
  • Biomedical Technology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Epidemiology (AREA)
  • Diabetes (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Hematology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Oncology (AREA)
  • Obesity (AREA)
  • Endocrinology (AREA)
  • Immunology (AREA)
  • Communicable Diseases (AREA)
  • Emergency Medicine (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Child & Adolescent Psychology (AREA)
  • Rheumatology (AREA)
DK14843226.3T 2013-11-22 2014-11-24 C/ebp alfa kort aktiverings-rna-sammensætninger og fremgangsmåder til anvendelse DK3071696T3 (da)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201361907732P 2013-11-22 2013-11-22
PCT/IB2014/003054 WO2015075557A2 (en) 2013-11-22 2014-11-24 C/ebp alpha compositions and methods of use

Publications (1)

Publication Number Publication Date
DK3071696T3 true DK3071696T3 (da) 2019-10-07

Family

ID=52649067

Family Applications (2)

Application Number Title Priority Date Filing Date
DK14843226.3T DK3071696T3 (da) 2013-11-22 2014-11-24 C/ebp alfa kort aktiverings-rna-sammensætninger og fremgangsmåder til anvendelse
DK19190263.4T DK3594348T3 (da) 2013-11-22 2014-11-24 Kort c/ebp-alpha til aktivering af rna-sammensætninger og anvendelsesfremgangsmåder

Family Applications After (1)

Application Number Title Priority Date Filing Date
DK19190263.4T DK3594348T3 (da) 2013-11-22 2014-11-24 Kort c/ebp-alpha til aktivering af rna-sammensætninger og anvendelsesfremgangsmåder

Country Status (10)

Country Link
US (4) US10202601B2 (da)
EP (3) EP3985118A1 (da)
JP (3) JP6621409B2 (da)
KR (3) KR102252561B1 (da)
CN (2) CN112220797B (da)
AU (3) AU2014351482B2 (da)
CA (2) CA3236835A1 (da)
DK (2) DK3071696T3 (da)
SI (2) SI3071696T1 (da)
WO (1) WO2015075557A2 (da)

Families Citing this family (89)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2013066438A2 (en) 2011-07-22 2013-05-10 President And Fellows Of Harvard College Evaluation and improvement of nuclease cleavage specificity
US20150044192A1 (en) 2013-08-09 2015-02-12 President And Fellows Of Harvard College Methods for identifying a target site of a cas9 nuclease
US9359599B2 (en) 2013-08-22 2016-06-07 President And Fellows Of Harvard College Engineered transcription activator-like effector (TALE) domains and uses thereof
US9526784B2 (en) 2013-09-06 2016-12-27 President And Fellows Of Harvard College Delivery system for functional nucleases
US9388430B2 (en) 2013-09-06 2016-07-12 President And Fellows Of Harvard College Cas9-recombinase fusion proteins and uses thereof
US9340799B2 (en) 2013-09-06 2016-05-17 President And Fellows Of Harvard College MRNA-sensing switchable gRNAs
CA3236835A1 (en) * 2013-11-22 2015-05-28 Mina Therapeutics Limited C/ebp alpha short activating rna compositions and methods of use
US20150166984A1 (en) 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting alpha-antitrypsin point mutations
EP3084008B1 (en) * 2013-12-17 2018-07-18 Csir A method for identification of anti-hiv human mirna mimics and mirna inhibitors and anti-hiv pharmaceutical compounds
US10441654B2 (en) 2014-01-24 2019-10-15 Children's Hospital Of Eastern Ontario Research Institute Inc. SMC combination therapy for the treatment of cancer
WO2016022363A2 (en) 2014-07-30 2016-02-11 President And Fellows Of Harvard College Cas9 proteins including ligand-dependent inteins
US20160060313A1 (en) 2014-08-27 2016-03-03 Daewoong Jo Development of Protein-Based Biotherapeutics That Penetrates Cell-Membrane and Induces Anti-Angiogenic Effect - Improved Cell-Permeable Suppressor of Cytokine Signaling (iCP-SOCS3) Proteins, Polynucleotides Encoding the Same, and Anti-Angiogenic Compositions Comprising the Same
WO2016054094A1 (en) 2014-09-30 2016-04-07 Research Institute At Nationwide Children's Hospital Compositions and methods for treating hepatic fibrosis
CN114525279A (zh) * 2015-04-22 2022-05-24 米纳治疗有限公司 C/EBP α SARNA组合物和使用方法
CA2991052A1 (en) * 2015-07-02 2017-01-05 City Of Hope Compounds and compositions including phosphorothioated oligodeoxynucleotide, and methods of use thereof
WO2017053428A1 (en) * 2015-09-21 2017-03-30 Intercept Pharmaceuticals, Inc. Methods of promoting hepatic regeneration
SG10202104041PA (en) 2015-10-23 2021-06-29 Harvard College Nucleobase editors and uses thereof
CN106636316B (zh) * 2015-10-30 2020-06-02 益善生物技术股份有限公司 卵巢癌相关microRNA检测试剂盒
CN106636312B (zh) * 2015-10-30 2020-06-02 益善生物技术股份有限公司 肝癌相关microRNA检测试剂盒
CN106636314B (zh) * 2015-10-30 2020-06-02 益善生物技术股份有限公司 乳腺癌相关microRNA检测试剂盒
CN106636313B (zh) * 2015-10-30 2020-06-02 益善生物技术股份有限公司 胃癌相关microRNA检测试剂盒
CN106749655B (zh) * 2015-11-19 2021-01-26 中国科学院生物物理研究所 一种针对蛋白e2-2的单克隆抗体
CN106729750A (zh) * 2015-11-20 2017-05-31 昆山彭济凯丰生物科技有限公司 通过miR-183治疗高血脂、脂肪肝、二型糖尿病和降低体重的方法和药物及它们的应用
EP3254683A1 (en) * 2016-06-06 2017-12-13 Medizinische Hochschule Hannover Medicament for the treatment of acute liver failure
CN106244588B (zh) * 2016-07-30 2019-07-12 内蒙古医科大学附属人民医院 激活肺癌细胞中RUNX3表达的saRNA及其应用
CN109715194B (zh) 2016-08-03 2024-06-11 南佛罗里达大学 用于治疗神经障碍的颤蛋白组合物
KR20250103795A (ko) 2016-08-03 2025-07-07 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 아데노신 핵염기 편집제 및 그의 용도
US11661590B2 (en) 2016-08-09 2023-05-30 President And Fellows Of Harvard College Programmable CAS9-recombinase fusion proteins and uses thereof
CN106086214A (zh) * 2016-08-11 2016-11-09 河南大学 miR‑149‑5p作为临床诊断或抑制肝癌的应用
WO2018039438A1 (en) 2016-08-24 2018-03-01 President And Fellows Of Harvard College Incorporation of unnatural amino acids into proteins using base editing
SG11201903089RA (en) 2016-10-14 2019-05-30 Harvard College Aav delivery of nucleobase editors
US11332713B2 (en) 2016-11-16 2022-05-17 KSQ Therapeutics, Inc. Gene-regulating compositions and methods for improved immunotherapy
EP3808380A1 (en) * 2016-12-08 2021-04-21 CureVac AG Rna for treatment or prophylaxis of a liver disease
WO2018119359A1 (en) 2016-12-23 2018-06-28 President And Fellows Of Harvard College Editing of ccr5 receptor gene to protect against hiv infection
US11898179B2 (en) 2017-03-09 2024-02-13 President And Fellows Of Harvard College Suppression of pain by gene editing
EP3592381A1 (en) 2017-03-09 2020-01-15 President and Fellows of Harvard College Cancer vaccine
WO2018165629A1 (en) 2017-03-10 2018-09-13 President And Fellows Of Harvard College Cytosine to guanine base editor
WO2018176009A1 (en) 2017-03-23 2018-09-27 President And Fellows Of Harvard College Nucleobase editors comprising nucleic acid programmable dna binding proteins
WO2018209320A1 (en) 2017-05-12 2018-11-15 President And Fellows Of Harvard College Aptazyme-embedded guide rnas for use with crispr-cas9 in genome editing and transcriptional activation
CN108938659A (zh) * 2017-05-19 2018-12-07 昆山彭济凯丰生物科技有限公司 一种调节食欲和体重的方法和药物及它们的应用
CN111801345A (zh) 2017-07-28 2020-10-20 哈佛大学的校长及成员们 使用噬菌体辅助连续进化(pace)的进化碱基编辑器的方法和组合物
EP3676376B1 (en) 2017-08-30 2025-01-15 President and Fellows of Harvard College High efficiency base editors comprising gam
CA3075205A1 (en) 2017-09-08 2019-03-14 Mina Therapeutics Limited Stabilized hnf4a sarna compositions and methods of use
EP4219715A3 (en) * 2017-09-08 2023-09-06 MiNA Therapeutics Limited Stabilized cebpa sarna compositions and methods of use
WO2019060613A2 (en) * 2017-09-22 2019-03-28 John Mansell COMPOSITIONS AND METHODS FOR TREATING SEPSIS-RELATED DISORDERS
US10676744B2 (en) * 2017-10-13 2020-06-09 University Of South Carolina Modulation of adipose tissue macrophage phenotype by use of MicroRNA-30
US11795443B2 (en) 2017-10-16 2023-10-24 The Broad Institute, Inc. Uses of adenosine base editors
CN107663541A (zh) * 2017-10-31 2018-02-06 温州医科大学 一种用于诊断原发性肝癌的特异性生物标志物及其筛选方法与应用
WO2019118949A1 (en) 2017-12-15 2019-06-20 The Broad Institute, Inc. Systems and methods for predicting repair outcomes in genetic engineering
US11007211B2 (en) 2018-01-07 2021-05-18 City Of Hope CCR7 aptamers and uses thereof
CN108559750B (zh) * 2018-01-16 2020-07-31 华南农业大学 Stat3在猪卵巢颗粒细胞中的应用
WO2019158720A1 (en) 2018-02-16 2019-08-22 Mina Therapeutics Limited C/ebp alpha sarna compositions and methods of use
WO2019178420A1 (en) 2018-03-15 2019-09-19 KSQ Therapeutics, Inc. Gene-regulating compositions and methods for improved immunotherapy
US12157760B2 (en) 2018-05-23 2024-12-03 The Broad Institute, Inc. Base editors and uses thereof
CN108959843B (zh) * 2018-06-06 2021-07-06 北京箭牧科技有限公司 靶向rna的化学小分子药物计算机筛选方法
US20210254069A1 (en) * 2018-06-15 2021-08-19 Mina Therapeutics Limited Combination therapies comprising c/ebp alpha sarna
EP3820495A4 (en) 2018-07-09 2022-07-20 The Broad Institute Inc. RNA-PROGRAMMABLE EPIGENETIC RNA MODIFIERS AND THEIR USES
WO2020051243A1 (en) * 2018-09-04 2020-03-12 Ohio State Innovation Foundation Lipid nanoparticles and methods of using thereof
WO2020092453A1 (en) 2018-10-29 2020-05-07 The Broad Institute, Inc. Nucleobase editors comprising geocas9 and uses thereof
US12351837B2 (en) 2019-01-23 2025-07-08 The Broad Institute, Inc. Supernegatively charged proteins and uses thereof
CN112912501B (zh) * 2019-01-30 2024-07-16 中美瑞康核酸技术(南通)研究院有限公司 寡核苷酸分子及其在肿瘤治疗中的应用
CN109837300A (zh) * 2019-03-04 2019-06-04 山东省千佛山医院 一种用于提高alpps诱导肝组织生长率的方法
EP3942043A2 (en) 2019-03-19 2022-01-26 The Broad Institute, Inc. Methods and compositions for editing nucleotide sequences
JPWO2020196736A1 (da) * 2019-03-28 2020-10-01
WO2020214842A1 (en) 2019-04-17 2020-10-22 The Broad Institute, Inc. Adenine base editors with reduced off-target effects
AU2020263502A1 (en) * 2019-04-24 2021-10-28 Nevada Research & Innovation Corporation Methods and compositions of miR-10 mimics and targets thereof
US20220396794A1 (en) * 2019-06-04 2022-12-15 Apterna Limited APTAMERS AGAINST TRANSFERRIN RECEPTOR (TfR)
EP4017539A1 (en) * 2019-08-19 2022-06-29 MiNA Therapeutics Limited Oligonucleotide conjugate compositions and methods of use
US12435330B2 (en) 2019-10-10 2025-10-07 The Broad Institute, Inc. Methods and compositions for prime editing RNA
WO2021087234A1 (en) * 2019-11-01 2021-05-06 The Board Of Trustees Of The Leland Stanford Junior University Lineage reprogramming as a cancer immunotherapy
IL320607A (en) * 2020-03-26 2025-07-01 Ramirez Fort Marigdalia Kaleth Ultraviolet radiation treatments
BR112022022603A2 (pt) 2020-05-08 2023-01-17 Broad Inst Inc Métodos e composições para edição simultânea de ambas as fitas de sequência alvo de nucleotídeos de fita dupla
GB202019692D0 (en) * 2020-12-14 2021-01-27 Apterna Ltd Aptamer-sirna fusions
CN112941076B (zh) * 2021-02-05 2023-07-07 中国药科大学 FXR靶向的saRNA及其应用
JP2024506882A (ja) * 2021-02-07 2024-02-15 ラクティゲン セラピューティクス 化学修飾された低分子活性化ｒｎａ
EP4329777A1 (en) 2021-04-28 2024-03-06 MiNA Therapeutics Limited Combination therapies comprising c/ebp alpha sarna
CN114306367B (zh) * 2021-08-27 2023-03-28 赵小洋 一种含有C/EBPα-saRNA的组合物
CN114349841B (zh) * 2021-10-26 2024-02-13 安徽农业大学 一种调控卵泡膜表面ovr基因表达活性的转录因子及其应用
CN114045294B (zh) * 2021-11-22 2023-03-24 昆明理工大学 一种脂质转运蛋白基因及其应用
CN113957157B (zh) * 2021-12-06 2023-10-10 湖北省农业科学院畜牧兽医研究所 一种与蛋鸡肝脏脂代谢相关的circRNA及其应用
CN114469996B (zh) * 2021-12-23 2023-10-20 中国医学科学院医学生物学研究所 一种包含miR-135b-5p的外泌体及在抗轮状病毒感染中的应用
CN114392266B (zh) * 2022-01-22 2022-11-15 暨南大学附属第一医院（广州华侨医院） 包含PPARγ抑制剂的药物组合物及其应用
CN114686424B (zh) * 2022-03-09 2023-09-22 海南医学院 一种调控C/EBPα基因表达的方法及C/EBPα表达抑制剂
WO2023214405A1 (en) * 2022-05-01 2023-11-09 Yeda Research And Development Co. Ltd. Reexpression of hnf4a to alleviate cancer-associated cachexia
CN120712103A (zh) * 2022-12-16 2025-09-26 普渡研究基金会 体内和离体产生car-中性粒细胞及其治疗癌症和对癌症进行成像的用途
WO2024165876A2 (en) * 2023-02-10 2024-08-15 Mina Therapeutics Limited Compositions and methods of using c/ebp alpha sarna
WO2024175887A1 (en) 2023-02-22 2024-08-29 Mina Therapeutics Limited Compositions and methods of using c/ebp alpha sarna
JP2026507228A (ja) 2023-03-03 2026-02-27 アーセナル バイオサイエンシズ インコーポレイテッド Ｐｓｍａ及びｃａ９を標的とするシステム
WO2026078664A1 (en) * 2024-10-10 2026-04-16 Instituto De Biologia Experimental E Tecnológica (Ibet) Transcription factor enhancing recombinant adeno-associated virus production, products, methods and uses thereof

Family Cites Families (189)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JPS5927900A (ja) 1982-08-09 1984-02-14 Wakunaga Seiyaku Kk 固定化オリゴヌクレオチド
FR2540122B1 (fr) 1983-01-27 1985-11-29 Centre Nat Rech Scient Nouveaux composes comportant une sequence d'oligonucleotide liee a un agent d'intercalation, leur procede de synthese et leur application
US4605735A (en) 1983-02-14 1986-08-12 Wakunaga Seiyaku Kabushiki Kaisha Oligonucleotide derivatives
US4948882A (en) 1983-02-22 1990-08-14 Syngene, Inc. Single-stranded labelled oligonucleotides, reactive monomers and methods of synthesis
US4824941A (en) 1983-03-10 1989-04-25 Julian Gordon Specific antibody to the native form of 2'5'-oligonucleotides, the method of preparation and the use as reagents in immunoassays or for binding 2'5'-oligonucleotides in biological systems
US4587044A (en) 1983-09-01 1986-05-06 The Johns Hopkins University Linkage of proteins to nucleic acids
US5118802A (en) 1983-12-20 1992-06-02 California Institute Of Technology DNA-reporter conjugates linked via the 2' or 5'-primary amino group of the 5'-terminal nucleoside
US5430136A (en) 1984-10-16 1995-07-04 Chiron Corporation Oligonucleotides having selectably cleavable and/or abasic sites
US5258506A (en) 1984-10-16 1993-11-02 Chiron Corporation Photolabile reagents for incorporation into oligonucleotide chains
US4828979A (en) 1984-11-08 1989-05-09 Life Technologies, Inc. Nucleotide analogs for nucleic acid labeling and detection
US4762779A (en) 1985-06-13 1988-08-09 Amgen Inc. Compositions and methods for functionalizing nucleic acids
US5317098A (en) 1986-03-17 1994-05-31 Hiroaki Shizuya Non-radioisotope tagging of fragments
JPS638396A (ja) 1986-06-30 1988-01-14 Wakunaga Pharmaceut Co Ltd ポリ標識化オリゴヌクレオチド誘導体
US4904582A (en) 1987-06-11 1990-02-27 Synthetic Genetics Novel amphiphilic nucleic acid conjugates
US5585481A (en) 1987-09-21 1996-12-17 Gen-Probe Incorporated Linking reagents for nucleotide probes
US5525465A (en) 1987-10-28 1996-06-11 Howard Florey Institute Of Experimental Physiology And Medicine Oligonucleotide-polyamide conjugates and methods of production and applications of the same
DE3738460A1 (de) 1987-11-12 1989-05-24 Max Planck Gesellschaft Modifizierte oligonukleotide
US5082830A (en) 1988-02-26 1992-01-21 Enzo Biochem, Inc. End labeled nucleotide probe
US5109124A (en) 1988-06-01 1992-04-28 Biogen, Inc. Nucleic acid probe linked to a label having a terminal cysteine
US5262536A (en) 1988-09-15 1993-11-16 E. I. Du Pont De Nemours And Company Reagents for the preparation of 5'-tagged oligonucleotides
US5512439A (en) 1988-11-21 1996-04-30 Dynal As Oligonucleotide-linked magnetic particles and uses thereof
US5457183A (en) 1989-03-06 1995-10-10 Board Of Regents, The University Of Texas System Hydroxylated texaphyrins
US5599923A (en) 1989-03-06 1997-02-04 Board Of Regents, University Of Tx Texaphyrin metal complexes having improved functionalization
US5391723A (en) 1989-05-31 1995-02-21 Neorx Corporation Oligonucleotide conjugates
US4958013A (en) 1989-06-06 1990-09-18 Northwestern University Cholesteryl modified oligonucleotides
US5451463A (en) 1989-08-28 1995-09-19 Clontech Laboratories, Inc. Non-nucleoside 1,3-diol reagents for labeling synthetic oligonucleotides
US5254469A (en) 1989-09-12 1993-10-19 Eastman Kodak Company Oligonucleotide-enzyme conjugate that can be used as a probe in hybridization assays and polymerase chain reaction procedures
US5292873A (en) 1989-11-29 1994-03-08 The Research Foundation Of State University Of New York Nucleic acids labeled with naphthoquinone probe
US5486603A (en) 1990-01-08 1996-01-23 Gilead Sciences, Inc. Oligonucleotide having enhanced binding affinity
US5578718A (en) 1990-01-11 1996-11-26 Isis Pharmaceuticals, Inc. Thiol-derivatized nucleosides
AU7579991A (en) 1990-02-20 1991-09-18 Gilead Sciences, Inc. Pseudonucleosides and pseudonucleotides and their polymers
US5214136A (en) 1990-02-20 1993-05-25 Gilead Sciences, Inc. Anthraquinone-derivatives oligonucleotides
ATE167523T1 (de) 1990-05-11 1998-07-15 Microprobe Corp Teststreifen zum eintauchen für nukleinsäure- hybridisierungsassays und verfahren zur kovalenten immobilisierung von oligonucleotiden
US5688941A (en) 1990-07-27 1997-11-18 Isis Pharmaceuticals, Inc. Methods of making conjugated 4' desmethyl nucleoside analog compounds
US5138045A (en) 1990-07-27 1992-08-11 Isis Pharmaceuticals Polyamine conjugated oligonucleotides
US5608046A (en) 1990-07-27 1997-03-04 Isis Pharmaceuticals, Inc. Conjugated 4'-desmethyl nucleoside analog compounds
US5218105A (en) 1990-07-27 1993-06-08 Isis Pharmaceuticals Polyamine conjugated oligonucleotides
US5245022A (en) 1990-08-03 1993-09-14 Sterling Drug, Inc. Exonuclease resistant terminally substituted oligonucleotides
US5512667A (en) 1990-08-28 1996-04-30 Reed; Michael W. Trifunctional intermediates for preparing 3'-tailed oligonucleotides
EP0556301B1 (en) 1990-11-08 2001-01-10 Hybridon, Inc. Incorporation of multiple reporter groups on synthetic oligonucleotides
US5371241A (en) 1991-07-19 1994-12-06 Pharmacia P-L Biochemicals Inc. Fluorescein labelled phosphoramidites
US5595726A (en) 1992-01-21 1997-01-21 Pharmacyclics, Inc. Chromophore probe for detection of nucleic acid
US5565552A (en) 1992-01-21 1996-10-15 Pharmacyclics, Inc. Method of expanded porphyrin-oligonucleotide conjugate synthesis
US5272250A (en) 1992-07-10 1993-12-21 Spielvogel Bernard F Boronated phosphoramidate compounds
US5574142A (en) 1992-12-15 1996-11-12 Microprobe Corporation Peptide linkers for improved oligonucleotide delivery
US5595756A (en) 1993-12-22 1997-01-21 Inex Pharmaceuticals Corporation Liposomal compositions for enhanced retention of bioactive agents
US5597696A (en) 1994-07-18 1997-01-28 Becton Dickinson And Company Covalent cyanine dye oligonucleotide conjugates
US5580731A (en) 1994-08-25 1996-12-03 Chiron Corporation N-4 modified pyrimidine deoxynucleotides and oligonucleotide probes synthesized therewith
US5795587A (en) 1995-01-23 1998-08-18 University Of Pittsburgh Stable lipid-comprising drug delivery complexes and methods for their production
US6086913A (en) 1995-11-01 2000-07-11 University Of British Columbia Liposomal delivery of AAV vectors
US20030073640A1 (en) 1997-07-23 2003-04-17 Ribozyme Pharmaceuticals, Inc. Novel compositions for the delivery of negatively charged molecules
JPH1160625A (ja) 1997-08-22 1999-03-02 Idemitsu Petrochem Co Ltd オレフィン重合用固体触媒成分、オレフィン重合用触 媒及びオレフィン重合体の製造方法
DE69837274T2 (de) 1997-10-10 2007-11-08 Inex Pharmaceuticals Corp., Burnaby Verfahren zur verkapselung von nukleinsäuren in lipiddoppelschichten
US6287586B1 (en) 1998-09-18 2001-09-11 The University Of British Columbia Pharmaceutical compositions of vanadium biguanide complexes and their use
EP1854880A1 (en) 1999-03-15 2007-11-14 University of British Columbia Methods and reagents for modulating cholesterol levels
US8193330B2 (en) 1999-04-06 2012-06-05 The Regents Of The University Of California Polynucleotides comprising Neurogenin3 promoter and bHLH encoding domains
US7094423B1 (en) 1999-07-15 2006-08-22 Inex Pharmaceuticals Corp. Methods for preparation of lipid-encapsulated therapeutic agents
US7189705B2 (en) 2000-04-20 2007-03-13 The University Of British Columbia Methods of enhancing SPLP-mediated transfection using endosomal membrane destabilizers
US6306655B1 (en) 2000-06-13 2001-10-23 Isis Pharmaceuticals, Inc. Antisense inhibition of C/EBP alpha expression
US6998115B2 (en) 2000-10-10 2006-02-14 Massachusetts Institute Of Technology Biodegradable poly(β-amino esters) and uses thereof
JP2004511572A (ja) 2000-10-25 2004-04-15 ザ　ユニバーシティ　オブ　ブリティッシュ　コロンビア 標的化送達のための脂質製剤
ATE303399T1 (de) 2001-03-12 2005-09-15 Intercept Pharmaceuticals Inc Steroide als agonisten für fxr
WO2003028657A2 (en) 2001-10-03 2003-04-10 The Johns Hopkins University Compositions for oral gene therapy and methods of using same
US7223887B2 (en) 2001-12-18 2007-05-29 The University Of British Columbia Multivalent cationic lipids and methods of using same in the production of lipid particles
DE10207178A1 (de) 2002-02-19 2003-09-04 Novosom Ag Komponenten für die Herstellung amphoterer Liposomen
US20050222064A1 (en) 2002-02-20 2005-10-06 Sirna Therapeutics, Inc. Polycationic compositions for cellular delivery of polynucleotides
WO2003092665A2 (en) 2002-05-02 2003-11-13 Massachusetts Eye And Ear Infirmary Ocular drug delivery systems and use thereof
AU2003266014B2 (en) 2002-05-06 2009-05-14 Alnylam Pharmaceuticals, Inc. Methods for delivery of nucleic acids
JP2006507841A (ja) 2002-11-14 2006-03-09 ダーマコン， インコーポレイテッド 機能的ｓｉＲＮＡおよび超機能的ｓｉＲＮＡ
EP1568706A1 (en) 2004-02-26 2005-08-31 Intercept Pharmaceuticals, Inc. Novel steroid agonist for FXR
ATE536418T1 (de) 2004-06-07 2011-12-15 Protiva Biotherapeutics Inc Lipidverkapselte interferenz-rna
JP5192234B2 (ja) 2004-08-10 2013-05-08 アルナイラム ファーマシューティカルズ， インコーポレイテッド 化学修飾オリゴヌクレオチド
US20060252670A1 (en) 2004-10-14 2006-11-09 Intercept Pharmaceuticals Inc. Method of reducing drug-induced adverse side effects in a patient
US8192718B1 (en) 2005-01-04 2012-06-05 Gp Medical, Inc. Pharmaceutical composition of nanoparticles
WO2007086883A2 (en) 2005-02-14 2007-08-02 Sirna Therapeutics, Inc. Cationic lipids and formulated molecular compositions containing them
US7404969B2 (en) 2005-02-14 2008-07-29 Sirna Therapeutics, Inc. Lipid nanoparticle based compositions and methods for the delivery of biologically active molecules
MX2007012157A (es) 2005-04-01 2008-03-14 Intezyne Technologies Llc Micelas polimericas para suministro de farmacos.
JP5362350B2 (ja) 2005-04-15 2013-12-11 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア 小分子活性化ｒｎａ分子及び使用方法
ITMI20050912A1 (it) 2005-05-19 2006-11-20 Erregierre Spa Processo di preparazione di acidi 3-a-ya(b)-diidrossi-6-a(b)-alchil-5b-colanici
US8357666B2 (en) 2005-08-01 2013-01-22 Nupotential, Inc. Reprogramming a cell by inducing a pluripotent gene through RNA interference
JP2007082430A (ja) * 2005-09-20 2007-04-05 Niigata Univ 脳腫瘍マーカーおよびその用途
CN101291987B (zh) 2005-10-20 2012-05-09 旭硝子株式会社 聚四氟乙烯水性分散液及其制品
EP2641970B1 (en) 2005-11-17 2014-12-24 Board of Regents, The University of Texas System Modulation of gene expression by oligomers targeted to chromosomal DNA
JP5713377B2 (ja) 2005-12-28 2015-05-07 ザ スクリプス リサーチ インスティテュート 薬物標的としての天然アンチセンスおよび非コードｒｎａ転写物
WO2007095174A2 (en) 2006-02-14 2007-08-23 Intercept Pharmaceuticals, Inc. Bile acid derivatives as fxr ligands for the prevention or treatment of fxr-mediated diseases or conditions
CN101522703B (zh) 2006-06-27 2013-04-17 英特塞普特医药品公司 胆酸派生物及其在制备预防或治疗fxr介导的疾病或状况的药物中的应用
WO2008042973A2 (en) 2006-10-03 2008-04-10 Alnylam Pharmaceuticals, Inc. Lipid containing formulations
EP2094238A2 (en) 2006-10-13 2009-09-02 Novosom AG Improvements in or relating to amphoteric liposomes
JP5292310B2 (ja) 2007-01-19 2013-09-18 インターセプト ファーマシューティカルズ， インコーポレイテッド Ｔｇｒ５調節剤としての２３−置換胆汁酸およびその使用方法
JP2010519203A (ja) 2007-02-16 2010-06-03 メルク・シャープ・エンド・ドーム・コーポレイション 生物活性分子の活性を強化するための組成物及び方法
CN101668774B (zh) 2007-02-28 2013-06-26 诺维莫尼公司 抗干扰素诱导蛋白-10的抗体及其使用方法
WO2008109449A1 (en) 2007-03-02 2008-09-12 Mdrna Inc. Nucleic acid compounds for inhibiting bcl2 gene expression and uses thereof
WO2008109556A1 (en) 2007-03-02 2008-09-12 Mdrna, Inc. Nucleic acid compounds for inhibiting telomerase gene expression and uses thereof
PT2164993T (pt) 2007-05-29 2017-01-12 Reid Christopher B Métodos para produção e usos de populações de células multipotentes, populações de células pluripotentes, populações de célula diferenciadas e populações de células resistentes a vih
SI2644192T1 (sl) 2007-09-28 2017-08-31 Pfizer Inc. Ciljanje rakavih celic z uporabo nanodelcev
WO2009046397A2 (en) 2007-10-04 2009-04-09 Board Of Regents, The University Of Texas System Modulating gene expression with agrna and gapmers targeting antisense transcripts
JP2011507554A (ja) 2007-12-28 2011-03-10 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア 遺伝子発現を増加させるための方法および組成物
EP2105145A1 (en) 2008-03-27 2009-09-30 ETH Zürich Method for muscle-specific delivery lipid-conjugated oligonucleotides
EP2276838A4 (en) 2008-04-07 2012-02-01 Nupotential Inc RE-PROGRAMMING A CELL BY INDUCING A PLURIPOTENTIC GENE THROUGH RNA INTERFERENCE
WO2009126927A2 (en) 2008-04-11 2009-10-15 Joslin Diabetes Center, Inc. Methods of generating insulin-producing cells
EP3604533A1 (en) 2008-04-11 2020-02-05 Arbutus Biopharma Corporation Site-specific delivery of nucleic acids by combining targeting ligands with endosomolytic components
NZ588583A (en) 2008-04-15 2012-08-31 Protiva Biotherapeutics Inc Novel lipid formulations for nucleic acid delivery
WO2009132131A1 (en) 2008-04-22 2009-10-29 Alnylam Pharmaceuticals, Inc. Amino lipid based improved lipid formulation
HUE035770T2 (en) 2008-06-16 2018-05-28 Pfizer Process for the preparation of diblock copolymers functionalized with targeting material for use in the preparation of therapeutic nanoparticles
WO2010005726A2 (en) 2008-06-16 2010-01-14 Bind Biosciences Inc. Therapeutic polymeric nanoparticles with mtor inhibitors and methods of making and using same
EA020954B1 (ru) 2008-06-16 2015-03-31 Бинд Терапьютикс, Инк. Загруженные лекарственным средством полимерные наночастицы, фармацевтическая композиция и способ лечения рака
EP2309991B1 (en) 2008-06-16 2019-03-06 Pfizer Inc Therapeutic polymeric nanoparticles comprising vinca alkaloids and methods of making and using same
EA020310B1 (ru) 2008-07-30 2014-10-30 Интерсепт Фармасьютикалз, Инк. Модуляторы рецептора tgr5 и их применение
EP2326331A4 (en) 2008-08-18 2013-05-15 Merck Sharp & Dohme NEW LIPID NANOPARTICLES AND NEW COMPONENTS FOR THE RELEASE OF NUCLEIC ACIDS
WO2010030763A2 (en) 2008-09-10 2010-03-18 Bind Biosciences, Inc. High throughput fabrication of nanoparticles
WO2010036962A1 (en) 2008-09-25 2010-04-01 Alnylam Pharmaceuticals, Inc. Lipid formulated compositions and methods for inhibiting expression of serum amyloid a gene
JP5777519B2 (ja) 2008-10-09 2015-09-09 テクミラ ファーマシューティカルズ コーポレイション 改良されたアミノ脂質および核酸の送達方法
WO2010047216A1 (ja) 2008-10-23 2010-04-29 国立大学法人 東京大学 microRNAの機能阻害法
JP2012508585A (ja) 2008-11-13 2012-04-12 ベイラー リサーチ インスティテュート インビボにおいて送達される膵島転写因子遺伝子による膵島の再生及び糖尿病の回復
ES2663948T3 (es) 2008-11-19 2018-04-17 Intercept Pharmaceuticals, Inc. Moduladores de TGR5 y método de uso de los mismos
EP2379064B1 (en) 2008-12-15 2020-02-26 Pfizer Inc. Long circulating nanoparticles for sustained release of therapeutic agents
WO2010080985A1 (en) * 2009-01-08 2010-07-15 Dana-Farber Cancer Institute, Inc. Compositions and methods for induced brown fat differentiation
WO2010080724A1 (en) 2009-01-12 2010-07-15 Merck Sharp & Dohme Corp. Novel lipid nanoparticles and novel components for delivery of nucleic acids
JP5596712B2 (ja) 2009-03-12 2014-09-24 イリノイ トゥール ワークス インコーポレイティド 給油ミス防止装置
US20120207744A1 (en) 2009-03-19 2012-08-16 Mendlein John D Reprogramming compositions and methods of using the same
JP5622254B2 (ja) 2009-03-31 2014-11-12 国立大学法人東京大学 二本鎖リボ核酸ポリイオンコンプレックス
US8815586B2 (en) 2009-04-24 2014-08-26 The Board Of Regents Of The University Of Texas System Modulation of gene expression using oligomers that target gene regions downstream of 3′ untranslated regions
US8287910B2 (en) 2009-04-30 2012-10-16 Intezyne Technologies, Inc. Polymeric micelles for polynucleotide encapsulation
SG176553A1 (en) 2009-05-05 2012-01-30 Muthiah Manoharan Lipid compositions
WO2010135329A2 (en) 2009-05-18 2010-11-25 Curna, Inc. Treatment of reprogramming factor related diseases by inhibition of natural antisense transcript to a reprogramming factor
WO2010138192A2 (en) 2009-05-27 2010-12-02 Selecta Biosciences, Inc. Nanocarriers possessing components with different rates of release
TR201811076T4 (tr) 2009-06-10 2018-08-27 Arbutus Biopharma Corp Geliştirilmiş lipit formulasyonu.
US9051567B2 (en) 2009-06-15 2015-06-09 Tekmira Pharmaceuticals Corporation Methods for increasing efficacy of lipid formulated siRNA
US8962583B2 (en) 2009-06-25 2015-02-24 The Brigham And Women's Hospital, Inc. Treatment of inflammatory diseases using miR-124
US8569256B2 (en) 2009-07-01 2013-10-29 Protiva Biotherapeutics, Inc. Cationic lipids and methods for the delivery of therapeutic agents
US8283333B2 (en) 2009-07-01 2012-10-09 Protiva Biotherapeutics, Inc. Lipid formulations for nucleic acid delivery
CN101941001B (zh) 2009-07-03 2014-04-02 3M创新有限公司 亲水涂层、制品、涂料组合物和方法
ES2693088T3 (es) 2009-07-20 2018-12-07 Janssen Biotech, Inc. Diferenciación de células madre embriónicas humanas
EP2464336A4 (en) 2009-08-14 2013-11-20 Alnylam Pharmaceuticals Inc COMPOSITIONS FORMULATED IN LIPIDS AND METHODS OF INHIBITING THE EXPRESSION OF EBOLA VIRUS GENE
WO2011022460A1 (en) 2009-08-20 2011-02-24 Merck Sharp & Dohme Corp. Novel cationic lipids with various head groups for oligonucleotide delivery
US20120264810A1 (en) 2009-09-22 2012-10-18 The University Of British Columbia Compositions and methods for enhancing cellular uptake and intracellular delivery of lipid particles
WO2011043913A2 (en) 2009-10-08 2011-04-14 Merck Sharp & Dohme Corp. Novel cationic lipids with short lipid chains for oligonucleotide delivery
WO2011056682A1 (en) 2009-10-27 2011-05-12 The University Of British Columbia Reverse head group lipids, lipid particle compositions comprising reverse headgroup lipids, and methods for the delivery of nucleic acids
CN107028886A (zh) 2009-11-04 2017-08-11 不列颠哥伦比亚大学 含有核酸的脂质粒子及相关的方法
WO2011071860A2 (en) 2009-12-07 2011-06-16 Alnylam Pharmaceuticals, Inc. 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
TR201906255T4 (tr) 2009-12-11 2019-05-21 Pfizer Terapötik partiküllerin liyofilize edilmesine yönelik stabil formülasyonlar.
EA201290497A1 (ru) 2009-12-15 2013-01-30 Байнд Байосайенсиз, Инк. Терапевтические полимерные наночастицы, включающие кортикостероиды, и способы получения таковых
WO2011084521A2 (en) 2009-12-15 2011-07-14 Bind Biosciences, Inc. Therapeutic polymeric nanoparticles comprising epothilone and methods of making and using same
ES2780156T3 (es) 2009-12-15 2020-08-24 Pfizer Composiciones terapéuticas de nanopartículas poliméricas con alta temperatura de transición vítrea o copolímeros de alto peso molecular
EP3494963A1 (en) 2009-12-18 2019-06-12 The University of British Columbia Methods and compositions for delivery of nucleic acids
CN102822342B (zh) 2010-01-06 2017-05-10 库尔纳公司 通过抑制胰腺发育基因的天然反义转录物而治疗胰腺发育基因相关疾病
EP2525781A1 (en) 2010-01-22 2012-11-28 Schering Corporation Novel cationic lipids for oligonucleotide delivery
WO2011127255A1 (en) 2010-04-08 2011-10-13 Merck Sharp & Dohme Corp. Preparation of lipid nanoparticles
US20110262491A1 (en) 2010-04-12 2011-10-27 Selecta Biosciences, Inc. Emulsions and methods of making nanocarriers
US20130156845A1 (en) 2010-04-29 2013-06-20 Alnylam Pharmaceuticals, Inc. Lipid formulated single stranded rna
WO2011143230A1 (en) 2010-05-10 2011-11-17 Alnylam Pharmaceuticals Methods and compositions for delivery of active agents
US8802863B2 (en) 2010-05-24 2014-08-12 Sirna Therapeutics, Inc. Amino alcohol cationic lipids for oligonucleotide delivery
US8748667B2 (en) 2010-06-04 2014-06-10 Sirna Therapeutics, Inc. Low molecular weight cationic lipids for oligonucleotide delivery
GB201010557D0 (en) 2010-06-23 2010-08-11 Mina Therapeutics Ltd RNA molecules and uses thereof
DE102010032758B4 (de) 2010-07-29 2012-02-23 Fujitsu Technology Solutions Intellectual Property Gmbh Computersystem, Verfahren zum Programmieren einer Echtzeituhr und Computerprogrammprodukt
US20130323269A1 (en) 2010-07-30 2013-12-05 Muthiah Manoharan Methods and compositions for delivery of active agents
WO2012016188A2 (en) 2010-07-30 2012-02-02 Alnylam Pharmaceuticals, Inc. Methods and compositions for delivery of active agents
US8968746B2 (en) 2010-07-30 2015-03-03 Curevac Gmbh Complexation of nucleic acids with disulfide-crosslinked cationic components for transfection and immunostimulation
MX349088B (es) 2010-09-20 2017-07-10 Merck Sharp & Dohme Lípidos catiónicos novedosos de bajo peso molecular para la entrega de oligonucleótidos.
WO2012047656A1 (en) 2010-09-27 2012-04-12 The University Of British Columbia Lipid a analog compositions
KR20130114115A (ko) 2010-09-30 2013-10-16 머크 샤프 앤드 돔 코포레이션 올리고뉴클레오티드 전달을 위한 저분자량 양이온성 지질
KR20130132795A (ko) 2010-10-08 2013-12-05 미나 테라퓨틱스 리미티드 짧은 rna 분자
CN103153347A (zh) 2010-10-21 2013-06-12 默沙东公司 用于寡核苷酸递送的新型低分子量阳离子脂质
EP2629760A4 (en) 2010-10-22 2014-04-02 Bind Therapeutics Inc THERAPEUTIC NANOPARTICLES CONTAINING COPOLYMERS OF HIGH MOLECULAR WEIGHT
WO2012061259A2 (en) 2010-11-05 2012-05-10 Merck Sharp & Dohme Corp. Novel low molecular weight cyclic amine containing cationic lipids for oligonucleotide delivery
AU2011325956B2 (en) 2010-11-12 2016-07-14 The General Hospital Corporation Polycomb-associated non-coding RNAs
US20120164120A1 (en) * 2010-12-23 2012-06-28 Food Industry Research And Development Institute Novel monascuspurpurones, preparation process thereof, and uses of the monascuspurpurones
WO2012092552A1 (en) 2010-12-30 2012-07-05 Selecta Biosciences, Inc. Synthetic nanocarriers with reactive groups that release biologically active agents
JP5902197B2 (ja) 2011-01-11 2016-04-13 アルニラム・ファーマシューティカルズ・インコーポレーテッド Ｐｅｇ化脂質および薬剤送達のためのそれらの使用
DE102011082231A1 (de) 2011-01-12 2012-07-12 Robert Bosch Gmbh Zündspule, insbesondere für kleinbauende Motoren
US20140066363A1 (en) 2011-02-07 2014-03-06 Arun K. Bhunia Carbohydrate nanoparticles for prolonged efficacy of antimicrobial peptide
EP2694040A4 (en) 2011-03-25 2014-09-03 Selecta Biosciences Inc SYNTHETIC NANOTRACKERS WITH OSMOTICALLY MEDIATED RELEASE
US10941399B2 (en) 2011-04-14 2021-03-09 Beth Israel Deaconess Medical Center, Inc. Methods and compositions for gene-specific demethylation by DNA methyltransferase (DNMT)-RNA interaction
KR20140041505A (ko) 2011-04-29 2014-04-04 셀렉타 바이오사이언시즈, 인크. 조절 b 세포 유도를 위한 관용원성 합성 나노운반체
US20140308363A1 (en) 2011-05-31 2014-10-16 Bind Therapeutics, Inc. Drug loaded polymeric nanoparticles and methods of making and using same
DK3336082T3 (da) 2011-06-08 2020-04-27 Translate Bio Inc Spaltelige lipider
PT2717893T (pt) 2011-06-08 2019-08-20 Translate Bio Inc Composições de nanopartículas lipídicas e métodos para transferência de arnm
RU2620980C2 (ru) 2011-06-09 2017-05-30 Курна, Инк. Лечение заболеваний, связанных с фратаксином (fxn), путем ингибирования природного антисмыслового транскрипта fxn
KR102039315B1 (ko) * 2011-06-21 2019-11-04 미나 테라퓨틱스 리미티드 알부민 생산 및 세포 증식
EP4014966A1 (en) 2011-07-06 2022-06-22 GlaxoSmithKline Biologicals S.A. Liposomes having useful n:p ratio for delivery of rna molecules
US20130028857A1 (en) 2011-07-29 2013-01-31 Selecta Biosciences, Inc. Synthetic nanocarriers comprising polymers comprising multiple immunomodulatory agents
EP2763701B1 (en) 2011-10-03 2018-12-19 Moderna Therapeutics, Inc. Modified nucleosides, nucleotides, and nucleic acids, and uses thereof
US9463247B2 (en) 2011-12-07 2016-10-11 Alnylam Pharmaceuticals, Inc. Branched alkyl and cycloalkyl terminated biodegradable lipids for the delivery of active agents
ES3065854T3 (en) 2011-12-07 2026-05-08 Alnylam Pharmaceuticals Inc Biodegradable lipids for the delivery of active agents
JP2015501844A (ja) 2011-12-16 2015-01-19 モデルナ セラピューティクス インコーポレイテッドＭｏｄｅｒｎａ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ． 修飾ヌクレオシド、ヌクレオチドおよび核酸組成物
SG11201503697TA (en) 2012-11-28 2015-06-29 Intercept Pharmaceuticals Inc Treatment of pulmonary disease
CA3236835A1 (en) * 2013-11-22 2015-05-28 Mina Therapeutics Limited C/ebp alpha short activating rna compositions and methods of use
CN114525279A (zh) * 2015-04-22 2022-05-24 米纳治疗有限公司 C/EBP α SARNA组合物和使用方法

Also Published As

Publication number Publication date
JP2016539127A (ja) 2016-12-15
KR102688720B1 (ko) 2024-07-29
CN107075515A (zh) 2017-08-18
WO2015075557A3 (en) 2015-11-26
EP3594348A1 (en) 2020-01-15
KR20160079824A (ko) 2016-07-06
KR102507624B1 (ko) 2023-03-09
AU2020270508B2 (en) 2022-12-15
US20240124874A1 (en) 2024-04-18
WO2015075557A2 (en) 2015-05-28
US20160298113A1 (en) 2016-10-13
KR20230037686A (ko) 2023-03-16
JP2020022513A (ja) 2020-02-13
EP3071696B1 (en) 2019-08-07
CN112220797A (zh) 2021-01-15
EP3594348B1 (en) 2021-09-08
KR20210047371A (ko) 2021-04-29
AU2014351482B2 (en) 2020-08-20
EP3985118A1 (en) 2022-04-20
US10633659B2 (en) 2020-04-28
JP6946399B2 (ja) 2021-10-06
CA3236835A1 (en) 2015-05-28
AU2014351482A1 (en) 2016-06-09
SI3071696T1 (sl) 2019-11-29
DK3594348T3 (da) 2021-10-11
AU2023201019A1 (en) 2023-08-03
US20200318114A1 (en) 2020-10-08
US20190249176A1 (en) 2019-08-15
AU2020270508A1 (en) 2020-12-17
SI3594348T1 (sl) 2022-04-29
KR102252561B1 (ko) 2021-05-20
CA2930973A1 (en) 2015-05-28
CN107075515B (zh) 2020-10-30
EP3071696A2 (en) 2016-09-28
JP6621409B2 (ja) 2019-12-18
CA2930973C (en) 2024-07-02
CN112220797B (zh) 2023-11-03
US10202601B2 (en) 2019-02-12
JP2022008404A (ja) 2022-01-13

Similar Documents

Publication Publication Date Title
DK3594348T3 (da) Kort c/ebp-alpha til aktivering af rna-sammensætninger og anvendelsesfremgangsmåder
DK3080274T3 (da) Fremgangsmåder og sammensætninger til genom-manipulation
DK2961831T3 (da) Sammensætninger og fremgangsmåder til immunterapi
KR20180084891A (ko) 구조 조성물 및 방법
DK4000608T3 (da) Farmaceutisk sammensætning, fremgangsmåder til behandling og anvendelser deraf
ZA201507006B (en) Compositions and methods of nucleic acid-targeting nucleic acids
DK3221363T3 (da) Antistoffer mod cd73 og anvendelser deraf
EP4036579C0 (en) BIOSENSOR MICROARRAY COMPOSITIONS AND METHODS
DK3099172T3 (da) Modificeret biologisk bekæmpelsesmiddel og anvendelser deraf
DK3008168T3 (da) Sc-celler og sammensætninger og fremgangsmåde til dannelse deraf
DK3043784T3 (da) Arylethere og anvendelser deraf
DK2929031T5 (da) Pcsk9-irna-sammensætninger og fremgangsmåder til anvendelse deraf
EP2953474A4 (en) COMPOSITIONS AND METHODS
DK3049056T4 (da) Topisk antisvampesammensætning og fremgangsmåder til behandling
HRP20170840T1 (hr) Supstituirani ksantini i metode njihove uporabe
DK3018183T3 (da) Halogenolefinbaseret sammensætning og anvendelse deraf
EP3039131C0 (en) ENZYME COMPOSITION AND USES
DK3058936T3 (da) Peptidsammensætning og anvendelser deraf
DK3077467T3 (da) Sikkativsammensætning og anvendelse deraf
DK3086807T3 (da) Antistoffer og fremgangsmåder til anvendelse
DK3007695T3 (da) Sammensætninger og fremgangsmåder til behandling af anæmi
FI20136131A7 (fi) Suoja, kaareva elementti ja suojan käyttö
DK2962100T3 (da) Tuberkulosebiomarkører og anvendelser deraf
DK3102200T3 (da) Terapeutisk forbindelse og sammensætning
DK3030549T3 (da) Pyridazinonforbindelser og anvendelser deraf