DK3594348T3 - Kort c/ebp-alpha til aktivering af rna-sammensætninger og anvendelsesfremgangsmåder - Google Patents

Kort c/ebp-alpha til aktivering af rna-sammensætninger og anvendelsesfremgangsmåder Download PDF

Info

Publication number
DK3594348T3
DK3594348T3 DK19190263.4T DK19190263T DK3594348T3 DK 3594348 T3 DK3594348 T3 DK 3594348T3 DK 19190263 T DK19190263 T DK 19190263T DK 3594348 T3 DK3594348 T3 DK 3594348T3
Authority
DK
Denmark
Prior art keywords
ebp
alpha
map
activation
methods
Prior art date
Application number
DK19190263.4T
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 DK3594348T3 publication Critical patent/DK3594348T3/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)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Genetics & Genomics (AREA)
  • Molecular Biology (AREA)
  • Biomedical Technology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Epidemiology (AREA)
  • Diabetes (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Hematology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Oncology (AREA)
  • Obesity (AREA)
  • Immunology (AREA)
  • Endocrinology (AREA)
  • Emergency Medicine (AREA)
  • Communicable Diseases (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Rheumatology (AREA)
  • Child & Adolescent Psychology (AREA)
DK19190263.4T 2013-11-22 2014-11-24 Kort c/ebp-alpha til aktivering af rna-sammensætninger og anvendelsesfremgangsmåder DK3594348T3 (da)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201361907732P 2013-11-22 2013-11-22
EP14843226.3A EP3071696B1 (en) 2013-11-22 2014-11-24 C/ebp alpha short activating rna compositions and methods of use

Publications (1)

Publication Number Publication Date
DK3594348T3 true DK3594348T3 (da) 2021-10-11

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 Before (1)

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

Country Status (10)

Country Link
US (4) US10202601B2 (da)
EP (3) EP3985118A1 (da)
JP (3) JP6621409B2 (da)
KR (3) KR102688720B1 (da)
CN (2) CN112220797B (da)
AU (3) AU2014351482B2 (da)
CA (2) CA3236835A1 (da)
DK (2) DK3071696T3 (da)
SI (2) SI3594348T1 (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
JP6261500B2 (ja) 2011-07-22 2018-01-17 プレジデント アンド フェローズ オブ ハーバード カレッジ ヌクレアーゼ切断特異性の評価および改善
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
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
US9526784B2 (en) 2013-09-06 2016-12-27 President And Fellows Of Harvard College Delivery system for functional nucleases
CA3236835A1 (en) * 2013-11-22 2015-05-28 Mina Therapeutics Limited C/ebp alpha short activating rna compositions and methods of use
US20150165054A1 (en) 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting caspase-9 point mutations
US20160319368A1 (en) * 2013-12-17 2016-11-03 Csir A Method for Identification of Anti-HIV Human miRNA Mimics and miRNA Inhibitors and Anti-HIV Pharmaceutical Compounds
WO2015109391A1 (en) 2014-01-24 2015-07-30 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
US20160060314A1 (en) * 2014-08-27 2016-03-03 Daewoong Jo Development of a Protein-Based Biotherapeutic Agent That Penetrates Cell-Membrane and Induces Anti-Tumor Effect in Solid Tumors - Improved Cell-Permeable Suppressor of Cytokine Signaling (iCP-SOCS3) Proteins, Polynucleotides Encoding the Same, and Anti-Tumor Compositions Comprising the Same
US20170298352A1 (en) 2014-09-30 2017-10-19 Research Institute at Nationwide Children's Hospit al Compositions and methods for treating hepatic fibrosis
US10912790B2 (en) * 2015-04-22 2021-02-09 Mina Therapeutics Limited C/EBP alpha saRNA compositions and methods of use
JP7073109B2 (ja) * 2015-07-02 2022-05-23 シティ・オブ・ホープ ホスホロチオエート化オリゴデオキシヌクレオチドを含む化合物及び組成物、ならびにそれらの使用法
CA2998876A1 (en) * 2015-09-21 2017-03-30 Intercept Pharmaceuticals, Inc. Methods of promoting hepatic regeneration
JP7109784B2 (ja) 2015-10-23 2022-08-01 プレジデント アンド フェローズ オブ ハーバード カレッジ 遺伝子編集のための進化したＣａｓ９蛋白質
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
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
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
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
KR102547316B1 (ko) 2016-08-03 2023-06-23 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 아데노신 핵염기 편집제 및 그의 용도
EP3506927A4 (en) 2016-08-03 2020-05-06 University of South Florida REELIN COMPOSITIONS FOR TREATING NEUROLOGICAL DISEASES
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
US11542509B2 (en) 2016-08-24 2023-01-03 President And Fellows Of Harvard College Incorporation of unnatural amino acids into proteins using base editing
EP3526320A1 (en) 2016-10-14 2019-08-21 President and Fellows of Harvard College Aav delivery of nucleobase editors
US11261428B2 (en) 2018-03-15 2022-03-01 KSQ Therapeutics, Inc. Gene-regulating compositions and methods for improved immunotherapy
US11332713B2 (en) 2016-11-16 2022-05-17 KSQ Therapeutics, Inc. Gene-regulating compositions and methods for improved immunotherapy
EP3551230A1 (en) 2016-12-08 2019-10-16 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
EP3592853A1 (en) 2017-03-09 2020-01-15 President and Fellows of Harvard College Suppression of pain by gene editing
JP2020510038A (ja) 2017-03-09 2020-04-02 プレジデント アンド フェローズ オブ ハーバード カレッジ がんワクチン
JP2020510439A (ja) 2017-03-10 2020-04-09 プレジデント アンド フェローズ オブ ハーバード カレッジ シトシンからグアニンへの塩基編集因子
CA3057192A1 (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
WO2019023680A1 (en) 2017-07-28 2019-01-31 President And Fellows Of Harvard College METHODS AND COMPOSITIONS FOR EVOLUTION OF BASIC EDITORS USING PHAGE-ASSISTED CONTINUOUS EVOLUTION (PACE)
EP3676376B1 (en) 2017-08-30 2025-01-15 President and Fellows of Harvard College High efficiency base editors comprising gam
WO2019048645A1 (en) * 2017-09-08 2019-03-14 Mina Therapeutics Limited STABILIZED COMPOSITIONS OF SMALL ACTIVATOR RNA (PARNA) FROM CEBPA AND METHODS OF USE
CA3075205A1 (en) 2017-09-08 2019-03-14 Mina Therapeutics Limited Stabilized hnf4a sarna compositions and methods of use
EP3684375A4 (en) * 2017-09-22 2021-09-22 John Mansell COMPOSITIONS AND METHODS OF TREATMENT OF 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
KR20250107288A (ko) 2017-10-16 2025-07-11 더 브로드 인스티튜트, 인코퍼레이티드 아데노신 염기 편집제의 용도
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
EP3797160A1 (en) 2018-05-23 2021-03-31 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
AU2019285344A1 (en) * 2018-06-15 2020-12-10 Mina Therapeutics Limited Combination therapies comprising C/EBP alpha saRNA
US12522807B2 (en) 2018-07-09 2026-01-13 The Broad Institute, Inc. RNA programmable epigenetic RNA modifiers and uses thereof
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
WO2020155534A1 (zh) * 2019-01-30 2020-08-06 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
JP7657726B2 (ja) 2019-03-19 2025-04-07 ザ ブロード インスティテュート，インコーポレーテッド 編集ヌクレオチド配列を編集するための方法および組成物
WO2020196736A1 (ja) * 2019-03-28 2020-10-01 日東電工株式会社 ＲＮＡｉ分子
US12473543B2 (en) 2019-04-17 2025-11-18 The Broad Institute, Inc. Adenine base editors with reduced off-target effects
KR20220004989A (ko) * 2019-04-24 2022-01-12 네바다 리서치 & 이노베이션 코포레이션 MiR-10 모사체 및 이의 표적의 방법 및 조성물
US20220396794A1 (en) * 2019-06-04 2022-12-15 Apterna Limited APTAMERS AGAINST TRANSFERRIN RECEPTOR (TfR)
CN114585633A (zh) * 2019-08-19 2022-06-03 米纳治疗有限公司 寡核苷酸缀合物组合物和使用方法
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
JP2023519915A (ja) * 2020-03-26 2023-05-15 ラミレス－フォート，マリグダリア，カレス 紫外線照射治療
DE112021002672T5 (de) 2020-05-08 2023-04-13 President And Fellows Of Harvard College Vefahren und zusammensetzungen zum gleichzeitigen editieren beider stränge einer doppelsträngigen nukleotid-zielsequenz
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
CN116829716A (zh) * 2021-02-07 2023-09-29 中美瑞康核酸技术(南通)研究院有限公司 一种化学修饰的小激活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
WO2024130257A2 (en) * 2022-12-16 2024-06-20 Purdue Research Foundation In vivo and ex vivo production of car-neutrophils and use thereof to treat and image cancer
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
KR20250158048A (ko) 2023-03-03 2025-11-05 아스널 바이오사이언시스, 인크. Psma 및 ca9을 표적으로 하는 시스템
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
US5258506A (en) 1984-10-16 1993-11-02 Chiron Corporation Photolabile reagents for incorporation into oligonucleotide chains
US5430136A (en) 1984-10-16 1995-07-04 Chiron Corporation Oligonucleotides having selectably cleavable and/or abasic sites
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
US5599923A (en) 1989-03-06 1997-02-04 Board Of Regents, University Of Tx Texaphyrin metal complexes having improved functionalization
US5457183A (en) 1989-03-06 1995-10-10 Board Of Regents, The University Of Texas System Hydroxylated texaphyrins
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
US5214136A (en) 1990-02-20 1993-05-25 Gilead Sciences, Inc. Anthraquinone-derivatives oligonucleotides
AU7579991A (en) 1990-02-20 1991-09-18 Gilead Sciences, Inc. Pseudonucleosides and pseudonucleotides and their polymers
ES2116977T3 (es) 1990-05-11 1998-08-01 Microprobe Corp Soportes solidos para ensayos de hibridacion de acidos nucleicos y metodos para inmovilizar oligonucleotidos de modo covalente.
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
US5218105A (en) 1990-07-27 1993-06-08 Isis Pharmaceuticals Polyamine conjugated oligonucleotides
US5608046A (en) 1990-07-27 1997-03-04 Isis Pharmaceuticals, Inc. Conjugated 4'-desmethyl nucleoside analog compounds
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
WO1992008728A1 (en) 1990-11-08 1992-05-29 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
US5565552A (en) 1992-01-21 1996-10-15 Pharmacyclics, Inc. Method of expanded porphyrin-oligonucleotide conjugate synthesis
US5595726A (en) 1992-01-21 1997-01-21 Pharmacyclics, Inc. Chromophore probe for detection of nucleic acid
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
ATE355826T1 (de) 1997-10-10 2007-03-15 Inex Pharmaceuticals Corp Verfahren zur verkapselung von nukleinsäuren in lipiddoppelschichten
AU5613999A (en) 1998-09-18 2000-04-10 University Of British Columbia, The 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
AU2002214854A1 (en) 2000-10-25 2002-05-06 Inex Pharmaceuticals Corporation Lipid formulations for target delivery
ES2248581T3 (es) 2001-03-12 2006-03-16 Intercept Pharmaceuticals, Inc. Esteroides como agonistas de 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
WO2003093449A2 (en) 2002-05-06 2003-11-13 Nucleonics, Inc. Methods for delivery of nucleic acids
EP2305812A3 (en) 2002-11-14 2012-06-06 Dharmacon, Inc. Fuctional and hyperfunctional sirna
EP1568706A1 (en) 2004-02-26 2005-08-31 Intercept Pharmaceuticals, Inc. Novel steroid agonist for FXR
US7799565B2 (en) 2004-06-07 2010-09-21 Protiva Biotherapeutics, Inc. Lipid encapsulated interfering RNA
EP1791567B1 (en) 2004-08-10 2015-07-29 Alnylam Pharmaceuticals Inc. Chemically modified oligonucleotides
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
US7404969B2 (en) 2005-02-14 2008-07-29 Sirna Therapeutics, Inc. Lipid nanoparticle based compositions and methods for the delivery of biologically active molecules
JP5042863B2 (ja) 2005-02-14 2012-10-03 サーナ・セラピューティクス・インコーポレイテッド 生物学的に活性な分子をデリバリーするための脂質ナノ粒子系組成物および方法
DE602006008625D1 (de) 2005-04-01 2009-10-01 Intezyne Technologies Inc Polymermicellen für die arzneistoffzufuhr
CA2604532C (en) 2005-04-15 2017-03-07 The Regents Of The University Of California Small activating rna molecules and methods of use
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
JP5141256B2 (ja) 2005-10-20 2013-02-13 旭硝子株式会社 ポリテトラフルオロエチレン水性分散液およびその製品
JP5066095B2 (ja) 2005-11-17 2012-11-07 ボード・オブ・リージエンツ，ザ・ユニバーシテイ・オブ・テキサス・システム 染色体ｄｎａに標的化されるオリゴマーによる遺伝子発現の調節
US8288354B2 (en) 2005-12-28 2012-10-16 The Scripps Research Institute Natural antisense and non-coding RNA transcripts as drug targets
CN101395170A (zh) 2006-02-14 2009-03-25 英特塞普特药品公司 用于预防或治疗fxr介导的疾病或状态的作为fxr配体的胆汁酸衍生物
US7932244B2 (en) 2006-06-27 2011-04-26 Intercept Pharmaceuticals, Inc. Bile acid derivatives as FXR ligands for the prevention or treatment of FXR-mediated diseases or conditions
EP3192788A1 (en) 2006-10-03 2017-07-19 Arbutus Biopharma Corporation Lipid containing formulations
JP5424885B2 (ja) 2006-10-13 2014-02-26 マリーナ バイオテック，インコーポレイテッド 両性リポソームにおけるまたは両性リポソームに関する改善、両性リポソームを処方する方法および両性リポソームに充填する方法
KR101555716B1 (ko) 2007-01-19 2015-09-25 인터셉트 파마슈티컬즈, 인크. Ｔｇｒ5 조절제로서의 23-치환 담즙산 및 이의 사용 방법
US20100015218A1 (en) 2007-02-16 2010-01-21 Vasant Jadhav Compositions and methods for potentiated activity of biologically active molecules
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
JP2010519910A (ja) 2007-03-02 2010-06-10 エムディーアールエヌエー，インコーポレイテッド Ｂｃｌ２遺伝子の発現を抑制するための核酸化合物およびその使用
WO2008109556A1 (en) 2007-03-02 2008-09-12 Mdrna, Inc. Nucleic acid compounds for inhibiting telomerase gene expression and uses thereof
EP2136788B1 (en) 2007-03-30 2011-10-26 Bind Biosciences, Inc. Cancer cell targeting using nanoparticles
SG10201509679XA (en) 2007-05-29 2015-12-30 Christopher B Reid Methods for production and uses of multipotent cell populations
EP2205746A4 (en) 2007-10-04 2010-12-22 Univ Texas MODULATION OF GENE EXPRESSION WITH AGRNA AND GAPS WITH ANTISENSE TRANSCRIPTS AS A TARGET
WO2009086428A2 (en) 2007-12-28 2009-07-09 The Regents Of The University Of California Methods and compositions for increasing gene expression
EP2105145A1 (en) 2008-03-27 2009-09-30 ETH Zürich Method for muscle-specific delivery lipid-conjugated oligonucleotides
AU2009234424A1 (en) 2008-04-07 2009-10-15 Nupotential, Inc. Reprogramming a cell by inducing a pluripotent gene through use of an HDAC modulator
WO2009126927A2 (en) 2008-04-11 2009-10-15 Joslin Diabetes Center, Inc. Methods of generating insulin-producing cells
WO2009126933A2 (en) 2008-04-11 2009-10-15 Alnylam Pharmaceuticals, Inc. Site-specific delivery of nucleic acids by combining targeting ligands with endosomolytic components
AU2009238175C1 (en) 2008-04-15 2023-11-30 Arbutus Biopharma Corporation Novel lipid formulations for nucleic acid delivery
WO2009132131A1 (en) 2008-04-22 2009-10-29 Alnylam Pharmaceuticals, Inc. Amino lipid based improved lipid formulation
ES2721850T3 (es) 2008-06-16 2019-08-05 Pfizer Nanopartículas poliméricas terapéuticas que comprenden alcaloides vinca y procedimientos de fabricación y uso de las mismas
US8613951B2 (en) 2008-06-16 2013-12-24 Bind Therapeutics, Inc. Therapeutic polymeric nanoparticles with mTor inhibitors and methods of making and using same
WO2010005740A2 (en) 2008-06-16 2010-01-14 Bind Biosciences, Inc. Methods for the preparation of targeting agent functionalized diblock copolymers for use in fabrication of therapeutic targeted nanoparticles
JP2012501965A (ja) 2008-06-16 2012-01-26 バインド バイオサイエンシズ インコーポレイテッド 薬剤を装填したポリマーナノ粒子及びその製造方法と使用方法
WO2010014836A2 (en) 2008-07-30 2010-02-04 Intercept Pharmaceuticals, Inc. Tgr5 modulators and methods of use thereof
US20110224447A1 (en) 2008-08-18 2011-09-15 Bowman Keith A Novel Lipid Nanoparticles and Novel Components for Delivery of Nucleic Acids
US20100087337A1 (en) 2008-09-10 2010-04-08 Bind Biosciences, Inc. High Throughput Fabrication of Nanoparticles
CA2739170A1 (en) 2008-09-25 2010-04-01 Alnylam Pharmaceuticals, Inc. Lipid formulated compositions and methods for inhibiting expression of serum amyloid a gene
CN104119242B (zh) 2008-10-09 2017-07-07 泰米拉制药公司 改善的氨基脂质和递送核酸的方法
EP2363467B1 (en) 2008-10-23 2015-12-16 The University of Tokyo Method for inhibiting function of micro-rna
NZ592821A (en) 2008-11-13 2012-06-29 Baylor Res Inst Regeneration of pancreatic islets and reversal of diabetes by islet transcription factor genes delivered in vivo
DK2698375T3 (da) 2008-11-19 2018-05-28 Intercept Pharmaceuticals Inc TGR5-modulatorer og fremgangsmåde til anvendelse deraf
US20100216804A1 (en) 2008-12-15 2010-08-26 Zale Stephen E Long Circulating Nanoparticles for Sustained Release of Therapeutic Agents
US9181315B2 (en) * 2009-01-08 2015-11-10 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
WO2010104661A1 (en) 2009-03-12 2010-09-16 Illinois Tool Works Inc. Mis-fuel inhibitor
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
EP2421972A2 (en) 2009-04-24 2012-02-29 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
WO2010127159A2 (en) 2009-04-30 2010-11-04 Intezyne Technologies, Incorporated Polymeric micelles for polynucleotide encapsulation
KR20180094137A (ko) 2009-05-05 2018-08-22 알닐람 파마슈티칼스 인코포레이티드 지질 조성물
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
CA2762653A1 (en) 2009-05-27 2010-12-02 Selecta Biosciences, Inc. Immunomodulatory agent-polymeric compounds
MX342785B (es) 2009-06-10 2016-10-12 Alnylam Pharmaceuticals Inc Formulacion mejorada de lipido.
WO2010147992A1 (en) 2009-06-15 2010-12-23 Alnylam Pharmaceuticals, Inc. 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
ES2613498T3 (es) 2009-07-01 2017-05-24 Protiva Biotherapeutics Inc. Nuevas formulaciones de lípidos para el suministro de agentes terapéuticos a tumores sólidos
US8569256B2 (en) 2009-07-01 2013-10-29 Protiva Biotherapeutics, Inc. Cationic lipids and methods for the delivery of therapeutic agents
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
SG177483A1 (en) 2009-07-20 2012-02-28 Janssen Biotech Inc Differentiation of human embryonic stem cells
US9029338B2 (en) 2009-08-14 2015-05-12 Alnylam Pharmaceuticals, Inc. Lipid formulated compositions and methods for inhibiting expression of a gene from the ebola virus
EP2467357B1 (en) 2009-08-20 2016-03-30 Sirna Therapeutics, Inc. Novel cationic lipids with various head groups for oligonucleotide delivery
WO2011036557A1 (en) 2009-09-22 2011-03-31 The University Of British Columbia Compositions and methods for enhancing cellular uptake and intracellular delivery of lipid particles
US20120253032A1 (en) 2009-10-08 2012-10-04 Merck Sharp & Dohme Corporation 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
CA2816925C (en) 2009-11-04 2023-01-10 The University Of British Columbia Nucleic acid-containing lipid particles and related methods
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
US8357401B2 (en) 2009-12-11 2013-01-22 Bind Biosciences, Inc. Stable formulations for lyophilizing therapeutic particles
JP6175237B2 (ja) 2009-12-15 2017-08-02 ファイザー・インク コルチコステロイドを含む治療用ポリマーナノ粒およびそれを製造かつ使用する方法
JP5898627B2 (ja) 2009-12-15 2016-04-06 バインド セラピューティックス インコーポレイテッド エポチロンを含む治療用ポリマーナノ粒子ならびにそれを製造および使用する方法
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
CA2784568A1 (en) 2009-12-18 2011-06-23 The University Of British Columbia Lipid particles for delivery of nucleic acids
KR101853509B1 (ko) 2010-01-06 2018-04-30 큐알엔에이, 인크. 췌장 발달 유전자에 대한 천연 안티센스 전사체의 억제에 의한 췌장 발달 유전자와 관련된 질환의 치료
US20130116419A1 (en) 2010-01-22 2013-05-09 Daniel Zewge Post-synthetic chemical modification of rna at the 2'-position of the ribose ring via "click" chemistry
EP2558074B1 (en) 2010-04-08 2018-06-06 The Trustees of Princeton University Preparation of lipid nanoparticles
US20110262491A1 (en) 2010-04-12 2011-10-27 Selecta Biosciences, Inc. Emulsions and methods of making nanocarriers
WO2011139911A2 (en) 2010-04-29 2011-11-10 Isis Pharmaceuticals, Inc. Lipid formulated single stranded rna
US9254327B2 (en) 2010-05-10 2016-02-09 Alnylam Pharmaceuticals, Inc. 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
WO2012016188A2 (en) 2010-07-30 2012-02-02 Alnylam Pharmaceuticals, Inc. Methods and compositions for delivery of active agents
CA2801523C (en) 2010-07-30 2021-08-03 Curevac Gmbh Complexation of nucleic acids with disulfide-crosslinked cationic components for transfection and immunostimulation
WO2012016184A2 (en) 2010-07-30 2012-02-02 Alnylam Pharmaceuticals, Inc. Methods and compositions for delivery of active agents
ES2888231T3 (es) 2010-09-20 2022-01-03 Sirna Therapeutics Inc Lípidos catiónicos de bajo peso molecular para el suministro de oligonucleótidos
WO2012047656A1 (en) 2010-09-27 2012-04-12 The University Of British Columbia Lipid a analog compositions
EP2621480B1 (en) 2010-09-30 2018-08-15 Sirna Therapeutics, Inc. Low molecular weight cationic lipids for oligonucleotide delivery
KR20130132795A (ko) 2010-10-08 2013-12-05 미나 테라퓨틱스 리미티드 짧은 rna 분자
JP2013545727A (ja) 2010-10-21 2013-12-26 メルク・シャープ・アンド・ドーム・コーポレーション オリゴヌクレオチド送達用の新規低分子量カチオン性脂質
EP2629760A4 (en) 2010-10-22 2014-04-02 Bind Therapeutics Inc THERAPEUTIC NANOPARTICLES CONTAINING COPOLYMERS OF HIGH MOLECULAR WEIGHT
EP2635265B1 (en) 2010-11-05 2018-04-04 Sirna Therapeutics, Inc. Novel low molecular weight cyclic amine containing cationic lipids for oligonucleotide delivery
EP3260540A1 (en) 2010-11-12 2017-12-27 The General Hospital Corporation Polycomb-associated non-coding rnas
TWI473612B (zh) * 2010-12-23 2015-02-21 Food Industry Res & Dev Inst 新穎紫紅麴酮、其製備方法及紫紅麴酮之應用
WO2012092552A1 (en) 2010-12-30 2012-07-05 Selecta Biosciences, Inc. Synthetic nanocarriers with reactive groups that release biologically active agents
CA2824526C (en) 2011-01-11 2020-07-07 Alnylam Pharmaceuticals, Inc. Pegylated lipids and their use for drug delivery
DE102011082231A1 (de) 2011-01-12 2012-07-12 Robert Bosch Gmbh Zündspule, insbesondere für kleinbauende Motoren
WO2012109121A1 (en) 2011-02-07 2012-08-16 Purdue Research Foundation Carbohydrate nanoparticles for prolonged efficacy of antimicrobial peptide
CA2830948A1 (en) 2011-03-25 2012-10-04 Selecta Biosciences, Inc. Osmotic mediated release synthetic nanocarriers
WO2012142480A1 (en) 2011-04-14 2012-10-18 Beth Israel Deaconess Medical Center, Inc. Chimeric rna oligonucleotides and uses thereof
KR20140041505A (ko) 2011-04-29 2014-04-04 셀렉타 바이오사이언시즈, 인크. 조절 b 세포 유도를 위한 관용원성 합성 나노운반체
WO2012166923A2 (en) 2011-05-31 2012-12-06 Bind Biosciences Drug loaded polymeric nanoparticles and methods of making and using same
EP3354644A1 (en) 2011-06-08 2018-08-01 Translate Bio, Inc. Cleavable lipids
PL3586861T3 (pl) 2011-06-08 2022-05-23 Translate Bio, Inc. Kompozycje nanocząstek lipidowych i sposoby dostarczania mrna
EP2718439B1 (en) 2011-06-09 2017-08-09 CuRNA, Inc. Treatment of frataxin (fxn) related diseases by inhibition of natural antisense transcript to fxn
EP4060043A1 (en) 2011-06-21 2022-09-21 MiNA Therapeutics Limited Albumin production and cell proliferation
US20140141070A1 (en) 2011-07-06 2014-05-22 Andrew Geall Liposomes having useful n:p ratio for delivery of rna molecules
JP2014521687A (ja) 2011-07-29 2014-08-28 セレクタ バイオサイエンシーズ インコーポレーテッド 体液性および細胞傷害性ｔリンパ球（ｃｔｌ）免疫応答を生成する合成ナノキャリア
RU2648950C2 (ru) 2011-10-03 2018-04-02 Модерна Терапьютикс, Инк. Модифицированные нуклеозиды, нуклеотиды и нуклеиновые кислоты и их применение
EP2788006B1 (en) 2011-12-07 2026-01-07 Alnylam Pharmaceuticals, Inc. Biodegradable lipids for the delivery of active agents
EP2788316B1 (en) 2011-12-07 2019-04-24 Alnylam Pharmaceuticals, Inc. Branched alkyl and cycloalkyl terminated biodegradable lipids for the delivery of active agents
CN104114572A (zh) 2011-12-16 2014-10-22 现代治疗公司 经修饰的核苷、核苷酸和核酸组合物
AU2013352288B2 (en) 2012-11-28 2017-11-23 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
US10912790B2 (en) * 2015-04-22 2021-02-09 Mina Therapeutics Limited C/EBP alpha saRNA compositions and methods of use

Also Published As

Publication number Publication date
DK3071696T3 (da) 2019-10-07
EP3071696B1 (en) 2019-08-07
EP3985118A1 (en) 2022-04-20
JP2020022513A (ja) 2020-02-13
CN112220797A (zh) 2021-01-15
US10633659B2 (en) 2020-04-28
CN107075515B (zh) 2020-10-30
AU2014351482B2 (en) 2020-08-20
JP2016539127A (ja) 2016-12-15
KR102252561B1 (ko) 2021-05-20
KR102688720B1 (ko) 2024-07-29
SI3594348T1 (sl) 2022-04-29
US20190249176A1 (en) 2019-08-15
KR20160079824A (ko) 2016-07-06
JP6946399B2 (ja) 2021-10-06
AU2020270508A1 (en) 2020-12-17
CA3236835A1 (en) 2015-05-28
AU2023201019A1 (en) 2023-08-03
KR20210047371A (ko) 2021-04-29
KR20230037686A (ko) 2023-03-16
US10202601B2 (en) 2019-02-12
US20200318114A1 (en) 2020-10-08
CN112220797B (zh) 2023-11-03
CA2930973C (en) 2024-07-02
JP2022008404A (ja) 2022-01-13
EP3594348A1 (en) 2020-01-15
CA2930973A1 (en) 2015-05-28
EP3594348B1 (en) 2021-09-08
SI3071696T1 (sl) 2019-11-29
US20240124874A1 (en) 2024-04-18
CN107075515A (zh) 2017-08-18
KR102507624B1 (ko) 2023-03-09
JP6621409B2 (ja) 2019-12-18
US20160298113A1 (en) 2016-10-13
WO2015075557A2 (en) 2015-05-28
EP3071696A2 (en) 2016-09-28
WO2015075557A3 (en) 2015-11-26
AU2020270508B2 (en) 2022-12-15
AU2014351482A1 (en) 2016-06-09

Similar Documents

Publication Publication Date Title
DK3594348T3 (da) Kort c/ebp-alpha til aktivering af rna-sammensætninger og anvendelsesfremgangsmåder
IL274064A (en) Compositions and methods for modulating apolipoprotein (a) expression
DK3556869T3 (da) Sammensætninger og fremgangsmåder til sekventering af polynukleotider
DK2971167T3 (da) Sammensætninger og fremgangsmåder til targeting af nucleinsyre med nucleinsyrer
DK3080274T3 (da) Fremgangsmåder og sammensætninger til genom-manipulation
DK2961831T3 (da) Sammensætninger og fremgangsmåder til immunterapi
DK3008168T3 (da) Sc-celler og sammensætninger og fremgangsmåde til dannelse deraf
DK3252160T3 (da) Systemer, fremgangsmåder og sammensætninger med crispr-cas-bestanddele til sekvensmanipulation
DK2929031T5 (da) Pcsk9-irna-sammensætninger og fremgangsmåder til anvendelse deraf
DK3087095T3 (da) FcRn-antagonist og fremgangsmåder til anvendelse
DK3428290T3 (da) Sammensætninger og fremgangsmåder til amplifikation af nukleinsyrer
DK2838917T3 (da) Fremgangsmåder og midler til frembringelse af heterodimere ig-lignende molekyler
DK2970456T3 (da) Fremgangsmåder og sammensætninger til levering af mrna-kodede antistoffer
DK3572527T3 (da) Fremgangsmåder og præparater til fremstilling af nukleinsyrebiblioteker
DK3019200T3 (da) Oligonukleotid-ligand-konjugater og fremgangsmåde til fremstiling deraf
DK2978419T3 (da) Sammensætninger til forbedring af cellelevedygtighed og fremgangsmåder til anvendelse deraf
DK4082530T5 (da) Proteasehæmmerholdige sammensætninger, sammensætninger omfattende samme og fremgangsmåder til fremstilling og anvendelse af samme
DK2948548T3 (da) Fremgangsmåde til specifik isolering af nukleinsyrer af interesse
DK3086807T3 (da) Antistoffer og fremgangsmåder til anvendelse
DK3071215T3 (da) Sammensætninger og fremgangsmåder til behandling af pulmonal hypertension
DK3007695T3 (da) Sammensætninger og fremgangsmåder til behandling af anæmi
DK2970123T3 (da) Salt af omecamtiv mecarbil og fremgangsmåde til fremstilling heraf
DK3050883T3 (da) Fremgangsmåde til fremstilling af diazabicyclooctanderivater og mellemprodukter
DK3363805T3 (da) Fremgangsmåder til syntese af sphingomyeliner og dihydrosphingomyeliner
DK3013544T3 (da) Fremgangsmåde til betonfremstilling