JP5933163B2 - 新規化合物 - Google Patents

新規化合物 Download PDF

Info

Publication number
JP5933163B2
JP5933163B2 JP2009531586A JP2009531586A JP5933163B2 JP 5933163 B2 JP5933163 B2 JP 5933163B2 JP 2009531586 A JP2009531586 A JP 2009531586A JP 2009531586 A JP2009531586 A JP 2009531586A JP 5933163 B2 JP5933163 B2 JP 5933163B2
Authority
JP
Japan
Prior art keywords
compound
formula
alkyl
lipid
acid
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Active
Application number
JP2009531586A
Other languages
English (en)
Japanese (ja)
Other versions
JP2010505873A5 (ja
JP2010505873A (ja
Inventor
マノハラン、ムシア
ジー． ラジェーブ、カランソータイル
ジー． ラジェーブ、カランソータイル
アキンク、アキン
ジャヤプラカシュ、ケイ．ナラヤナネア
ジャイラマン、ムスサミー
エイ． マイヤー、マーティン
エイ． マイヤー、マーティン
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Arbutus Biopharma Corp
Original Assignee
Tekmira Pharmaceuticals Corp
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 Tekmira Pharmaceuticals Corp filed Critical Tekmira Pharmaceuticals Corp
Publication of JP2010505873A publication Critical patent/JP2010505873A/ja
Publication of JP2010505873A5 publication Critical patent/JP2010505873A5/ja
Application granted granted Critical
Publication of JP5933163B2 publication Critical patent/JP5933163B2/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Classifications

    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07C—ACYCLIC OR CARBOCYCLIC COMPOUNDS
    • C07C219/00—Compounds containing amino and esterified hydroxy groups bound to the same carbon skeleton
    • C07C219/02—Compounds containing amino and esterified hydroxy groups bound to the same carbon skeleton having esterified hydroxy groups and amino groups bound to acyclic carbon atoms of the same carbon skeleton
    • C07C219/04—Compounds containing amino and esterified hydroxy groups bound to the same carbon skeleton having esterified hydroxy groups and amino groups bound to acyclic carbon atoms of the same carbon skeleton the carbon skeleton being acyclic and saturated
    • C07C219/06—Compounds containing amino and esterified hydroxy groups bound to the same carbon skeleton having esterified hydroxy groups and amino groups bound to acyclic carbon atoms of the same carbon skeleton the carbon skeleton being acyclic and saturated having the hydroxy groups esterified by carboxylic acids having the esterifying carboxyl groups bound to hydrogen atoms or to acyclic carbon atoms of an acyclic saturated carbon skeleton
    • 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
    • A61K31/325—Carbamic acids; Thiocarbamic acids; Anhydrides or salts thereof
    • 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
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/10—Dispersions; Emulsions
    • A61K9/127—Synthetic bilayered vehicles, e.g. liposomes or liposomes with cholesterol as the only non-phosphatidyl surfactant
    • A61K9/1271—Non-conventional liposomes, e.g. PEGylated liposomes or liposomes coated or grafted with polymers
    • A61K9/1272—Non-conventional liposomes, e.g. PEGylated liposomes or liposomes coated or grafted with polymers comprising non-phosphatidyl surfactants as bilayer-forming substances, e.g. cationic lipids or non-phosphatidyl liposomes coated or grafted with polymers
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/10—Dispersions; Emulsions
    • A61K9/127—Synthetic bilayered vehicles, e.g. liposomes or liposomes with cholesterol as the only non-phosphatidyl surfactant
    • A61K9/1271—Non-conventional liposomes, e.g. PEGylated liposomes or liposomes coated or grafted with polymers
    • A61K9/1273—Polymersomes; Liposomes with polymerisable or polymerised bilayer-forming substances
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07C—ACYCLIC OR CARBOCYCLIC COMPOUNDS
    • C07C209/00—Preparation of compounds containing amino groups bound to a carbon skeleton
    • C07C209/68—Preparation of compounds containing amino groups bound to a carbon skeleton from amines, by reactions not involving amino groups, e.g. reduction of unsaturated amines, aromatisation, or substitution of the carbon skeleton
    • C07C209/78—Preparation of compounds containing amino groups bound to a carbon skeleton from amines, by reactions not involving amino groups, e.g. reduction of unsaturated amines, aromatisation, or substitution of the carbon skeleton from carbonyl compounds, e.g. from formaldehyde, and amines having amino groups bound to carbon atoms of six-membered aromatic rings, with formation of methylene-diarylamines
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07C—ACYCLIC OR CARBOCYCLIC COMPOUNDS
    • C07C233/00—Carboxylic acid amides
    • C07C233/01—Carboxylic acid amides having carbon atoms of carboxamide groups bound to hydrogen atoms or to acyclic carbon atoms
    • C07C233/34—Carboxylic acid amides having carbon atoms of carboxamide groups bound to hydrogen atoms or to acyclic carbon atoms having the nitrogen atom of at least one of the carboxamide groups bound to a carbon atom of a hydrocarbon radical substituted by amino groups
    • C07C233/35—Carboxylic acid amides having carbon atoms of carboxamide groups bound to hydrogen atoms or to acyclic carbon atoms having the nitrogen atom of at least one of the carboxamide groups bound to a carbon atom of a hydrocarbon radical substituted by amino groups with the substituted hydrocarbon radical bound to the nitrogen atom of the carboxamide group by an acyclic carbon atom
    • C07C233/36—Carboxylic acid amides having carbon atoms of carboxamide groups bound to hydrogen atoms or to acyclic carbon atoms having the nitrogen atom of at least one of the carboxamide groups bound to a carbon atom of a hydrocarbon radical substituted by amino groups with the substituted hydrocarbon radical bound to the nitrogen atom of the carboxamide group by an acyclic carbon atom having the carbon atom of the carboxamide group bound to a hydrogen atom or to a carbon atom of an acyclic saturated carbon skeleton
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07C—ACYCLIC OR CARBOCYCLIC COMPOUNDS
    • C07C271/00—Derivatives of carbamic acids, i.e. compounds containing any of the groups, the nitrogen atom not being part of nitro or nitroso groups
    • C07C271/06—Esters of carbamic acids
    • C07C271/08—Esters of carbamic acids having oxygen atoms of carbamate groups bound to acyclic carbon atoms
    • C07C271/10—Esters of carbamic acids having oxygen atoms of carbamate groups bound to acyclic carbon atoms with the nitrogen atoms of the carbamate groups bound to hydrogen atoms or to acyclic carbon atoms
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07C—ACYCLIC OR CARBOCYCLIC COMPOUNDS
    • C07C323/00—Thiols, sulfides, hydropolysulfides or polysulfides substituted by halogen, oxygen or nitrogen atoms, or by sulfur atoms not being part of thio groups
    • C07C323/23—Thiols, sulfides, hydropolysulfides or polysulfides substituted by halogen, oxygen or nitrogen atoms, or by sulfur atoms not being part of thio groups containing thio groups and nitrogen atoms, not being part of nitro or nitroso groups, bound to the same carbon skeleton
    • C07C323/24—Thiols, sulfides, hydropolysulfides or polysulfides substituted by halogen, oxygen or nitrogen atoms, or by sulfur atoms not being part of thio groups containing thio groups and nitrogen atoms, not being part of nitro or nitroso groups, bound to the same carbon skeleton having the sulfur atoms of the thio groups bound to acyclic carbon atoms of the carbon skeleton
    • C07C323/25—Thiols, sulfides, hydropolysulfides or polysulfides substituted by halogen, oxygen or nitrogen atoms, or by sulfur atoms not being part of thio groups containing thio groups and nitrogen atoms, not being part of nitro or nitroso groups, bound to the same carbon skeleton having the sulfur atoms of the thio groups bound to acyclic carbon atoms of the carbon skeleton the carbon skeleton being acyclic and saturated
    • 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
    • A61K47/30—Macromolecular organic or inorganic compounds, e.g. inorganic polyphosphates
    • A61K47/34—Macromolecular compounds obtained otherwise than by reactions only involving carbon-to-carbon unsaturated bonds, e.g. polyesters, polyamino acids, polysiloxanes, polyphosphazines, copolymers of polyalkylene glycol or poloxamers

Landscapes

  • Chemical & Material Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Epidemiology (AREA)
  • Dispersion Chemistry (AREA)
  • Molecular Biology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Biophysics (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Biochemistry (AREA)
  • Biotechnology (AREA)
  • Genetics & Genomics (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Organic Low-Molecular-Weight Compounds And Preparation Thereof (AREA)
  • Low-Molecular Organic Synthesis Reactions Using Catalysts (AREA)
JP2009531586A 2006-10-03 2007-10-03 新規化合物 Active JP5933163B2 (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US82802206P 2006-10-03 2006-10-03
US60/828,022 2006-10-03
US87045706P 2006-12-18 2006-12-18
US60/870,457 2006-12-18
PCT/US2007/080331 WO2008042973A2 (en) 2006-10-03 2007-10-03 Lipid containing formulations

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2013123084A Division JP5833597B2 (ja) 2006-10-03 2013-06-11 会合複合体及びそれを形成する方法

Publications (3)

Publication Number Publication Date
JP2010505873A JP2010505873A (ja) 2010-02-25
JP2010505873A5 JP2010505873A5 (ja) 2010-11-25
JP5933163B2 true JP5933163B2 (ja) 2016-06-08

Family

ID=39269192

Family Applications (8)

Application Number Title Priority Date Filing Date
JP2009531586A Active JP5933163B2 (ja) 2006-10-03 2007-10-03 新規化合物
JP2013123084A Expired - Fee Related JP5833597B2 (ja) 2006-10-03 2013-06-11 会合複合体及びそれを形成する方法
JP2015157142A Pending JP2015227370A (ja) 2006-10-03 2015-08-07 会合複合体及びそれを形成する方法
JP2016219673A Pending JP2017036330A (ja) 2006-10-03 2016-11-10 製剤用化合物
JP2018236147A Pending JP2019048887A (ja) 2006-10-03 2018-12-18 製剤用化合物
JP2020182378A Active JP7127098B2 (ja) 2006-10-03 2020-10-30 製剤用脂質ナノ粒子
JP2022129999A Pending JP2022164716A (ja) 2006-10-03 2022-08-17 製剤用脂質ナノ粒子
JP2024119315A Pending JP2024150681A (ja) 2006-10-03 2024-07-25 製剤用脂質ナノ粒子

Family Applications After (7)

Application Number Title Priority Date Filing Date
JP2013123084A Expired - Fee Related JP5833597B2 (ja) 2006-10-03 2013-06-11 会合複合体及びそれを形成する方法
JP2015157142A Pending JP2015227370A (ja) 2006-10-03 2015-08-07 会合複合体及びそれを形成する方法
JP2016219673A Pending JP2017036330A (ja) 2006-10-03 2016-11-10 製剤用化合物
JP2018236147A Pending JP2019048887A (ja) 2006-10-03 2018-12-18 製剤用化合物
JP2020182378A Active JP7127098B2 (ja) 2006-10-03 2020-10-30 製剤用脂質ナノ粒子
JP2022129999A Pending JP2022164716A (ja) 2006-10-03 2022-08-17 製剤用脂質ナノ粒子
JP2024119315A Pending JP2024150681A (ja) 2006-10-03 2024-07-25 製剤用脂質ナノ粒子

Country Status (12)

Country Link
US (6) US8034376B2 (enExample)
EP (3) EP2695608B1 (enExample)
JP (8) JP5933163B2 (enExample)
KR (1) KR101129509B1 (enExample)
CN (3) CN110066224A (enExample)
AU (1) AU2007303205A1 (enExample)
CA (4) CA2848238C (enExample)
DK (1) DK2068886T3 (enExample)
ES (2) ES2430206T3 (enExample)
IN (1) IN2015DN00255A (enExample)
MX (4) MX363224B (enExample)
WO (1) WO2008042973A2 (enExample)

Families Citing this family (378)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20020142304A1 (en) * 2001-03-09 2002-10-03 Anderson Daniel G. Uses and methods of making microarrays of polymeric biomaterials
US9228186B2 (en) 2002-11-14 2016-01-05 Thermo Fisher Scientific Inc. Methods and compositions for selecting siRNA of improved functionality
EP1735009A4 (en) 2004-03-12 2011-03-30 Alnylam Pharmaceuticals Inc RNAI AGENTS TARGETING THE VASCULAR ENDOTHELIUM GROWTH FACTOR (VEGF)
JP5777846B2 (ja) * 2005-06-15 2015-09-09 マサチューセッツ インスティテュート オブ テクノロジー アミン含有脂質およびその使用
CN101484588B (zh) 2006-05-11 2013-11-06 阿尔尼拉姆医药品有限公司 抑制pcsk9基因表达的组合物和方法
US8598333B2 (en) * 2006-05-26 2013-12-03 Alnylam Pharmaceuticals, Inc. SiRNA silencing of genes expressed in cancer
ES2430206T3 (es) * 2006-10-03 2013-11-19 Tekmira Pharmaceuticals Corporation Formulación que contiene lípidos
EP2494993B1 (en) 2007-05-04 2018-08-01 Marina Biotech, Inc. Amino acid lipids and uses thereof
WO2009076400A2 (en) 2007-12-10 2009-06-18 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of factor vii gene
CA2711240C (en) * 2008-01-02 2021-10-26 Alnylam Pharmaceuticals, Inc. Liver screening method
EP2245039A4 (en) * 2008-01-31 2012-06-06 Alnylam Pharmaceuticals Inc OPTIMIZED METHODS FOR EXPORING DSRNA TO TARGET THE PCSK9 GEN
NZ588280A (en) 2008-03-05 2012-11-30 Alnylam Pharmaceuticals Inc Compositions and methods for inhibiting expression of eg5 and vegf genes
EP2690175B1 (en) 2008-09-02 2016-12-28 Alnylam Pharmaceuticals, Inc. Compositions and methods for combined inhibition of mutant EGFR gene and IL-6 expression
EP3584320A1 (en) 2008-09-25 2019-12-25 Alnylam Pharmaceuticals, Inc. Lipid formulated compositions and methods for inhibiting expression of serum amyloid a gene
CA2740000C (en) * 2008-10-09 2017-12-12 Tekmira Pharmaceuticals Corporation Improved amino lipids and methods for the delivery of nucleic acids
US8603532B2 (en) 2008-10-20 2013-12-10 Massachusetts Institute Of Technology Nanostructures for drug delivery
AU2009307677C1 (en) 2008-10-20 2022-09-22 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of transthyretin
EP2358398A2 (en) 2008-10-24 2011-08-24 Isis Pharmaceuticals, Inc. Oligomeric compounds and methods
CA3006395C (en) 2008-11-07 2022-05-31 Massachusetts Institute Of Technology Aminoalcohol lipidoids and uses thereof
EP2184054A1 (en) * 2008-11-08 2010-05-12 Lipoxen Technologies Limited Small Interfering RNA Delivery
PL4241767T3 (pl) 2008-11-10 2025-03-03 Arbutus Biopharma Corporation Nowe lipidy i kompozycje do dostarczania środków terapeutycznych
US20120016006A1 (en) * 2008-11-17 2012-01-19 Alnylam Pharmaceuticals ,Inc. a corporation Compositions And Methods For Increasing Cellular Uptake Of RNAi Via SID-1
CA2746514C (en) 2008-12-10 2018-11-27 Alnylam Pharmaceuticals, Inc. Gnaq targeted dsrna compositions and methods for inhibiting expression
EP2391343B1 (en) 2009-01-29 2017-03-01 Arbutus Biopharma Corporation Improved lipid formulation for the delivery of nucleic acids
WO2010099341A1 (en) 2009-02-26 2010-09-02 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of mig-12 gene
JP6032724B2 (ja) 2009-03-12 2016-11-30 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Ａｌｎｙｌａｍ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ， Ｉｎｃ． 脂質製剤組成物およびＥｇ５遺伝子とＶＥＧＦ遺伝子の発現を阻害する方法
PT2414322T (pt) * 2009-03-20 2022-12-07 Egen Inc Derivados de poliamina
WO2010120385A1 (en) * 2009-04-18 2010-10-21 Massachusetts Institute Of Technology pH SENSITIVE BIODEGRADABLE POLYMERIC PARTICLES FOR DRUG DELIVERY
AU2017200272A1 (en) * 2009-05-05 2017-02-02 Arbutus Biopharma Corporation Lipid compositions
KR20180094137A (ko) 2009-05-05 2018-08-22 알닐람 파마슈티칼스 인코포레이티드 지질 조성물
AU2010245933B2 (en) 2009-05-05 2016-06-16 Arbutus Biopharma Corporation Methods of delivering oligonucleotides to immune cells
CA2763016C (en) 2009-05-20 2018-04-17 Eth Zurich Targeting micrornas for metabolic disorders
EP2438168B1 (en) 2009-06-01 2020-02-12 Halo-Bio Rnai Therapeutics, Inc. Polynucleotides for multivalent rna interference, compositions and methods of use thereof
PL3431076T3 (pl) 2009-06-10 2022-01-31 Arbutus Biopharma Corporation Ulepszona formulacja lipidowa
WO2010147992A1 (en) 2009-06-15 2010-12-23 Alnylam Pharmaceuticals, Inc. Methods for increasing efficacy of lipid formulated sirna
KR20120050429A (ko) 2009-06-15 2012-05-18 알닐람 파마슈티칼스 인코포레이티드 Ｐｃｓｋ9 유전자를 표적으로 하는 지질 제형된 ｄｓｒｎａ
WO2011017548A1 (en) * 2009-08-05 2011-02-10 Alnylam Pharmaceuticals, Inc. Lipid formulated compositions and methods for inhibiting expression of eg5 and vegf genes
AP2015008874A0 (en) 2009-08-14 2015-11-30 Alnylam Pharmaceuticals Inc Lipid formulated compositions and methods for inhibiting expression of a gene from the ebola virus
CN104758254B (zh) 2009-08-31 2018-10-30 那野伽利阿株式会社 颗粒组合物和含有其的医药组合物
US9187746B2 (en) 2009-09-22 2015-11-17 Alnylam Pharmaceuticals, Inc. Dual targeting siRNA agents
WO2011056883A1 (en) 2009-11-03 2011-05-12 Alnylam Pharmaceuticals, Inc. Lipid formulated compositions and methods for inhibiting expression of transthyretin (ttr)
AU2010326132B9 (en) 2009-12-01 2014-10-02 Translate Bio, Inc. Delivery of mRNA for the augmentation of proteins and enzymes in human genetic diseases
CA2784568A1 (en) 2009-12-18 2011-06-23 Martin A. Maier Lipid particles for delivery of nucleic acids
KR101762466B1 (ko) * 2009-12-23 2017-07-27 노파르티스 아게 지질, 지질 조성물 및 이의 사용 방법
EP2525780B1 (en) * 2010-01-19 2022-09-07 Northwestern University Synthetic nanostructures including nucleic acids and/or other entities
WO2011090965A1 (en) * 2010-01-22 2011-07-28 Merck Sharp & Dohme Corp. Novel cationic lipids for oligonucleotide delivery
AU2011235276B2 (en) 2010-03-29 2015-09-03 Alnylam Pharmaceuticals, Inc. SiRNA therapy for transthyretin (TTR) related ocular amyloidosis
CA2792561C (en) 2010-04-06 2021-10-26 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of cd274/pd-l1 gene
WO2011133871A2 (en) 2010-04-22 2011-10-27 Alnylam Pharmaceuticals, Inc. 5'-end derivatives
EP3091027B1 (en) 2010-04-28 2018-01-17 Ionis Pharmaceuticals, Inc. 5' modified nucleosides and oligomeric compounds prepared therefrom
CN103154014B (zh) 2010-04-28 2015-03-25 Isis制药公司 修饰核苷、其类似物以及由它们制备的寡聚化合物
EP2606057B1 (en) 2010-04-28 2016-06-15 Ionis Pharmaceuticals, Inc. Modified 5' diphosphate nucleosides and oligomeric compounds prepared therefrom
WO2011141704A1 (en) 2010-05-12 2011-11-17 Protiva Biotherapeutics, Inc Novel cyclic cationic lipids and methods of use
WO2011141705A1 (en) 2010-05-12 2011-11-17 Protiva Biotherapeutics, Inc. Novel cationic lipids and methods of use thereof
CA2801066C (en) 2010-06-02 2021-02-09 Alnylam Pharmaceuticals, Inc. Compositions and methods directed to treating liver fibrosis
NZ605079A (en) 2010-06-03 2015-08-28 Alnylam Pharmaceuticals Inc Biodegradable lipids for the delivery of active agents
US9518259B2 (en) 2010-06-15 2016-12-13 Ionis Pharmaceuticals, Inc. Compounds and methods for modulating interaction between proteins and target nucleic acids
WO2012016184A2 (en) 2010-07-30 2012-02-02 Alnylam Pharmaceuticals, Inc. Methods and compositions for delivery of active agents
US20130202652A1 (en) * 2010-07-30 2013-08-08 Alnylam Pharmaceuticals, Inc. Methods and compositions for delivery of active agents
WO2012017208A1 (en) 2010-08-04 2012-02-09 Cizzle Biotechnology Limited Methods and compounds for the diagnosis and treatment of
CA2807552A1 (en) 2010-08-06 2012-02-09 Moderna Therapeutics, Inc. Engineered nucleic acids and methods of use thereof
WO2012027675A2 (en) 2010-08-26 2012-03-01 Massachusetts Institute Of Technology Poly(beta-amino alcohols), their preparation, and uses thereof
US9549901B2 (en) 2010-09-03 2017-01-24 The Brigham And Women's Hospital, Inc. Lipid-polymer hybrid particles
PL3590949T3 (pl) 2010-10-01 2022-08-29 Modernatx, Inc. Kwasy rybonukleinowe zawierające n1-metylo-pseudouracyle i ich zastosowania
CN110123830A (zh) 2010-11-09 2019-08-16 阿尔尼拉姆医药品有限公司 用于抑制Eg5和VEGF基因的表达的脂质配制的组合物和方法
US20120136073A1 (en) * 2010-11-15 2012-05-31 Life Technologies Corporation Amine-Containing Transfection Reagents and methods for making and using same
US9127275B2 (en) 2010-12-10 2015-09-08 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of klf-1 and bcl11a genes
US9193973B2 (en) 2010-12-10 2015-11-24 Alynylam Pharmaceuticals, Inc. Compositions and methods for increasing erythropoietin (EPO) production
KR101308591B1 (ko) 2010-12-30 2013-09-13 주식회사 삼양바이오팜 양이온성 지질을 포함하는 음이온성 약물 전달체 및 그 제조방법
DK2663548T3 (en) * 2011-01-11 2017-07-24 Alnylam Pharmaceuticals Inc PEGYLED LIPIDS AND THEIR USE FOR PHARMACEUTICAL SUPPLY
PL2691443T3 (pl) 2011-03-28 2021-08-30 Massachusetts Institute Of Technology Sprzężone lipomery i ich zastosowania
CA3217805A1 (en) 2011-03-29 2012-10-04 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of tmprss6 gene
DE12722942T1 (de) 2011-03-31 2021-09-30 Modernatx, Inc. Freisetzung und formulierung von manipulierten nukleinsäuren
WO2012145374A1 (en) 2011-04-19 2012-10-26 Regulus Therapeutics Inc. TARGETING miR-378 FAMILY MEMBERS FOR THE TREATMENT OF METABOLIC DISORDERS
US20120270928A1 (en) 2011-04-25 2012-10-25 Regulus Therapeutics Inc. Microrna compounds and methods for modulating mir-21 activity
EP4043025A1 (en) 2011-06-08 2022-08-17 Translate Bio, Inc. Lipid nanoparticle compositions and methods for mrna delivery
WO2012177906A1 (en) 2011-06-21 2012-12-27 Alnylam Pharmaceuticals, Inc. Assays and methods for determining activity of a therapeutic agent in a subject
EP3693464A3 (en) 2011-06-21 2020-12-09 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibition of expression of apolipoprotein c-iii (apoc3) genes
WO2012177949A2 (en) 2011-06-21 2012-12-27 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibition of expression of protein c (proc) genes
KR102232287B1 (ko) 2011-06-21 2021-03-29 알닐람 파마슈티칼스 인코포레이티드 안지오포이에틴-유사 3(ANGPTL3) iRNA 조성물 및 그 사용 방법
EP4134433A1 (en) 2011-06-23 2023-02-15 Alnylam Pharmaceuticals, Inc. Serpina1 sirnas: compositions of matter and methods of treatment
US9464124B2 (en) 2011-09-12 2016-10-11 Moderna Therapeutics, Inc. Engineered nucleic acids and methods of use thereof
EP2760477B1 (en) 2011-09-27 2018-08-08 Alnylam Pharmaceuticals, Inc. Di-aliphatic substituted pegylated lipids
RS62993B1 (sr) 2011-10-03 2022-03-31 Modernatx Inc Modifikovani nukleozidi, nukleotidi, i nukleinske kiseline, i njihove upotrebe
PE20142362A1 (es) 2011-11-18 2015-01-30 Alnylam Pharmaceuticals Inc Agentes de iarn, composiciones y metodos de uso de los mismos para tratar enfermedades asociadas con transtiretina (ttr)
US20140308304A1 (en) 2011-12-07 2014-10-16 Alnylam Pharmaceuticals, Inc. Lipids for the delivery of active agents
US9463247B2 (en) 2011-12-07 2016-10-11 Alnylam Pharmaceuticals, Inc. Branched alkyl and cycloalkyl terminated biodegradable lipids for the delivery of active agents
EP2788006B1 (en) 2011-12-07 2026-01-07 Alnylam Pharmaceuticals, Inc. Biodegradable lipids for the delivery of active agents
PT2791160T (pt) 2011-12-16 2022-07-04 Modernatx Inc Composições arnm modificado
US9035039B2 (en) 2011-12-22 2015-05-19 Protiva Biotherapeutics, Inc. Compositions and methods for silencing SMAD4
AU2013222179B2 (en) 2012-02-24 2017-08-24 Arbutus Biopharma Corporat ion Trialkyl cationic lipids and methods of use thereof
US10501512B2 (en) 2012-04-02 2019-12-10 Modernatx, Inc. Modified polynucleotides
AU2013243955B2 (en) 2012-04-02 2018-02-22 Modernatx, Inc. Modified polynucleotides for the production of oncology-related proteins and peptides
US9283287B2 (en) 2012-04-02 2016-03-15 Moderna Therapeutics, Inc. Modified polynucleotides for the production of nuclear proteins
US9572897B2 (en) 2012-04-02 2017-02-21 Modernatx, Inc. Modified polynucleotides for the production of cytoplasmic and cytoskeletal proteins
WO2013154799A1 (en) 2012-04-09 2013-10-17 Isis Pharmaceuticals, Inc. Tricyclic nucleosides and oligomeric compounds prepared therefrom
US9133461B2 (en) 2012-04-10 2015-09-15 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of the ALAS1 gene
AU2013249548A1 (en) 2012-04-19 2014-11-06 Sirna Therapeutics, Inc. Novel diester and triester based low molecular weight, biodegradable cationic lipids for oligonucleotide delivery
CN104254607B (zh) 2012-04-25 2020-10-27 赛诺菲 用于调节mir-21活性的微小rna化合物及方法
US9127274B2 (en) 2012-04-26 2015-09-08 Alnylam Pharmaceuticals, Inc. Serpinc1 iRNA compositions and methods of use thereof
US9255154B2 (en) 2012-05-08 2016-02-09 Alderbio Holdings, Llc Anti-PCSK9 antibodies and use thereof
WO2013172358A1 (ja) * 2012-05-14 2013-11-21 公立大学法人大阪府立大学 機能性化合物及びその化合物を含有する分子集合体、並びにそれらを含有する組成物及びキット並びにそれらの使用
EP3536787A1 (en) 2012-06-08 2019-09-11 Translate Bio, Inc. Nuclease resistant polynucleotides and uses thereof
AU2013274101B2 (en) 2012-06-15 2017-09-07 The Brigham And Women's Hospital, Inc. Compositions for treating cancer and methods for making the same
WO2014018375A1 (en) 2012-07-23 2014-01-30 Xenon Pharmaceuticals Inc. Cyp8b1 and uses thereof in therapeutic and diagnostic methods
CA2884870C (en) 2012-08-13 2022-03-29 Massachusetts Institute Of Technology Amine-containing lipidoids and uses thereof
UA116639C2 (uk) 2012-10-09 2018-04-25 Рег'Юлес Терап'Ютікс Інк. Способи лікування синдрому альпорта
CA2892529C (en) 2012-11-26 2023-04-25 Moderna Therapeutics, Inc. Terminally modified rna
MX367076B (es) 2012-12-05 2019-08-05 Alnylam Pharmaceuticals Inc Composiciones de arni de proteina convertasa subtilisina kexina 9 (pcsk9) y metodos de uso de las mismas.
WO2014089486A1 (en) * 2012-12-07 2014-06-12 Shire Human Genetic Therapies, Inc. Lipidic nanoparticles for mrna delivering
WO2014093537A1 (en) 2012-12-11 2014-06-19 Isis Pharmaceuticals, Inc. Competitive modulation of micrornas
US9765022B2 (en) 2013-02-28 2017-09-19 Tufts University Disulfide compounds for delivery of pharmaceutical agents
AU2014236250B2 (en) 2013-03-14 2019-01-03 Dicerna Pharmaceuticals, Inc. Process for formulating an anionic agent
TR201901310T4 (tr) 2013-03-14 2019-02-21 Translate Bio Inc Mesajcı RNA'nın saflaştırılması yöntemleri.
SG10201700239UA (en) 2013-03-14 2017-02-27 Alnylam Pharmaceuticals Inc COMPLEMENT COMPONENT C5 iRNA COMPOSITIONS AND METHODS OF USE THEREOF
LT2968586T (lt) 2013-03-14 2018-11-26 Translate Bio, Inc. Cft mrnr kompozicijos bei su jomis susiję būdai ir panaudojimai
EP2971010B1 (en) 2013-03-14 2020-06-10 ModernaTX, Inc. Formulation and delivery of modified nucleoside, nucleotide, and nucleic acid compositions
US8980864B2 (en) 2013-03-15 2015-03-17 Moderna Therapeutics, Inc. Compositions and methods of altering cholesterol levels
TWI680767B (zh) 2013-05-01 2020-01-01 美商雷格勒斯治療公司 用於增強的細胞攝取之化合物及方法
US9315472B2 (en) 2013-05-01 2016-04-19 Massachusetts Institute Of Technology 1,3,5-triazinane-2,4,6-trione derivatives and uses thereof
NZ630890A (en) 2013-05-01 2017-09-29 Regulus Therapeutics Inc Microrna compounds and methods for modulating mir-122
WO2014182661A2 (en) 2013-05-06 2014-11-13 Alnylam Pharmaceuticals, Inc Dosages and methods for delivering lipid formulated nucleic acid molecules
US20160082126A1 (en) 2013-05-13 2016-03-24 Tufts University Nanocomplexes for delivery of saporin
UY40064A (es) 2013-05-22 2023-02-28 Alnylam Pharmaceuticals Inc COMPOSICIONES DE ARNi DE SERPINA1 Y SUS MÉTODOS DE USO
SG10202103166XA (en) 2013-05-22 2021-04-29 Alnylam Pharmaceuticals Inc Tmprss6 irna compositions and methods of use thereof
WO2015011633A1 (en) 2013-07-23 2015-01-29 Protiva Biotherapeutics, Inc. Compositions and methods for delivering messenger rna
EP3052106A4 (en) 2013-09-30 2017-07-19 ModernaTX, Inc. Polynucleotides encoding immune modulating polypeptides
EP3052626A1 (en) 2013-10-02 2016-08-10 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of the lect2 gene
EP3052521A1 (en) 2013-10-03 2016-08-10 Moderna Therapeutics, Inc. Polynucleotides encoding low density lipoprotein receptor
UA124961C2 (uk) 2013-10-04 2021-12-22 Елнілем Фармасьютикалз, Інк. ДВОНИТКОВА РИБОНУКЛЕЇНОВА КИСЛОТА (dsRNA) ДЛЯ ІНГІБУВАННЯ ЕКСПРЕСІЇ ALAS1
ES2865699T3 (es) 2013-10-22 2021-10-15 Translate Bio Inc Formulaciones de lípidos para la administración de ARN mensajero
JP6608815B2 (ja) 2013-10-22 2019-11-20 トランスレイト バイオ， インコーポレイテッド アルギニノコハク酸合成酵素欠損症のｍＲＮＡ治療
JP6506749B2 (ja) 2013-10-22 2019-04-24 シャイアー ヒューマン ジェネティック セラピーズ インコーポレイテッド フェニルケトン尿症のためのｍＲＮＡ療法
US9994846B2 (en) 2013-10-25 2018-06-12 Regulus Therapeutics Inc. MicroRNA compounds and methods for modulating miR-21 activity
EP3985118A1 (en) 2013-11-22 2022-04-20 MiNA Therapeutics Limited C/ebp alpha short activating rna compositions and methods of use
KR20240170595A (ko) 2013-12-12 2024-12-03 알닐람 파마슈티칼스 인코포레이티드 보체 성분 ｉＲＮＡ 조성물 및 이의 이용 방법
KR20230152154A (ko) 2014-02-11 2023-11-02 알닐람 파마슈티칼스 인코포레이티드 케토헥소키나제(ＫＨＫ) ｉＲＮＡ 조성물 및 그의 사용 방법
KR20160132496A (ko) 2014-04-03 2016-11-18 인빅터스 온콜로지 피비티. 엘티디. 초분자 조합 치료제
CA3177878A1 (en) 2014-04-23 2015-10-29 Modernatx, Inc. Nucleic acid vaccines
CN106164248B (zh) 2014-04-25 2019-10-15 川斯勒佰尔公司 信使rna的纯化方法
EP3146049B1 (en) 2014-05-22 2020-02-26 Alnylam Pharmaceuticals, Inc. Angiotensinogen (agt) irna compositions and methods of use thereof
MA48050A (fr) 2014-05-30 2020-02-12 Translate Bio Inc Lipides biodégradables pour l'administration d'acides nucléiques
CN116059170A (zh) 2014-06-24 2023-05-05 川斯勒佰尔公司 用于递送核酸的立体化学富集组合物
EP3766916B1 (en) 2014-06-25 2022-09-28 Acuitas Therapeutics Inc. Novel lipids and lipid nanoparticle formulations for delivery of nucleic acids
US9840479B2 (en) 2014-07-02 2017-12-12 Massachusetts Institute Of Technology Polyamine-fatty acid derived lipidoids and uses thereof
AU2015301057A1 (en) 2014-08-07 2017-01-19 Regulus Therapeutics Inc. Targeting micrornas for metabolic disorders
US10060921B2 (en) 2014-08-29 2018-08-28 Alnylam Pharmaceuticals, Inc. Methods of treating transthyretin (TTR) mediated amyloidosis
EP3191591A1 (en) 2014-09-12 2017-07-19 Alnylam Pharmaceuticals, Inc. Polynucleotide agents targeting complement component c5 and methods of use thereof
EP3197904B1 (en) 2014-09-25 2022-08-10 Cold Spring Harbor Laboratory Treatment of rett syndrome
EP3201338B1 (en) 2014-10-02 2021-11-03 Arbutus Biopharma Corporation Compositions and methods for silencing hepatitis b virus gene expression
JOP20200115A1 (ar) 2014-10-10 2017-06-16 Alnylam Pharmaceuticals Inc تركيبات وطرق لتثبيط التعبير الجيني عن hao1 (حمض أوكسيداز هيدروكسيلي 1 (أوكسيداز جليكولات))
WO2016061487A1 (en) 2014-10-17 2016-04-21 Alnylam Pharmaceuticals, Inc. Polynucleotide agents targeting aminolevulinic acid synthase-1 (alas1) and uses thereof
EP3904519A1 (en) 2014-10-30 2021-11-03 Genzyme Corporation Polynucleotide agents targeting serpinc1 (at3) and methods of use thereof
JOP20200092A1 (ar) 2014-11-10 2017-06-16 Alnylam Pharmaceuticals Inc تركيبات iRNA لفيروس الكبد B (HBV) وطرق لاستخدامها
JP2017535552A (ja) 2014-11-17 2017-11-30 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Ａｌｎｙｌａｍ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ， Ｉｎｃ． アポリポタンパク質Ｃ３（ＡＰＯＣ３）ｉＲＮＡ組成物およびその使用方法
US20170369872A1 (en) 2014-12-18 2017-12-28 Alnylam Pharmaceuticals, Inc. Reversir tm compounds
EP3256587A2 (en) 2015-02-13 2017-12-20 Alnylam Pharmaceuticals, Inc. Patatin-like phospholipase domain containing 3 (pnpla3) irna compositions and methods of use thereof
US20180245074A1 (en) 2015-06-04 2018-08-30 Protiva Biotherapeutics, Inc. Treating hepatitis b virus infection using crispr
WO2016201301A1 (en) 2015-06-12 2016-12-15 Alnylam Pharmaceuticals, Inc. Complement component c5 irna compositions and methods of use thereof
WO2016205323A1 (en) 2015-06-18 2016-12-22 Alnylam Pharmaceuticals, Inc. Polynucleotde agents targeting hydroxyacid oxidase (glycolate oxidase, hao1) and methods of use thereof
WO2016209862A1 (en) 2015-06-23 2016-12-29 Alnylam Pharmaceuticals, Inc. Glucokinase (gck) irna compositions and methods of use thereof
HRP20240865T1 (hr) 2015-06-29 2024-10-11 Acuitas Therapeutics Inc. Lipidi i formulacije lipidnih nanočestica za isporuku nukleinskih kiselina
WO2017011286A1 (en) 2015-07-10 2017-01-19 Alnylam Pharmaceuticals, Inc. Insulin-like growth factor binding protein, acid labile subunit (igfals) and insulin-like growth factor 1 (igf-1) irna compositions and methods of use thereof
EP3329003A2 (en) 2015-07-29 2018-06-06 Arbutus Biopharma Corporation Compositions and methods for silencing hepatitis b virus gene expression
KR20240074895A (ko) 2015-07-31 2024-05-28 알닐람 파마슈티칼스 인코포레이티드 트랜스티레틴(TTR) iRNA 조성물 및 TTR-관련 질병을 치료하거나, 예방하기 위한 그의 사용 방법
US11564893B2 (en) 2015-08-17 2023-01-31 Modernatx, Inc. Methods for preparing particles and related compositions
CN108366966A (zh) 2015-08-24 2018-08-03 光环生物干扰疗法公司 用于调节基因表达的多核苷酸纳米颗粒及其用途
MA44836A (fr) * 2015-08-26 2018-07-04 Univ Texas Procédés de traitement du syndrome polykystique des reins
CA2996873A1 (en) 2015-09-02 2017-03-09 Alnylam Pharmaceuticals, Inc. Programmed cell death 1 ligand 1 (pd-l1) irna compositions and methods of use thereof
AU2016324310B2 (en) 2015-09-17 2021-04-08 Modernatx, Inc. Compounds and compositions for intracellular delivery of therapeutic agents
CN113636947A (zh) 2015-10-28 2021-11-12 爱康泰生治疗公司 用于递送核酸的新型脂质和脂质纳米颗粒制剂
DK3386484T3 (da) 2015-12-10 2022-07-04 Modernatx Inc Sammensætninger og fremgangsmåder til afgivelse af terapeutiske midler
EP4036079B1 (en) 2015-12-22 2025-11-19 ModernaTX, Inc. Compounds and compositions for intracellular delivery of agents
KR101949453B1 (ko) 2015-12-30 2019-02-18 주식회사 삼양바이오팜 양이온성 지질의 선택적 합성 방법
WO2017147335A1 (en) * 2016-02-24 2017-08-31 Rutgers, The State University Of New Jersey Bacterial efflux pump inhibitors
IL302398B2 (en) 2016-03-03 2025-06-01 Univ Massachusetts Closed-ended linear duplex dna for non-viral gene transfer
MA45295A (fr) 2016-04-19 2019-02-27 Alnylam Pharmaceuticals Inc Composition d'arni de protéine de liaison de lipoprotéines haute densité (hdlbp/vigiline) et procédés pour les utiliser
CN115837014A (zh) 2016-05-18 2023-03-24 摩登纳特斯有限公司 编码松弛素的多核苷酸
WO2017214518A1 (en) 2016-06-10 2017-12-14 Alnylam Pharmaceuticals, Inc. COMPLETMENT COMPONENT C5 iRNA COMPOSTIONS AND METHODS OF USE THEREOF FOR TREATING PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH)
CN109563511A (zh) 2016-06-30 2019-04-02 阿布特斯生物制药公司 用于递送信使rna的组合物和方法
EP4485466A3 (en) 2016-08-17 2025-04-02 The Broad Institute Inc. Novel crispr enzymes and systems
US20200283743A1 (en) 2016-08-17 2020-09-10 The Broad Institute, Inc. Novel crispr enzymes and systems
WO2018039131A1 (en) 2016-08-22 2018-03-01 Protiva Biotherapeutics, Inc. Anti-pd-1 antibodies, or fragments thereof, for treating hepatitis b
US12491261B2 (en) 2016-10-26 2025-12-09 Acuitas Therapeutics, Inc. Lipid nanoparticle formulations
EP3538067A1 (en) 2016-11-08 2019-09-18 Modernatx, Inc. Stabilized formulations of lipid nanoparticles
TW202313978A (zh) 2016-11-23 2023-04-01 美商阿尼拉製藥公司 絲胺酸蛋白酶抑制因子Ａ１ ｉＲＮＡ組成物及其使用方法
CA3043768A1 (en) 2016-11-29 2018-06-07 PureTech Health LLC Exosomes for delivery of therapeutic agents
EA201991360A1 (ru) 2016-12-05 2019-11-29 Модифицированные олигонуклеотиды для лечения поликистозной болезни почек
US20190345494A1 (en) 2016-12-05 2019-11-14 Regulus Therapeutics Inc. Methods for treatment of polycystic kidney disease
SG10201913552UA (en) 2016-12-16 2020-03-30 Alnylam Pharmaceuticals Inc Methods for treating or preventing ttr-associated diseases using transthyretin (ttr) irna compositions
AU2018224326B2 (en) 2017-02-27 2024-01-04 Translate Bio, Inc. Novel codon-optimized CFTR mRNA
WO2018165612A1 (en) 2017-03-10 2018-09-13 Rutgers, The State University Of New Jersey Bacterial efflux pump inhibitors
US11938114B2 (en) 2017-03-10 2024-03-26 Rutgers, The State University Of New Jersey Bacterial efflux pump inhibitors
IL269210B2 (en) 2017-03-10 2025-05-01 Univ Rutgers Indole derivatives as efflux pump inhibitors
SI3596041T1 (sl) 2017-03-15 2023-02-28 Modernatx, Inc. Spojina in sestavki za intracelično dostavljanje terapevtskih sredstev
JP7332478B2 (ja) 2017-03-15 2023-08-23 モデルナティエックス インコーポレイテッド 脂質ナノ粒子製剤
SI3596042T1 (sl) 2017-03-15 2022-04-29 Modernatx, Inc. Kristalne oblike amino lipidov
US11357856B2 (en) 2017-04-13 2022-06-14 Acuitas Therapeutics, Inc. Lipids for delivery of active agents
US12350368B2 (en) 2017-04-14 2025-07-08 The Broad Institute, Inc. Delivery of large payloads
AU2018254437B2 (en) 2017-04-18 2025-05-08 Alnylam Pharmaceuticals, Inc. Methods for the treatment of subjects having a hepatitis B virus (HBV) infection
IL301115A (en) 2017-04-28 2023-05-01 Acuitas Therapeutics Inc Novel carbonyl lipids and lipid nanoparticle formulations for delivery of nucleic acids
CA3063531A1 (en) 2017-05-16 2018-11-22 Translate Bio, Inc. Treatment of cystic fibrosis by delivery of codon-optimized mrna encoding cftr
JP7281822B2 (ja) 2017-05-26 2023-05-26 ラトガーズ、ザ ステイト ユニバーシティ オブ ニュージャージー 細菌の排出ポンプ阻害剤
EP3638678B1 (en) 2017-06-14 2025-12-03 ModernaTX, Inc. Compounds and compositions for intracellular delivery of agents
WO2018232357A1 (en) 2017-06-15 2018-12-20 Modernatx, Inc. Rna formulations
US11458121B2 (en) 2017-06-26 2022-10-04 Rutgers, The State University Of New Jersey Therapeutic compounds and methods to treat infection
US20210228738A1 (en) 2017-07-17 2021-07-29 INSERM (Institut National de la Santé et de la Recherche Médicale) Compositions and methods for increasing or enhancing transduction of gene therapy vectors and for removing or reducing immunoglobulins
CA3073020A1 (en) 2017-08-16 2019-02-21 Acuitas Therapeutics, Inc. Lipids for use in lipid nanoparticle formulations
US11524932B2 (en) 2017-08-17 2022-12-13 Acuitas Therapeutics, Inc. Lipids for use in lipid nanoparticle formulations
CA3073018A1 (en) 2017-08-17 2019-02-21 Acuitas Therapeutics, Inc. Lipids for use in lipid nanoparticle formulations
WO2019036028A1 (en) 2017-08-17 2019-02-21 Acuitas Therapeutics, Inc. LIPIDS FOR USE IN LIPID NANOPARTICULAR FORMULATIONS
MX2020002348A (es) 2017-08-31 2020-10-08 Modernatx Inc Métodos de elaboración de nanopartículas lipídicas.
EP4183882A1 (en) 2017-09-08 2023-05-24 MiNA Therapeutics Limited Stabilized hnf4a sarna compositions and methods of use
WO2019048645A1 (en) 2017-09-08 2019-03-14 Mina Therapeutics Limited STABILIZED COMPOSITIONS OF SMALL ACTIVATOR RNA (PARNA) FROM CEBPA AND METHODS OF USE
KR20250046363A (ko) 2017-09-19 2025-04-02 알닐람 파마슈티칼스 인코포레이티드 트랜스타이레틴(ttr) 매개 아밀로이드증을 치료하기 위한 조성물 및 방법
US12227742B2 (en) 2017-10-23 2025-02-18 The Broad Institute, Inc. Nucleic acid modifiers
WO2019089922A1 (en) 2017-11-01 2019-05-09 Alnylam Pharmaceuticals, Inc. Complement component c3 irna compositions and methods of use thereof
US12018080B2 (en) 2017-11-13 2024-06-25 The Broad Institute, Inc. Methods and compositions for treating cancer by targeting the CLEC2D-KLRB1 pathway
EP3714054A1 (en) 2017-11-20 2020-09-30 Alnylam Pharmaceuticals, Inc. Serum amyloid p component (apcs) irna compositions and methods of use thereof
WO2019109074A1 (en) 2017-12-01 2019-06-06 Shepherd Therapeutics, Inc. Mebendazole cancer therapies and methods of use
US10933061B2 (en) 2017-12-21 2021-03-02 Shepherd Therapeutics, Inc. Pyrvinium pamoate therapies and methods of use
US10968257B2 (en) 2018-04-03 2021-04-06 The Broad Institute, Inc. Target recognition motifs and uses thereof
WO2019197845A1 (en) 2018-04-12 2019-10-17 Mina Therapeutics Limited Sirt1-sarna compositions and methods of use
US20210317461A1 (en) 2018-08-09 2021-10-14 Verseau Therapeutics, Inc. Oligonucleotide compositions for targeting ccr2 and csf1r and uses thereof
AR115960A1 (es) 2018-08-16 2021-03-17 Alnylam Pharmaceuticals Inc Composiciones y métodos para inhibir la expresión del gen lect2
CA3108544A1 (en) 2018-08-24 2020-02-27 Translate Bio, Inc. Methods for purification of messenger rna
EP3853364A1 (en) 2018-09-18 2021-07-28 Alnylam Pharmaceuticals, Inc. Ketohexokinase (khk) irna compositions and methods of use thereof
WO2020061284A1 (en) 2018-09-19 2020-03-26 Modernatx, Inc. Peg lipids and uses thereof
EP4509118A3 (en) 2018-09-19 2025-05-14 ModernaTX, Inc. High-purity peg lipids and uses thereof
JP7410135B2 (ja) 2018-09-19 2024-01-09 モデルナティエックス インコーポレイテッド 治療薬の細胞内送達のための化合物及び組成物
JP2022501367A (ja) 2018-09-20 2022-01-06 モデルナティエックス インコーポレイテッドModernaTX, Inc. 脂質ナノ粒子の調製及びその投与方法
IL322436A (en) 2018-09-21 2025-09-01 Acuitas Therapeutics Inc Systems and methods for producing lipid nanoparticles and liposomes
JP7387745B2 (ja) 2018-10-01 2023-11-28 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 活性物質の送達のための生分解性脂質
US10913951B2 (en) 2018-10-31 2021-02-09 University of Pittsburgh—of the Commonwealth System of Higher Education Silencing of HNF4A-P2 isoforms with siRNA to improve hepatocyte function in liver failure
WO2020097493A1 (en) 2018-11-09 2020-05-14 Arbutus Biopharma Corporation Negatively charged peg-lipid conjugates
TW202031268A (zh) 2018-11-13 2020-09-01 美商雷格勒斯治療公司 用於調節mir-10b 活性之微小rna 化合物及方法
AU2019384557B2 (en) 2018-11-21 2025-07-17 Translate Bio, Inc. Treatment of cystic fibrosis by delivery of nebulized mRNA encoding CFTR
CN109432504B (zh) * 2018-11-27 2021-11-16 中国人民解放军总医院第四医学中心 一种成骨基因干预功能材料及其制备方法
EP3894559A4 (en) 2018-12-03 2023-04-05 Triplet Therapeutics, Inc. METHODS OF TREATMENT OF TRINUCLEOTIDE REPEAT EXPANSION DISORDERS RELATED TO MLH3 ACTIVITY
US20220056455A1 (en) 2018-12-20 2022-02-24 Praxis Precision Medicines, Inc. Compositions and methods for the treatment of kcnt1 related disorders
WO2020128816A2 (en) 2018-12-20 2020-06-25 Pfizer Inc. Pharmaceutical compositions and methods comprising a combination of a benzoxazole transthyretin stabilizer and an additional therapeutic agent
CN113474328B (zh) 2019-01-11 2025-07-11 爱康泰生治疗公司 用于脂质纳米颗粒递送活性剂的脂质
US12215382B2 (en) 2019-03-01 2025-02-04 The General Hospital Corporation Liver protective MARC variants and uses thereof
WO2020186213A1 (en) 2019-03-14 2020-09-17 The Broad Institute, Inc. Novel nucleic acid modifiers
US20220177863A1 (en) 2019-03-18 2022-06-09 The Broad Institute, Inc. Type vii crispr proteins and systems
WO2020208361A1 (en) 2019-04-12 2020-10-15 Mina Therapeutics Limited Sirt1-sarna compositions and methods of use
US20220220469A1 (en) 2019-05-20 2022-07-14 The Broad Institute, Inc. Non-class i multi-component nucleic acid targeting systems
JP7692829B2 (ja) 2019-07-30 2025-06-16 塩野義製薬株式会社 Ｍｕｒｆ１を標的とする核酸医薬
EP4013870A1 (en) 2019-08-13 2022-06-22 Alnylam Pharmaceuticals, Inc. Small ribosomal protein subunit 25 (rps25) irna agent compositions and methods of use thereof
MX2022002689A (es) 2019-09-03 2022-04-07 Alnylam Pharmaceuticals Inc Composiciones y metodos para inhibir la expresion del gen de quimiotaxina derivada de celulas de leucocitos 2 (lect2).
WO2021046265A1 (en) 2019-09-06 2021-03-11 Generation Bio Co. Lipid nanoparticle compositions comprising closed-ended dna and cleavable lipids and methods of use thereof
AU2020350759A1 (en) 2019-09-19 2022-03-31 Modernatx, Inc. Branched tail lipid compounds and compositions for intracellular delivery of therapeutic agents
US11987791B2 (en) 2019-09-23 2024-05-21 Omega Therapeutics, Inc. Compositions and methods for modulating hepatocyte nuclear factor 4-alpha (HNF4α) gene expression
CN114391040A (zh) 2019-09-23 2022-04-22 欧米茄治疗公司 用于调节载脂蛋白b(apob)基因表达的组合物和方法
US12297426B2 (en) 2019-10-01 2025-05-13 The Broad Institute, Inc. DNA damage response signature guided rational design of CRISPR-based systems and therapies
WO2021067747A1 (en) 2019-10-04 2021-04-08 Alnylam Pharmaceuticals, Inc. Compositions and methods for silencing ugt1a1 gene expression
US12378560B2 (en) 2019-10-29 2025-08-05 Northwestern University Sequence multiplicity within spherical nucleic acids
AR120341A1 (es) 2019-11-01 2022-02-09 Alnylam Pharmaceuticals Inc COMPOSICIONES DE AGENTES DE ARNi CONTRA LA HUNTINGTINA (HTT) Y SUS MÉTODOS DE USO
EP4051796A1 (en) 2019-11-01 2022-09-07 Alnylam Pharmaceuticals, Inc. Compositions and methods for silencing dnajb1-prkaca fusion gene expression
WO2021102373A1 (en) 2019-11-22 2021-05-27 Alnylam Pharmaceuticals, Inc. Ataxin3 (atxn3) rnai agent compositions and methods of use thereof
AU2020402885A1 (en) 2019-12-13 2022-06-16 Alnylam Pharmaceuticals, Inc. Human chromosome 9 open reading frame 72 (C9orf72) iRNA agent compositions and methods of use thereof
WO2021154941A1 (en) 2020-01-31 2021-08-05 Alnylam Pharmaceuticals, Inc. Complement component c5 irna compositions for use in the treatment of amyotrophic lateral sclerosis (als)
US20230285297A1 (en) * 2020-01-31 2023-09-14 Modernatx, Inc. Methods of preparing lipid nanoparticles
US20230136787A1 (en) 2020-02-10 2023-05-04 Alnylam Pharmaceuticals, Inc. Compositions and methods for silencing vegf-a expression
EP4114947A1 (en) 2020-03-05 2023-01-11 Alnylam Pharmaceuticals, Inc. Complement component c3 irna compositions and methods of use thereof for treating or preventing complement component c3-associated diseases
AU2021234302A1 (en) 2020-03-11 2022-11-10 Omega Therapeutics, Inc. Compositions and methods for modulating forkhead box p3 (foxp3) gene expression
CN115667530A (zh) 2020-03-24 2023-01-31 世代生物公司 非病毒dna载体和其用于表达因子ix治疗剂的用途
EP4127186A1 (en) 2020-03-24 2023-02-08 Generation Bio Co. Non-viral dna vectors and uses thereof for expressing gaucher therapeutics
TW202204615A (zh) 2020-03-26 2022-02-01 美商阿尼拉製藥公司 冠狀病毒ｉＲＮＡ組成物及其使用方法
EP4127171A2 (en) 2020-03-30 2023-02-08 Alnylam Pharmaceuticals, Inc. Compositions and methods for silencing dnajc15 gene expression
KR20230008729A (ko) 2020-04-06 2023-01-16 알닐람 파마슈티칼스 인코포레이티드 Myoc 발현을 사일런싱하기 위한 조성물 및 방법
WO2021206917A1 (en) 2020-04-07 2021-10-14 Alnylam Pharmaceuticals, Inc. ANGIOTENSIN-CONVERTING ENZYME 2 (ACE2) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
EP4133079A1 (en) 2020-04-07 2023-02-15 Alnylam Pharmaceuticals, Inc. Compositions and methods for silencing scn9a expression
EP4133077A1 (en) 2020-04-07 2023-02-15 Alnylam Pharmaceuticals, Inc. Transmembrane serine protease 2 (tmprss2) irna compositions and methods of use thereof
BR112022021813A2 (pt) 2020-04-27 2023-01-17 Alnylam Pharmaceuticals Inc Composições de agente de apolipoproteína e (apoe) irna e métodos de uso das mesmas
WO2021237097A1 (en) 2020-05-21 2021-11-25 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting marc1 gene expression
US11408000B2 (en) 2020-06-03 2022-08-09 Triplet Therapeutics, Inc. Oligonucleotides for the treatment of nucleotide repeat expansion disorders associated with MSH3 activity
CN116075592A (zh) 2020-06-09 2023-05-05 阿尔尼拉姆医药品有限公司 用于沉默gpam(线粒体甘油-3-磷酸酰基转移酶1)表达的sirna组合物和方法
EP4162050A1 (en) 2020-06-09 2023-04-12 Alnylam Pharmaceuticals, Inc. Rnai compositions and methods of use thereof for delivery by inhalation
AU2021308681A1 (en) 2020-07-16 2023-03-09 Acuitas Therapeutics, Inc. Cationic lipids for use in lipid nanoparticles
WO2022023284A1 (en) 2020-07-27 2022-02-03 Anjarium Biosciences Ag Compositions of dna molecules, methods of making therefor, and methods of use thereof
WO2022066847A1 (en) 2020-09-24 2022-03-31 Alnylam Pharmaceuticals, Inc. Dipeptidyl peptidase 4 (dpp4) irna compositions and methods of use thereof
TW202229552A (zh) 2020-10-05 2022-08-01 美商艾拉倫製藥股份有限公司 Ｇ蛋白－偶合受體７５（ＧＰＲ７５）ｉＲＮＡ組成物及其使用方法
WO2022087041A1 (en) 2020-10-21 2022-04-28 Alnylam Pharmaceuticals, Inc. Methods and compositions for treating primary hyperoxaluria
WO2022087329A1 (en) 2020-10-23 2022-04-28 Alnylam Pharmaceuticals, Inc. Mucin 5b (muc5b) irna compositions and methods of use thereof
AU2021393417A1 (en) 2020-12-01 2023-06-29 Alnylam Pharmaceuticals, Inc. Methods and compositions for inhibition of hao1 (hydroxyacid oxidase 1 (glycolate oxidase)) gene expression
AU2022220704A1 (en) 2021-02-12 2022-08-18 Alnylam Pharmaceuticals, Inc. Superoxide dismutase 1 (sod1) irna compositions and methods of use thereof for treating or preventing superoxide dismutase 1- (sod1-) associated neurodegenerative diseases
US11524023B2 (en) 2021-02-19 2022-12-13 Modernatx, Inc. Lipid nanoparticle compositions and methods of formulating the same
WO2022182864A1 (en) 2021-02-25 2022-09-01 Alnylam Pharmaceuticals, Inc. Prion protein (prnp) irna compositions and methods and methods of use thereof
EP4305169A1 (en) 2021-03-12 2024-01-17 Alnylam Pharmaceuticals, Inc. Glycogen synthase kinase 3 alpha (gsk3a) irna compositions and methods of use thereof
WO2022200810A1 (en) 2021-03-26 2022-09-29 Mina Therapeutics Limited Tmem173 sarna compositions and methods of use
CN117203336A (zh) 2021-03-29 2023-12-08 阿尔尼拉姆医药品有限公司 亨廷顿蛋白(HTT)iRNA药剂组合物以及其使用方法
CA3214538A1 (en) 2021-04-20 2022-10-27 Joel DE BEER Compositions of dna molecules encoding amylo-alpha-1, 6-glucosidase, 4-alpha-glucanotransferase, methods of making thereof, and methods of use thereof
US20240226324A1 (en) 2021-04-27 2024-07-11 Generation Bio Co. Non-viral dna vectors expressing therapeutic antibodies and uses thereof
EP4329884A1 (en) 2021-04-27 2024-03-06 Generation Bio Co. Non-viral dna vectors expressing anti-coronavirus antibodies and uses thereof
WO2022232343A1 (en) 2021-04-29 2022-11-03 Alnylam Pharmaceuticals, Inc. Signal transducer and activator of transcription factor 6 (stat6) irna compositions and methods of use thereof
EP4341401A1 (en) 2021-05-18 2024-03-27 Alnylam Pharmaceuticals, Inc. Sodium-glucose cotransporter-2 (sglt2) irna compositions and methods of use thereof
JP7720925B2 (ja) * 2021-05-20 2025-08-08 北京理工大学 化合物、リポソーム及びその使用
JP2024521907A (ja) 2021-06-04 2024-06-04 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 ヒト９番染色体オープンリーディングフレーム７２（Ｃ９ＯＲＦ７２）ｉＲＮＡ剤組成物及びその使用方法
EP4367242A2 (en) 2021-07-07 2024-05-15 Omega Therapeutics, Inc. Compositions and methods for modulating secreted frizzled receptor protein 1 (sfrp1) gene expression
EP4381067A1 (en) 2021-08-03 2024-06-12 Alnylam Pharmaceuticals, Inc. Transthyretin (ttr) irna compositions and methods of use thereof
CA3230404A1 (en) 2021-08-31 2023-03-09 Alnylam Pharmaceuticals, Inc. Cell death-inducing dffa-like effector b (cideb) irna compositions and methods of use thereof
WO2023044343A1 (en) 2021-09-14 2023-03-23 Renagade Therapeutics Management Inc. Acyclic lipids and methods of use thereof
TW202328067A (zh) 2021-09-14 2023-07-16 美商雷納嘉德醫療管理公司 環狀脂質及其使用方法
EP4413132A1 (en) 2021-10-08 2024-08-14 Regulus Therapeutics Inc. Methods and compositions for treatment of polycystic kidney disease
EP4413133A2 (en) 2021-10-08 2024-08-14 Regulus Therapeutics Inc. Methods and compositions for avoiding off-target effects
JP2024541974A (ja) 2021-10-29 2024-11-13 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 ハンチンチン（ＨＴＴ）ｉＲＮＡ剤組成物およびその使用方法
TW202334080A (zh) 2021-11-08 2023-09-01 美商歐納醫療公司 用於遞送環狀聚核苷酸之脂質奈米粒子組合物
US20230159930A1 (en) 2021-11-19 2023-05-25 Sanegene Bio Usa Inc. Double stranded rna targeting angiopoietin-like 3 (angptl-3) and methods of use thereof
WO2023099884A1 (en) 2021-12-01 2023-06-08 Mina Therapeutics Limited Pax6 sarna compositions and methods of use
CN118647600A (zh) 2021-12-16 2024-09-13 爱康泰生治疗公司 用于脂质纳米颗粒制剂的脂质
CA3242118A1 (en) 2021-12-22 2023-06-29 Alfica Sehgal Modulation of gene transcription using antisense oligonucleotides targeting regulatory rnas
KR102852074B1 (ko) * 2021-12-23 2025-08-28 주식회사 삼양홀딩스 약물 전달용 지질 및 이를 포함하는 나노입자, 및 이 나노입자를 포함하는 약물 전달용 조성물
KR20230096311A (ko) * 2021-12-23 2023-06-30 주식회사 삼양홀딩스 약물의 폐 전달용 나노입자 조성물
WO2023122752A1 (en) 2021-12-23 2023-06-29 Renagade Therapeutics Management Inc. Constrained lipids and methods of use thereof
US20250381294A1 (en) 2022-01-14 2025-12-18 Anjarium Biosciences Ag Compositions of DNA Molecules Encoding Factor VIII, Methods of Making Thereof, and Methods of Use Thereof
EP4469575A2 (en) 2022-01-24 2024-12-04 Alnylam Pharmaceuticals, Inc. Heparin sulfate biosynthesis pathway enzyme irna agent compositions and methods of use thereof
WO2023143601A1 (zh) * 2022-01-30 2023-08-03 康希诺生物股份公司 一种用于核酸递送的新型可电离脂质及其lnp组合物和疫苗
CN116554042A (zh) * 2022-01-30 2023-08-08 康希诺生物股份公司 一种用于核酸递送的新型可电离脂质及其lnp组合物和疫苗
WO2023170435A1 (en) 2022-03-07 2023-09-14 Mina Therapeutics Limited Il10 sarna compositions and methods of use
JP2025509607A (ja) 2022-03-14 2025-04-11 ジェネレーション バイオ カンパニー 異種プライムブーストワクチン組成物及び使用の方法
AU2023250649A1 (en) 2022-04-04 2024-11-14 The Regents Of The University Of California Genetic complementation compositions and methods
CA3255619A1 (en) 2022-04-07 2023-10-12 Renagade Therapeutics Management Inc. CYCLIC LIPIDS AND LIPID NANOPARTICLES (LPNs) FOR THE SUPPLY OF NUCLEIC ACIDS OR PEPTIDES INTENDED FOR USE IN VACCINATION AGAINST INFECTIOUS AGENTS
WO2023220561A1 (en) 2022-05-09 2023-11-16 Sanegene Bio Usa Inc. Double stranded rna targeting 17-beta hydroxysteroiddehydrogenase 13 (hsd17b13) and methods of use thereof
CN120092091A (zh) 2022-05-30 2025-06-03 上海环码生物医药有限公司 合成的环状rna组合物及其使用方法
AU2023283348A1 (en) 2022-06-07 2025-01-09 Generation Bio Co. Lipid nanoparticle compositions and uses thereof
CN117247359A (zh) * 2022-06-09 2023-12-19 中国科学院广州生物医药与健康研究院 一种树状样脂质化合物、脂质体、脂质复合物、脂质纳米颗粒及其应用
IL317425A (en) 2022-06-10 2025-02-01 Camp4 Therapeutics Corp Methods of modulating progranulin expression using antisense oligonucleotides targeting regulatory RNAs
TW202435912A (zh) 2022-08-03 2024-09-16 美商航海家醫療公司 用於穿過血腦屏障之組合物及方法
KR20250046303A (ko) 2022-08-05 2025-04-02 사네진 바이오 유에스에이 인크. 안지오텐시노겐 (agt)을 표적화하는 이중 가닥 rna 및 그의 사용 방법
WO2024040222A1 (en) 2022-08-19 2024-02-22 Generation Bio Co. Cleavable closed-ended dna (cedna) and methods of use thereof
TW202424193A (zh) 2022-09-15 2024-06-16 美商艾拉倫製藥股份有限公司 第１３型１７β－羥基類固醇去氫酶（ＨＳＤ１７Ｂ１３）ｉＲＮＡ組成物及其使用方法
KR20250110209A (ko) 2022-09-23 2025-07-18 엔크로마 바이오, 인크. Hbv 유전자 발현의 에피제네틱 조절을 위한 조성물 및 방법
EP4602164A2 (en) 2022-10-14 2025-08-20 Sanegene Bio USA Inc. Small interfering rna targeting c3 and uses thereof
KR102775606B1 (ko) * 2022-11-01 2025-03-06 주식회사 삼양홀딩스 아마이드 및 에스터 작용기를 갖는 지질 및 이의 제조방법
WO2024102677A1 (en) 2022-11-08 2024-05-16 Orna Therapeutics, Inc. Circular rna compositions
TW202425959A (zh) 2022-11-08 2024-07-01 美商歐納醫療公司 遞送多核苷酸的脂質及奈米顆粒組合物
WO2024102762A1 (en) 2022-11-08 2024-05-16 Orna Therapeutics, Inc. Lipids and lipid nanoparticle compositions for delivering polynucleotides
CN115745788B (zh) * 2022-11-21 2025-06-24 国家纳米科学中心 一种可电离脂质化合物及其制备方法和应用
WO2024119145A1 (en) 2022-12-01 2024-06-06 Camp4 Therapeutics Corporation Modulation of syngap1 gene transcription using antisense oligonucleotides targeting regulatory rnas
JP2025540076A (ja) 2022-12-01 2025-12-11 ジェネレーション バイオ カンパニー 核酸、イオン化可能な脂質、ステロール、脂質アンカーポリマー、及びヘルパー脂質を含む脂質ナノ粒子、それらの使用
IL320873A (en) 2022-12-01 2025-07-01 Generation Bio Co Novel polyglycerol-conjugated lipids and lipid nanoparticle compositions containing them
EP4626402A1 (en) 2022-12-01 2025-10-08 Generation Bio Co. Lipid nanoparticles comprising nucleic acids and lipid-anchored polymers
AU2023406483A1 (en) 2022-12-01 2025-05-29 Generation Bio Co. Stealth lipid nanoparticle compositions for cell targeting
CN120359199A (zh) * 2022-12-12 2025-07-22 星锐医药(苏州)有限公司 新型化合物及其靶向递送用途
CN121219420A (zh) 2022-12-14 2025-12-26 普罗维登斯治疗控股公司 用于感染性疾病的组合物和方法
EP4638743A2 (en) 2022-12-19 2025-10-29 Sanegene Bio USA Inc. Small interfering rna targeting cfb and uses thereof
WO2024134199A1 (en) 2022-12-22 2024-06-27 Mina Therapeutics Limited Chemically modified sarna compositions and methods of use
AU2024217843A1 (en) 2023-02-09 2025-07-31 Alnylam Pharmaceuticals, Inc. Reversir molecules and methods of use thereof
US20240309383A1 (en) 2023-02-24 2024-09-19 Suzhou Sanegene Bio Inc. Small interfering rna targeting hbv and uses thereof
US20240309380A1 (en) 2023-03-03 2024-09-19 Sanegene Bio Usa Inc. Small interfering rna targeting apoc3 and uses thereof
KR20250163337A (ko) 2023-03-22 2025-11-20 레굴루스 테라퓨틱스 인크 신경계 장애의 치료 방법
WO2024205657A2 (en) 2023-03-29 2024-10-03 Orna Therapeutics, Inc. Lipids and lipid nanoparticle compositions for delivering polynucleotides
AU2024255359A1 (en) 2023-04-12 2025-09-11 Regulus Therapeutics Inc. Modified oligonucleotide for treating polycystic kidney disease
WO2024233308A2 (en) 2023-05-05 2024-11-14 Orna Therapeutics, Inc. Circular rna compositions and methods
AU2024270764A1 (en) 2023-05-15 2025-12-04 Nchroma Bio, Inc. Compositions and methods for epigenetic regulation of hbv gene expression
WO2024238700A1 (en) 2023-05-15 2024-11-21 Chroma Medicine, Inc. Compositions and methods for epigenetic regulation of hbv gene expression
WO2024243438A2 (en) 2023-05-23 2024-11-28 Omega Therapeutics, Inc. Compositions and methods for reducing cxcl9, cxcl10, and cxcl11 gene expression
US20250027089A1 (en) 2023-06-21 2025-01-23 Sanegene Bio Usa Inc. Double stranded rna targeting proprotein convertase subtilisin kexin 9 (pcsk9) and methods of use thereof
AU2024287308A1 (en) 2023-07-13 2025-12-18 Korro Bio, Inc. Rna-editing oligonucleotides and uses thereof
WO2025015338A1 (en) 2023-07-13 2025-01-16 Korro Bio, Inc. Rna-editing oligonucleotides and uses thereof
WO2025049690A1 (en) 2023-08-29 2025-03-06 Orna Therapeutics, Inc. Circular polyethylene glycol lipids
WO2025052180A2 (en) 2023-09-07 2025-03-13 Axelyf ehf. Lipids and lipid nanoparticles
WO2025072383A1 (en) 2023-09-25 2025-04-03 The Broad Institute, Inc. Viral open reading frames, uses thereof, and methods of detecting the same
WO2025096809A1 (en) 2023-10-31 2025-05-08 Korro Bio, Inc. Oligonucleotides comprising phosphoramidate internucleotide linkages
WO2025097055A2 (en) 2023-11-02 2025-05-08 The Broad Institute, Inc. Compositions and methods of use of t cells in immunotherapy
WO2025101501A1 (en) 2023-11-07 2025-05-15 Orna Therapeutics, Inc. Circular rna compositions
WO2025129128A1 (en) 2023-12-15 2025-06-19 Vivasor, Inc. Compositions and methods for non-viral delivery of therapeutic compounds
WO2025129158A1 (en) 2023-12-15 2025-06-19 The Broad Institute, Inc. Engineered arc delivery vesicles and uses thereof
WO2025166323A2 (en) 2024-02-02 2025-08-07 Editas Medicine, Inc. Crispr-related methods and compositions targeting lipoprotein (a) expression
WO2025193584A1 (en) 2024-03-11 2025-09-18 Regulus Therapeutics Inc. Compound for use in methods for treatment of polycystic kidney disease
WO2025199231A2 (en) 2024-03-20 2025-09-25 Vertex Pharmaceuticals Incorporated Mucin-5b (muc5b) targeted sirna and antisense oligonucleotides and methods of use thereof
WO2025213138A1 (en) 2024-04-05 2025-10-09 Editas Medicine, Inc. Crispr/rna-guided nuclease related methods and compositions for treating primary open angle glaucoma
WO2025228441A2 (en) 2024-04-30 2025-11-06 Sanegene Bio Usa Inc. Small interfering rna targeting inhbe and uses thereof
WO2025231432A1 (en) 2024-05-03 2025-11-06 Caribou Biosciences, Inc. In vivo gene editing with crispr systems
WO2025250808A1 (en) 2024-05-29 2025-12-04 The Brigham And Women’S Hospital, Inc. Anti-crispr delivery compositions and methods
WO2025255388A1 (en) 2024-06-05 2025-12-11 Camp4 Therapeutics Corporation Modulation of syngap1 gene transcription using antisense oligonucleotides targeting regulatory rnas
WO2026003582A2 (en) 2024-06-27 2026-01-02 Axelyf ehf. Lipids and lipid nanoparticles

Family Cites Families (120)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US3233734A (en) 1961-04-19 1966-02-08 Muller Hans Filtering apparatus
US3223734A (en) * 1961-04-26 1965-12-14 Archer Daniels Midland Co Process for producing tertiary amines
US3608046A (en) 1968-09-30 1971-09-21 Nasa Technique of duplicating fragile core
US3687808A (en) 1969-08-14 1972-08-29 Univ Leland Stanford Junior Synthetic polynucleotides
GB1561617A (en) * 1976-10-18 1980-02-27 Texaco Development Corp High tertiary aminecontent compositions useful as polyurethane catalysts
DE2820892A1 (de) * 1978-05-12 1979-11-22 Nattermann A & Cie Neue strukturanaloga von glyveriden und verfahren zu deren herstellung
US4469863A (en) 1980-11-12 1984-09-04 Ts O Paul O P Nonionic nucleic acid alkyl and aryl phosphonates and processes for manufacture and use thereof
US5023243A (en) 1981-10-23 1991-06-11 Molecular Biosystems, Inc. Oligonucleotide therapeutic agent and method of making same
US4476301A (en) 1982-04-29 1984-10-09 Centre National De La Recherche Scientifique Oligonucleotides, a process for preparing the same and their application as mediators of the action of interferon
DD237512A1 (de) * 1984-04-11 1986-07-16 Rainer Noack Verfahren zur polymerisation von isocyanaten
US5550111A (en) 1984-07-11 1996-08-27 Temple University-Of The Commonwealth System Of Higher Education Dual action 2',5'-oligoadenylate antiviral derivatives and uses thereof
US5405938A (en) 1989-12-20 1995-04-11 Anti-Gene Development Group Sequence-specific binding polymers for duplex nucleic acids
US5185444A (en) 1985-03-15 1993-02-09 Anti-Gene Deveopment Group Uncharged morpolino-based polymers having phosphorous containing chiral intersubunit linkages
US5166315A (en) 1989-12-20 1992-11-24 Anti-Gene Development Group Sequence-specific binding polymers for duplex nucleic acids
US5034506A (en) 1985-03-15 1991-07-23 Anti-Gene Development Group Uncharged morpholino-based polymers having achiral intersubunit linkages
US5235033A (en) 1985-03-15 1993-08-10 Anti-Gene Development Group Alpha-morpholino ribonucleoside derivatives and polymers thereof
US5264423A (en) 1987-03-25 1993-11-23 The United States Of America As Represented By The Department Of Health And Human Services Inhibitors for replication of retroviruses and for the expression of oncogene products
US5276019A (en) 1987-03-25 1994-01-04 The United States Of America As Represented By The Department Of Health And Human Services Inhibitors for replication of retroviruses and for the expression of oncogene products
US4933470A (en) * 1987-10-05 1990-06-12 Neorx Corporation Method of synthesis of vicinal diamines
US5188897A (en) 1987-10-22 1993-02-23 Temple University Of The Commonwealth System Of Higher Education Encapsulated 2',5'-phosphorothioate oligoadenylates
US4924624A (en) 1987-10-22 1990-05-15 Temple University-Of The Commonwealth System Of Higher Education 2,',5'-phosphorothioate oligoadenylates and plant antiviral uses thereof
JPH02767A (ja) * 1987-12-17 1990-01-05 Shionogi & Co Ltd 脂質誘導体
EP0406309A4 (en) 1988-03-25 1992-08-19 The University Of Virginia Alumni Patents Foundation Oligonucleotide n-alkylphosphoramidates
US5278302A (en) 1988-05-26 1994-01-11 University Patents, Inc. Polynucleotide phosphorodithioates
US5216141A (en) 1988-06-06 1993-06-01 Benner Steven A Oligonucleotide analogs containing sulfur linkages
JP2805649B2 (ja) 1988-12-24 1998-09-30 靖雄 菊川 ジアシルアミド化合物およびアミド類の製造法
US5045338A (en) 1989-09-19 1991-09-03 Nabisco Brands, Inc. Secondary amide esters as low calorie fat mimetics
US5399676A (en) 1989-10-23 1995-03-21 Gilead Sciences Oligonucleotides with inverted polarity
US5264562A (en) 1989-10-24 1993-11-23 Gilead Sciences, Inc. Oligonucleotide analogs with novel linkages
US5264564A (en) 1989-10-24 1993-11-23 Gilead Sciences Oligonucleotide analogs with novel linkages
US5177198A (en) 1989-11-30 1993-01-05 University Of N.C. At Chapel Hill Process for preparing oligoribonucleoside and oligodeoxyribonucleoside boranophosphates
US5223168A (en) 1989-12-12 1993-06-29 Gary Holt Surface cleaner and treatment
US5506351A (en) 1992-07-23 1996-04-09 Isis Pharmaceuticals Process for the preparation of 2'-O-alkyl guanosine and related compounds
US5459255A (en) 1990-01-11 1995-10-17 Isis Pharmaceuticals, Inc. N-2 substituted purines
US5578718A (en) 1990-01-11 1996-11-26 Isis Pharmaceuticals, Inc. Thiol-derivatized nucleosides
US5457191A (en) 1990-01-11 1995-10-10 Isis Pharmaceuticals, Inc. 3-deazapurines
US5212295A (en) 1990-01-11 1993-05-18 Isis Pharmaceuticals Monomers for preparation of oligonucleotides having chiral phosphorus linkages
US5587361A (en) 1991-10-15 1996-12-24 Isis Pharmaceuticals, Inc. Oligonucleotides having phosphorothioate linkages of high chiral purity
US5387470A (en) 1990-03-02 1995-02-07 W. R. Grace & Co.-Conn. Packaging film
US5321131A (en) 1990-03-08 1994-06-14 Hybridon, Inc. Site-specific functionalization of oligodeoxynucleotides for non-radioactive labelling
US5470967A (en) 1990-04-10 1995-11-28 The Dupont Merck Pharmaceutical Company Oligonucleotide analogs with sulfamate linkages
DE4013632A1 (de) * 1990-04-27 1991-10-31 Max Planck Gesellschaft Liposomen mit positiver ueberschussladung
US5677437A (en) 1990-07-27 1997-10-14 Isis Pharmaceuticals, Inc. Heteroatomic oligonucleoside linkages
US5218105A (en) 1990-07-27 1993-06-08 Isis Pharmaceuticals Polyamine conjugated oligonucleotides
US5541307A (en) 1990-07-27 1996-07-30 Isis Pharmaceuticals, Inc. Backbone modified oligonucleotide analogs and solid phase synthesis thereof
US5489677A (en) 1990-07-27 1996-02-06 Isis Pharmaceuticals, Inc. Oligonucleoside linkages containing adjacent oxygen and nitrogen atoms
US5138045A (en) 1990-07-27 1992-08-11 Isis Pharmaceuticals Polyamine conjugated oligonucleotides
US5623070A (en) 1990-07-27 1997-04-22 Isis Pharmaceuticals, Inc. Heteroatomic oligonucleoside linkages
US5378825A (en) 1990-07-27 1995-01-03 Isis Pharmaceuticals, Inc. Backbone modified oligonucleotide analogs
US5602240A (en) 1990-07-27 1997-02-11 Ciba Geigy Ag. Backbone modified oligonucleotide analogs
US5610289A (en) 1990-07-27 1997-03-11 Isis Pharmaceuticals, Inc. Backbone modified oligonucleotide analogues
US5608046A (en) 1990-07-27 1997-03-04 Isis Pharmaceuticals, Inc. Conjugated 4'-desmethyl nucleoside analog compounds
US5618704A (en) 1990-07-27 1997-04-08 Isis Pharmacueticals, Inc. Backbone-modified oligonucleotide analogs and preparation thereof through radical coupling
US5386023A (en) 1990-07-27 1995-01-31 Isis Pharmaceuticals Backbone modified oligonucleotide analogs and preparation thereof through reductive coupling
DK0541722T3 (da) 1990-08-03 1996-04-22 Sterling Winthrop Inc Forbindelser og fremgangsmåder til inhibering af genekspression
US5212293A (en) 1990-08-06 1993-05-18 American Cyanamid Company Process for the preparation of deoxynucleosides
US6262241B1 (en) 1990-08-13 2001-07-17 Isis Pharmaceuticals, Inc. Compound for detecting and modulating RNA activity and gene expression
US5177196A (en) 1990-08-16 1993-01-05 Microprobe Corporation Oligo (α-arabinofuranosyl nucleotides) and α-arabinofuranosyl precursors thereof
US5214134A (en) 1990-09-12 1993-05-25 Sterling Winthrop Inc. Process of linking nucleosides with a siloxane bridge
US5561225A (en) 1990-09-19 1996-10-01 Southern Research Institute Polynucleotide analogs containing sulfonate and sulfonamide internucleoside linkages
AU662298B2 (en) 1990-09-20 1995-08-31 Gilead Sciences, Inc. Modified internucleoside linkages
US5539082A (en) 1993-04-26 1996-07-23 Nielsen; Peter E. Peptide nucleic acids
JP3034993B2 (ja) 1991-06-17 2000-04-17 旭電化工業株式会社 透明性の改善された合成樹脂組成物
WO1993002340A1 (en) 1991-07-25 1993-02-04 The Whitaker Corporation Liquid level sensor
US5571799A (en) 1991-08-12 1996-11-05 Basco, Ltd. (2'-5') oligoadenylate analogues useful as inhibitors of host-v5.-graft response
US5239113A (en) * 1991-10-15 1993-08-24 Monsanto Company Substituted β-amino acid derivatives useful as platelet aggregation inhibitors and intermediates thereof
US5599797A (en) 1991-10-15 1997-02-04 Isis Pharmaceuticals, Inc. Oligonucleotides having phosphorothioate linkages of high chiral purity
ATE239484T1 (de) 1991-10-24 2003-05-15 Isis Pharmaceuticals Inc Derivatisierte oligonukleotide mit verbessertem aufnahmevermögen
US5399460A (en) * 1991-12-04 1995-03-21 E. I. Du Pont De Nemours And Company Negative photoresists containing aminoacrylate salts
JP3122520B2 (ja) * 1992-03-13 2001-01-09 生化学工業株式会社 ２−アミノピリジン誘導体、その製造方法及び蛍光標識剤
US5633360A (en) 1992-04-14 1997-05-27 Gilead Sciences, Inc. Oligonucleotide analogs capable of passive cell membrane permeation
US5434257A (en) 1992-06-01 1995-07-18 Gilead Sciences, Inc. Binding compentent oligomers containing unsaturated 3',5' and 2',5' linkages
US5476925A (en) 1993-02-01 1995-12-19 Northwestern University Oligodeoxyribonucleotides including 3'-aminonucleoside-phosphoramidate linkages and terminal 3'-amino groups
GB9304618D0 (en) 1993-03-06 1993-04-21 Ciba Geigy Ag Chemical compounds
JPH08508491A (ja) 1993-03-31 1996-09-10 スターリング ウインスロップ インコーポレイティド ホスホジエステル結合をアミド結合に置き換えたオリゴヌクレオチド
US5571902A (en) 1993-07-29 1996-11-05 Isis Pharmaceuticals, Inc. Synthesis of oligonucleotides
US5625050A (en) 1994-03-31 1997-04-29 Amgen Inc. Modified oligonucleotides and intermediates useful in nucleic acid therapeutics
US5554746A (en) 1994-05-16 1996-09-10 Isis Pharmaceuticals, Inc. Lactam nucleic acids
JP2982621B2 (ja) * 1994-07-22 1999-11-29 味の素株式会社 光増感機能をもつ新規アミノ酸
US5830430A (en) * 1995-02-21 1998-11-03 Imarx Pharmaceutical Corp. Cationic lipids and the use thereof
US5646241A (en) 1995-05-12 1997-07-08 Quantum Materials, Inc. Bleed resistant cyanate ester-containing compositions
US5679852A (en) 1995-06-02 1997-10-21 Schering Aktiengesellschaft Process for the production of DTPA-monoamides of the central carboxylic acid and their use as pharmaceutical agents
AU7078396A (en) * 1995-09-27 1997-04-30 Regents Of The University Of California, The Polyfunctional cationic cytofectins, formulations and methods for generating active cytofectin:polynucleotide transfection complexes
US5869715A (en) * 1995-09-27 1999-02-09 The Reagents Of The University Of California Polyfunctional cationic cytofectins
EP0941120A4 (en) * 1996-11-05 2004-08-18 Bristol Myers Squibb Co BRANCHED PEPTIDLINKERS
US6210707B1 (en) 1996-11-12 2001-04-03 The Regents Of The University Of California Methods of forming protein-linked lipidic microparticles, and compositions thereof
JP4656675B2 (ja) * 1997-05-14 2011-03-23 ユニバーシティー オブ ブリティッシュ コロンビア 脂質小胞への荷電した治療剤の高率封入
JP2001055327A (ja) * 1999-06-11 2001-02-27 Fuji Chemical Industries Ltd 新規なヒドロキサム酸誘導体を含む医薬
GB9914045D0 (en) 1999-06-16 1999-08-18 Smithkline Beecham Plc Novel compounds
CA2379060A1 (en) * 1999-07-14 2001-01-25 Alza Corporation Neutral lipopolymer and liposomal compositions containing same
US6458876B1 (en) * 1999-08-09 2002-10-01 Air Products And Chemicals, Inc. Ink jet paper coatings containing polyvinyl alcohol-alkylated polyamine blends
WO2003070918A2 (en) 2002-02-20 2003-08-28 Ribozyme Pharmaceuticals, Incorporated Rna interference by modified short interfering nucleic acid
MXPA02012198A (es) 2000-06-09 2004-08-19 Teni Boulikas Encapsulacion de polinucleotidos y drogas en liposomas objetivos.
MY133479A (en) * 2001-02-24 2007-11-30 Boehringer Ingelheim Pharma Xanthine derivatives, the preparation thereof and their use as pharmaceutical compositions
US20060211642A1 (en) * 2001-05-18 2006-09-21 Sirna Therapeutics, Inc. RNA inteference mediated inhibition of hepatitis C virus (HVC) gene expression using short interfering nucleic acid (siNA)
GB0118517D0 (en) * 2001-07-30 2001-09-19 Mitsubishi Tokyo Pharm Inc Compound
US20030194445A1 (en) * 2001-11-12 2003-10-16 Kuhner Carla H. Compositions and methods of use of peptides in combination with biocides and/or germicides
JP4493970B2 (ja) * 2002-10-16 2010-06-30 武田薬品工業株式会社 持続性製剤
CA2517517C (en) * 2003-03-03 2012-12-18 Array Biopharma, Inc. P38 inhibitors and methods of use thereof
EP1648519B1 (en) * 2003-07-16 2014-10-08 Protiva Biotherapeutics Inc. Lipid encapsulated interfering rna
CA2551022C (en) * 2003-09-15 2013-06-04 Protiva Biotherapeutics, Inc. Polyethyleneglycol-modified lipid compounds and uses thereof
JP2008502583A (ja) * 2003-10-01 2008-01-31 バイエル・ヘルスケア・アクチェンゲゼルシャフト 抗細菌性アミド大員環
JP4764426B2 (ja) 2004-06-07 2011-09-07 プロチバ バイオセラピューティクス インコーポレイティッド カチオン性脂質および使用方法
EP1766035B1 (en) * 2004-06-07 2011-12-07 Protiva Biotherapeutics Inc. Lipid encapsulated interfering rna
AU2005259799A1 (en) 2004-07-02 2006-01-12 Protiva Biotherapeutics, Inc. Immunostimulatory siRNA molecules and uses therefor
SI1765289T1 (sl) * 2004-07-07 2009-02-28 Statens Seruminstitut Sestavki in postopki za stabiliziranje dodatnih pripravkov na osnovi lipidov z uporabo glikolipidov
JP2006091276A (ja) * 2004-09-22 2006-04-06 Fuji Photo Film Co Ltd 反射防止フィルム、偏光板、画像表示装置、及び反射防止フィルムの製造方法
GB0422877D0 (en) * 2004-10-14 2004-11-17 Univ Glasgow Bioactive polymers
WO2006051314A2 (en) * 2004-11-12 2006-05-18 Ucl Business Plc Guanidine derivatives as inhibitors of ddah
GB0425555D0 (en) * 2004-11-19 2004-12-22 Glaxo Group Ltd Novel compounds
GB0425556D0 (en) * 2004-11-19 2004-12-22 Glaxo Group Ltd Novel compounds
DE102004058925A1 (de) 2004-12-07 2006-06-08 Siemens Ag Hochfrequenz-Plasmazündvorrichtung für Verbrennungskraftmaschinen, insbesondere für direkt einspritzende Otto-Motoren
WO2006074546A1 (en) 2005-01-13 2006-07-20 Protiva Biotherapeutics, Inc. Lipid encapsulated interfering rna
GB0502482D0 (en) * 2005-02-07 2005-03-16 Glaxo Group Ltd Novel compounds
ITRM20050090A1 (it) * 2005-03-02 2006-09-03 Sigma Tau Ind Farmaceutiche Riunite Spa Derivati dell'acido ammino-butanoico inibitore della cpt.
US20060228406A1 (en) * 2005-03-17 2006-10-12 Invitrogen Corporation Transfection reagent for non-adherent suspension cells
JP5777846B2 (ja) * 2005-06-15 2015-09-09 マサチューセッツ インスティテュート オブ テクノロジー アミン含有脂質およびその使用
JP2007230789A (ja) * 2006-02-27 2007-09-13 Fujifilm Corp 無機酸化物微粒子の製造方法、それにより得られる無機酸化微粒子、ならびにそれを用いた組成物および光学フィルム
ES2430206T3 (es) 2006-10-03 2013-11-19 Tekmira Pharmaceuticals Corporation Formulación que contiene lípidos
US20110117125A1 (en) * 2008-01-02 2011-05-19 Tekmira Pharmaceuticals Corporation Compositions and methods for the delivery of nucleic acids

Also Published As

Publication number Publication date
AU2007303205A1 (en) 2008-04-10
MX2019003005A (es) 2019-08-05
US20200361854A1 (en) 2020-11-19
CA2665225A1 (en) 2008-04-10
ES2430206T3 (es) 2013-11-19
JP2024150681A (ja) 2024-10-23
EP2068886A4 (en) 2010-12-22
JP2022164716A (ja) 2022-10-27
CA3144493A1 (en) 2008-04-10
MX363224B (es) 2019-03-15
WO2008042973A2 (en) 2008-04-10
US8034376B2 (en) 2011-10-11
DK2068886T3 (da) 2013-11-18
CN101616677A (zh) 2009-12-30
JP2010505873A (ja) 2010-02-25
US20090023673A1 (en) 2009-01-22
EP2695608B1 (en) 2016-11-23
CA2848238C (en) 2016-07-19
MX2009003548A (es) 2009-04-15
EP2068886B1 (en) 2013-09-18
US8642076B2 (en) 2014-02-04
US20180065918A1 (en) 2018-03-08
KR101129509B1 (ko) 2012-04-13
JP5833597B2 (ja) 2015-12-16
EP2068886A2 (en) 2009-06-17
JP2013213036A (ja) 2013-10-17
CN105030654A (zh) 2015-11-11
US20140121393A1 (en) 2014-05-01
IN2015DN00255A (enExample) 2015-06-12
JP2019048887A (ja) 2019-03-28
JP2021011503A (ja) 2021-02-04
EP2695608A3 (en) 2014-05-21
WO2008042973A3 (en) 2008-08-28
ES2611924T3 (es) 2017-05-11
CA2848238A1 (en) 2008-04-10
US20120046478A1 (en) 2012-02-23
EP3192788A1 (en) 2017-07-19
JP2017036330A (ja) 2017-02-16
US20230174459A1 (en) 2023-06-08
JP7127098B2 (ja) 2022-08-29
CN110066224A (zh) 2019-07-30
MX2022013691A (es) 2022-12-13
CN101616677B (zh) 2015-07-22
KR20090088362A (ko) 2009-08-19
EP2695608A2 (en) 2014-02-12
CA2927045A1 (en) 2008-04-10
US11420931B2 (en) 2022-08-23
CA2665225C (en) 2015-06-30
JP2015227370A (ja) 2015-12-17

Similar Documents

Publication Publication Date Title
JP7127098B2 (ja) 製剤用脂質ナノ粒子
JP5475753B2 (ja) 核酸送達用の脂質製剤
US20090163705A1 (en) Cationic lipids
EP3395797B1 (en) Compounds as cationic lipids
AU2014200910B2 (en) Lipid containing formulations
US11471533B2 (en) Compound usable as cationic lipid
US20140294978A1 (en) Cationic lipid

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20101001

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20101001

RD04 Notification of resignation of power of attorney

Free format text: JAPANESE INTERMEDIATE CODE: A7424

Effective date: 20120127

A977 Report on retrieval

Free format text: JAPANESE INTERMEDIATE CODE: A971007

Effective date: 20121115

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20121211

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20130214

A602 Written permission of extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A602

Effective date: 20130221

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20130611

A711 Notification of change in applicant

Free format text: JAPANESE INTERMEDIATE CODE: A711

Effective date: 20130611

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20130725

A02 Decision of refusal

Free format text: JAPANESE INTERMEDIATE CODE: A02

Effective date: 20140212

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20151104

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20160217

A61 First payment of annual fees (during grant procedure)

Free format text: JAPANESE INTERMEDIATE CODE: A61

Effective date: 20160502

R150 Certificate of patent or registration of utility model

Ref document number: 5933163

Country of ref document: JP

Free format text: JAPANESE INTERMEDIATE CODE: R150

R250 Receipt of annual fees

Free format text: JAPANESE INTERMEDIATE CODE: R250

R250 Receipt of annual fees

Free format text: JAPANESE INTERMEDIATE CODE: R250

R250 Receipt of annual fees

Free format text: JAPANESE INTERMEDIATE CODE: R250

R250 Receipt of annual fees

Free format text: JAPANESE INTERMEDIATE CODE: R250

R250 Receipt of annual fees

Free format text: JAPANESE INTERMEDIATE CODE: R250

R250 Receipt of annual fees

Free format text: JAPANESE INTERMEDIATE CODE: R250

R250 Receipt of annual fees

Free format text: JAPANESE INTERMEDIATE CODE: R250