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DK2068886T3 - Lipidholdige præparater - Google Patents

Lipidholdige præparater

Info

Publication number
DK2068886T3
DK2068886T3 DK07853756.0T DK07853756T DK2068886T3 DK 2068886 T3 DK2068886 T3 DK 2068886T3 DK 07853756 T DK07853756 T DK 07853756T DK 2068886 T3 DK2068886 T3 DK 2068886T3
Authority
DK
Denmark
Prior art keywords
lipid
containing preparations
preparations
Prior art date
Application number
DK07853756.0T
Other languages
English (en)
Inventor
Muthiah Manoharan
Kallanthottahil G Rajeev
Akin Akinc
Muthusamy Jayraman
Martin A Maier
Jayaprakash K Narayanannair
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
Application granted granted Critical
Publication of DK2068886T3 publication Critical patent/DK2068886T3/da

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)
DK07853756.0T 2006-10-03 2007-10-03 Lipidholdige præparater DK2068886T3 (da)

Applications Claiming Priority (3)

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

Publications (1)

Publication Number Publication Date
DK2068886T3 true DK2068886T3 (da) 2013-11-18

Family

ID=39269192

Family Applications (1)

Application Number Title Priority Date Filing Date
DK07853756.0T DK2068886T3 (da) 2006-10-03 2007-10-03 Lipidholdige præparater

Country Status (12)

Country Link
US (6) US8034376B2 (da)
EP (3) EP2068886B1 (da)
JP (8) JP5933163B2 (da)
KR (1) KR101129509B1 (da)
CN (3) CN105030654A (da)
AU (1) AU2007303205A1 (da)
CA (4) CA2848238C (da)
DK (1) DK2068886T3 (da)
ES (2) ES2611924T3 (da)
IN (1) IN2015DN00255A (da)
MX (4) MX2009003548A (da)
WO (1) WO2008042973A2 (da)

Families Citing this family (381)

* 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
EP2636739B1 (en) 2004-03-12 2014-12-10 Alnylam Pharmaceuticals Inc. iRNA agents targeting VEGF
AU2006259415B2 (en) * 2005-06-15 2012-08-30 Massachusetts Institute Of Technology Amine-containing lipids and uses thereof
EP2584047B1 (en) 2006-05-11 2014-11-19 Alnylam Pharmaceuticals Inc. Compositions and methods for inhibiting expression of the PCSK9 gene
US8598333B2 (en) * 2006-05-26 2013-12-03 Alnylam Pharmaceuticals, Inc. SiRNA silencing of genes expressed in cancer
CA2848238C (en) * 2006-10-03 2016-07-19 Tekmira Pharmaceuticals Corporation Lipid containing formulations
HUE040417T2 (hu) 2007-05-04 2019-03-28 Marina Biotech Inc Aminosavlipidek és alkalmazásuk
AU2008335202A1 (en) 2007-12-10 2009-06-18 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of Factor VII gene
AU2008347251A1 (en) * 2008-01-02 2009-07-16 Tekmira Pharmaceuticals Corporation Liver screening method
AU2009241591A1 (en) * 2008-01-31 2009-11-05 Alnylam Pharmaceuticals, Inc. Optimized methods for delivery of DSRNA targeting the PCSK9 gene
KR101397407B1 (ko) 2008-03-05 2014-06-19 알닐람 파마슈티칼스 인코포레이티드 Ｅｇ5 및 ＶＥＧＦ 유전자의 발현을 억제하기 위한 조성물 및 방법
WO2010028054A1 (en) 2008-09-02 2010-03-11 Alnylam Europe Ag. Compositions and methods for inhibiting expression of mutant egfr gene
AU2009296395A1 (en) 2008-09-25 2010-04-01 Alnylam Pharmaceuticals, Inc. Lipid formulated compositions and methods for inhibiting expression of Serum Amyloid A gene
AU2009303345B2 (en) 2008-10-09 2015-08-20 Arbutus Biopharma Corporation Improved amino lipids and methods for the delivery of nucleic acids
IL302142B2 (en) 2008-10-20 2024-07-01 Alnylam Pharmaceuticals Inc Compounds and methods for inhibiting transthyretin expression
WO2010047765A2 (en) 2008-10-20 2010-04-29 Massachussetts Institute Of Technology Nanostructures for drug delivery
US8987435B2 (en) 2008-10-24 2015-03-24 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
CA3033577A1 (en) 2008-11-10 2010-05-14 Arbutus Biopharma Corporation Novel lipids and compositions for the delivery of therapeutics
WO2010057217A1 (en) * 2008-11-17 2010-05-20 Alnylam Pharmaceuticals, Inc. Compositions and methods for increasing cellular uptake of rnai via sid-1
NZ593618A (en) 2008-12-10 2013-02-22 Alnylam Pharmaceuticals Inc Gnaq targeted dsrna compositions and methods for inhibiting expression
WO2010088537A2 (en) 2009-01-29 2010-08-05 Alnylam Pharmaceuticals, Inc. Improved lipid formulation
US20120041051A1 (en) 2009-02-26 2012-02-16 Kevin Fitzgerald Compositions And Methods For Inhibiting Expression Of MIG-12 Gene
CA2754043A1 (en) * 2009-03-12 2010-09-16 Alnylam Pharmaceuticals, Inc. Lipid formulated compositions and methods for inhibiting expression of eg5 and vegf genes
KR101906392B1 (ko) * 2009-03-20 2018-10-10 씨엘에스엔 래버러토리스, 인코퍼레이티드 폴리아민 유도체
WO2010120385A1 (en) * 2009-04-18 2010-10-21 Massachusetts Institute Of Technology pH SENSITIVE BIODEGRADABLE POLYMERIC PARTICLES FOR DRUG DELIVERY
WO2010129709A1 (en) 2009-05-05 2010-11-11 Alnylam Pharmaceuticals, Inc. Lipid compositions
AU2017200272A1 (en) * 2009-05-05 2017-02-02 Arbutus Biopharma Corporation Lipid compositions
EP3504967A1 (en) 2009-05-05 2019-07-03 Arbutus Biopharma Corporation Methods of delivering oligonucleotides to immune cells
JP2012527444A (ja) 2009-05-20 2012-11-08 イーティーエイチ チューリッヒ 代謝障害に関するターゲッティングマイクロｒｎａ
JP5875976B2 (ja) 2009-06-01 2016-03-02 ヘイロー−バイオ アールエヌエーアイ セラピューティクス， インコーポレイテッド 多価ｒｎａ干渉のためのポリヌクレオチド、組成物およびそれらの使用方法
SMT201800499T1 (it) 2009-06-10 2018-11-09 Arbutus Biopharma Corp Formulazione lipidica migliorata
NZ597504A (en) 2009-06-15 2013-10-25 Alnylam Pharmaceuticals Inc Lipid formulated dsrna targeting the pcsk9 gene
US9051567B2 (en) 2009-06-15 2015-06-09 Tekmira Pharmaceuticals Corporation Methods for increasing efficacy of lipid formulated siRNA
WO2011017548A1 (en) * 2009-08-05 2011-02-10 Alnylam Pharmaceuticals, Inc. Lipid formulated compositions and methods for inhibiting expression of eg5 and vegf genes
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
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
WO2011038031A1 (en) 2009-09-22 2011-03-31 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)
HUE038039T2 (hu) 2009-12-01 2018-09-28 Translate Bio Inc mRNS bejuttatása fehérjék és enzimek kiegészítésére humán genetikai betegségekben
WO2011075656A1 (en) 2009-12-18 2011-06-23 The University Of British Columbia Methods and compositions for delivery of nucleic acids
CA3009891C (en) * 2009-12-23 2020-09-15 Novartis Ag Lipids, lipid compositions, and methods of using them
EP2525780B1 (en) * 2010-01-19 2022-09-07 Northwestern University Synthetic nanostructures including nucleic acids and/or other entities
EP2526113B1 (en) * 2010-01-22 2016-08-10 Sirna Therapeutics, Inc. Post-synthetic chemical modification of rna at the 2'-position of the ribose ring via "click" chemistry
WO2011123468A1 (en) 2010-03-29 2011-10-06 Alnylam Pharmaceuticals, Inc. Sirna therapy for transthyretin (ttr) related ocular amyloidosis
JP2013523162A (ja) 2010-04-06 2013-06-17 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 Ｃｄ２７４／ｐｄ−ｌ１遺伝子の発現を阻害するための組成物および方法
WO2011133871A2 (en) 2010-04-22 2011-10-27 Alnylam Pharmaceuticals, Inc. 5'-end derivatives
EP2625186B1 (en) 2010-04-28 2016-07-27 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
JP2013527856A (ja) 2010-05-12 2013-07-04 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 陽イオン性脂質およびその使用方法
US9944671B2 (en) 2010-06-02 2018-04-17 Alnylam Pharmaceuticals, Inc. Compositions and methods directed to treating liver fibrosis
IL300109A (en) 2010-06-03 2023-03-01 Alnylam Pharmaceuticals Inc Biodegradable lipids for the transfer of active substances
WO2011159836A2 (en) 2010-06-15 2011-12-22 Isis Pharmaceuticals, Inc. Compounds and methods for modulating interaction between proteins and target nucleic acids
US20130323269A1 (en) 2010-07-30 2013-12-05 Muthiah Manoharan Methods and compositions for delivery of active agents
WO2012016188A2 (en) 2010-07-30 2012-02-02 Alnylam Pharmaceuticals, Inc. Methods and compositions for delivery of active agents
KR20140016230A (ko) 2010-08-04 2014-02-07 시즐 바이오테크놀로지 리미티드 암의 진단 및 치료를 위한 방법 및 화합물
CA2807552A1 (en) 2010-08-06 2012-02-09 Moderna Therapeutics, Inc. Engineered nucleic acids and methods of use thereof
US9193827B2 (en) 2010-08-26 2015-11-24 Massachusetts Institute Of Technology Poly(beta-amino alcohols), their preparation, and uses thereof
WO2012031205A2 (en) 2010-09-03 2012-03-08 The Brigham And Women's Hospital, Inc. Lipid-polymer hybrid particles
MX2013003681A (es) 2010-10-01 2013-11-20 Moderna Therapeutics Inc Ácidos nucleicos manipulados y métodos de uso de los mismos.
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
EP3470395A1 (en) * 2010-11-15 2019-04-17 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
EP2649182A4 (en) 2010-12-10 2015-05-06 Alnylam Pharmaceuticals Inc Compositions and methods for increasing erythropoietin (epo) production
WO2012091523A2 (en) 2010-12-30 2012-07-05 Samyang Biopharmaceuticals Corporation Carrier for negatively charged drugs comprising a cationic lipid and a preparation method thereof
EP2663548B1 (en) * 2011-01-11 2017-04-05 Alnylam Pharmaceuticals, Inc. Pegylated lipids and their use for drug delivery
PL2691443T3 (pl) 2011-03-28 2021-08-30 Massachusetts Institute Of Technology Sprzężone lipomery i ich zastosowania
US9175290B2 (en) 2011-03-29 2015-11-03 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
ES2621863T3 (es) 2011-04-25 2017-07-05 Regulus Therapeutics Inc. Compuestos de microARN y métodos para modular la actividad de miR-21
ME03491B (me) 2011-06-08 2020-01-20 Translate Bio Inc Kompozicije lipidnih nanočestica i postupci za isporuku irnk
EP4092120A1 (en) 2011-06-21 2022-11-23 Alnylam Pharmaceuticals, Inc. Angiopoietin-like 3 (anglptl3) irna compositions and methods of use thereof
US20140275211A1 (en) 2011-06-21 2014-09-18 Alnylam Pharmaceuticals, Inc. Assays and methods for determining activity of a therapeutic agent in a subject
EP3388068A1 (en) 2011-06-21 2018-10-17 Alnylam Pharmaceuticals, Inc. Composition and methods for inhibition of expression of protein c (proc) genes
US9315813B2 (en) 2011-06-21 2016-04-19 Alnylam Pharmaceuticals, Inc Compositions and methods for inhibition of expression of apolipoprotein C-III (APOC3) genes
US20140235693A1 (en) 2011-06-23 2014-08-21 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
US9701623B2 (en) 2011-09-27 2017-07-11 Alnylam Pharmaceuticals, Inc. Di-aliphatic substituted pegylated lipids
SI3682905T1 (sl) 2011-10-03 2022-04-29 Modernatx, Inc. Modificirani nukleozidi, nukleotidi in nukleinske kisline ter njihove uporabe
WO2013075035A1 (en) 2011-11-18 2013-05-23 Alnylam Pharmaceuticals Rnai agents, compositions and methods of use thereof for treating transthyretin (ttr) associated diseases
WO2013086322A1 (en) 2011-12-07 2013-06-13 Alnylam Pharmaceuticals, Inc. Branched alkyl and cycloalkyl terminated biodegradable lipids for the delivery of active agents
WO2013086373A1 (en) 2011-12-07 2013-06-13 Alnylam Pharmaceuticals, Inc. Lipids for the delivery of active agents
EP3988537A1 (en) 2011-12-07 2022-04-27 Alnylam Pharmaceuticals, Inc. Biodegradable lipids for the delivery of active agents
US20130156849A1 (en) 2011-12-16 2013-06-20 modeRNA Therapeutics Modified nucleoside, nucleotide, and nucleic acid compositions
US9035039B2 (en) 2011-12-22 2015-05-19 Protiva Biotherapeutics, Inc. Compositions and methods for silencing SMAD4
EP3988104A1 (en) 2012-02-24 2022-04-27 Arbutus Biopharma Corporation Trialkyl cationic lipids and methods of use thereof
US10501512B2 (en) 2012-04-02 2019-12-10 Modernatx, Inc. Modified polynucleotides
US9283287B2 (en) 2012-04-02 2016-03-15 Moderna Therapeutics, Inc. Modified polynucleotides for the production of nuclear proteins
US9572897B2 (en) 2012-04-02 2017-02-21 Modernatx, Inc. Modified polynucleotides for the production of cytoplasmic and cytoskeletal proteins
HK1206601A1 (en) 2012-04-02 2016-01-15 Moderna Therapeutics, Inc. Modified polynucleotides for the production of biologics and proteins associated with human disease
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
AU2013251696B2 (en) 2012-04-25 2018-11-15 Sanofi MicroRNA compounds and methods for modulating miR-21 activity
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
EP2859102A4 (en) 2012-06-08 2016-05-11 Shire Human Genetic Therapies NUCLEASE RESISTANT POLYNUCLEOTIDES AND USES THEREOF
BR112014031421A2 (pt) 2012-06-15 2017-06-27 Brigham & Womens Hospital Inc composições para tratamento de câncer e métodos para produção das mesmas
WO2014018375A1 (en) 2012-07-23 2014-01-30 Xenon Pharmaceuticals Inc. Cyp8b1 and uses thereof in therapeutic and diagnostic methods
EP2882706A1 (en) 2012-08-13 2015-06-17 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
SI2922554T1 (sl) 2012-11-26 2022-06-30 Modernatx, Inc. Terminalno modificirana RNA
EP3336187A1 (en) 2012-12-05 2018-06-20 Alnylam Pharmaceuticals, Inc. Pcsk9 irna compositions and methods of use thereof
US20150366997A1 (en) * 2012-12-07 2015-12-24 Shire Human Genetics Therapies, Inc. COMPOSITIONS AND METHODS FOR mRNA DELIVERY
US9695475B2 (en) 2012-12-11 2017-07-04 Ionis Pharmaceuticals, Inc. Competitive modulation of microRNAs
JP6762094B2 (ja) 2013-02-28 2020-09-30 タフツ ユニバーシティー 薬剤のデリバリーのためのジスルフィド化合物
US10258698B2 (en) 2013-03-14 2019-04-16 Modernatx, Inc. Formulation and delivery of modified nucleoside, nucleotide, and nucleic acid compositions
EP3467108B1 (en) 2013-03-14 2024-05-22 Translate Bio, Inc. Methods for purification of messenger rna
WO2014160129A2 (en) 2013-03-14 2014-10-02 Alnylam Pharmaceuticals, Inc. Complement component c5 irna compositions and methods of use thereof
CN105142614A (zh) 2013-03-14 2015-12-09 迪克纳制药公司 用于配制阴离子试剂的方法
EA037922B1 (ru) 2013-03-14 2021-06-07 Шир Хьюман Дженетик Терапис, Инк. мРНК CFTR И КОМПОЗИЦИИ ДЛЯ ЛЕЧЕНИЯ МУКОВИСЦИДОЗА У МЛЕКОПИТАЮЩЕГО
US8980864B2 (en) 2013-03-15 2015-03-17 Moderna Therapeutics, Inc. Compositions and methods of altering cholesterol levels
EP2992095B1 (en) 2013-05-01 2019-01-09 Regulus Therapeutics Inc. Microrna compounds and methods for modulating mir-122
JP6649248B2 (ja) 2013-05-01 2020-02-19 レグルス セラピューティクス インコーポレイテッド 細胞取り込みの向上のための化合物および方法
WO2014179562A1 (en) 2013-05-01 2014-11-06 Massachusetts Institute Of Technology 1,3,5-triazinane-2,4,6-trione derivatives and uses thereof
TW201515650A (zh) 2013-05-06 2015-05-01 艾爾妮蘭製藥公司 遞送經脂質調配之核酸分子之劑量及方法
WO2014186366A1 (en) * 2013-05-13 2014-11-20 Tufts University Nanocomplexes for delivery of saporin
AU2014268529C1 (en) 2013-05-22 2020-10-01 Alnylam Pharmaceuticals, Inc. TMPRSS6 iRNA compositions and methods of use thereof
PT2999785T (pt) 2013-05-22 2018-07-09 Alnylam Pharmaceuticals Inc Composições de irna de serpina1 e métodos de uso das mesmas
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
SG11201602503TA (en) 2013-10-03 2016-04-28 Moderna Therapeutics Inc Polynucleotides encoding low density lipoprotein receptor
JP6613227B2 (ja) 2013-10-04 2019-11-27 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 Ａｌａｓ１遺伝子の発現を阻害する組成物および方法
EP3060258A1 (en) 2013-10-22 2016-08-31 Shire Human Genetic Therapies, Inc. Mrna therapy for phenylketonuria
MX2016005238A (es) 2013-10-22 2016-08-12 Shire Human Genetic Therapies Formulaciones de lipidos para la administracion de acido ribonucleico mensajero.
AU2014340092B2 (en) 2013-10-22 2019-09-19 Translate Bio, Inc. mRNA therapy for Argininosuccinate Synthetase Deficiency
WO2015061536A1 (en) 2013-10-25 2015-04-30 Regulus Therapeutics Inc. Microrna compounds and methods for modulating mir-21 activity
SI3071696T1 (sl) 2013-11-22 2019-11-29 Mina Therapeutics Ltd C/EBP alfa kratko delujoči RNA sestavki in postopki uporabe
WO2015089368A2 (en) 2013-12-12 2015-06-18 Alnylam Pharmaceuticals, Inc. Complement component irna compositions and methods of use thereof
CN118845818A (zh) 2014-02-11 2024-10-29 阿尔尼拉姆医药品有限公司 己酮糖激酶（KHK）iRNA组合物及其使用方法
AU2015241198A1 (en) 2014-04-03 2016-11-17 Invictus Oncology Pvt. Ltd. Supramolecular combinatorial therapeutics
AU2015249553B2 (en) 2014-04-23 2021-03-04 Modernatx, Inc. Nucleic acid vaccines
EP3636742B1 (en) 2014-04-25 2025-11-05 Translate Bio, Inc. Methods for purification of messenger rna
CN112852809A (zh) 2014-05-22 2021-05-28 阿尔尼拉姆医药品有限公司 血管紧张素原(AGT)iRNA组合物及其使用方法
CA2949106C (en) 2014-05-30 2023-10-24 Shire Human Genetic Therapies, Inc. Biodegradable lipids for delivery of nucleic acids
EA033966B1 (ru) 2014-06-24 2019-12-13 Транслейт Био, Инк. Стереохимически обогащенные композиции для доставки нуклеиновых кислот
CA3179824A1 (en) 2014-06-25 2015-12-30 Acuitas Therapeutics Inc. Lipids and lipid nanoparticle formulations for delivery of nucleic acids
EP3164379A1 (en) 2014-07-02 2017-05-10 Massachusetts Institute of Technology Polyamine-fatty acid derived lipidoids and uses thereof
TW201613949A (en) 2014-08-07 2016-04-16 Regulus Therapeutics Inc Targeting microRNAs for metabolic disorders
EP4137138A1 (en) 2014-08-29 2023-02-22 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
WO2016049110A1 (en) 2014-09-25 2016-03-31 Cold Spring Harbor Laboratory Treatment of rett syndrome
CA2963271A1 (en) 2014-10-02 2016-04-07 Protiva Biotherapeutics, Inc. 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
AU2015364508A1 (en) 2014-12-18 2017-07-06 Alnylam Pharmaceuticals, Inc. ReversirTM compounds
CA2976445A1 (en) 2015-02-13 2016-08-18 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
DK3313829T3 (da) 2015-06-29 2024-06-17 Acuitas Therapeutics Inc Lipider og lipide nanopartikelformuleringer til levering af nukleinsyrer
US10494632B2 (en) 2015-07-10 2019-12-03 Alnylam Pharmaceuticals, Inc. Insulin-like growth factor binding protein, acid labile subunit (IGFALS) 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
SI3329002T1 (sl) 2015-07-31 2021-02-26 Alnylam Pharmaceuticals, Inc. Sestavki transtiretin (TTR) IRNA in sestavki in postopki njihove uporabe za zdravljenje ali preprečevanje s TTR povezanih bolezni
US11564893B2 (en) 2015-08-17 2023-01-31 Modernatx, Inc. Methods for preparing particles and related compositions
WO2017035278A1 (en) 2015-08-24 2017-03-02 Halo-Bio Rnai Therapeutics, Inc. Polynucleotide nanoparticles for the modulation of gene expression and uses thereof
MA44836A (fr) 2015-08-26 2018-07-04 Univ Texas Procédés de traitement du syndrome polykystique des reins
CN114525280A (zh) 2015-09-02 2022-05-24 阿尔尼拉姆医药品有限公司 程序性细胞死亡1配体1（PD-L1）的iRNA组合物及其使用方法
SI3350157T1 (sl) 2015-09-17 2022-04-29 Modernatx, Inc. Sestave za doziranje terapevtskih sredstev v celice
ES2938557T3 (es) 2015-10-28 2023-04-12 Acuitas Therapeutics Inc Lípidos y formulaciones de nanopartículas lipídicas novedosos para la entrega de ácidos nucleicos
WO2017099823A1 (en) 2015-12-10 2017-06-15 Modernatx, Inc. Compositions and methods for delivery of therapeutic agents
PL3394030T3 (pl) 2015-12-22 2022-04-11 Modernatx, Inc. Związki i kompozycje do wewnątrzkomórkowego dostarczania środków
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
JP6994140B2 (ja) 2016-03-03 2022-02-03 ユニバーシティ オブ マサチューセッツ 非ウイルス的遺伝子導入のための閉鎖型直鎖状二重鎖ｄｎａ
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
EP3458034A4 (en) 2016-05-18 2020-01-01 ModernaTX, Inc. POLYNUCLEOTIDES ENCODING RELAXIN
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)
JP7086870B2 (ja) * 2016-06-30 2022-06-20 アルブータス・バイオファーマー・コーポレイション メッセンジャーｒｎａを送達するための組成物及び方法
US11352647B2 (en) 2016-08-17 2022-06-07 The Broad Institute, Inc. Crispr enzymes and systems
CN110312799A (zh) 2016-08-17 2019-10-08 博德研究所 新型crispr酶和系统
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
US11583504B2 (en) 2016-11-08 2023-02-21 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
WO2018102397A1 (en) 2016-11-29 2018-06-07 PureTech Health LLC Exosomes for delivery of therapeutic agents
MX2019006337A (es) 2016-12-05 2019-09-26 Regulus Therapeutics Inc Métodos para el tratamiento de una enfermedad poliquística renal.
IL322679A (en) 2016-12-05 2025-10-01 Regulus Therapeutics Inc Modified oligonucleotides for the treatment of polycystic kidney disease
TWI790217B (zh) 2016-12-16 2023-01-21 美商阿尼拉製藥公司 使用甲狀腺素運載蛋白（ＴＴＲ）ｉＲＮＡ組成物於治療或預防ＴＴＲ相關疾病之方法
EP3585417B1 (en) 2017-02-27 2023-02-22 Translate Bio, Inc. Method of making a codon-optimized cftr mrna
WO2018165614A1 (en) 2017-03-10 2018-09-13 Rutgers, The State University Of New Jersey Bacterial efflux pump inhibitors
US11180459B2 (en) 2017-03-10 2021-11-23 Rutgers, The State University Of New Jersey Bacterial efflux pump inhibitors
CN110770224B (zh) 2017-03-10 2022-11-18 罗格斯新泽西州立大学 作为外排泵抑制剂的吲哚衍生物
ES3020935T3 (en) 2017-03-15 2025-05-23 Modernatx Inc Lipid nanoparticle formulation
EP3596041B1 (en) 2017-03-15 2022-11-02 ModernaTX, Inc. Compound and compositions for intracellular delivery of therapeutic agents
DK3596042T3 (da) 2017-03-15 2022-04-11 Modernatx Inc Krystalformer af aminolipider
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
KR102720270B1 (ko) 2017-04-18 2024-10-23 알닐람 파마슈티칼스 인코포레이티드 B형 간염 바이러스 (hbv)에 감염된 대상체의 치료 방법
ES2981244T3 (es) 2017-04-28 2024-10-07 Acuitas Therapeutics Inc Novedosas formulaciones de nanopartículas de lípidos de carbonilo y lípidos para la administración de ácidos nucleicos
CA3063531A1 (en) 2017-05-16 2018-11-22 Translate Bio, Inc. Treatment of cystic fibrosis by delivery of codon-optimized mrna encoding cftr
WO2018218192A1 (en) 2017-05-26 2018-11-29 Rutgers, The State University Of New Jersey Bacterial efflux pump inhibitors
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
WO2019005841A1 (en) 2017-06-26 2019-01-03 Rutgers, The State University Of New Jersey THERAPEUTIC COMPOUNDS AND METHODS FOR TREATING 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
JP7355731B2 (ja) 2017-08-16 2023-10-03 アクイタス セラピューティクス インコーポレイテッド 脂質ナノ粒子製剤における使用のための脂質
WO2019036030A1 (en) 2017-08-17 2019-02-21 Acuitas Therapeutics, Inc. LIPIDS FOR USE IN LIPID NANOPARTICLE FORMULATIONS
US11542225B2 (en) 2017-08-17 2023-01-03 Acuitas Therapeutics, Inc. Lipids for use in lipid nanoparticle formulations
US12065396B2 (en) 2017-08-17 2024-08-20 Acuitas Therapeutics, Inc. Lipids for use in lipid nanoparticle formulations
AU2018326799A1 (en) 2017-08-31 2020-02-27 Modernatx, Inc. Methods of making lipid nanoparticles
WO2019048645A1 (en) 2017-09-08 2019-03-14 Mina Therapeutics Limited STABILIZED COMPOSITIONS OF SMALL ACTIVATOR RNA (PARNA) FROM CEBPA AND METHODS OF USE
CA3075219A1 (en) 2017-09-08 2019-03-14 Mina Therapeutics Limited Hnf4a sarna compositions 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
EP3704252A1 (en) 2017-11-01 2020-09-09 Alnylam Pharmaceuticals, Inc. Complement component c3 irna compositions and methods of use thereof
EP3710039A4 (en) 2017-11-13 2021-08-04 The Broad Institute, Inc. METHODS AND COMPOSITIONS FOR TREATMENT OF CANCER BY TARGETING THE CLEC2D-KLRB1 PATH
WO2019100039A1 (en) 2017-11-20 2019-05-23 Alnylam Pharmaceuticals, Inc. Serum amyloid p component (apcs) irna compositions and methods of use thereof
US20190175560A1 (en) 2017-12-01 2019-06-13 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
EP3775211B1 (en) 2018-04-12 2023-04-05 MiNA Therapeutics Limited Sirt1-sarna compositions and methods of use
JP2021534101A (ja) 2018-08-09 2021-12-09 ヴェルソー セラピューティクス， インコーポレイテッド Ｃｃｒ２及びｃｓｆ１ｒを標的とするためのオリゴヌクレオチド組成物ならびにその使用
AR115960A1 (es) 2018-08-16 2021-03-17 Alnylam Pharmaceuticals Inc Composiciones y métodos para inhibir la expresión del gen lect2
CN118421617A (zh) 2018-08-24 2024-08-02 川斯勒佰尔公司 用于纯化信使rna的方法
CA3105385A1 (en) 2018-09-18 2020-03-26 Alnylam Pharmaceuticals, Inc. Ketohexokinase (khk) irna compositions and methods of use thereof
MA53652A (fr) 2018-09-19 2021-07-28 Modernatx Inc Lipides peg de haute pureté et leurs utilisations
EP3853202A1 (en) 2018-09-19 2021-07-28 ModernaTX, Inc. Compounds and compositions for intracellular delivery of therapeutic agents
JP7526168B2 (ja) 2018-09-19 2024-07-31 モデルナティエックス インコーポレイテッド Ｐｅｇ脂質及びそれらの使用
US12090235B2 (en) 2018-09-20 2024-09-17 Modernatx, Inc. Preparation of lipid nanoparticles and methods of administration thereof
EP3852911B1 (en) 2018-09-21 2025-01-22 Acuitas Therapeutics, Inc. Systems and methods for manufacturing lipid nanoparticles and liposomes
AU2019351809C1 (en) 2018-10-01 2025-09-11 Alnylam Pharmaceuticals, Inc. Biodegradable lipids for the delivery of active agents
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
EP3876999A4 (en) 2018-11-09 2022-08-31 Arbutus Biopharma Corporation NEGATIVELY CHARGED PEG-LIPID CONJUGATES
CN112996568A (zh) 2018-11-13 2021-06-18 莱古路斯治疗法股份有限公司 用于调节mir-10b活性的微小rna化合物和方法
MX2021005969A (es) 2018-11-21 2021-09-14 Translate Bio Inc Tratamiento de la fibrosis quística mediante el suministro de arnm que codifica cftr nebulizado.
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
JP2022515744A (ja) 2018-12-20 2022-02-22 プラクシス プレシジョン メディシンズ， インコーポレイテッド Ｋｃｎｔ１関連障害の治療のための組成物及び方法
US12268669B2 (en) 2018-12-20 2025-04-08 Pfizer Inc. Pharmaceutical compositions and methods comprising a combination of a benzoxazole transthyretin stabilizer and an additional therapeutic agent
EP4450487A3 (en) 2019-01-11 2024-12-25 Acuitas Therapeutics, Inc. Lipids for lipid nanoparticle delivery of active agents
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
US12534714B2 (en) 2019-03-18 2026-01-27 The Broad Institute, Inc. Type VII CRISPR proteins and systems
EP3953473A1 (en) 2019-04-12 2022-02-16 MiNA Therapeutics Limited Sirt1-sarna compositions and methods of use
WO2020236972A2 (en) 2019-05-20 2020-11-26 The Broad Institute, Inc. Non-class i multi-component nucleic acid targeting systems
US20220267778A1 (en) 2019-07-30 2022-08-25 Shionogi & Co., Ltd. Nucleic acid drug targeting murf1
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
JP7805286B2 (ja) 2019-09-03 2026-01-23 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 Ｌｅｃｔ２遺伝子の発現を阻害するための組成物および方法
JP7700101B2 (ja) * 2019-09-06 2025-06-30 ジェネレーション バイオ カンパニー 閉端ｄｎａおよび切断可能脂質を含む脂質ナノ粒子組成物ならびにそれらの使用方法
JP7638972B2 (ja) 2019-09-19 2025-03-04 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分岐状尾部脂質化合物及び組成物
CN114729376A (zh) 2019-09-23 2022-07-08 欧米茄治疗公司 用于调节肝细胞核因子4α(HNF4α)基因表达的组合物和方法
AU2020355000A1 (en) 2019-09-23 2022-03-17 Omega Therapeutics, Inc. Compositions and methods for modulating apolipoprotein B (APOB) gene expression
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
US12503699B2 (en) 2019-10-04 2025-12-23 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
EP4061945A1 (en) 2019-11-22 2022-09-28 Alnylam Pharmaceuticals, Inc. Ataxin3 (atxn3) rnai agent compositions and methods of use thereof
BR112022011417A2 (pt) 2019-12-13 2022-08-30 Alnylam Pharmaceuticals Inc Composições do agente de irna da fase de leitura aberta 72 do cromossomo humano 9 (c9orf72) e métodos de uso das mesmas
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)
WO2021155274A1 (en) * 2020-01-31 2021-08-05 Modernatx, Inc. Methods of preparing lipid nanoparticles
WO2021163066A1 (en) 2020-02-10 2021-08-19 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
CN116096886A (zh) 2020-03-11 2023-05-09 欧米茄治疗公司 用于调节叉头框p3(foxp3)基因表达的组合物和方法
AU2021244555A1 (en) 2020-03-24 2022-11-24 Generation Bio Co. Non-viral dna vectors and uses thereof for expressing factor ix therapeutics
IL296662A (en) 2020-03-24 2022-11-01 Generation Bio Co Non-viral dna vectors and uses thereof for expressing gaucher therapeutics
JP2023519274A (ja) 2020-03-26 2023-05-10 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 コロナウイルスｉＲＮＡ組成物およびその使用方法
WO2021202443A2 (en) 2020-03-30 2021-10-07 Alnylam Pharmaceucticals, Inc. Compositions and methods for silencing dnajc15 gene expression
WO2021202902A1 (en) 2020-04-01 2021-10-07 Alnylam Pharmaceuticals, Inc. ALPHA-2A ADRENERGIC RECEPTOR (ADRA2A) iRNA AGENT COMPOSITIONS AND METHODS OF USE THEREOF
AU2021252545A1 (en) 2020-04-06 2022-11-03 Alnylam Pharmaceuticals, Inc. Compositions and methods for silencing myoc expression
EP4133077A1 (en) 2020-04-07 2023-02-15 Alnylam Pharmaceuticals, Inc. Transmembrane serine protease 2 (tmprss2) irna compositions and methods of use thereof
WO2021206917A1 (en) 2020-04-07 2021-10-14 Alnylam Pharmaceuticals, Inc. ANGIOTENSIN-CONVERTING ENZYME 2 (ACE2) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
KR20230008078A (ko) 2020-04-07 2023-01-13 알닐람 파마슈티칼스 인코포레이티드 Scn9a 발현을 사일런싱하기 위한 조성물 및 방법
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
AR122534A1 (es) 2020-06-03 2022-09-21 Triplet Therapeutics Inc Métodos para el tratamiento de los trastornos de expansión por repetición de nucleótidos asociados con la actividad de msh3
EP4162050A1 (en) 2020-06-09 2023-04-12 Alnylam Pharmaceuticals, Inc. Rnai compositions and methods of use thereof for delivery by inhalation
EP4649951A2 (en) 2020-06-09 2025-11-19 Alnylam Pharmaceuticals, Inc. Sirna compositions and methods for silencing gpam (glycerol-3-phosphate acyltransferase 1, mitochondrial) expression
EP4182297B1 (en) 2020-07-16 2025-09-03 Acuitas Therapeutics, Inc. Cationic lipids for use in lipid nanoparticles
KR20230056669A (ko) 2020-07-27 2023-04-27 안자리움 바이오사이언시스 아게 Dna 분자의 조성물, 이의 제조 방법 및 이의 사용 방법
EP4217489A1 (en) 2020-09-24 2023-08-02 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
EP4232582A1 (en) 2020-10-23 2023-08-30 Alnylam Pharmaceuticals, Inc. Mucin 5b (muc5b) irna compositions and methods of use thereof
EP4256053A1 (en) 2020-12-01 2023-10-11 Alnylam Pharmaceuticals, Inc. Methods and compositions for inhibition of hao1 (hydroxyacid oxidase 1 (glycolate oxidase)) gene expression
CA3210763A1 (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
TW202305133A (zh) 2021-03-26 2023-02-01 英商米納治療有限公司 Tmem173 sarna組合物及其使用方法
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
KR20240012370A (ko) 2021-04-20 2024-01-29 안자리움 바이오사이언시스 아게 아밀로-알파-1, 6-글루코시다제, 4-알파-글루카노트랜스퍼라제를 인코딩하는 dna 분자의 조성물, 이를 제조하는 방법 및 이를 사용하는 방법
JP2024515788A (ja) 2021-04-27 2024-04-10 ジェネレーション バイオ カンパニー 治療抗体を発現する非ウイルスｄｎａベクター及びその使用
EP4329884A1 (en) 2021-04-27 2024-03-06 Generation Bio Co. Non-viral dna vectors expressing anti-coronavirus antibodies and uses thereof
EP4330396A1 (en) 2021-04-29 2024-03-06 Alnylam Pharmaceuticals, Inc. Signal transducer and activator of transcription factor 6 (stat6) irna compositions and methods of use thereof
JP2024522068A (ja) 2021-05-18 2024-06-11 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 ナトリウム－グルコース共輸送体２（ｓｇｌｔ２）ｉｒｎａ組成物およびその使用方法
EP4342881A4 (en) * 2021-05-20 2025-08-27 Beijing Institute Tech COMPOUND, LIPOSOME AND THEIR USES
CN117561334A (zh) 2021-06-04 2024-02-13 阿尔尼拉姆医药品有限公司 人染色体9开放阅读框72（C9ORF72）iRNA药剂组合物和其使用方法
EP4367242A2 (en) 2021-07-07 2024-05-15 Omega Therapeutics, Inc. Compositions and methods for modulating secreted frizzled receptor protein 1 (sfrp1) gene expression
JP2024530018A (ja) 2021-08-03 2024-08-14 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 トランスサイレチン（ＴＴＲ）ｉＲＮＡ組成物およびその使用方法
IL311030A (en) 2021-08-31 2024-04-01 Alnylam Pharmaceuticals Inc DFFA-like Cell Death Inducer B (CIDEB) IRNA compositions and methods of using them
WO2023044343A1 (en) 2021-09-14 2023-03-23 Renagade Therapeutics Management Inc. Acyclic lipids and methods of use thereof
EP4402123A1 (en) 2021-09-14 2024-07-24 Renagade Therapeutics Management Inc. Cyclic lipids and methods of use thereof
KR20240067943A (ko) 2021-09-24 2024-05-17 알닐람 파마슈티칼스 인코포레이티드 미소관 연관 단백질 타우(MAPT) iRNA 제제 조성물 및 이의 사용 방법
CA3234547A1 (en) 2021-10-08 2023-04-13 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
TW202334418A (zh) 2021-10-29 2023-09-01 美商艾拉倫製藥股份有限公司 杭丁頓（ＨＴＴ）ｉＲＮＡ劑組成物及其使用方法
JP2024542155A (ja) 2021-11-08 2024-11-13 オルナ セラピューティクス、インコーポレイテッド 環状ポリヌクレオチドを送達するための脂質ナノ粒子組成物
WO2023092102A1 (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
AU2022421244A1 (en) 2021-12-22 2024-07-11 Camp4 Therapeutics Corporation 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
EP4452928A1 (en) 2021-12-23 2024-10-30 Renagade Therapeutics Management Inc. Constrained lipids and methods of use thereof
CN118414149A (zh) * 2021-12-23 2024-07-30 三养控股公司 用于肺的药物递送的纳米颗粒组合物
JP2025504404A (ja) 2022-01-14 2025-02-12 アンジャリウム バイオサイエンシズ エージー Ｖｉｉｉ因子をコードするｄｎａ分子の組成物、それを作製する方法、及びその使用方法
WO2023141314A2 (en) 2022-01-24 2023-07-27 Alnylam Pharmaceuticals, Inc. Heparin sulfate biosynthesis pathway enzyme irna agent compositions and methods of use thereof
CN116514672B (zh) * 2022-01-30 2025-06-27 康希诺生物股份公司 一种用于核酸递送的可电离脂质及其lnp组合物和疫苗
WO2023143591A1 (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
WO2023170435A1 (en) 2022-03-07 2023-09-14 Mina Therapeutics Limited Il10 sarna compositions and methods of use
CA3245737A1 (en) 2022-03-14 2023-09-21 Generation Bio Co. PRIME-BOOST HETEROLOGICAL VACCINE COMPOSITIONS AND METHODS OF USE
CA3255225A1 (en) 2022-04-04 2023-10-12 The Regents Of The University Of California COMPOSITIONS AND METHODS OF GENETIC COMPLEMENTATION
AU2023251104A1 (en) 2022-04-07 2024-10-17 Renagade Therapeutics Management Inc. Cyclic lipids and lipid nanoparticles (lnp) for the delivery of nucleic acids or peptides for use in vaccinating against infectious agents
US20240002857A1 (en) 2022-05-09 2024-01-04 Sanegene Bio Usa Inc. Double stranded rna targeting 17-beta hydroxysteroiddehydrogenase 13 (hsd17b13) and methods of use thereof
JP2025523368A (ja) 2022-05-30 2025-07-23 シャンハイ サーコード バイオメッド カンパニー，リミティド 合成環状ｒｎａ組成物及びその使用方法
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
AU2023283551A1 (en) 2022-06-10 2024-12-19 Camp4 Therapeutics Corporation Methods of modulating progranulin expression using antisense oligonucleotides targeting regulatory rnas
EP4565597A2 (en) 2022-08-03 2025-06-11 Voyager Therapeutics, Inc. Compositions and methods for crossing the blood brain barrier
AU2023320578A1 (en) 2022-08-05 2025-02-13 Sanegene Bio Usa Inc. Double stranded rna targeting angiotensinogen (agt) and methods of use thereof
WO2024040222A1 (en) 2022-08-19 2024-02-22 Generation Bio Co. Cleavable closed-ended dna (cedna) and methods of use thereof
MA71735A (fr) 2022-09-15 2025-05-30 Regeneron Pharmaceuticals, Inc. Compositions d'arni de 17b-hydroxystéroïde déshydrogénase de type 13 (hsd17b13) et leurs procédés d'utilisation
CA3268237A1 (en) 2022-09-23 2024-03-28 Nchroma Bio, Inc. COMPOSITIONS AND METHODS FOR THE EPIGENETIC REGULATION OF HBV GENE EXPRESSION
JP2025534701A (ja) 2022-10-14 2025-10-17 セーンジーン バイオ ユーエスエー インコーポレイティド Ｃ３を標的とする低分子干渉ｒｎａおよびその使用
AU2023373632A1 (en) * 2022-11-01 2025-05-01 Samyang Holdings Corporation Lipid having amide and ester functional groups, and method for producing same
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
EP4615517A1 (en) 2022-11-08 2025-09-17 Orna Therapeutics, Inc. Circular rna compositions
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
JP2025540101A (ja) 2022-12-01 2025-12-11 キャンプ４ セラピューティクス コーポレイション 調節ｒｎａを標的とするアンチセンスオリゴヌクレオチドを用いたｓｙｎｇａｐ１遺伝子転写の調節
CN120641085A (zh) 2022-12-01 2025-09-12 世代生物公司 包含核酸、可电离脂质、固醇、脂质锚定聚合物和辅助脂质的脂质纳米颗粒、其用途
WO2024119103A1 (en) 2022-12-01 2024-06-06 Generation Bio Co. Lipid nanoparticles comprising nucleic acids and lipid-anchored polymers
CN120641465A (zh) 2022-12-01 2025-09-12 世代生物公司 新颖的聚甘油缀合脂质和包含其的脂质纳米颗粒组合物
EP4626401A1 (en) 2022-12-01 2025-10-08 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
WO2024168010A2 (en) 2023-02-09 2024-08-15 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
WO2024196998A1 (en) 2023-03-22 2024-09-26 Regulus Therapeutics, Inc. Methods for treating nervous system disorders
WO2024205657A2 (en) 2023-03-29 2024-10-03 Orna Therapeutics, Inc. Lipids and lipid nanoparticle compositions for delivering polynucleotides
TW202442241A (zh) 2023-04-12 2024-11-01 美商雷格勒斯治療公司 用於治療多囊性腎病之方法
WO2024233308A2 (en) 2023-05-05 2024-11-14 Orna Therapeutics, Inc. Circular rna compositions and methods
WO2024238700A1 (en) 2023-05-15 2024-11-21 Chroma Medicine, Inc. Compositions and methods for epigenetic regulation of hbv gene expression
AU2024270764A1 (en) 2023-05-15 2025-12-04 Nchroma Bio, 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
WO2024263915A2 (en) 2023-06-21 2024-12-26 Sanegene Bio Usa Inc. Double stranded rna targeting proprotein convertase subtilisin kexin 9 (pcsk9) and methods of use thereof
WO2025015335A1 (en) 2023-07-13 2025-01-16 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
WO2025129158A1 (en) 2023-12-15 2025-06-19 The Broad Institute, Inc. Engineered arc delivery vesicles and uses thereof
WO2025129128A1 (en) 2023-12-15 2025-06-19 Vivasor, Inc. Compositions and methods for non-viral delivery of therapeutic compounds
WO2025166325A1 (en) 2024-02-02 2025-08-07 Editas Medicine, Inc. MODIFIED GUIDE RNAs
TW202539703A (zh) 2024-03-11 2025-10-16 美商雷格勒斯治療公司 治療多囊性腎病之方法
US20250313840A1 (en) 2024-03-20 2025-10-09 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
WO2026027887A2 (en) 2024-08-02 2026-02-05 Mina Therapeutics Limited Hbg1/2-sarna compositions and methods of use

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
GB1561618A (en) * 1976-10-18 1980-02-27 Texaco Development Corp Polyurethane formation using high tertiary amine content 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
US5166315A (en) 1989-12-20 1992-11-24 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
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
US5405938A (en) 1989-12-20 1995-04-11 Anti-Gene Development Group Sequence-specific binding polymers for duplex nucleic acids
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
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
US4933470A (en) * 1987-10-05 1990-06-12 Neorx Corporation Method of synthesis of vicinal diamines
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
US5188897A (en) 1987-10-22 1993-02-23 Temple University Of The Commonwealth System Of Higher Education Encapsulated 2',5'-phosphorothioate oligoadenylates
JPH02767A (ja) * 1987-12-17 1990-01-05 Shionogi & Co Ltd 脂質誘導体
JPH03503894A (ja) 1988-03-25 1991-08-29 ユニバーシィティ　オブ　バージニア　アランミ　パテンツ　ファウンデイション オリゴヌクレオチド　ｎ‐アルキルホスホラミデート
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
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
US5587361A (en) 1991-10-15 1996-12-24 Isis Pharmaceuticals, Inc. Oligonucleotides having phosphorothioate linkages of high chiral purity
US5506351A (en) 1992-07-23 1996-04-09 Isis Pharmaceuticals Process for the preparation of 2'-O-alkyl guanosine and related compounds
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
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
US5378825A (en) 1990-07-27 1995-01-03 Isis Pharmaceuticals, Inc. Backbone modified oligonucleotide analogs
US5386023A (en) 1990-07-27 1995-01-31 Isis Pharmaceuticals Backbone modified oligonucleotide analogs and preparation thereof through reductive coupling
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
US5618704A (en) 1990-07-27 1997-04-08 Isis Pharmacueticals, Inc. Backbone-modified oligonucleotide analogs and preparation thereof through radical coupling
US5541307A (en) 1990-07-27 1996-07-30 Isis Pharmaceuticals, Inc. Backbone modified oligonucleotide analogs and solid phase synthesis thereof
US5602240A (en) 1990-07-27 1997-02-11 Ciba Geigy Ag. Backbone modified oligonucleotide analogs
US5623070A (en) 1990-07-27 1997-04-22 Isis Pharmaceuticals, Inc. Heteroatomic oligonucleoside linkages
US5608046A (en) 1990-07-27 1997-03-04 Isis Pharmaceuticals, Inc. Conjugated 4'-desmethyl nucleoside analog compounds
US5610289A (en) 1990-07-27 1997-03-11 Isis Pharmaceuticals, Inc. Backbone modified oligonucleotide analogues
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
IL113519A (en) 1990-08-03 1997-11-20 Sterling Winthrop Inc Oligonucleoside sequences of from about 6 to about 200 bases having a three atom internucleoside linkage, their preparation and pharmaceutical compositions for inhibiting gene expression containing said oligonucleosides
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
WO1992005186A1 (en) 1990-09-20 1992-04-02 Gilead Sciences 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
EP0621944B1 (en) 1991-07-25 1997-03-05 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
CA2122030C (en) 1991-10-24 1997-03-04 Muthiah Manoharan Derivatized oligonucleotides having improved uptake and other properties
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
WO1994022891A1 (en) 1993-03-31 1994-10-13 Sterling Winthrop Inc. Oligonucleotides with amide linkages replacing phosphodiester linkages
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
WO1998051278A2 (en) * 1997-05-14 1998-11-19 Inex Pharmaceuticals Corporation High efficiency encapsulation of charged therapeutic agents in lipid vesicles
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
CZ2002140A3 (cs) 1999-07-14 2002-05-15 Alza Corporation Neutrální lipopolymer a lipozomové kompozice, které jej obsahují
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
EP1292284A2 (en) 2000-06-09 2003-03-19 Teni Boulikas Encapsulation of polynucleotides and drugs into targeted liposomes
NZ528216A (en) * 2001-02-24 2006-12-22 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
US20050064595A1 (en) * 2003-07-16 2005-03-24 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
CA2540646A1 (en) * 2003-10-01 2005-04-14 Bayer Healthcare Ag Antibacterial amide macrocycles
EP1766035B1 (en) * 2004-06-07 2011-12-07 Protiva Biotherapeutics Inc. Lipid encapsulated interfering rna
US7745651B2 (en) 2004-06-07 2010-06-29 Protiva Biotherapeutics, Inc. Cationic lipids and methods of use
AU2005259799A1 (en) 2004-07-02 2006-01-12 Protiva Biotherapeutics, Inc. Immunostimulatory siRNA molecules and uses therefor
AU2005259685B2 (en) * 2004-07-07 2010-04-08 Statens Serum Institut Compositions and methods for stabilizing lipid based adjuvant formulations using glycolipids
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
AU2005303610B2 (en) * 2004-11-12 2011-07-21 Ucl Business Plc Guanidine derivatives as inhibitors of DDAH
GB0425556D0 (en) * 2004-11-19 2004-12-22 Glaxo Group Ltd Novel compounds
GB0425555D0 (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.
WO2006102163A2 (en) * 2005-03-17 2006-09-28 Invitrogen Corporation Transfection reagents for non-adherent suspension cells
AU2006259415B2 (en) * 2005-06-15 2012-08-30 Massachusetts Institute Of Technology Amine-containing lipids and uses thereof
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
CA2848238C (en) 2006-10-03 2016-07-19 Tekmira Pharmaceuticals Corporation Lipid containing formulations
EP3100718B1 (en) * 2008-01-02 2019-11-27 Arbutus Biopharma Corporation Improved compositions and methods for the delivery of nucleic acids

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