JP5749494B2 - 核酸の送達のための改善された組成物および方法 - Google Patents

核酸の送達のための改善された組成物および方法 Download PDF

Info

Publication number
JP5749494B2
JP5749494B2 JP2010541545A JP2010541545A JP5749494B2 JP 5749494 B2 JP5749494 B2 JP 5749494B2 JP 2010541545 A JP2010541545 A JP 2010541545A JP 2010541545 A JP2010541545 A JP 2010541545A JP 5749494 B2 JP5749494 B2 JP 5749494B2
Authority
JP
Japan
Prior art keywords
lipid
peg
sirna
nucleic acid
dma
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.)
Expired - Fee Related
Application number
JP2010541545A
Other languages
English (en)
Japanese (ja)
Other versions
JP2011509258A (ja
JP2011509258A5 (enExample
Inventor
マイケル ジェイ． ホープ，
マイケル ジェイ． ホープ，
ショーン シー． センプル，
ショーン シー． センプル，
チャンシン チェン，
チャンシン チェン，
マデン，　トーマス　ディー．
トーマス ディー． マデン，
バーバラ ムイ，
バーバラ ムイ，
ピーター アール． カリス，
ピーター アール． カリス，
マルコ エー． シウフォリーニ，
マルコ エー． シウフォリーニ，
キム エフ． ワン，
キム エフ． ワン，
マティアー マノハラン，
マティアー マノハラン，
ラジーブ ジー． カランソッタティル，
ラジーブ ジー． カランソッタティル，
スリニバスル マスナ，
スリニバスル マスナ，
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 JP2011509258A publication Critical patent/JP2011509258A/ja
Publication of JP2011509258A5 publication Critical patent/JP2011509258A5/ja
Application granted granted Critical
Publication of JP5749494B2 publication Critical patent/JP5749494B2/ja
Expired - Fee Related legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Classifications

    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • 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
    • 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
    • A61K9/1275—Lipoproteins or protein-free species thereof, e.g. chylomicrons; Artificial high-density lipoproteins [HDL], low-density lipoproteins [LDL] or very-low-density lipoproteins [VLDL]; Precursors thereof
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/04—Antibacterial agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12—Antivirals
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P33/00—Antiparasitic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00—Drugs for immunological or allergic disorders
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00—Drugs for immunological or allergic disorders
    • A61P37/02—Immunomodulators
    • A61P37/04—Immunostimulants
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07C—ACYCLIC OR CARBOCYCLIC COMPOUNDS
    • C07C215/00—Compounds containing amino and hydroxy groups bound to the same carbon skeleton
    • C07C215/02—Compounds containing amino and hydroxy groups bound to the same carbon skeleton having hydroxy groups and amino groups bound to acyclic carbon atoms of the same carbon skeleton
    • C07C215/04—Compounds containing amino and hydroxy groups bound to the same carbon skeleton having hydroxy groups and amino groups bound to acyclic carbon atoms of the same carbon skeleton the carbon skeleton being saturated
    • C07C215/06—Compounds containing amino and hydroxy groups bound to the same carbon skeleton having hydroxy groups and amino groups bound to acyclic carbon atoms of the same carbon skeleton the carbon skeleton being saturated and acyclic
    • C07C215/10—Compounds containing amino and hydroxy groups bound to the same carbon skeleton having hydroxy groups and amino groups bound to acyclic carbon atoms of the same carbon skeleton the carbon skeleton being saturated and acyclic with one amino group and at least two hydroxy groups bound to the carbon skeleton
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07C—ACYCLIC OR CARBOCYCLIC COMPOUNDS
    • C07C217/00—Compounds containing amino and etherified hydroxy groups bound to the same carbon skeleton
    • C07C217/02—Compounds containing amino and etherified hydroxy groups bound to the same carbon skeleton having etherified hydroxy groups and amino groups bound to acyclic carbon atoms of the same carbon skeleton
    • C07C217/04—Compounds containing amino and etherified hydroxy groups bound to the same carbon skeleton having etherified hydroxy groups and amino groups bound to acyclic carbon atoms of the same carbon skeleton the carbon skeleton being acyclic and saturated
    • C07C217/06—Compounds containing amino and etherified hydroxy groups bound to the same carbon skeleton having etherified hydroxy groups and amino groups bound to acyclic carbon atoms of the same carbon skeleton the carbon skeleton being acyclic and saturated having only one etherified hydroxy group and one amino group bound to the carbon skeleton, which is not further substituted
    • C07C217/08—Compounds containing amino and etherified hydroxy groups bound to the same carbon skeleton having etherified hydroxy groups and amino groups bound to acyclic carbon atoms of the same carbon skeleton the carbon skeleton being acyclic and saturated having only one etherified hydroxy group and one amino group bound to the carbon skeleton, which is not further substituted the oxygen atom of the etherified hydroxy group being further bound to an acyclic carbon atom
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07C—ACYCLIC OR CARBOCYCLIC COMPOUNDS
    • C07C217/00—Compounds containing amino and etherified hydroxy groups bound to the same carbon skeleton
    • C07C217/02—Compounds containing amino and etherified hydroxy groups bound to the same carbon skeleton having etherified hydroxy groups and amino groups bound to acyclic carbon atoms of the same carbon skeleton
    • C07C217/04—Compounds containing amino and etherified hydroxy groups bound to the same carbon skeleton having etherified hydroxy groups and amino groups bound to acyclic carbon atoms of the same carbon skeleton the carbon skeleton being acyclic and saturated
    • C07C217/28—Compounds containing amino and etherified hydroxy groups bound to the same carbon skeleton having etherified hydroxy groups and amino groups bound to acyclic carbon atoms of the same carbon skeleton the carbon skeleton being acyclic and saturated having one amino group and at least two singly-bound oxygen atoms, with at least one being part of an etherified hydroxy group, bound to the carbon skeleton, e.g. ethers of polyhydroxy amines
    • 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
    • C07C219/08—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 at least one of the hydroxy groups esterified by a carboxylic acid having the esterifying carboxyl group bound to an acyclic carbon atom of an acyclic unsaturated carbon skeleton
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07C—ACYCLIC OR CARBOCYCLIC COMPOUNDS
    • C07C229/00—Compounds containing amino and carboxyl groups bound to the same carbon skeleton
    • C07C229/02—Compounds containing amino and carboxyl groups bound to the same carbon skeleton having amino and carboxyl groups bound to acyclic carbon atoms of the same carbon skeleton
    • C07C229/04—Compounds containing amino and carboxyl groups bound to the same carbon skeleton having amino and carboxyl groups bound to acyclic carbon atoms of the same carbon skeleton the carbon skeleton being acyclic and saturated
    • C07C229/06—Compounds containing amino and carboxyl groups bound to the same carbon skeleton having amino and carboxyl groups bound to acyclic carbon atoms of the same carbon skeleton the carbon skeleton being acyclic and saturated having only one amino and one carboxyl group bound to the carbon skeleton
    • C07C229/10—Compounds containing amino and carboxyl groups bound to the same carbon skeleton having amino and carboxyl groups bound to acyclic carbon atoms of the same carbon skeleton the carbon skeleton being acyclic and saturated having only one amino and one carboxyl group bound to the carbon skeleton the nitrogen atom of the amino group being further bound to acyclic carbon atoms or to carbon atoms of rings other than six-membered aromatic rings
    • C07C229/12—Compounds containing amino and carboxyl groups bound to the same carbon skeleton having amino and carboxyl groups bound to acyclic carbon atoms of the same carbon skeleton the carbon skeleton being acyclic and saturated having only one amino and one carboxyl group bound to the carbon skeleton the nitrogen atom of the amino group being further bound to acyclic carbon atoms or to carbon atoms of rings other than six-membered aromatic rings to carbon atoms of acyclic carbon skeletons
    • 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
    • C07C271/12—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 to hydrogen atoms or to carbon atoms of unsubstituted hydrocarbon radicals
    • 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
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D317/00—Heterocyclic compounds containing five-membered rings having two oxygen atoms as the only ring hetero atoms
    • C07D317/08—Heterocyclic compounds containing five-membered rings having two oxygen atoms as the only ring hetero atoms having the hetero atoms in positions 1 and 3
    • C07D317/10—Heterocyclic compounds containing five-membered rings having two oxygen atoms as the only ring hetero atoms having the hetero atoms in positions 1 and 3 not condensed with other rings
    • C07D317/14—Heterocyclic compounds containing five-membered rings having two oxygen atoms as the only ring hetero atoms having the hetero atoms in positions 1 and 3 not condensed with other rings with substituted hydrocarbon radicals attached to ring carbon atoms
    • C07D317/28—Radicals substituted by nitrogen atoms

Landscapes

  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • General Health & Medical Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Epidemiology (AREA)
  • Immunology (AREA)
  • Dispersion Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Communicable Diseases (AREA)
  • Oncology (AREA)
  • Molecular Biology (AREA)
  • Virology (AREA)
  • Tropical Medicine & Parasitology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
  • Organic Low-Molecular-Weight Compounds And Preparation Thereof (AREA)
  • Peptides Or Proteins (AREA)
JP2010541545A 2008-01-02 2008-12-31 核酸の送達のための改善された組成物および方法 Expired - Fee Related JP5749494B2 (ja)

Applications Claiming Priority (9)

Application Number Priority Date Filing Date Title
US1861608P 2008-01-02 2008-01-02
US1862708P 2008-01-02 2008-01-02
US61/018,627 2008-01-02
US61/018,616 2008-01-02
US3974808P 2008-03-26 2008-03-26
US61/039,748 2008-03-26
US4956808P 2008-05-01 2008-05-01
US61/049,568 2008-05-01
PCT/US2008/088676 WO2009086558A1 (en) 2008-01-02 2008-12-31 Improved compositions and methods for the delivery of nucleic acids

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2014029321A Division JP2014132014A (ja) 2008-01-02 2014-02-19 核酸の送達のための改善された組成物および方法

Publications (3)

Publication Number Publication Date
JP2011509258A JP2011509258A (ja) 2011-03-24
JP2011509258A5 JP2011509258A5 (enExample) 2012-12-27
JP5749494B2 true JP5749494B2 (ja) 2015-07-15

Family

ID=40824751

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2010541545A Expired - Fee Related JP5749494B2 (ja) 2008-01-02 2008-12-31 核酸の送達のための改善された組成物および方法
JP2014029321A Withdrawn JP2014132014A (ja) 2008-01-02 2014-02-19 核酸の送達のための改善された組成物および方法

Family Applications After (1)

Application Number Title Priority Date Filing Date
JP2014029321A Withdrawn JP2014132014A (ja) 2008-01-02 2014-02-19 核酸の送達のための改善された組成物および方法

Country Status (5)

Country Link
US (1) US20110117125A1 (enExample)
EP (2) EP3100718B1 (enExample)
JP (2) JP5749494B2 (enExample)
CA (2) CA3044134A1 (enExample)
WO (1) WO2009086558A1 (enExample)

Families Citing this family (471)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US9228186B2 (en) 2002-11-14 2016-01-05 Thermo Fisher Scientific Inc. Methods and compositions for selecting siRNA of improved functionality
US7605251B2 (en) 2006-05-11 2009-10-20 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of the PCSK9 gene
CA3144493A1 (en) * 2006-10-03 2008-04-10 Arbutus Biopharma Corporation Lipid containing formulations
JP5350360B2 (ja) 2007-03-29 2013-11-27 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 エボラ由来の遺伝子の発現を阻害するための組成物および方法
US9006191B2 (en) 2007-12-27 2015-04-14 Protiva Biotherapeutics, Inc. Silencing of polo-like kinase expression using interfering RNA
CA2713379A1 (en) * 2008-01-31 2009-11-05 Alnylam Pharmaceuticals, Inc. Optimized methods for delivery of dsrna targeting the pcsk9 gene
WO2009129319A2 (en) 2008-04-15 2009-10-22 Protiva Biotherapeutics, Inc. Silencing of csn5 gene expression using interfering rna
EP2279254B1 (en) 2008-04-15 2017-07-05 Protiva Biotherapeutics Inc. Novel lipid formulations for nucleic acid delivery
WO2009132131A1 (en) * 2008-04-22 2009-10-29 Alnylam Pharmaceuticals, Inc. Amino lipid based improved lipid formulation
US8546554B2 (en) 2008-09-25 2013-10-01 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
EP2937418B1 (en) 2008-10-20 2017-10-18 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of transthyretin
AU2009320006A1 (en) 2008-10-30 2010-06-03 Peixuan Guo Membrane-integrated viral DNA-packaging motor protein connector biosensor for DNA sequencing and other uses
WO2010054384A1 (en) 2008-11-10 2010-05-14 Alnylam Pharmaceuticals, Inc. Lipids and compositions for the delivery of therapeutics
EP3699172A3 (en) 2008-11-10 2020-11-18 Arbutus Biopharma Corporation Novel lipids and compositions for the delivery of therapeutics
EP2373382B1 (en) 2008-12-10 2017-02-15 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
CA2754043A1 (en) * 2009-03-12 2010-09-16 Alnylam Pharmaceuticals, Inc. Lipid formulated compositions and methods for inhibiting expression of eg5 and vegf genes
CA2760706C (en) * 2009-05-05 2019-08-06 Alnylam Pharmaceuticals, Inc. Methods of delivering oligonucleotides to immune cells
KR20220038506A (ko) 2009-06-10 2022-03-28 알닐람 파마슈티칼스 인코포레이티드 향상된 지질 조성물
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
ES2613498T3 (es) 2009-07-01 2017-05-24 Protiva Biotherapeutics Inc. Nuevas formulaciones de lípidos para el suministro de agentes terapéuticos a tumores sólidos
EP2449106B1 (en) * 2009-07-01 2015-04-08 Protiva Biotherapeutics Inc. Compositions and methods for silencing apolipoprotein b
AU2010270337B2 (en) 2009-07-09 2016-09-22 Biontech Delivery Technologies Gmbh Amphoteric liposomes comprising imino lipids
WO2011011447A1 (en) 2009-07-20 2011-01-27 Protiva Biotherapeutics, Inc. Compositions and methods for silencing ebola virus gene expression
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
US9181295B2 (en) 2009-08-20 2015-11-10 Sirna Therapeutics, Inc. Cationic lipids with various head groups for oligonucleotide delivery
US9187746B2 (en) 2009-09-22 2015-11-17 Alnylam Pharmaceuticals, Inc. Dual targeting siRNA agents
WO2011038160A2 (en) 2009-09-23 2011-03-31 Protiva Biotherapeutics, Inc. Compositions and methods for silencing genes expressed in cancer
WO2011056682A1 (en) * 2009-10-27 2011-05-12 The University Of British Columbia Reverse head group lipids, lipid particle compositions comprising reverse headgroup lipids, and methods for the delivery of nucleic acids
JP5723378B2 (ja) 2009-11-03 2015-05-27 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Ａｌｎｙｌａｍ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ， Ｉｎｃ． トランスサイレチン（ｔｔｒ）を阻害する脂質製剤化組成物及び方法
RU2573409C2 (ru) 2009-11-04 2016-01-20 Дзе Юниверсити Оф Бритиш Коламбиа Содержащие нуклеиновые кислоты липидные частицы и относящиеся к ним способы
SI2506857T1 (en) 2009-12-01 2018-08-31 Translate Bio, Inc. Delivery of mRNA for the enrichment of proteins and enzymes in human genetic diseases
CA3044884A1 (en) * 2009-12-07 2011-06-16 Arbutus Biopharma Corporation Compositions for nucleic acid delivery
US20130017223A1 (en) 2009-12-18 2013-01-17 The University Of British Columbia Methods and compositions for delivery of nucleic acids
WO2011076807A2 (en) * 2009-12-23 2011-06-30 Novartis Ag Lipids, lipid compositions, and methods of using them
US8846631B2 (en) 2010-01-14 2014-09-30 Regulus Therapeutics Inc. MicroRNA compositions and methods
US20130116419A1 (en) 2010-01-22 2013-05-09 Daniel Zewge Post-synthetic chemical modification of rna at the 2'-position of the ribose ring via "click" chemistry
EP3214174B1 (en) 2010-03-04 2019-10-16 InteRNA Technologies B.V. A mirna molecule defined by its source and its diagnostic and therapeutic uses in diseases or conditions associated with emt
US8349308B2 (en) 2010-03-26 2013-01-08 Mersana Therapeutics, Inc. Modified polymers for delivery of polynucleotides, method of manufacture, and methods of use thereof
EP3391877A1 (en) 2010-04-08 2018-10-24 The Trustees of Princeton University Preparation of lipid nanoparticles
KR101773643B1 (ko) 2010-04-28 2017-08-31 교와 핫꼬 기린 가부시키가이샤 양이온성 지질
US20130129811A1 (en) * 2010-04-28 2013-05-23 Takeshi Kuboyama Cationic lipid
US10077232B2 (en) 2010-05-12 2018-09-18 Arbutus Biopharma Corporation Cyclic cationic lipids and methods of use
US20130123338A1 (en) * 2010-05-12 2013-05-16 Protiva Biotherapeutics, Inc. Novel cationic lipids and methods of use thereof
EP4481047A3 (en) 2010-06-03 2025-04-16 Alnylam Pharmaceuticals, Inc. Biodegradable lipids for the delivery of active agents
US9006417B2 (en) 2010-06-30 2015-04-14 Protiva Biotherapeutics, Inc. Non-liposomal systems for nucleic acid delivery
FI4005592T3 (fi) 2010-07-06 2023-01-13 Virionin kaltaisia kuljetuspartikkeleita itsereplikoituville rna-molekyyleille
PT3243526T (pt) 2010-07-06 2020-03-04 Glaxosmithkline Biologicals Sa Distribuição de arn para despoletar múltiplas vias imunitárias
US10487332B2 (en) 2010-07-06 2019-11-26 Glaxosmithkline Biologicals Sa Immunisation of large mammals with low doses of RNA
CN103153284B (zh) 2010-07-06 2015-11-25 诺华股份有限公司 含有其pKa值有利于RNA递送的脂质的脂质体
EP2591106A1 (en) 2010-07-06 2013-05-15 InteRNA Technologies B.V. Mirna and its diagnostic and therapeutic uses in diseases or conditions associated with melanoma, or in diseases or conditions associated with activated braf pathway
WO2012016184A2 (en) 2010-07-30 2012-02-02 Alnylam Pharmaceuticals, Inc. 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
CN103068980B (zh) * 2010-08-02 2017-04-05 瑟纳治疗公司 使用短干扰核酸（siNA）的RNA干扰介导的联蛋白（钙粘蛋白关联蛋白质），β1（CTNNB1）基因表达的抑制
CN103328500B (zh) 2010-08-04 2018-01-26 西兹尔生物技术有限公司 用于癌症的诊断和治疗的方法和化合物
US20130253168A1 (en) 2010-08-31 2013-09-26 Steven L. Colletti Novel single chemical entities and methods for delivery of oligonucleotides
RS63890B1 (sr) 2010-08-31 2023-02-28 Glaxosmithkline Biologicals Sa Mali lipozomi za isporuku rnk koja kodira imunogen
JP5911870B2 (ja) * 2010-08-31 2016-04-27 ノバルティス アーゲー 免疫原をコードするｒｎａの送達のためのｐｅｇ化リポソーム
WO2012031205A2 (en) 2010-09-03 2012-03-08 The Brigham And Women's Hospital, Inc. Lipid-polymer hybrid particles
US8466122B2 (en) 2010-09-17 2013-06-18 Protiva Biotherapeutics, Inc. Trialkyl cationic lipids and methods of use thereof
ES2987915T3 (es) 2010-09-20 2024-11-18 Sirna Therapeutics Inc Nuevos lípidos catiónicos de bajo peso molecular para el suministro de oligonucleótidos
EP2621480B1 (en) 2010-09-30 2018-08-15 Sirna Therapeutics, Inc. Low molecular weight cationic lipids for oligonucleotide delivery
KR102266691B1 (ko) 2010-10-11 2021-06-23 노파르티스 아게 항원 전달 플랫폼
WO2012054365A2 (en) 2010-10-21 2012-04-26 Merck Sharp & Dohme Corp. Novel low molecular weight cationic lipids for oligonucleotide delivery
DK2635265T3 (en) 2010-11-05 2018-07-16 Sirna Therapeutics Inc New low molecular weight cyclic amine-containing cationic lipids for oligonucleotide delivery
ES2702428T3 (es) 2010-11-15 2019-02-28 Life Technologies Corp Reactivos de transfección que contienen amina y métodos para prepararlos y usarlos
EP2474617A1 (en) 2011-01-11 2012-07-11 InteRNA Technologies BV Mir for treating neo-angiogenesis
US9999673B2 (en) 2011-01-11 2018-06-19 Alnylam Pharmaceuticals, Inc. PEGylated lipids and their use for drug delivery
EP2532649B1 (en) * 2011-06-07 2015-04-08 Incella GmbH Amino lipids, their synthesis and uses thereof
KR20220025112A (ko) 2011-06-08 2022-03-03 샤이어 휴먼 지네틱 테라피즈 인크. Mrna 전달을 위한 지질 나노입자 조성물 및 방법
RU2014104090A (ru) * 2011-07-06 2015-08-20 Новартис Аг Липосомы с эффективным соотношением n:p для доставки молекул phk
JP2014522842A (ja) 2011-07-06 2014-09-08 ノバルティス アーゲー 免疫原性組み合わせ組成物およびその使用
PT2750707T (pt) * 2011-08-31 2019-01-23 Glaxosmithkline Biologicals Sa Lipossomas peguilados para distribuição de arn que codifica imunogénio
US9126966B2 (en) * 2011-08-31 2015-09-08 Protiva Biotherapeutics, Inc. Cationic lipids and methods of use thereof
JP6250543B2 (ja) 2011-09-27 2017-12-20 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 ジ脂肪族置換ｐｅｇ化脂質
US20140328759A1 (en) 2011-10-25 2014-11-06 The University Of British Columbia Limit size lipid nanoparticles and related methods
JP2013095755A (ja) 2011-11-02 2013-05-20 Kyowa Hakko Kirin Co Ltd カチオン性脂質
CA2853689C (en) 2011-11-04 2020-06-30 Nitto Denko Corporation Method of producing lipid nanoparticles for drug delivery
UA118649C2 (uk) 2011-11-18 2019-02-25 Елнілем Фармасьютікалз, Інк. ДВОЛАНЦЮГОВИЙ ЗАСІБ ДЛЯ PHKі ТА ФАРМАЦЕВТИЧНА КОМПОЗИЦІЯ ДЛЯ ІНГІБУВАННЯ ЕКСПРЕСІЇ ТРАНСТИРЕТИНУ (TTR)
WO2013086322A1 (en) 2011-12-07 2013-06-13 Alnylam Pharmaceuticals, Inc. Branched alkyl and cycloalkyl terminated biodegradable lipids for the delivery of active agents
DE21212055T1 (de) 2011-12-07 2022-08-04 Alnylam Pharmaceuticals, Inc. Biologisch abbaubare lipide zur freisetzung von wirkstoffen
US20140308304A1 (en) 2011-12-07 2014-10-16 Alnylam Pharmaceuticals, Inc. Lipids for the delivery of active agents
JP6182457B2 (ja) 2011-12-12 2017-08-16 協和発酵キリン株式会社 カチオン性脂質を含有するドラックデリバリーシステムのための脂質ナノ粒子
CN104159615B (zh) 2011-12-12 2017-08-18 协和发酵麒麟株式会社 含有阳离子性脂质的组合的脂质纳米粒子
EP3369818B1 (en) 2011-12-22 2021-06-09 InteRNA Technologies B.V. Mirna for treating head and neck cancer
US9035039B2 (en) 2011-12-22 2015-05-19 Protiva Biotherapeutics, Inc. Compositions and methods for silencing SMAD4
US9352042B2 (en) 2012-02-24 2016-05-31 Protiva Biotherapeutics, Inc. Trialkyl cationic lipids and methods of use thereof
WO2013151664A1 (en) * 2012-04-02 2013-10-10 modeRNA Therapeutics Modified polynucleotides for the production of proteins
WO2013158579A1 (en) 2012-04-19 2013-10-24 Merck Sharp & Dohme Corp. Novel diester and triester based low molecular weight, biodegradable cationic lipids for oligonucleotide delivery
JP6108197B2 (ja) * 2012-07-02 2017-04-05 日油株式会社 三級アミノ基含有脂質の製造方法
US9415109B2 (en) 2012-07-06 2016-08-16 Alnylam Pharmaceuticals, Inc. Stable non-aggregating nucleic acid lipid particle formulations
TW201726599A (zh) 2012-07-06 2017-08-01 協和醱酵麒麟有限公司 陽離子性脂質
AU2013291125A1 (en) 2012-07-16 2015-01-22 Dicerna Pharmaceuticals, Inc. RNAi pharmaceutical composition capable of suppressing expression of KRAS gene
US10201556B2 (en) 2012-11-06 2019-02-12 Interna Technologies B.V. Combination for use in treating diseases or conditions associated with melanoma, or treating diseases or conditions associated with activated B-raf pathway
BR112015013105B1 (pt) 2012-12-05 2022-02-08 Alnylam Pharmaceuticals, Inc Agente de rnai de fita dupla capaz de inibir a expressão de pcsk9, seus usos, composição farmacêutica e método de inibição da expressão de pcsk9 em uma célula in vitro
JP2014147365A (ja) * 2013-02-04 2014-08-21 Osaka City Univ オリゴヌクレオチド構造体および遺伝子発現抑制方法
EP3932947A1 (en) 2013-03-14 2022-01-05 Translate Bio MA, Inc. Methods and compositions for delivering mrna coded antibodies
CN105143456A (zh) 2013-03-15 2015-12-09 不列颠哥伦比亚大学 用于转染的脂质纳米粒子和相关方法
WO2015011633A1 (en) 2013-07-23 2015-01-29 Protiva Biotherapeutics, Inc. Compositions and methods for delivering messenger rna
KR20160067219A (ko) 2013-10-03 2016-06-13 모더나 세라퓨틱스, 인코포레이티드 저밀도 지단백질 수용체를 암호화하는 폴리뉴클레오타이드
WO2015130584A2 (en) 2014-02-25 2015-09-03 Merck Sharp & Dohme Corp. Lipid nanoparticle vaccine adjuvants and antigen delivery systems
WO2015186770A1 (ja) 2014-06-04 2015-12-10 協和発酵キリン株式会社 ＣＫＡＰ５遺伝子発現抑制ＲＮＡｉ医薬組成物
CA2953341C (en) 2014-06-25 2023-01-24 Acuitas Therapeutics Inc. Lipids and lipid nanoparticle formulations for delivery of nucleic acids
CN106794141B (zh) 2014-07-16 2021-05-28 诺华股份有限公司 将核酸包封在脂质纳米粒主体中的方法
KR102410072B1 (ko) 2014-08-29 2022-06-20 알닐람 파마슈티칼스 인코포레이티드 트랜스타이레틴(ttr) 매개 아밀로이드증의 치료 방법
US10415037B2 (en) 2014-10-02 2019-09-17 Arbutus Biopharma Corporation Compositions and methods for silencing hepatitis B virus gene expression
WO2016093205A1 (ja) * 2014-12-08 2016-06-16 日油株式会社 カチオン性脂質の製造方法
US20170369872A1 (en) 2014-12-18 2017-12-28 Alnylam Pharmaceuticals, Inc. Reversir tm compounds
HK1244030A1 (en) * 2014-12-29 2018-07-27 东丽株式会社 Composition containing nucleic acid molecule stably
WO2016197132A1 (en) 2015-06-04 2016-12-08 Protiva Biotherapeutics Inc. Treating hepatitis b virus infection using crispr
HUE067372T2 (hu) 2015-06-29 2024-10-28 Acuitas Therapeutics Inc Lipidek és nanoszemcsés lipid formulázások nukleinsavak beadására
CN117965536A (zh) * 2015-07-22 2024-05-03 波涛生命科学有限公司 寡核苷酸组合物及其方法
CN108350455A (zh) 2015-07-29 2018-07-31 阿布特斯生物制药公司 用于使b型肝炎病毒基因表达沉默的组合物和方法
LT3329002T (lt) 2015-07-31 2021-01-11 Alnylam Pharmaceuticals, Inc. Transtiretino (ttr) irnr kompozicijos ir jų naudojimo būdai, skirti naudoti su ttr susijusių ligų gydymui arba prevencijai
MX2018002090A (es) 2015-08-24 2018-09-12 Halo Bio Rnai Therapeutics Inc Nanoparticulas de polinucleótido para modulación de expresión génica y sus usos.
HRP20220156T1 (hr) * 2015-09-17 2022-04-15 Modernatx, Inc. Spojevi i pripravci za unutarstaničnu isporuku terapeutskih sredstava
WO2018081480A1 (en) 2016-10-26 2018-05-03 Acuitas Therapeutics, Inc. Lipid nanoparticle formulations
CN108368028B (zh) 2015-10-28 2021-09-03 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
EP3369816B1 (en) * 2015-10-30 2024-03-13 Toray Industries, Inc. Composition stably containing single-stranded nucleic acid molecule that suppresses expression of tgf-beta1 gene
RS63051B1 (sr) 2015-12-22 2022-04-29 Modernatx Inc Jedinjenja i kompozicije za intracelularnu isporuku agenasa
US10525138B2 (en) 2015-12-25 2020-01-07 Kyowa Hakko Kirin Co., Ltd. Compound as cationic lipid
EP3442590A2 (en) * 2016-04-13 2019-02-20 Modernatx, Inc. Lipid compositions and their uses for intratumoral polynucleotide delivery
PL3458083T3 (pl) 2016-05-18 2023-03-13 Modernatx, Inc. Polinukleotydy kodujące interleukinę-12 (il12) i ich zastosowania
WO2017205566A1 (en) * 2016-05-27 2017-11-30 Preceres Inc. Oxidized aminolipidoids and uses thereof
US11191849B2 (en) 2016-06-30 2021-12-07 Arbutus Biopharma Corporation Compositions and methods for delivering messenger RNA
CN109715205A (zh) 2016-08-19 2019-05-03 库瑞瓦格股份公司 用于癌症治疗的rna
WO2018039131A1 (en) 2016-08-22 2018-03-01 Protiva Biotherapeutics, Inc. Anti-pd-1 antibodies, or fragments thereof, for treating hepatitis b
WO2018064755A1 (en) 2016-10-03 2018-04-12 Precision Nanosystems Inc. Compositions for transfecting resistant cell types
KR20230010826A (ko) 2016-10-14 2023-01-19 프리시젼 바이오사이언시스 인코포레이티드 B형 간염 바이러스 게놈 내의 인식 서열에 대해 특이적인 조작된 메가뉴클레아제
FR3058061A1 (fr) * 2016-10-27 2018-05-04 Selexel Nouvelle utilisation d'oligonucleotides double brin
EP3538067A1 (en) 2016-11-08 2019-09-18 Modernatx, Inc. Stabilized formulations of lipid nanoparticles
WO2018104538A1 (en) 2016-12-08 2018-06-14 Curevac Ag Rna for treatment or prophylaxis of a liver disease
WO2018104540A1 (en) 2016-12-08 2018-06-14 Curevac Ag Rnas for wound healing
AU2017379901B2 (en) 2016-12-21 2024-02-15 Arbutus Biopharma Corporation Methods for ameliorating infusion reactions
WO2018115525A1 (en) 2016-12-23 2018-06-28 Curevac Ag Lassa virus vaccine
WO2018115507A2 (en) 2016-12-23 2018-06-28 Curevac Ag Henipavirus vaccine
WO2018115527A2 (en) 2016-12-23 2018-06-28 Curevac Ag Mers coronavirus vaccine
US10927084B2 (en) * 2017-01-26 2021-02-23 Tosoh Corporation Alkanolamine, friction-reducing agent, and lubricating oil composition
RS63953B1 (sr) 2017-03-15 2023-02-28 Modernatx Inc Jedinjenje i kompozicije za intracelularnu isporuku terapeutskih sredstava
WO2018170270A1 (en) * 2017-03-15 2018-09-20 Modernatx, Inc. Varicella zoster virus (vzv) vaccine
WO2018170322A1 (en) 2017-03-15 2018-09-20 Modernatx, Inc. Crystal forms of amino lipids
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
WO2018170256A1 (en) * 2017-03-15 2018-09-20 Modernatx, Inc. Herpes simplex virus vaccine
US11338045B2 (en) 2017-03-17 2022-05-24 Newcastle University Adeno-associated virus vector delivery of a fragment of micro-dystrophin to treat muscular dystrophy
CA3050614A1 (en) 2017-03-17 2018-09-20 Curevac Ag Rna vaccine and immune checkpoint inhibitors for combined anticancer therapy
MX2019011215A (es) 2017-03-24 2020-02-12 Curevac Ag Ácidos nucleicos que codifican proteínas asociadas a crispr y usos de los mismos.
WO2018191719A1 (en) 2017-04-13 2018-10-18 Acuitas Therapeutics, Inc. Lipid delivery of therapeutic agents to adipose tissue
ES2994451T3 (en) 2017-04-20 2025-01-24 Atyr Pharma Inc Compositions for treating lung inflammation
EP3630200A4 (en) 2017-05-31 2021-02-24 Arcturus Therapeutics, Inc. THERAPEUTICS FOR PHENYLKETONURIA
US11015204B2 (en) 2017-05-31 2021-05-25 Arcturus Therapeutics, Inc. Synthesis and structure of high potency RNA therapeutics
KR102636537B1 (ko) 2017-05-31 2024-02-15 울트라제닉스 파마수티컬 인코포레이티드 글리코겐 축적 질환 유형 iii에 대한 치료제
EP3638678B1 (en) 2017-06-14 2025-12-03 ModernaTX, Inc. Compounds and compositions for intracellular delivery of agents
JP7722629B2 (ja) 2017-07-04 2025-08-13 キュアバック エスイー 新規核酸分子
US11602557B2 (en) 2017-08-22 2023-03-14 Cure Vac SE Bunyavirales vaccine
WO2019048645A1 (en) 2017-09-08 2019-03-14 Mina Therapeutics Limited STABILIZED COMPOSITIONS OF SMALL ACTIVATOR RNA (PARNA) FROM CEBPA AND METHODS OF USE
EP3684931A1 (en) 2017-09-19 2020-07-29 Alnylam Pharmaceuticals, Inc. Compositions and methods for treating transthyretin (ttr) mediated amyloidosis
BR112020004351A2 (pt) 2017-10-19 2020-09-08 Curevac Ag moléculas de ácido nucleico artificial
WO2019089828A1 (en) 2017-10-31 2019-05-09 Acuitas Therapeutics, Inc. Lamellar lipid nanoparticles
AU2018358016A1 (en) 2017-11-03 2020-05-07 Interna Technologies B.V. MiRNA molecule, equivalent, antagomir, or source thereof for treating and/or diagnosing a condition and/or a disease associated with neuronal deficiency or for neuronal (re)generation
SG11202003247RA (en) 2017-11-08 2020-05-28 Curevac Ag Rna sequence adaptation
US11931406B2 (en) 2017-12-13 2024-03-19 CureVac SE Flavivirus vaccine
CN108152274A (zh) * 2017-12-25 2018-06-12 汕头大学医学院 一种利用RNase ONE核酸酶和化学发光技术对血清miRNA进行定量检测的方法
EP3773702A2 (en) 2018-04-05 2021-02-17 CureVac AG Novel yellow fever nucleic acid molecules for vaccination
US11566246B2 (en) 2018-04-12 2023-01-31 Mina Therapeutics Limited SIRT1-saRNA compositions and methods of use
US11142750B2 (en) 2018-04-12 2021-10-12 Precision Biosciences, Inc. Optimized engineered meganucleases having specificity for a recognition sequence in the Hepatitis B virus genome
CA3091558A1 (en) 2018-04-17 2019-10-24 Curevac Ag Novel rsv rna molecules and compositions for vaccination
IL324506A (en) 2018-06-18 2026-01-01 Res Inst Nationwide Childrens Hospital Adeno-associated virus vector for delivery of muscle-specific microdystrophin for the treatment of muscular dystrophy
US20210260178A1 (en) 2018-06-27 2021-08-26 Curevac Ag Novel lassa virus rna molecules and compositions for vaccination
EP3853202A1 (en) 2018-09-19 2021-07-28 ModernaTX, Inc. Compounds and compositions for intracellular delivery of therapeutic agents
EP3852911B1 (en) 2018-09-21 2025-01-22 Acuitas Therapeutics, Inc. Systems and methods for manufacturing lipid nanoparticles and liposomes
ES2951836T3 (es) 2018-10-01 2023-10-25 Alnylam Pharmaceuticals Inc Lípidos biodegradables para el suministro de agentes activos
CA3116576A1 (en) 2018-10-18 2020-04-23 Acuitas Therapeutics, Inc. Lipids for lipid nanoparticle delivery of active agents
CA3122080A1 (en) 2018-12-06 2020-06-11 Arcturus Therapeutics, Inc. Compositions and methods for treating ornithine transcarbamylase deficiency
TW202039534A (zh) 2018-12-14 2020-11-01 美商美國禮來大藥廠 KRAS變體mRNA分子
AU2019410737A1 (en) 2018-12-21 2021-06-10 CureVac SE RNA for malaria vaccines
PL3908568T3 (pl) 2019-01-11 2024-12-02 Acuitas Therapeutics, Inc. Lipidy do dostarczania środków czynnych w nanocząstkach lipidowych
EP3920950A1 (en) 2019-02-08 2021-12-15 CureVac AG Coding rna administered into the suprachoroidal space in the treatment of ophtalmic diseases
MA55321A (fr) 2019-03-15 2022-01-19 Modernatx Inc Vaccins à base d'arn contre le vih
EP3953473A1 (en) 2019-04-12 2022-02-16 MiNA Therapeutics Limited Sirt1-sarna compositions and methods of use
WO2020219876A1 (en) * 2019-04-25 2020-10-29 Intellia Therapeutics, Inc. Ionizable amine lipids and lipid nanoparticles
SG11202113253SA (en) 2019-06-07 2021-12-30 Scribe Therapeutics Inc Engineered casx systems
EP3986452A1 (en) 2019-06-18 2022-04-27 CureVac AG Rotavirus mrna vaccine
JP7416096B2 (ja) * 2019-06-20 2024-01-17 プレシジョン ナノシステムズ ユーエルシー 核酸送達のためのイオン化可能な脂質
KR102928289B1 (ko) * 2019-06-29 2026-02-20 글로벌 라이프 사이언시즈 솔루션즈 캐나다 유엘씨 핵산 전달을 위한 이온화 가능한 지질
CA3147875A1 (en) 2019-07-19 2021-01-28 Flagship Pioneering Innovations Vi, Llc Recombinase compositions and methods of use
GB2600859B (en) 2019-08-14 2024-04-03 Acuitas Therapeutics Inc Improved lipid nanoparticles for delivery of nucleic acids
WO2021028439A1 (en) 2019-08-14 2021-02-18 Curevac Ag Rna combinations and compositions with decreased immunostimulatory properties
CN114929205B (zh) 2019-09-06 2024-09-03 世代生物公司 包括末端封闭式dna和可切割脂质的脂质纳米颗粒组合物及其使用方法
AU2020351220A1 (en) 2019-09-19 2022-04-07 Modernatx, Inc. Carbonate containing lipid compounds and compositions for intracellular delivery of therapeutic agents
EP4031524A1 (en) 2019-09-19 2022-07-27 ModernaTX, Inc. Branched tail lipid compounds and compositions for intracellular delivery of therapeutic agents
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 Sepia Therapeutics, Inc. Compositions and methods for modulating apolipoprotein B (APOB) gene expression
AU2020385378A1 (en) 2019-11-22 2022-04-07 Generation Bio Co. Ionizable lipids and nanoparticle compositions thereof
US12410418B2 (en) 2019-12-06 2025-09-09 Precision Biosciences, Inc. Optimized engineered meganucleases having specificity for a recognition sequence in the Hepatitis B virus genome
CA3162019A1 (en) 2019-12-20 2021-06-24 Gemma NAVARRO Lipid nanoparticles for delivery of nucleic acids
DE202021004130U1 (de) 2020-02-04 2022-10-26 Curevac Ag Coronavirus-Vakzine
EP4114470A4 (en) 2020-03-03 2024-04-17 Arcturus Therapeutics, Inc. COMPOSITIONS AND METHODS FOR TREATING ORNITHINE TRANSCARBAMYLASE DEFICIENCY
JP2023517644A (ja) 2020-03-09 2023-04-26 アークトゥラス・セラピューティクス・インコーポレイテッド コロナウイルスワクチン組成物及び方法
WO2021183720A1 (en) 2020-03-11 2021-09-16 Omega Therapeutics, Inc. Compositions and methods for modulating forkhead box p3 (foxp3) gene expression
IL296660A (en) 2020-03-24 2022-11-01 Generation Bio Co Non-viral DNA vectors and their use for therapeutic expression of factor ix
IL296662A (en) 2020-03-24 2022-11-01 Generation Bio Co Non-viral dna vectors and uses thereof for expressing gaucher therapeutics
US12534435B2 (en) 2020-03-27 2026-01-27 Generation Bio Co. Lipids and nanoparticle compositions thereof
AU2021250814A1 (en) 2020-03-30 2022-10-13 BioNTech SE RNA compositions targeting Claudin-18.2
US12194157B2 (en) 2020-04-09 2025-01-14 Finncure Oy Carrier for targeted delivery to a host
AU2021252164B2 (en) 2020-04-09 2026-07-02 Finncure Oy Mimetic nanoparticles for preventing the spreading and lowering the infection rate of novel coronaviruses
WO2021222801A2 (en) 2020-05-01 2021-11-04 Arcturus Therapeutics, Inc. Nucleic acids and methods of treatment for cystic fibrosis
IL298363A (en) 2020-05-20 2023-01-01 Flagship Pioneering Innovations Vi Llc Immunogenic compositions and uses thereof
AU2021275223A1 (en) 2020-05-20 2023-02-02 Flagship Pioneering Innovations Vi, Llc. Coronavirus antigen compositions and their uses
US20230203510A1 (en) 2020-05-29 2023-06-29 Flagship Pioneering Innovations Vi, Llc Trem compositions and methods relating thereto
WO2021239880A1 (en) 2020-05-29 2021-12-02 Curevac Ag Nucleic acid based combination vaccines
EP4158032A2 (en) 2020-05-29 2023-04-05 Flagship Pioneering Innovations VI, LLC Trem compositions and methods relating thereto
EP4164668A1 (en) 2020-06-15 2023-04-19 Research Institute at Nationwide Children's Hospital Adeno-associated virus vector delivery for muscular dystrophies
WO2025056938A1 (en) 2023-09-11 2025-03-20 BioNTech SE Rna compositions for delivery of incretin agents
IL299571A (en) 2020-07-08 2023-02-01 Janssen Sciences Ireland Unlimited Co Rna replicon vaccines against hbv
KR20230051172A (ko) 2020-07-16 2023-04-17 아퀴타스 테라퓨틱스 인크. 지질 나노 입자에 사용하기 위한 양이온성 지질
MX2023001109A (es) 2020-07-27 2023-04-24 Anjarium Biosciences Ag Composiciones de moleculas de adn, metodos para elaborar las mismas, y metodos de usos de las mismas.
EP4172194A1 (en) 2020-07-31 2023-05-03 CureVac SE Nucleic acid encoded antibody mixtures
US12178921B2 (en) 2020-08-14 2024-12-31 Arcturus Therapeutics, Inc. Method of lyophilizing lipid nanoparticles
WO2022043551A2 (en) 2020-08-31 2022-03-03 Curevac Ag Multivalent nucleic acid based coronavirus vaccines
IL300947A (en) 2020-09-03 2023-04-01 Flagship Pioneering Innovations Vi Llc Immunogenic compositions and uses thereof
IL301253A (en) 2020-09-13 2023-05-01 Arcturus Therapeutics Inc As a mass of large RNA lipid nanoparticles
KR20250099412A (ko) 2020-11-12 2025-07-01 프리시젼 바이오사이언시스 인코포레이티드 디스트로핀 유전자 내의 인식 서열에 대해 특이성을 갖는 조작된 메가뉴클레아제
CN117098541A (zh) 2020-11-25 2023-11-21 阿卡格拉医药公司 用于递送核酸的脂质纳米粒及相关使用方法
AU2021392719B2 (en) 2020-12-03 2025-08-14 Scribe Therapeutics Inc. Engineered class 2 type v crispr systems
PH12023500013A1 (en) 2020-12-04 2024-03-11 Tidal Therapeutics Inc Ionizable cationic lipids and lipi nanoparticles, and methods of synthesis and use thereof
GB2603454A (en) 2020-12-09 2022-08-10 Ucl Business Ltd Novel therapeutics for the treatment of neurodegenerative disorders
MX2023007574A (es) 2020-12-22 2023-09-29 CureVac SE "vacuna de arn contra variantes de sars-cov-2.
US20240299309A1 (en) 2020-12-22 2024-09-12 CureVac SE Pharmaceutical composition comprising lipid-based carriers encapsulating rna for multidose administration
KR20230135585A (ko) 2020-12-23 2023-09-25 플래그쉽 파이어니어링 이노베이션스 브이아이, 엘엘씨 변형된 trem의 조성물 및 이의 용도
AU2021412833A1 (en) 2020-12-28 2023-07-06 Arcturus Therapeutics, Inc. Transcription activator-like effector nucleases (talens) targeting hbv
EP4277929A1 (en) * 2021-01-14 2023-11-22 Translate Bio, Inc. Methods and compositions for delivering mrna coded antibodies
CA3170747A1 (en) 2021-01-27 2022-08-04 Moritz THRAN Method of reducing the immunostimulatory properties of in vitro transcribed rna
TW202245835A (zh) 2021-02-04 2022-12-01 美商默沙東有限責任公司 用於肺炎球菌結合物疫苗之奈米乳化液佐劑組合物
EP4313152A1 (en) 2021-03-26 2024-02-07 GlaxoSmithKline Biologicals S.A. Immunogenic compositions
US20240175033A1 (en) 2021-03-26 2024-05-30 Mina Therapeutics Limited TMEM173 saRNA Compositions and Methods of Use
EP4312988A2 (en) 2021-03-31 2024-02-07 CureVac SE Syringes containing pharmaceutical compositions comprising rna
EP4313109A1 (en) 2021-03-31 2024-02-07 Flagship Pioneering Innovations V, Inc. Thanotransmission polypeptides and their use in treating cancer
EP4319803B1 (en) 2021-04-08 2025-11-26 Vaxthera SAS Coronavirus vaccine comprising a mosaic protein
JP2024517427A (ja) 2021-04-20 2024-04-22 アンジャリウム バイオサイエンシズ エージー アミロ－α－１，６－グルコシダーゼ、４－α－グルカノトランスフェラーゼをコードするＤＮＡ分子の組成物、その作製の方法、及びその使用の方法
US20240216535A1 (en) 2021-04-27 2024-07-04 Generation Bio Co. Non-viral dna vectors expressing anti-coronavirus antibodies and uses thereof
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
CN113402404B (zh) * 2021-04-30 2022-03-11 苏州科锐迈德生物医药科技有限公司 脂质化合物及包含其的脂质载体、核酸脂质纳米粒组合物和药物制剂
CA3171589A1 (en) 2021-05-03 2022-11-03 Moritz THRAN Improved nucleic acid sequence for cell type specific expression
CN117460542A (zh) 2021-06-11 2024-01-26 纳米智能生物医学工程有限公司 包含用于将寡核苷酸递送至靶细胞内的肽-脂质缀合物的纳米颗粒以及包含其的药物组合物
CN117642380A (zh) 2021-06-14 2024-03-01 世代生物公司 阳离子脂质及其组合物
WO2023283359A2 (en) 2021-07-07 2023-01-12 Omega Therapeutics, Inc. Compositions and methods for modulating secreted frizzled receptor protein 1 (sfrp1) gene expression
US20230046606A1 (en) 2021-07-15 2023-02-16 Turn Biotechnologies, Inc. Synthetic, persistent rna constructs with on/off mechanism for controlled expression and methods of use
US20230042860A1 (en) 2021-07-15 2023-02-09 Turn Biotechnologies, Inc. Polycistronic expression vectors
US20230044997A1 (en) 2021-07-15 2023-02-09 Turn Biotechnologies, Inc. Synthetic, persistent rna constructs and methods of use for cell rejuvenation and for treatment
WO2023009547A1 (en) 2021-07-26 2023-02-02 Flagship Pioneering Innovations Vi, Llc Trem compositions and uses thereof
EP4377331A2 (en) 2021-07-30 2024-06-05 CureVac SE Mrnas for treatment or prophylaxis of liver diseases
AR126675A1 (es) 2021-08-03 2023-11-01 Alnylam Pharmaceuticals Inc COMPOSICIONES DE ARNi CONTRA LA TRANSTIRRETINA (TTR) Y SUS MÉTODOS DE USO
WO2023023055A1 (en) 2021-08-16 2023-02-23 Renagade Therapeutics Management Inc. Compositions and methods for optimizing tropism of delivery systems for rna
EP4763287A2 (en) 2021-09-03 2026-06-24 CureVac SE Novel lipid nanoparticles for delivery of nucleic acids
IL309502A (en) 2021-09-03 2024-02-01 CureVac SE Lipid nanoparticles for nucleic acid delivery involving phosphatidylserine
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
WO2023044343A1 (en) 2021-09-14 2023-03-23 Renagade Therapeutics Management Inc. Acyclic lipids and methods of use thereof
AR127073A1 (es) 2021-09-17 2023-12-13 Flagship Pioneering Innovations Vi Llc Composiciones y métodos para producir polirribonucleótidos circulares
IL311610A (en) 2021-09-21 2024-05-01 Scribe Therapeutics Inc Engineered CASX suppressor systems
CN115894281B (zh) * 2021-09-22 2025-07-01 广州谷森制药有限公司 阳离子脂质化合物、其制备方法、组合物及应用
EP4419706A1 (en) 2021-10-18 2024-08-28 Flagship Pioneering Innovations VI, LLC Compositions and methods for purifying polyribonucleotides
EP4422698A1 (en) 2021-10-29 2024-09-04 CureVac SE Improved circular rna for expressing therapeutic proteins
US20250043269A1 (en) 2021-11-03 2025-02-06 The J. David Gladstone Institutes, a testamentary trust established under the Will of J. David Glad Precise Genome Editing Using Retrons
WO2023081526A1 (en) 2021-11-08 2023-05-11 Orna Therapeutics, Inc. Lipid nanoparticle compositions for delivering circular polynucleotides
JP2024546431A (ja) 2021-11-11 2024-12-24 アークトゥラス・セラピューティクス・インコーポレイテッド Ｒｎａ送達のためのイオン化可能なカチオン性脂質
US20250049948A1 (en) 2021-11-16 2025-02-13 Sail Biomedicines, Inc. Novel ionizable lipids and lipid nanoparticles and methods of using the same
EP4436549A1 (en) 2021-11-22 2024-10-02 Sail Biomedicines, Inc. Novel ionizable lipids and lipid nanoparticles and methods of using the same
MX2024006407A (es) 2021-11-24 2024-08-19 Flagship Pioneering Innovations Vi Llc Composiciones de inmunogenos del virus varicela-zoster y sus usos.
IL312965A (en) 2021-11-24 2024-07-01 Flagship Pioneering Innovations Vi Llc Immunogenic compositions and their uses
WO2023096990A1 (en) 2021-11-24 2023-06-01 Flagship Pioneering Innovation Vi, Llc Coronavirus immunogen compositions and their uses
WO2023099884A1 (en) 2021-12-01 2023-06-08 Mina Therapeutics Limited Pax6 sarna compositions and methods of use
GB202117758D0 (en) 2021-12-09 2022-01-26 Ucl Business Ltd Therapeutics for the treatment of neurodegenerative disorders
WO2023114943A2 (en) 2021-12-16 2023-06-22 Acuitas Therapeutics, Inc. Lipids for use in lipid nanoparticle formulations
CN118510896A (zh) 2021-12-17 2024-08-16 旗舰创业创新六公司 用于在变性条件下富集环状rna的方法
EP4453201A1 (en) 2021-12-22 2024-10-30 Flagship Pioneering Innovations VI, LLC Compositions and methods for purifying polyribonucleotides
WO2023122752A1 (en) 2021-12-23 2023-06-29 Renagade Therapeutics Management Inc. Constrained lipids and methods of use thereof
AU2022420620A1 (en) 2021-12-23 2024-07-04 Flagship Pioneering Innovations Vi, Llc Circular polyribonucleotides encoding antifusogenic polypeptides
WO2023135273A2 (en) 2022-01-14 2023-07-20 Anjarium Biosciences Ag Compositions of dna molecules encoding factor viii, methods of making thereof, and methods of use thereof
WO2023141602A2 (en) 2022-01-21 2023-07-27 Renagade Therapeutics Management Inc. Engineered retrons and methods of use
WO2023144193A1 (en) 2022-01-25 2023-08-03 CureVac SE Mrnas for treatment of hereditary tyrosinemia type i
JP2025508332A (ja) 2022-01-27 2025-03-26 ビオンテック エスエー 単純ヘルペスウイルス抗原の送達のための薬学的組成物及び関連方法
EP4469091A1 (en) 2022-01-28 2024-12-04 CureVac SE Nucleic acid encoded transcription factor inhibitors
CN114213295B (zh) * 2022-02-22 2022-05-20 中国科学院基础医学与肿瘤研究所(筹) 一种阳离子化合物、制备方法及其复合物和用途
WO2023170435A1 (en) 2022-03-07 2023-09-14 Mina Therapeutics Limited Il10 sarna compositions and methods of use
EP4493212A1 (en) 2022-03-14 2025-01-22 Generation Bio Co. Heterologous prime boost vaccine compositions and methods of use
CA3245624A1 (en) 2022-03-25 2023-09-28 Sail Biomedicines, Inc. NEW IONIZABLE LIPIDS AND LIPID NANOPARTICLES AND THEIR METHODS OF USE
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
JP2025511756A (ja) 2022-04-08 2025-04-16 フラッグシップ パイオニアリング イノベーションズ セブン，エルエルシー ワクチン及び関連する方法
WO2023220083A1 (en) 2022-05-09 2023-11-16 Flagship Pioneering Innovations Vi, Llc Trem compositions and methods of use for treating proliferative disorders
WO2023218420A1 (en) 2022-05-13 2023-11-16 Janssen Pharmaceuticals, Inc. Mrna compositions for inducing latent hiv-1 reversal
JP2025516638A (ja) 2022-05-13 2025-05-30 フラッグシップ パイオニアリング イノベーションズ セブン，エルエルシー 二本鎖ｄｎａ組成物及び関連する方法
AU2023268051A1 (en) 2022-05-13 2025-01-02 BioNTech SE Rna compositions targeting hiv
WO2023230587A2 (en) 2022-05-25 2023-11-30 Akagera Medicines, Inc. Lipid nanoparticles for delivery of nucleic acids and methods of use thereof
CA3248209A1 (en) 2022-05-25 2023-11-30 CureVac SE Escherichia coli Antigenic Polypeptide-Based Nucleic Acid Vaccine (FIMH)
WO2023230295A1 (en) 2022-05-25 2023-11-30 BioNTech SE Rna compositions for delivery of monkeypox antigens and related methods
US20250375534A1 (en) 2022-05-30 2025-12-11 BioNTech SE Complexes for delivery of nucleic acids
CA3257824A1 (en) 2022-06-02 2023-12-07 Scribe Therapeutics Inc. MODIFIED TYPE V, CLASS 2 CRISPR SYSTEMS
EP4536819A1 (en) 2022-06-07 2025-04-16 Scribe Therapeutics Inc. Compositions and methods for the targeting of pcsk9
WO2023239756A1 (en) 2022-06-07 2023-12-14 Generation Bio Co. Lipid nanoparticle compositions and uses thereof
CN119998446A (zh) 2022-06-07 2025-05-13 斯克里贝治疗公司 用于靶向pcsk9的组合物和方法
AR129560A1 (es) 2022-06-08 2024-09-04 Tidal Therapeutics Inc Lípidos catiónicos ionizables y nanopartículas lipídicas, y métodos de síntesis y uso de los mismos
EP4544043A1 (en) 2022-06-22 2025-04-30 Flagship Pioneering Innovations VI, LLC Compositions of modified trems and uses thereof
US20240042021A1 (en) 2022-08-01 2024-02-08 Flagship Pioneering Innovations Vii, Llc Immunomodulatory proteins and related methods
CA3263767A1 (en) 2022-08-03 2024-02-08 Voyager Therapeutics, Inc. COMPOSITIONS AND PROCESSES ALLOWING CROSSING THE BLOOD-BRAIN BARRIER
EP4565209A1 (en) * 2022-08-04 2025-06-11 Sanofi Pasteur Inc. Quantitative assessment of rna encapsulation
EP4569112A1 (en) 2022-08-12 2025-06-18 Remix Therapeutics Inc. Methods and compositions for modulating splicing at alternative splice sites
WO2024040222A1 (en) 2022-08-19 2024-02-22 Generation Bio Co. Cleavable closed-ended dna (cedna) and methods of use thereof
EP4581012A2 (en) 2022-08-31 2025-07-09 Sail Biomedicines, Inc. Novel ionizable lipids and lipid nanoparticles and methods of using the same
CN120152962A (zh) 2022-09-06 2025-06-13 国立大学法人九州大学 脂质纳米粒子
CA3172967A1 (en) 2022-09-13 2024-03-13 Universite Laval Monoglycerol acrylate based polymer and uses thereof
US20260151470A1 (en) 2022-09-23 2026-06-04 BioNTech SE Compositions for delivery of plasmodium csp antigens and related methods
WO2024063788A1 (en) 2022-09-23 2024-03-28 BioNTech SE Compositions for delivery of malaria antigens and related methods
WO2024063789A1 (en) 2022-09-23 2024-03-28 BioNTech SE Compositions for delivery of malaria antigens and related methods
IL319414A (en) 2022-09-23 2025-05-01 BioNTech SE Compositions for delivering antigens at the liver level and related methods
CA3267272A1 (en) 2022-09-26 2024-04-04 Glaxosmithkline Biologicals Sa FLU VIRUS VACCINES
WO2024077191A1 (en) 2022-10-05 2024-04-11 Flagship Pioneering Innovations V, Inc. Nucleic acid molecules encoding trif and additionalpolypeptides and their use in treating cancer
WO2024074211A1 (en) 2022-10-06 2024-04-11 BioNTech SE Rna compositions targeting claudin-18.2
CN120129702A (zh) 2022-10-06 2025-06-10 生物技术公司 靶向密蛋白-18.2的rna组合物
WO2024089638A1 (en) 2022-10-28 2024-05-02 Glaxosmithkline Biologicals Sa Nucleic acid based vaccine
EP4612296A1 (en) 2022-10-31 2025-09-10 Flagship Pioneering Innovations VI, LLC Compositions and methods for purifying polyribonucleotides
WO2024102762A1 (en) 2022-11-08 2024-05-16 Orna Therapeutics, Inc. Lipids and lipid nanoparticle compositions for delivering polynucleotides
WO2024102799A1 (en) 2022-11-08 2024-05-16 Flagship Pioneering Innovations Vi, Llc Compositions and methods for producing circular polyribonucleotides
TW202428289A (zh) 2022-11-08 2024-07-16 美商歐納醫療公司 環狀rna組合物
WO2024102730A1 (en) 2022-11-08 2024-05-16 Orna Therapeutics, Inc. Lipids and nanoparticle compositions for delivering polynucleotides
EP4626402A1 (en) 2022-12-01 2025-10-08 Generation Bio Co. Lipid nanoparticles comprising nucleic acids and lipid-anchored polymers
US20260207525A1 (en) 2022-12-01 2026-07-23 Generation Bio Co. Lipid nanoparticles comprising nucleic acids, ionizable lipids, sterols, lipid anchored polymers and helper lipids, their uses
AU2023406321A1 (en) 2022-12-01 2025-05-29 Generation Bio Co. Novel polyglycerol-conjugated lipids and lipid nanoparticle compositions comprising the same
JP2025541726A (ja) 2022-12-01 2025-12-23 ジェネレーション バイオ カンパニー 細胞標的化のためのステルス脂質ナノ粒子組成物
JP2025541845A (ja) 2022-12-09 2025-12-23 ソウル大学校産学協力団 Ｂ細胞及びＴ細胞内へｍＲＮＡを伝達するためのペプチド基盤結合体を含むナノ粒子及びこの用途
WO2024123139A1 (ko) 2022-12-09 2024-06-13 주식회사 나이벡 타겟 세포 내로 올리고뉴클레오티드를 전달하기 위한 펩타이드 기반 결합체를 포함하는 나노입자 및 이를 포함하는 약학적 조성물
TW202440929A (zh) 2022-12-14 2024-10-16 加拿大商普羅維登斯治療控股公司 用於感染性疾病的組合物和方法
TW202430215A (zh) 2022-12-14 2024-08-01 美商旗艦先鋒創新有限責任（Ｖｉｉ）公司 用於將治療劑遞送至骨之組成物和方法
US20260218178A1 (en) 2022-12-22 2026-07-30 Mina Therapeutics Limited Chemically Modified saRNA Compositions and Methods of Use
CN121127490A (zh) 2022-12-28 2025-12-12 生物技术欧洲股份公司 靶向hiv的rna组合物
WO2024141784A2 (en) 2022-12-29 2024-07-04 Popvax Private Limited Broadly protective betacoronavirus vaccines and compositions
EP4642481A2 (en) 2022-12-29 2025-11-05 Popvax Private Limited Multitarget vaccines and therapeutics
US20240238473A1 (en) 2023-01-09 2024-07-18 Beth Israel Deaconess Medical Center, Inc. Recombinant nucleic acid molecules and their use in wound healing
US20240252520A1 (en) 2023-01-09 2024-08-01 Beth Israel Deaconess Medical Center, Inc. Therapeutic agents and their use for treating chronic wounds
WO2024151687A1 (en) 2023-01-09 2024-07-18 Flagship Pioneering Innovations V, Inc. Genetic switches and their use in treating cancer
EP4648794A2 (en) 2023-01-09 2025-11-19 Flagship Pioneering Innovations VII, LLC Vaccines and related methods
IL322368A (en) 2023-02-03 2025-09-01 Genzyme Corp HSC-specific antibody-conjugated lipid nanoparticles and their uses
WO2024160936A1 (en) 2023-02-03 2024-08-08 Glaxosmithkline Biologicals Sa Rna formulation
TW202438515A (zh) 2023-02-06 2024-10-01 美商旗艦先鋒創新有限責任（Ｖｉｉ）公司 免疫調節組合物及相關方法
IL322261A (en) 2023-02-09 2025-09-01 Alnylam Pharmaceuticals Inc Reverser molecules and methods of using them
US20240293318A1 (en) 2023-02-13 2024-09-05 Flagship Pioneering Innovations Vii, Llc Cleavable linker-containing ionizable lipids and lipid carriers for therapeutic compositions
GB202302092D0 (en) 2023-02-14 2023-03-29 Glaxosmithkline Biologicals Sa Analytical method
TW202446956A (zh) 2023-02-17 2024-12-01 美商旗艦先鋒創新有限責任（Ｖｉｉ）公司 包含經修飾的胞嘧啶的ｄｎａ組成物
KR20250151437A (ko) 2023-02-17 2025-10-21 플래그쉽 파이어니어링 이노베이션스 Vii, 엘엘씨 변형된 우라실을 포함하는 dna 조성물
KR20250153298A (ko) 2023-03-08 2025-10-24 큐어백 에스이 핵산 전달을 위한 신규의 지질 나노입자 제형
EP4680750A1 (en) 2023-03-15 2026-01-21 Flagship Pioneering Innovations VI, LLC Compositions comprising polyribonucleotides and uses thereof
EP4680276A1 (en) 2023-03-15 2026-01-21 Flagship Pioneering Innovations VI, LLC Immunogenic compositions and uses thereof
TW202444914A (zh) 2023-03-29 2024-11-16 美商斯奎柏治療公司 抑制子融合蛋白系統
WO2024206555A1 (en) 2023-03-29 2024-10-03 Scribe Therapeutics Inc. Compositions and methods for the targeting of pcsk9
WO2024205657A2 (en) 2023-03-29 2024-10-03 Orna Therapeutics, Inc. Lipids and lipid nanoparticle compositions for delivering polynucleotides
WO2024206676A1 (en) 2023-03-29 2024-10-03 Scribe Therapeutics Inc. Compositions and methods for the targeting of lpa
AU2024255972A1 (en) 2023-04-12 2025-10-23 Flagship Pioneering Innovations Vi, Llc Modified trems, compositions, and related methods thereof
WO2024216128A1 (en) 2023-04-12 2024-10-17 Flagship Pioneering Innovations Vi, Llc Trems for use in correction of missense mutations
WO2024220746A2 (en) 2023-04-21 2024-10-24 Flagship Pioneering Innovations Vii, Llc Rnai agents targeting fatty acid synthase and related methods
EP4701657A1 (en) 2023-04-27 2026-03-04 GlaxoSmithKline Biologicals S.A. Influenza virus vaccines
JP2026518411A (ja) 2023-04-27 2026-06-08 グラクソスミスクライン バイオロジカルズ ソシエテ アノニム インフルエンザウイルスワクチン
AU2024266138A1 (en) 2023-05-03 2025-11-13 Manifold Biotechnologies, Inc. Methods and compositions for high-throughput protein delivery, screening, and detection
WO2024228044A1 (en) 2023-05-03 2024-11-07 BioNTech SE Optimized csp variants and related methods
CN121038807A (zh) 2023-05-03 2025-11-28 生物技术欧洲股份公司 优化的csp变体及相关方法
WO2024233308A2 (en) 2023-05-05 2024-11-14 Orna Therapeutics, Inc. Circular rna compositions and methods
WO2024230934A1 (en) 2023-05-11 2024-11-14 CureVac SE Therapeutic nucleic acid for the treatment of ophthalmic diseases
DE102023001946A1 (de) 2023-05-12 2024-11-14 Friedrich-Schiller-Universität Jena, Körperschaft des öffentlichen Rechts Nanopartikel für den Transport von Wirkstoffen mit anionischen Gruppen, Verfahren zu deren Herstellung und deren Verwendung
EP4716741A2 (en) 2023-05-23 2026-04-01 Flagship Labs 114, Inc. Compositions and methods for reducing cxcl9, cxcl10, and cxcl11 gene expression
KR20240174509A (ko) * 2023-06-07 2024-12-17 포항공과대학교 산학협력단 핵산 치료제에 사용되는 기능성 지질 라이브러리를 합성하기 위한 단일 연속 유동 합성 방법
WO2024258829A1 (en) 2023-06-12 2024-12-19 Flagship Pioneering Innovations Vii, Llc Sars-cov-2 vaccine compositions and related methods
WO2025006684A1 (en) 2023-06-28 2025-01-02 Flagship Pioneering Innovations Vi, Llc Circular polyribonucleotides encoding antifusogenic polypeptides
WO2025008774A1 (en) 2023-07-05 2025-01-09 Takeda Pharmaceutical Company Limited Viral vectors encoding recombinant fviii variants with increased expression for gene therapy of hemophilia a
CN121532514A (zh) 2023-07-07 2026-02-13 上海环码生物医药有限公司 用于季节性流感的环状rna疫苗及使用方法
TW202515531A (zh) 2023-07-07 2025-04-16 美商輝瑞股份有限公司 兩親性ｔｌｒ７／８佐劑及其用途
EP4743577A1 (en) 2023-07-13 2026-05-20 Korro Bio, Inc. RNA EDITING OLIGONUCLEOTIDES AND THEIR USES
AU2024287308A1 (en) 2023-07-13 2025-12-18 Korro Bio, Inc. Rna-editing oligonucleotides and uses thereof
EP4746902A2 (en) 2023-07-21 2026-05-27 BioNTech SE Compositions for delivery of plasmodium antigens and related methods
WO2025024324A1 (en) 2023-07-21 2025-01-30 BioNTech SE Compositions for delivery of plasmodium antigens and related methods
EP4750488A1 (en) 2023-07-24 2026-06-03 BioNTech SE Compositions for delivery of plasmodium antigens and related methods
KR20260044217A (ko) 2023-07-25 2026-04-01 플래그쉽 파이어니어링 이노베이션스 Vii, 엘엘씨 Cas 엔도뉴클레아제 및 관련 방법
WO2025026545A1 (en) 2023-08-01 2025-02-06 BioNTech SE Ionizable thioplipids and uses thereof
TW202519509A (zh) 2023-08-01 2025-05-16 德商拜恩技術股份公司 可離子化硫脂質及其用途
WO2025030154A1 (en) 2023-08-03 2025-02-06 The Trustees Of The University Of Pennsylvania Pharmaceutical compositions for delivery of herpes simplex virus glycoprotein b antigens and related methods
AU2024316307A1 (en) 2023-08-03 2026-02-26 BioNTech SE Pharmaceutical compositions for delivery of herpes simplex virus antigens and related methods
CN121866336A (zh) 2023-08-03 2026-04-14 百欧恩泰欧洲股份公司 靶向hiv的rna组合物
EP4754130A2 (en) 2023-08-03 2026-06-10 BioNTech SE Rna compositions targeting hiv
WO2025031498A1 (zh) 2023-08-10 2025-02-13 浙江海昶生物医药技术有限公司 Toll样受体激动剂及其应用
WO2025036297A1 (zh) 2023-08-11 2025-02-20 浙江海昶生物医药技术有限公司 一种具有靶向性的脂质组合物及其制备方法
WO2025042786A1 (en) 2023-08-18 2025-02-27 Flagship Pioneering Innovations Vi, Llc Compositions comprising circular polyribonucleotides and uses thereof
WO2025049690A1 (en) 2023-08-29 2025-03-06 Orna Therapeutics, Inc. Circular polyethylene glycol lipids
EP4771042A1 (en) 2023-08-29 2026-07-08 Shanghai Circode Biomed Co., Ltd Circular rna encoding vegf polypeptides, formulations, and methods of uses
WO2025054236A2 (en) 2023-09-06 2025-03-13 Flagship Pioneering Innovations Vii, Llc Sars-cov-2 vaccine compositions and related methods
WO2025054556A1 (en) 2023-09-07 2025-03-13 BioNTech SE Rna compositions for delivery of mpox antigens and related methods
WO2025052180A2 (en) 2023-09-07 2025-03-13 Axelyf ehf. Lipids and lipid nanoparticles
WO2025051994A1 (en) 2023-09-07 2025-03-13 Coave Therapeutics Ionizable lipid nanoparticles
CN121844046A (zh) 2023-09-11 2026-04-10 C·米库尔卡 用于递送肠促胰岛素剂的rna组合物
IL327035A (en) 2023-09-18 2026-05-01 Flagship Pioneering Innovations Vii Llc Ionizable lipidoid compositions and their medical uses
CN122270430A (zh) 2023-09-18 2026-06-23 约翰内斯堡威特沃特斯兰德大学 使腰果壳液（cnsl）和/或其组分增值的方法
WO2025064850A1 (en) 2023-09-22 2025-03-27 BioNTech SE Rna constructs with n-terminal degrons to enhance an immune response
WO2025072331A1 (en) 2023-09-26 2025-04-03 Flagship Pioneering Innovations Vii, Llc Cas nucleases and related methods
WO2025081042A1 (en) 2023-10-12 2025-04-17 Renagade Therapeutics Management Inc. Nickase-retron template-based precision editing system and methods of use
WO2025096809A1 (en) 2023-10-31 2025-05-08 Korro Bio, Inc. Oligonucleotides comprising phosphoramidate internucleotide linkages
WO2025096807A2 (en) 2023-10-31 2025-05-08 Flagship Pioneering Innovations Vii, Llc Novel therapeutic dna forms
CN122497525A (zh) 2023-11-07 2026-07-31 欧纳医疗公司 环状rna组合物
KR20260113064A (ko) 2023-11-14 2026-07-21 플래그쉽 파이어니어링 이노베이션스 Vii, 엘엘씨 이온화 가능한 리피도이드 조성물 및 이의 치료적 용도
WO2025106754A1 (en) 2023-11-15 2025-05-22 BioNTech SE Coronavirus vaccine
US20250161347A1 (en) 2023-11-22 2025-05-22 Flagship Pioneering Innovations Vii, Llc Methods and compositions for treating non-alcoholic fatty liver disease
WO2025114520A1 (en) 2023-12-01 2025-06-05 Coave Therapeutics Ionizable lipid nanoparticles
WO2025117877A2 (en) 2023-12-01 2025-06-05 Flagship Pioneering Innovations Vii, Llc Cas nucleases and related methods
WO2025124711A1 (en) 2023-12-13 2025-06-19 BioNTech SE Glycolipid compositions
WO2025133105A1 (en) 2023-12-21 2025-06-26 Biontech Delivery Technologies Gmbh Compositions and methods
WO2025132839A1 (en) 2023-12-21 2025-06-26 Glaxosmithkline Biologicals Sa Influenza virus vaccines
WO2025134062A2 (en) 2023-12-21 2025-06-26 Biontech Delivery Technologies Gmbh Ionizable lipids
WO2025134066A1 (en) 2023-12-21 2025-06-26 Biontech Delivery Technologies Gmbh Ionizable lipids
AU2024409925A1 (en) 2023-12-26 2026-08-06 Emmune, Inc. Systems for nucleic acid transfer
WO2025149492A1 (en) 2024-01-08 2025-07-17 BioNTech SE Rna encoding an immune inhibitory il-1 family member
WO2025155753A2 (en) 2024-01-17 2025-07-24 Renagade Therapeutics Management Inc. Improved gene editing system, guides, and methods
US20250375499A1 (en) 2024-01-26 2025-12-11 Flagship Pioneering Innovations Vii, Llc Immunoreceptor inhibitory proteins and related methods
TW202543581A (zh) 2024-02-12 2025-11-16 美商艾伊拉醫療公司 遞送組成物
TW202543583A (zh) 2024-03-01 2025-11-16 加拿大商阿奎特斯醫療公司 用於將治療劑包覆於脂質奈米顆粒中之材料及方法
WO2025194019A1 (en) 2024-03-14 2025-09-18 Flagship Pioneering Innovations Vii, Llc Methods for treating liver fibrosis and non-alcoholic fatty liver disease
WO2025202937A1 (en) 2024-03-26 2025-10-02 BioNTech SE Cancer vaccines
GB202404607D0 (en) 2024-03-29 2024-05-15 Glaxosmithkline Biologicals Sa RNA formulation
WO2025213131A1 (en) 2024-04-05 2025-10-09 BioNTech SE Rna compositions for delivery of orthopox antigens and related methods
WO2025217275A2 (en) 2024-04-10 2025-10-16 Flagship Pioneering Innovations Vii, Llc Immune cell targeted compositions and related methods
TW202613158A (zh) 2024-05-16 2026-04-01 美商旗艦先鋒創新有限責任（Ｖｉｉ）公司 免疫受體抑制蛋白及相關方法
WO2025240940A1 (en) 2024-05-17 2025-11-20 Scribe Therapeutics Inc. Compositions and methods for the targeting of apolipoprotein c3
WO2025245188A2 (en) 2024-05-21 2025-11-27 Flagship Pioneering Innovations Vii, Llc Methods of treating liver steatosis and non-alcoholic fatty liver disease
WO2025245111A1 (en) 2024-05-22 2025-11-27 Flagship Pioneering Innovations Vii, Llc Immunoreceptor targeting proteins and related methods
WO2025259931A1 (en) 2024-06-14 2025-12-18 Orbital Therapeutics, Inc. Compositions and methods for rna circularization
WO2025262460A1 (en) 2024-06-21 2025-12-26 BioNTech SE Lipid compositions for nucleic acid delivery
WO2026006203A2 (en) 2024-06-24 2026-01-02 Orbital Therapeutics, Inc. Compositions and methods for making circular rna
US20260000702A1 (en) 2024-06-26 2026-01-01 Flagship Pioneering Innovations Vii, Llc Therapeutic circular dna forms
WO2026003582A2 (en) 2024-06-27 2026-01-02 Axelyf ehf. Lipids and lipid nanoparticles
WO2026015882A1 (en) 2024-07-12 2026-01-15 BioNTech SE Hsv antigen fragments and related methods
WO2026018213A1 (en) 2024-07-18 2026-01-22 Vaxthera Sas A broadly protective mosaic vaccine against highly pathogenic avian influenza viruses
WO2026020171A2 (en) 2024-07-19 2026-01-22 Aera Therapeutics, Inc. Chimeric antigen receptor constructs
WO2026028165A1 (en) 2024-08-01 2026-02-05 Precision Biosciences, Inc. Engineered meganucleases having specificity for recognition sequences in the c9orf72 gene
WO2026027887A2 (en) 2024-08-02 2026-02-05 Mina Therapeutics Limited Hbg1/2-sarna compositions and methods of use
WO2026050243A1 (en) 2024-08-26 2026-03-05 Korro Bio, Inc. Galnac conjugated oligonucleotides for rna editing
WO2026047603A1 (en) 2024-08-28 2026-03-05 BioNTech SE Sars-cov-2 immunogenic compositions
WO2026055547A1 (en) 2024-09-06 2026-03-12 Flagship Pioneering Innovations Vii, Llc Dna compositions and related methods
WO2026055543A1 (en) 2024-09-06 2026-03-12 Flagship Pioneering Innovations Vii, Llc Modified dna compositions and related methods
WO2026057625A1 (en) 2024-09-11 2026-03-19 BioNTech SE Peptidoglycan hydrolases having a chap domain with engineered active site
WO2026057626A1 (en) 2024-09-11 2026-03-19 BioNTech SE Peptidoglycan hydrolases having multiple enzymatically active domains
WO2026064313A1 (en) 2024-09-17 2026-03-26 Flagship Pioneering Innovations Vii, Llc Rna compositions and related methods
WO2026064512A1 (en) 2024-09-18 2026-03-26 Generation Bio Co. Polyglycerol-conjugated lipids and lipid nanoparticle compositions comprising the same
WO2026072557A2 (en) 2024-09-24 2026-04-02 Genzyme Corporation Ionizable cationic lipids and lipid nanoparticles, and methods of synthesis and use thereof
WO2026068864A1 (en) 2024-09-30 2026-04-02 BioNTech SE Engineered ch1 and cl domains for the prevention of chain mispairing
WO2026085300A1 (en) 2024-10-16 2026-04-23 Flagship Pioneering Innovations Vii, Llc Compositions and methods
WO2026082899A2 (en) 2024-10-16 2026-04-23 BioNTech SE Rna for preventing or treating tuberculosis
WO2026085495A1 (en) 2024-10-18 2026-04-23 Generation Bio Co. Synthetic partially single-stranded nucleic acid compositions and uses and methods therefor
WO2026088120A1 (en) 2024-10-24 2026-04-30 Pfizer Inc. Lipidated tlr7/8 modulators as adjuvants and uses thereof
WO2026088119A1 (en) 2024-10-24 2026-04-30 Pfizer Inc. Lipidated tlr7/8 modulators as adjuvants and uses thereof
WO2026096919A1 (en) 2024-11-01 2026-05-07 Sail Biomedicines, Inc. Novel dipepetide core lipids, lipid carriers, and methods of using thereof
WO2026102108A1 (en) 2024-11-07 2026-05-15 Flagship Pioneering Innovations Vii, Llc Dna production methods
WO2026112559A1 (en) 2024-11-25 2026-05-28 Genzyme Corporation Ionizable cationic lipids and lipid nanoparticles, and methods of synthesis and use thereof
WO2026119817A1 (en) 2024-12-02 2026-06-11 BioNTech SE Agents and methods for targeted delivery of immune effector cells
WO2026122478A1 (en) 2024-12-02 2026-06-11 Korro Bio, Inc. Extrahepatic delivery formulations and methods
WO2026122747A2 (en) 2024-12-05 2026-06-11 Flagship Pioneering Innovations Vii, Llc Novel immunomodulatory proteins and related methods
WO2026125590A1 (en) 2024-12-13 2026-06-18 BioNTech SE Measuring nucleic acid integrity
WO2026125598A1 (en) 2024-12-13 2026-06-18 BioNTech SE Measuring nucleic acid integrity
WO2026148356A1 (en) 2025-01-06 2026-07-09 BioNTech SE Coronavirus and influenza immunogenic combinations
WO2026152023A1 (en) 2025-01-10 2026-07-16 Sail Biomedicines, Inc. Lipid compounds, lipid nanoparticle compositions and use thereof with polyribonucleotides
WO2026151879A1 (en) 2025-01-10 2026-07-16 Flagship Pioneering Innovations Vii, Llc Vaccine compositions and methods
WO2026150132A1 (en) 2025-01-12 2026-07-16 BioNTech SE Combination therapy comprising an rna cancer vaccine and a multispecific binding agent targeting pd-1/pd-l1 and vegf/vegfr for cancer treatment
WO2026152164A1 (en) 2025-01-13 2026-07-16 Sail Biomedicines, Inc. Cd5-targeted lipid nanoparticles encapsulating polyribonucleotides encoding chimeric antigen receptors, and methods to generate car-expressing t cells in vivo
WO2026161677A1 (en) 2025-01-24 2026-07-30 Flagship Pioneering Innovations Vii, Llc Compositions and methods
WO2026162682A1 (en) 2025-01-29 2026-08-06 BioNTech SE Compositions for delivery of plasmodium rh5 antigens and related methods
WO2026162552A1 (en) 2025-01-31 2026-08-06 BioNTech SE Agents and methods for targeted delivery to cells

Family Cites Families (153)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US3687808A (en) 1969-08-14 1972-08-29 Univ Leland Stanford Junior Synthetic polynucleotides
US3993754A (en) 1974-10-09 1976-11-23 The United States Of America As Represented By The United States Energy Research And Development Administration Liposome-encapsulated actinomycin for cancer chemotherapy
US4086257A (en) 1976-10-12 1978-04-25 Sears Barry D Phosphatidyl quaternary ammonium compounds
CH624011A5 (enExample) 1977-08-05 1981-07-15 Battelle Memorial Institute
US4235871A (en) 1978-02-24 1980-11-25 Papahadjopoulos Demetrios P Method of encapsulating biologically active materials in lipid vesicles
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
US4522803A (en) 1983-02-04 1985-06-11 The Liposome Company, Inc. Stable plurilamellar vesicles, their preparation and use
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
US4603044A (en) 1983-01-06 1986-07-29 Technology Unlimited, Inc. Hepatocyte Directed Vesicle delivery system
US4588578A (en) 1983-08-08 1986-05-13 The Liposome Company, Inc. Lipid vesicles prepared in a monophase
US5118800A (en) 1983-12-20 1992-06-02 California Institute Of Technology Oligonucleotides possessing a primary amino group in the terminal nucleotide
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
FR2567892B1 (fr) 1984-07-19 1989-02-17 Centre Nat Rech Scient Nouveaux oligonucleotides, leur procede de preparation et leurs applications comme mediateurs dans le developpement des effets des interferons
US5367066A (en) 1984-10-16 1994-11-22 Chiron Corporation Oligonucleotides with selectably cleavable and/or abasic sites
FR2575751B1 (fr) 1985-01-08 1987-04-03 Pasteur Institut Nouveaux nucleosides de derives de l'adenosine, leur preparation et leurs applications biologiques
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
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
US5405938A (en) 1989-12-20 1995-04-11 Anti-Gene Development Group Sequence-specific binding polymers for duplex nucleic acids
US4737323A (en) 1986-02-13 1988-04-12 Liposome Technology, Inc. Liposome extrusion method
US5453566A (en) 1986-03-28 1995-09-26 Calgene, Inc. Antisense regulation of gene expression in plant/cells
US4987071A (en) 1986-12-03 1991-01-22 University Patents, Inc. RNA ribozyme polymerases, dephosphorylases, restriction endoribonucleases and methods
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
EP0366685B1 (en) 1987-06-24 1994-10-19 Howard Florey Institute Of Experimental Physiology And Medicine Nucleoside derivatives
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
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
US5175273A (en) 1988-07-01 1992-12-29 Genentech, Inc. Nucleic acid intercalating agents
CA1340323C (en) 1988-09-20 1999-01-19 Arnold E. Hampel Rna catalyst for cleaving specific rna sequences
GB8822492D0 (en) 1988-09-24 1988-10-26 Considine J Apparatus for removing tumours from hollow organs of body
US4957773A (en) 1989-02-13 1990-09-18 Syracuse University Deposition of boron-containing films from decaborane
US5134066A (en) 1989-08-29 1992-07-28 Monsanto Company Improved probes using nucleosides containing 3-dezauracil analogs
CA2039718C (en) 1989-08-31 2003-02-25 John J. Rossi Chimeric dna-rna catalytic sequences
US5591722A (en) 1989-09-15 1997-01-07 Southern Research Institute 2'-deoxy-4'-thioribonucleosides and their antiviral activity
US5286634A (en) 1989-09-28 1994-02-15 Stadler Joan K Synergistic method for host cell transformation
US5013556A (en) 1989-10-20 1991-05-07 Liposome Technology, Inc. Liposomes with enhanced circulation time
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
WO1991006556A1 (en) 1989-10-24 1991-05-16 Gilead Sciences, Inc. 2' modified oligonucleotides
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
US5130302A (en) 1989-12-20 1992-07-14 Boron Bilogicals, Inc. Boronated nucleoside, nucleotide and oligonucleotide compounds, compositions and methods for using same
US5459255A (en) 1990-01-11 1995-10-17 Isis Pharmaceuticals, Inc. N-2 substituted purines
US5681941A (en) 1990-01-11 1997-10-28 Isis Pharmaceuticals, Inc. Substituted purines and oligonucleotide cross-linking
US5670633A (en) 1990-01-11 1997-09-23 Isis Pharmaceuticals, Inc. Sugar modified oligonucleotides that detect and modulate gene expression
US5587361A (en) 1991-10-15 1996-12-24 Isis Pharmaceuticals, Inc. Oligonucleotides having phosphorothioate linkages of high chiral purity
US5646265A (en) 1990-01-11 1997-07-08 Isis Pharmceuticals, Inc. Process for the preparation of 2'-O-alkyl purine phosphoramidites
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
GB9009980D0 (en) 1990-05-03 1990-06-27 Amersham Int Plc Phosphoramidite derivatives,their preparation and the use thereof in the incorporation of reporter groups on synthetic oligonucleotides
EP0455905B1 (en) 1990-05-11 1998-06-17 Microprobe Corporation Dipsticks for nucleic acid hybridization assays and methods for covalently immobilizing oligonucleotides
US6365730B1 (en) 1990-06-19 2002-04-02 Gene Shears Pty. Limited DNA-Armed ribozymes and minizymes
US5618704A (en) 1990-07-27 1997-04-08 Isis Pharmacueticals, Inc. Backbone-modified oligonucleotide analogs and preparation thereof through radical coupling
US5608046A (en) 1990-07-27 1997-03-04 Isis Pharmaceuticals, Inc. Conjugated 4'-desmethyl nucleoside analog compounds
US5541307A (en) 1990-07-27 1996-07-30 Isis Pharmaceuticals, Inc. Backbone modified oligonucleotide analogs and solid phase synthesis thereof
US5610289A (en) 1990-07-27 1997-03-11 Isis Pharmaceuticals, Inc. Backbone modified oligonucleotide analogues
JPH0874B2 (ja) 1990-07-27 1996-01-10 アイシス・ファーマシューティカルス・インコーポレーテッド 遺伝子発現を検出および変調するヌクレアーゼ耐性、ピリミジン修飾オリゴヌクレオチド
US5623070A (en) 1990-07-27 1997-04-22 Isis Pharmaceuticals, Inc. Heteroatomic oligonucleoside linkages
US5489677A (en) 1990-07-27 1996-02-06 Isis Pharmaceuticals, Inc. Oligonucleoside linkages containing adjacent oxygen and nitrogen atoms
US5677437A (en) 1990-07-27 1997-10-14 Isis Pharmaceuticals, Inc. Heteroatomic oligonucleoside linkages
US5602240A (en) 1990-07-27 1997-02-11 Ciba Geigy Ag. Backbone modified oligonucleotide analogs
JPH06502300A (ja) 1990-08-03 1994-03-17 サノフィ 遺伝子発現の抑制のための化合物及び方法
US5789573A (en) 1990-08-14 1998-08-04 Isis Pharmaceuticals, Inc. Antisense inhibition of ICAM-1, E-selectin, and CMV IE1/IE2
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
US5432272A (en) 1990-10-09 1995-07-11 Benner; Steven A. Method for incorporating into a DNA or RNA oligonucleotide using nucleotides bearing heterocyclic bases
AU649074B2 (en) 1990-10-12 1994-05-12 Max-Planck-Gesellschaft Zur Forderung Der Wissenschaften E.V. Modified ribozymes
US5539082A (en) 1993-04-26 1996-07-23 Nielsen; Peter E. Peptide nucleic acids
US5719262A (en) 1993-11-22 1998-02-17 Buchardt, Deceased; Ole Peptide nucleic acids having amino acid side chains
US5714331A (en) 1991-05-24 1998-02-03 Buchardt, Deceased; Ole Peptide nucleic acids having enhanced binding affinity, sequence specificity and solubility
DE4216134A1 (de) 1991-06-20 1992-12-24 Europ Lab Molekularbiolog Synthetische katalytische oligonukleotidstrukturen
US5571799A (en) 1991-08-12 1996-11-05 Basco, Ltd. (2'-5') oligoadenylate analogues useful as inhibitors of host-v5.-graft response
DE59208572D1 (de) 1991-10-17 1997-07-10 Ciba Geigy Ag Bicyclische Nukleoside, Oligonukleotide, Verfahren zu deren Herstellung und Zwischenprodukte
US5594121A (en) 1991-11-07 1997-01-14 Gilead Sciences, Inc. Enhanced triple-helix and double-helix formation with oligomers containing modified purines
US5484908A (en) 1991-11-26 1996-01-16 Gilead Sciences, Inc. Oligonucleotides containing 5-propynyl pyrimidines
US5359044A (en) 1991-12-13 1994-10-25 Isis Pharmaceuticals Cyclobutyl oligonucleotide surrogates
US5652094A (en) 1992-01-31 1997-07-29 University Of Montreal Nucleozymes
FR2687679B1 (fr) 1992-02-05 1994-10-28 Centre Nat Rech Scient Oligothionucleotides.
US5633360A (en) 1992-04-14 1997-05-27 Gilead Sciences, Inc. Oligonucleotide analogs capable of passive cell membrane permeation
CA2135646A1 (en) 1992-05-11 1993-11-25 Kenneth G. Draper Method and reagent for inhibiting viral replication
US5434257A (en) 1992-06-01 1995-07-18 Gilead Sciences, Inc. Binding compentent oligomers containing unsaturated 3',5' and 2',5' linkages
EP0577558A2 (de) 1992-07-01 1994-01-05 Ciba-Geigy Ag Carbocyclische Nukleoside mit bicyclischen Ringen, Oligonukleotide daraus, Verfahren zu deren Herstellung, deren Verwendung und Zwischenproduckte
JPH07509133A (ja) 1992-07-17 1995-10-12 リボザイム・ファーマシューティカルズ・インコーポレイテッド 動物疾患の処置のための方法および剤
US5334761A (en) * 1992-08-28 1994-08-02 Life Technologies, Inc. Cationic lipids
IL108367A0 (en) 1993-01-27 1994-04-12 Hektoen Inst For Medical Resea Antisense polynzcleotide inhibition of human growth factor-sensitive cancer cells
US5476925A (en) 1993-02-01 1995-12-19 Northwestern University Oligodeoxyribonucleotides including 3'-aminonucleoside-phosphoramidate linkages and terminal 3'-amino groups
AU6044994A (en) * 1993-02-19 1994-09-14 Nippon Shinyaku Co. Ltd. Glycerol derivative, device and pharmaceutical composition
GB9304618D0 (en) 1993-03-06 1993-04-21 Ciba Geigy Ag Chemical compounds
EP0691968B1 (en) 1993-03-30 1997-07-16 Sanofi Acyclic nucleoside analogs and oligonucleotide sequences containing them
HU9501974D0 (en) 1993-03-31 1995-09-28 Sterling Winthrop Inc Oligonucleotides with amide linkages replacing phosphodiester linkages
DE4311944A1 (de) 1993-04-10 1994-10-13 Degussa Umhüllte Natriumpercarbonatpartikel, Verfahren zu deren Herstellung und sie enthaltende Wasch-, Reinigungs- und Bleichmittelzusammensetzungen
WO1995002698A1 (en) * 1993-07-12 1995-01-26 Life Technologies, Inc. Composition and methods for transfecting eukaryotic cells
US5532130A (en) 1993-07-20 1996-07-02 Dyad Pharmaceutical Corporation Methods and compositions for sequence-specific hybridization of RNA by 2'-5' oligonucleotides
US5502177A (en) 1993-09-17 1996-03-26 Gilead Sciences, Inc. Pyrimidine derivatives for labeled binding partners
US5801154A (en) 1993-10-18 1998-09-01 Isis Pharmaceuticals, Inc. Antisense oligonucleotide modulation of multidrug resistance-associated protein
US5457187A (en) 1993-12-08 1995-10-10 Board Of Regents University Of Nebraska Oligonucleotides containing 5-fluorouracil
US5446137B1 (en) 1993-12-09 1998-10-06 Behringwerke Ag Oligonucleotides containing 4'-substituted nucleotides
US5674908A (en) * 1993-12-20 1997-10-07 Life Technologies, Inc. Highly packed polycationic ammonium, sulfonium and phosphonium lipids
US5519134A (en) 1994-01-11 1996-05-21 Isis Pharmaceuticals, Inc. Pyrrolidine-containing monomers and oligomers
US6989434B1 (en) * 1994-02-11 2006-01-24 Invitrogen Corporation Reagents for intracellular delivery of macromolecules
US6075012A (en) * 1994-02-11 2000-06-13 Life Technologies, Inc. Reagents for intracellular delivery of macromolecules
US5591317A (en) 1994-02-16 1997-01-07 Pitts, Jr.; M. Michael Electrostatic device for water treatment
US5631359A (en) 1994-10-11 1997-05-20 Ribozyme Pharmaceuticals, Inc. Hairpin ribozymes
US5596091A (en) 1994-03-18 1997-01-21 The Regents Of The University Of California Antisense oligonucleotides comprising 5-aminoalkyl pyrimidine nucleotides
US5627053A (en) 1994-03-29 1997-05-06 Ribozyme Pharmaceuticals, Inc. 2'deoxy-2'-alkylnucleotide containing nucleic acid
US5625050A (en) 1994-03-31 1997-04-29 Amgen Inc. Modified oligonucleotides and intermediates useful in nucleic acid therapeutics
US5525711A (en) 1994-05-18 1996-06-11 The United States Of America As Represented By The Secretary Of The Department Of Health And Human Services Pteridine nucleotide analogs as fluorescent DNA probes
US5534499A (en) 1994-05-19 1996-07-09 The University Of British Columbia Lipophilic drug derivatives for use in liposomes
US5597909A (en) 1994-08-25 1997-01-28 Chiron Corporation Polynucleotide reagents containing modified deoxyribose moieties, and associated methods of synthesis and use
US5753613A (en) * 1994-09-30 1998-05-19 Inex Pharmaceuticals Corporation Compositions for the introduction of polyanionic materials into cells
US5885613A (en) 1994-09-30 1999-03-23 The University Of British Columbia Bilayer stabilizing components and their use in forming programmable fusogenic liposomes
AU3559695A (en) 1994-09-30 1996-04-26 Inex Pharmaceuticals Corp. Glycosylated protein-liposome conjugates and methods for their preparation
US5820873A (en) 1994-09-30 1998-10-13 The University Of British Columbia Polyethylene glycol modified ceramide lipids and liposome uses thereof
AU3889595A (en) 1994-10-05 1996-05-02 Amgen, Inc. Method for inhibiting smooth muscle cell proliferation and oligonucleotides for use therein
US5627159A (en) * 1994-10-27 1997-05-06 Life Technologies, Inc. Enhancement of lipid cationic transfections in the presence of serum
US5783683A (en) 1995-01-10 1998-07-21 Genta Inc. Antisense oligonucleotides which reduce expression of the FGFRI gene
US6051429A (en) * 1995-06-07 2000-04-18 Life Technologies, Inc. Peptide-enhanced cationic lipid transfections
JP4335310B2 (ja) 1995-06-07 2009-09-30 ザ ユニバーシティ オブ ブリティッシュ コロンビア 疎水性脂質−核酸複合中間体を通して調製される脂質−核酸粒子、及び遺伝子移送のための使用
JPH11507815A (ja) * 1995-06-07 1999-07-13 ジンタ・インコーポレイテッド ホスホン酸基本カチオン性脂質
US5747470A (en) 1995-06-07 1998-05-05 Gen-Probe Incorporated Method for inhibiting cellular proliferation using antisense oligonucleotides to gp130 mRNA
WO1996040961A1 (en) * 1995-06-07 1996-12-19 Life Technologies, Inc. Peptide-enhanced cationic lipid transfections
US20030069173A1 (en) * 1998-03-16 2003-04-10 Life Technologies, Inc. Peptide-enhanced transfections
CA2223088A1 (en) * 1995-06-07 1996-12-19 Bob Dale Brown Novel carbamate-based cationic lipids
US6339173B1 (en) * 1996-07-22 2002-01-15 Promega Biosciences, Inc. Amide-based cationic lipids
US6020526A (en) * 1995-07-21 2000-02-01 Genta, Incorporated Amide-based cationic lipids
US5739119A (en) 1996-11-15 1998-04-14 Galli; Rachel L. Antisense oligonucleotides specific for the muscarinic type 2 acetylcholine receptor MRNA
US5877220A (en) * 1997-03-06 1999-03-02 Genta, Incorporated Amide-based oligomeric cationic lipids
US6034135A (en) * 1997-03-06 2000-03-07 Promega Biosciences, Inc. Dimeric cationic lipids
US6406705B1 (en) 1997-03-10 2002-06-18 University Of Iowa Research Foundation Use of nucleic acids containing unmethylated CpG dinucleotide as an adjuvant
US6235310B1 (en) * 1997-04-04 2001-05-22 Valentis, Inc. Methods of delivery using cationic lipids and helper lipids
CA2289702C (en) 1997-05-14 2008-02-19 Inex Pharmaceuticals Corp. High efficiency encapsulation of charged therapeutic agents in lipid vesicles
US6320017B1 (en) 1997-12-23 2001-11-20 Inex Pharmaceuticals Corp. Polyamide oligomers
CA2350882C (en) * 1998-11-12 2011-02-22 Life Technologies Corporation Transfection reagents
AU780535B2 (en) 1999-08-27 2005-03-24 Inex Pharmaceuticals Corp. Compositions for stimulating cytokine secretion and inducing an immune response
CA2427068A1 (en) * 2000-10-27 2002-05-02 Invitrogen Corporation Method for introducing antisense oligonucleotides into eucaryotic cells
WO2002069369A2 (en) 2000-12-08 2002-09-06 Coley Pharmaceutical Gmbh Cpg-like nucleic acids and methods of use thereof
JP2004003808A (ja) * 2002-03-27 2004-01-08 Osaka Gas Co Ltd 冷凍用熱搬送システムの摩擦低減方法
DE10361170A1 (de) * 2003-06-13 2005-01-05 Henkel Kgaa Lagerstabiles Polyelektrolytkapselsystem auf Basis von Peroxycarbonsäuren
CN1882693B (zh) * 2003-09-15 2012-08-15 普洛体维生物治疗公司 聚乙二醇修饰的脂质化合物及其应用
CA2569664C (en) * 2004-06-07 2013-07-16 Protiva Biotherapeutics, Inc. Lipid encapsulated interfering rna
US7745651B2 (en) * 2004-06-07 2010-06-29 Protiva Biotherapeutics, Inc. Cationic lipids and methods of use
WO2006074546A1 (en) * 2005-01-13 2006-07-20 Protiva Biotherapeutics, Inc. Lipid encapsulated interfering rna
WO2006102163A2 (en) * 2005-03-17 2006-09-28 Invitrogen Corporation Transfection reagents for non-adherent suspension cells
US9006191B2 (en) * 2007-12-27 2015-04-14 Protiva Biotherapeutics, Inc. Silencing of polo-like kinase expression using interfering RNA
CA2760706C (en) * 2009-05-05 2019-08-06 Alnylam Pharmaceuticals, Inc. Methods of delivering oligonucleotides to immune cells
ES2702428T3 (es) * 2010-11-15 2019-02-28 Life Technologies Corp Reactivos de transfección que contienen amina y métodos para prepararlos y usarlos

Also Published As

Publication number Publication date
JP2011509258A (ja) 2011-03-24
EP2224912A1 (en) 2010-09-08
EP2224912B1 (en) 2016-05-11
CA2709875C (en) 2019-07-16
EP2224912A4 (en) 2012-01-04
WO2009086558A1 (en) 2009-07-09
EP3100718B1 (en) 2019-11-27
CA3044134A1 (en) 2009-07-09
JP2014132014A (ja) 2014-07-17
US20110117125A1 (en) 2011-05-19
EP3100718A1 (en) 2016-12-07
CA2709875A1 (en) 2009-07-09

Similar Documents

Publication Publication Date Title
JP6505912B2 (ja) 改良されたアミノ脂質および核酸の送達方法
EP3100718B1 (en) Improved compositions and methods for the delivery of nucleic acids
AU2015210364B2 (en) Improved amino lipids and methods for the delivery of nucleic acids
AU2010208035B2 (en) Improved lipid formulation for the delivery of nucleic acids
WO2010048536A2 (en) Processes for preparing lipids
WO2009132131A1 (en) Amino lipid based improved lipid formulation
HK1160459B (en) Improved amino lipids and methods for the delivery of nucleic acids

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20111118

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20111118

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20111227

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20121106

A977 Report on retrieval

Free format text: JAPANESE INTERMEDIATE CODE: A971007

Effective date: 20130822

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20130826

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20140219

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20140403

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20140626

A602 Written permission of extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A602

Effective date: 20140703

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20141003

TRDD Decision of grant or rejection written
A01 Written decision to grant a patent or to grant a registration (utility model)

Free format text: JAPANESE INTERMEDIATE CODE: A01

Effective date: 20150318

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20150331

A61 First payment of annual fees (during grant procedure)

Free format text: JAPANESE INTERMEDIATE CODE: A61

Effective date: 20150514

R150 Certificate of patent or registration of utility model

Ref document number: 5749494

Country of ref document: JP

Free format text: JAPANESE INTERMEDIATE CODE: R150

R250 Receipt of annual fees

Free format text: JAPANESE INTERMEDIATE CODE: R250

RD04 Notification of resignation of power of attorney

Free format text: JAPANESE INTERMEDIATE CODE: R3D04

R250 Receipt of annual fees

Free format text: JAPANESE INTERMEDIATE CODE: R250

LAPS Cancellation because of no payment of annual fees