SG11202106987WA - Lipids for lipid nanoparticle delivery of active agents - Google Patents

Lipids for lipid nanoparticle delivery of active agents

Info

Publication number
SG11202106987WA
SG11202106987WA SG11202106987WA SG11202106987WA SG11202106987WA SG 11202106987W A SG11202106987W A SG 11202106987WA SG 11202106987W A SG11202106987W A SG 11202106987WA SG 11202106987W A SG11202106987W A SG 11202106987WA SG 11202106987W A SG11202106987W A SG 11202106987WA
Authority
SG
Singapore
Prior art keywords
lipids
active agents
lipid nanoparticle
nanoparticle delivery
delivery
Prior art date
Application number
SG11202106987WA
Inventor
Xinyao Du
Original Assignee
Acuitas Therapeutics Inc
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 Acuitas Therapeutics Inc filed Critical Acuitas Therapeutics Inc
Publication of SG11202106987WA publication Critical patent/SG11202106987WA/en

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Classifications

    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07C—ACYCLIC OR CARBOCYCLIC COMPOUNDS
    • C07C233/00—Carboxylic acid amides
    • C07C233/01—Carboxylic acid amides having carbon atoms of carboxamide groups bound to hydrogen atoms or to acyclic carbon atoms
    • C07C233/45—Carboxylic acid amides having carbon atoms of carboxamide groups bound to hydrogen atoms or to acyclic carbon atoms having the nitrogen atom of at least one of the carboxamide groups bound to a carbon atom of a hydrocarbon radical substituted by carboxyl groups
    • C07C233/46—Carboxylic acid amides having carbon atoms of carboxamide groups bound to hydrogen atoms or to acyclic carbon atoms having the nitrogen atom of at least one of the carboxamide groups bound to a carbon atom of a hydrocarbon radical substituted by carboxyl groups with the substituted hydrocarbon radical bound to the nitrogen atom of the carboxamide group by an acyclic carbon atom
    • C07C233/47—Carboxylic acid amides having carbon atoms of carboxamide groups bound to hydrogen atoms or to acyclic carbon atoms having the nitrogen atom of at least one of the carboxamide groups bound to a carbon atom of a hydrocarbon radical substituted by carboxyl groups with the substituted hydrocarbon radical bound to the nitrogen atom of the carboxamide group by an acyclic carbon atom having the carbon atom of the carboxamide group bound to a hydrogen atom or to a carbon atom of an acyclic saturated carbon skeleton
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07C—ACYCLIC OR CARBOCYCLIC COMPOUNDS
    • C07C237/00—Carboxylic acid amides, the carbon skeleton of the acid part being further substituted by amino groups
    • C07C237/02—Carboxylic acid amides, the carbon skeleton of the acid part being further substituted by amino groups having the carbon atoms of the carboxamide groups bound to acyclic carbon atoms of the carbon skeleton
    • C07C237/04—Carboxylic acid amides, the carbon skeleton of the acid part being further substituted by amino groups having the carbon atoms of the carboxamide groups bound to acyclic carbon atoms of the carbon skeleton the carbon skeleton being acyclic and saturated
    • C07C237/08—Carboxylic acid amides, the carbon skeleton of the acid part being further substituted by amino groups having the carbon atoms of the carboxamide groups bound to acyclic carbon atoms of the carbon skeleton the carbon skeleton being acyclic and saturated having the nitrogen atom of at least one of the carboxamide groups bound to an acyclic carbon atom of a hydrocarbon radical substituted by singly-bound oxygen atoms
    • 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—Liposomes
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07C—ACYCLIC OR CARBOCYCLIC COMPOUNDS
    • C07C237/00—Carboxylic acid amides, the carbon skeleton of the acid part being further substituted by amino groups
    • C07C237/02—Carboxylic acid amides, the carbon skeleton of the acid part being further substituted by amino groups having the carbon atoms of the carboxamide groups bound to acyclic carbon atoms of the carbon skeleton
    • C07C237/04—Carboxylic acid amides, the carbon skeleton of the acid part being further substituted by amino groups having the carbon atoms of the carboxamide groups bound to acyclic carbon atoms of the carbon skeleton the carbon skeleton being acyclic and saturated
    • C07C237/12—Carboxylic acid amides, the carbon skeleton of the acid part being further substituted by amino groups having the carbon atoms of the carboxamide groups bound to acyclic carbon atoms of the carbon skeleton the carbon skeleton being acyclic and saturated having the nitrogen atom of at least one of the carboxamide groups bound to an acyclic carbon atom of a hydrocarbon radical substituted by carboxyl groups
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D207/00—Heterocyclic compounds containing five-membered rings not condensed with other rings, with one nitrogen atom as the only ring hetero atom
    • C07D207/02—Heterocyclic compounds containing five-membered rings not condensed with other rings, with one nitrogen atom as the only ring hetero atom with only hydrogen or carbon atoms directly attached to the ring nitrogen atom
    • C07D207/04—Heterocyclic compounds containing five-membered rings not condensed with other rings, with one nitrogen atom as the only ring hetero atom with only hydrogen or carbon atoms directly attached to the ring nitrogen atom having no double bonds between ring members or between ring members and non-ring members
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D295/00—Heterocyclic compounds containing polymethylene-imine rings with at least five ring members, 3-azabicyclo [3.2.2] nonane, piperazine, morpholine or thiomorpholine rings, having only hydrogen atoms directly attached to the ring carbon atoms
    • C07D295/04—Heterocyclic compounds containing polymethylene-imine rings with at least five ring members, 3-azabicyclo [3.2.2] nonane, piperazine, morpholine or thiomorpholine rings, having only hydrogen atoms directly attached to the ring carbon atoms with substituted hydrocarbon radicals attached to ring nitrogen atoms
    • C07D295/14—Heterocyclic compounds containing polymethylene-imine rings with at least five ring members, 3-azabicyclo [3.2.2] nonane, piperazine, morpholine or thiomorpholine rings, having only hydrogen atoms directly attached to the ring carbon atoms with substituted hydrocarbon radicals attached to ring nitrogen atoms substituted by carbon atoms having three bonds to hetero atoms with at the most one bond to halogen, e.g. ester or nitrile radicals
    • C07D295/145—Heterocyclic compounds containing polymethylene-imine rings with at least five ring members, 3-azabicyclo [3.2.2] nonane, piperazine, morpholine or thiomorpholine rings, having only hydrogen atoms directly attached to the ring carbon atoms with substituted hydrocarbon radicals attached to ring nitrogen atoms substituted by carbon atoms having three bonds to hetero atoms with at the most one bond to halogen, e.g. ester or nitrile radicals with the ring nitrogen atoms and the carbon atoms with three bonds to hetero atoms attached to the same carbon chain, which is not interrupted by carbocyclic rings
    • C07D295/15—Heterocyclic compounds containing polymethylene-imine rings with at least five ring members, 3-azabicyclo [3.2.2] nonane, piperazine, morpholine or thiomorpholine rings, having only hydrogen atoms directly attached to the ring carbon atoms with substituted hydrocarbon radicals attached to ring nitrogen atoms substituted by carbon atoms having three bonds to hetero atoms with at the most one bond to halogen, e.g. ester or nitrile radicals with the ring nitrogen atoms and the carbon atoms with three bonds to hetero atoms attached to the same carbon chain, which is not interrupted by carbocyclic rings to an acyclic saturated chain
    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering N.A.
SG11202106987WA 2019-01-11 2020-01-10 Lipids for lipid nanoparticle delivery of active agents SG11202106987WA (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962791566P 2019-01-11 2019-01-11
US201962890469P 2019-08-22 2019-08-22
PCT/US2020/013196 WO2020146805A1 (en) 2019-01-11 2020-01-10 Lipids for lipid nanoparticle delivery of active agents

Publications (1)

Publication Number Publication Date
SG11202106987WA true SG11202106987WA (en) 2021-07-29

Family

ID=69469244

Family Applications (1)

Application Number Title Priority Date Filing Date
SG11202106987WA SG11202106987WA (en) 2019-01-11 2020-01-10 Lipids for lipid nanoparticle delivery of active agents

Country Status (12)

Country Link
US (2) US11453639B2 (en)
EP (1) EP3908568A1 (en)
JP (1) JP2022517783A (en)
KR (1) KR20210138569A (en)
CN (1) CN113474328A (en)
AU (1) AU2020205717A1 (en)
BR (1) BR112021013654A2 (en)
CA (1) CA3125485A1 (en)
IL (1) IL284535A (en)
MX (1) MX2021008358A (en)
SG (1) SG11202106987WA (en)
WO (1) WO2020146805A1 (en)

Families Citing this family (34)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA3179824A1 (en) 2014-06-25 2015-12-30 Acuitas Therapeutics Inc. Lipids and lipid nanoparticle formulations for delivery of nucleic acids
JP7072386B2 (en) 2015-06-29 2022-05-20 アクイタス セラピューティクス インコーポレイテッド Lipid and Lipid Nanoparticle Formulations for Nucleic Acid Delivery
PL3368507T3 (en) 2015-10-28 2023-03-27 Acuitas Therapeutics Inc. Novel lipids and lipid nanoparticle formulations for delivery of nucleic acids
WO2018191657A1 (en) 2017-04-13 2018-10-18 Acuitas Therapeutics, Inc. Lipids for delivery of active agents
CA3061612A1 (en) 2017-04-28 2018-11-01 Acuitas Therapeutics, Inc. Novel carbonyl lipids and lipid nanoparticle formulations for delivery of nucleic acids
US11639329B2 (en) 2017-08-16 2023-05-02 Acuitas Therapeutics, Inc. Lipids for use in lipid nanoparticle formulations
US11542225B2 (en) 2017-08-17 2023-01-03 Acuitas Therapeutics, Inc. Lipids for use in lipid nanoparticle formulations
US11524932B2 (en) 2017-08-17 2022-12-13 Acuitas Therapeutics, Inc. Lipids for use in lipid nanoparticle formulations
JP2022548399A (en) 2019-09-23 2022-11-18 オメガ セラピューティクス， インコーポレイテッド Compositions and methods for modulating hepatocyte nuclear factor 4-alpha (HNF4α) gene expression
WO2021061707A1 (en) 2019-09-23 2021-04-01 Omega Therapeutics, Inc. Compositions and methods for modulating apolipoprotein b (apob) gene expression
JP2023517326A (en) 2020-03-11 2023-04-25 オメガ セラピューティクス， インコーポレイテッド Compositions and methods for modulating forkhead box P3 (FOXP3) gene expression
CN113874507A (en) 2020-04-09 2021-12-31 苏州艾博生物科技有限公司 Nucleic acid vaccine for coronavirus
BR112022020203A2 (en) 2020-04-09 2022-11-22 Suzhou Abogen Biosciences Co Ltd COMPOSITION OF LIPID NANOPARTICLES
EP4132478A1 (en) 2020-04-09 2023-02-15 Finncure Oy Mimetic nanoparticles for preventing the spreading and lowering the infection rate of novel coronaviruses
US11524023B2 (en) 2021-02-19 2022-12-13 Modernatx, Inc. Lipid nanoparticle compositions and methods of formulating the same
WO2023283359A2 (en) 2021-07-07 2023-01-12 Omega Therapeutics, Inc. Compositions and methods for modulating secreted frizzled receptor protein 1 (sfrp1) gene expression
AU2022337090A1 (en) 2021-09-03 2024-02-15 Glaxosmithkline Biologicals Sa Substitution of nucleotide bases in self-amplifying messenger ribonucleic acids
CA3230031A1 (en) 2021-09-03 2023-03-09 Patrick Baumhof Novel lipid nanoparticles for delivery of nucleic acids
AR127312A1 (en) 2021-10-08 2024-01-10 Suzhou Abogen Biosciences Co Ltd LIPID COMPOUNDS AND LIPID NANOPARTICLE COMPOSITIONS
AU2022361755A1 (en) 2021-10-08 2024-04-04 Pfizer Inc. Immunogenic lnp compositions and methods thereof
WO2023069498A1 (en) 2021-10-22 2023-04-27 Senda Biosciences, Inc. Mrna vaccine composition
WO2023073228A1 (en) 2021-10-29 2023-05-04 CureVac SE Improved circular rna for expressing therapeutic proteins
US20230181482A1 (en) * 2021-11-18 2023-06-15 Astrazeneca Ab Lipids for delivery of nucleic acid segments
WO2023096858A1 (en) 2021-11-23 2023-06-01 Senda Biosciences, Inc. A bacteria-derived lipid composition and use thereof
WO2023111907A1 (en) 2021-12-17 2023-06-22 Pfizer Inc. Polynucleotide compositions and uses thereof
WO2023122080A1 (en) 2021-12-20 2023-06-29 Senda Biosciences, Inc. Compositions comprising mrna and lipid reconstructed plant messenger packs
WO2023144330A1 (en) 2022-01-28 2023-08-03 CureVac SE Nucleic acid encoded transcription factor inhibitors
WO2023160702A1 (en) * 2022-02-28 2023-08-31 深圳深信生物科技有限公司 Amino lipid compound, preparation method therefor, composition thereof and use thereof
WO2023177904A1 (en) 2022-03-18 2023-09-21 Modernatx, Inc. Sterile filtration of lipid nanoparticles and filtration analysis thereof for biological applications
CN116813493A (en) * 2022-03-21 2023-09-29 苏州科锐迈德生物医药科技有限公司 Lipid compound, lipid carrier based on lipid compound, nucleic acid lipid nanoparticle composition and pharmaceutical preparation
WO2023227608A1 (en) 2022-05-25 2023-11-30 Glaxosmithkline Biologicals Sa Nucleic acid based vaccine encoding an escherichia coli fimh antigenic polypeptide
WO2023242817A2 (en) 2022-06-18 2023-12-21 Glaxosmithkline Biologicals Sa Recombinant rna molecules comprising untranslated regions or segments encoding spike protein from the omicron strain of severe acute respiratory coronavirus-2
WO2023248128A1 (en) 2022-06-23 2023-12-28 Pfizer Inc. Use of a spray freeze-drying process for the lyophilization of a mrna-encapsulating lipid nanoparticles formulation
WO2024044147A1 (en) 2022-08-23 2024-02-29 Modernatx, Inc. Methods for purification of ionizable lipids

Family Cites Families (278)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6346516B1 (en) 1998-11-09 2002-02-12 Council Of Scientific & Industrial Research Cationic amphiphiles containing N-hydroxyalkyl group for intracellular delivery of biologically active molecules
US2856420A (en) 1955-12-15 1958-10-14 Minnesota Mining & Mfg Perfluoro- and perfluorochlorocarboxylic acid esters of amino alcohols
US3340299A (en) 1964-03-27 1967-09-05 Air Reduction Tetrasubstituted ethylene diamines
GB1277947A (en) 1968-08-22 1972-06-14 Armour Ind Chem Co Compositions and method for controlling insect pests
US3729564A (en) 1970-12-16 1973-04-24 Pfizer N-secondary alkyl alkanediamines and derivatives thereof as anti-inflammatory agents
JPS5122416B2 (en) 1972-11-11 1976-07-09
DE2633615C3 (en) 1976-07-27 1981-08-13 Bayer Ag, 5090 Leverkusen Process for dyeing synthetic polyamide fiber materials
DE3050800C2 (en) 1979-03-22 1989-06-22 Continental Pharma Inc., Bruessel/Bruxelles, Be
US4897355A (en) 1985-01-07 1990-01-30 Syntex (U.S.A.) Inc. N[ω,(ω-1)-dialkyloxy]- and N-[ω,(ω-1)-dialkenyloxy]-alk-1-yl-N,N,N-tetrasubstituted ammonium lipids and uses therefor
US5049386A (en) 1985-01-07 1991-09-17 Syntex (U.S.A.) Inc. N-ω,(ω-1)-dialkyloxy)- and N-(ω,(ω-1)-dialkenyloxy)Alk-1-YL-N,N,N-tetrasubstituted ammonium lipids and uses therefor
US4946787A (en) 1985-01-07 1990-08-07 Syntex (U.S.A.) Inc. N-(ω,(ω-1)-dialkyloxy)- and N-(ω,(ω-1)-dialkenyloxy)-alk-1-yl-N,N,N-tetrasubstituted ammonium lipids and uses therefor
US5422251A (en) 1986-11-26 1995-06-06 Princeton University Triple-stranded nucleic acids
US5176996A (en) 1988-12-20 1993-01-05 Baylor College Of Medicine Method for making synthetic oligonucleotides which bind specifically to target sites on duplex DNA molecules, by forming a colinear triplex, the synthetic oligonucleotides and methods of use
US6077509A (en) 1990-03-30 2000-06-20 Autoimmune, Inc. Peptide fragments of myelin basic protein
US5420032A (en) 1991-12-23 1995-05-30 Universitge Laval Homing endonuclease which originates from chlamydomonas eugametos and recognizes and cleaves a 15, 17 or 19 degenerate double stranded nucleotide sequence
US5792632A (en) 1992-05-05 1998-08-11 Institut Pasteur Nucleotide sequence encoding the enzyme I-SceI and the uses thereof
JP2588339B2 (en) 1992-06-02 1997-03-05 花王株式会社 Novel diamino diester and method for producing the same
US6140466A (en) 1994-01-18 2000-10-31 The Scripps Research Institute Zinc finger protein derivatives and methods therefor
ATE310812T1 (en) 1994-01-18 2005-12-15 Scripps Research Inst DERIVATIVES OF ZINC FINGER PROTEINS AND METHODS
US6242568B1 (en) 1994-01-18 2001-06-05 The Scripps Research Institute Zinc finger protein derivatives and methods therefor
DE69535853D1 (en) 1994-03-23 2008-11-20 Univ Case Western Reserve COMPACT NUCLEIC ACID AND THEIR ADMINISTRATION IN CELLS
US6197553B1 (en) 1994-07-15 2001-03-06 Merck & Co., Inc. Method for large scale plasmid purification
GB9824544D0 (en) 1998-11-09 1999-01-06 Medical Res Council Screening system
USRE45721E1 (en) 1994-08-20 2015-10-06 Gendaq, Ltd. Relating to binding proteins for recognition of DNA
FR2727679B1 (en) 1994-12-05 1997-01-03 Rhone Poulenc Rorer Sa NEW TRANSFECTION AGENTS AND THEIR PHARMACEUTICAL APPLICATIONS
US5795587A (en) 1995-01-23 1998-08-18 University Of Pittsburgh Stable lipid-comprising drug delivery complexes and methods for their production
US5789538A (en) 1995-02-03 1998-08-04 Massachusetts Institute Of Technology Zinc finger proteins with high affinity new DNA binding specificities
US5705385A (en) 1995-06-07 1998-01-06 Inex Pharmaceuticals Corporation Lipid-nucleic acid particles prepared via a hydrophobic lipid-nucleic acid complex intermediate and use for gene transfer
US5976567A (en) 1995-06-07 1999-11-02 Inex Pharmaceuticals Corp. Lipid-nucleic acid particles prepared via a hydrophobic lipid-nucleic acid complex intermediate and use for gene transfer
US5981501A (en) 1995-06-07 1999-11-09 Inex Pharmaceuticals Corp. Methods for encapsulating plasmids in lipid bilayers
US7422902B1 (en) 1995-06-07 2008-09-09 The University Of British Columbia Lipid-nucleic acid particles prepared via a hydrophobic lipid-nucleic acid complex intermediate and use for gene transfer
EP0869937A4 (en) 1995-07-21 2004-07-21 Promega Biosciences Inc Novel amide-based cationic lipids
DE19605175A1 (en) 1996-02-13 1997-08-14 Sourovoi Andrej Dr Lipid compounds and their use
US5925523A (en) 1996-08-23 1999-07-20 President & Fellows Of Harvard College Intraction trap assay, reagents and uses thereof
JP2001505934A (en) 1996-10-11 2001-05-08 インフィニューム ホールディングス ベスローテン フェンノートシャップ Fuel composition
CA2217550A1 (en) 1996-10-22 1998-04-22 F. Hoffmann-La Roche Ag Cationic lipids for gene therapy
US6884430B1 (en) 1997-02-10 2005-04-26 Aventis Pharma S.A. Formulation of stabilized cationic transfection agent(s) /nucleic acid particles
GB9703369D0 (en) 1997-02-18 1997-04-09 Lindqvist Bjorn H Process
GB2338237B (en) 1997-02-18 2001-02-28 Actinova Ltd In vitro peptide or protein expression library
US5965542A (en) 1997-03-18 1999-10-12 Inex Pharmaceuticals Corp. Use of temperature to control the size of cationic liposome/plasmid DNA complexes
US5756785A (en) 1997-03-21 1998-05-26 Lambent Technologies, Inc. Guerbet betaines
GB9710807D0 (en) 1997-05-23 1997-07-23 Medical Res Council Nucleic acid binding proteins
GB9710809D0 (en) 1997-05-23 1997-07-23 Medical Res Council Nucleic acid binding proteins
FR2763943B1 (en) 1997-05-28 1999-07-09 Rhone Poulenc Rorer Sa COMPOUNDS, THEIR PREPARATION AND THEIR USE FOR TRANSFERRING NUCLEIC ACIDS INTO CELLS
US6395713B1 (en) 1997-07-23 2002-05-28 Ribozyme Pharmaceuticals, Inc. Compositions for the delivery of negatively charged molecules
US20030073640A1 (en) 1997-07-23 2003-04-17 Ribozyme Pharmaceuticals, Inc. Novel compositions for the delivery of negatively charged molecules
US6989264B2 (en) 1997-09-05 2006-01-24 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
WO1999033493A1 (en) 1997-12-23 1999-07-08 Inex Pharmaceuticals Corporation Polyamide oligomers
US6410248B1 (en) 1998-01-30 2002-06-25 Massachusetts Institute Of Technology General strategy for selecting high-affinity zinc finger proteins for diverse DNA target sites
US6410328B1 (en) 1998-02-03 2002-06-25 Protiva Biotherapeutics Inc. Sensitizing cells to compounds using lipid-mediated gene and compound delivery
JP4309051B2 (en) 1998-03-02 2009-08-05 マサチューセッツ インスティテュート オブ テクノロジー Polyzinc finger protein with improved linker
US6986902B1 (en) 1998-04-28 2006-01-17 Inex Pharmaceuticals Corporation Polyanionic polymers which enhance fusogenicity
US6013813A (en) 1998-06-17 2000-01-11 Hansotech Inc Guerbet based sorbitan esters
US6140081A (en) 1998-10-16 2000-10-31 The Scripps Research Institute Zinc finger binding domains for GNN
US6656498B1 (en) 1998-11-25 2003-12-02 Vanderbilt University Cationic liposomes for gene transfer
US5919743A (en) 1998-12-28 1999-07-06 Petroferm Inc. Guerbet branched quaternary compounds in personal care applications
US6599692B1 (en) 1999-09-14 2003-07-29 Sangamo Bioscience, Inc. Functional genomics using zinc finger proteins
US6453242B1 (en) 1999-01-12 2002-09-17 Sangamo Biosciences, Inc. Selection of sites for targeting by zinc finger proteins and methods of designing zinc finger proteins to bind to preselected sites
US7013219B2 (en) 1999-01-12 2006-03-14 Sangamo Biosciences, Inc. Regulation of endogenous gene expression in cells using zinc finger proteins
US6503717B2 (en) 1999-12-06 2003-01-07 Sangamo Biosciences, Inc. Methods of using randomized libraries of zinc finger proteins for the identification of gene function
US7070934B2 (en) 1999-01-12 2006-07-04 Sangamo Biosciences, Inc. Ligand-controlled regulation of endogenous gene expression
US6534261B1 (en) 1999-01-12 2003-03-18 Sangamo Biosciences, Inc. Regulation of endogenous gene expression in cells using zinc finger proteins
US7030215B2 (en) 1999-03-24 2006-04-18 Sangamo Biosciences, Inc. Position dependent recognition of GNN nucleotide triplets by zinc fingers
US6794136B1 (en) 2000-11-20 2004-09-21 Sangamo Biosciences, Inc. Iterative optimization in the design of binding proteins
US7112337B2 (en) 1999-04-23 2006-09-26 Alza Corporation Liposome composition for delivery of nucleic acid
US6211140B1 (en) 1999-07-26 2001-04-03 The Procter & Gamble Company Cationic charge boosting systems
GB9930533D0 (en) 1999-12-23 2000-02-16 Mitsubishi Tokyo Pharm Inc Nucleic acid delivery
WO2001059450A2 (en) 2000-02-08 2001-08-16 Sangamo Biosciences, Inc. Cells expressing zinc finger protein for drug discovery
US20020061512A1 (en) 2000-02-18 2002-05-23 Kim Jin-Soo Zinc finger domains and methods of identifying same
ATE374249T1 (en) 2000-04-28 2007-10-15 Sangamo Biosciences Inc DATABASES OF REGULATORY SEQUENCES, METHODS OF THEIR PRODUCTION AND USE
US7001768B2 (en) 2000-04-28 2006-02-21 Sangamo Biosciences, Inc. Targeted modification of chromatin structure
US7923542B2 (en) 2000-04-28 2011-04-12 Sangamo Biosciences, Inc. Libraries of regulatory sequences, methods of making and using same
US20030044787A1 (en) 2000-05-16 2003-03-06 Joung J. Keith Methods and compositions for interaction trap assays
JP2001338416A (en) 2000-05-25 2001-12-07 Sony Corp Discoid magnetic recording medium
JP2002060786A (en) 2000-08-23 2002-02-26 Kao Corp Germicidal stainproofing agent for hard surface
US20040142474A1 (en) 2000-09-14 2004-07-22 Expression Genetics, Inc. Novel cationic lipopolymer as a biocompatible gene delivery agent
US7067317B2 (en) 2000-12-07 2006-06-27 Sangamo Biosciences, Inc. Regulation of angiogenesis with zinc finger proteins
GB0108491D0 (en) 2001-04-04 2001-05-23 Gendaq Ltd Engineering zinc fingers
JP4527394B2 (en) 2001-06-01 2010-08-18 バイオジェン・アイデック・エムエイ・インコーポレイテッド Molecules and methods for inhibiting KIM-1 disruption
WO2003016496A2 (en) 2001-08-20 2003-02-27 The Scripps Research Institute Zinc finger binding domains for cnn
US6723551B2 (en) 2001-11-09 2004-04-20 The United States Of America As Represented By The Department Of Health And Human Services Production of adeno-associated virus in insect cells
EP1572893B1 (en) 2001-11-09 2009-01-07 THE GOVERNMENT OF THE UNITED STATES OF AMERICA as represented by THE SECRETARY of the DEPARTMENT OF HEALTH AND HUMAN SERVICES Production of adeno-associated virus in insect cells
US7262054B2 (en) 2002-01-22 2007-08-28 Sangamo Biosciences, Inc. Zinc finger proteins for DNA binding and gene regulation in plants
AU2003251286B2 (en) 2002-01-23 2007-08-16 The University Of Utah Research Foundation Targeted chromosomal mutagenesis using zinc finger nucleases
US20050222064A1 (en) 2002-02-20 2005-10-06 Sirna Therapeutics, Inc. Polycationic compositions for cellular delivery of polynucleotides
ES2292994T3 (en) 2002-03-15 2008-03-16 Cellectis SIMPLE HYBRID AND CHAIN MEGANUCLEASES AND ITS USE.
CA2479858A1 (en) 2002-03-21 2003-10-02 Monika Liljedahl Methods and compositions for using zinc finger endonucleases to enhance homologous recombination
US7074596B2 (en) 2002-03-25 2006-07-11 Board Of Supervisors Of Louisiana State University And Agricultural And Mechanical College Synthesis and use of anti-reverse mRNA cap analogues
JP4111856B2 (en) 2002-04-12 2008-07-02 昭和電工株式会社 Stabilized ascorbic acid derivatives
US7419817B2 (en) 2002-05-17 2008-09-02 The United States Of America As Represented By The Secretary Department Of Health And Human Services, Nih. Scalable purification of AAV2, AAV4 or AAV5 using ion-exchange chromatography
WO2004002453A1 (en) 2002-06-28 2004-01-08 Protiva Biotherapeutics Ltd. Method and apparatus for producing liposomes
US6620794B1 (en) 2002-07-08 2003-09-16 Colonial Chemical Inc. Guerbet functionalized phospholipids
US7361635B2 (en) 2002-08-29 2008-04-22 Sangamo Biosciences, Inc. Simultaneous modulation of multiple genes
AU2003298574B2 (en) 2002-09-05 2008-04-24 California Institute Of Technology Use of chimeric nucleases to stimulate gene targeting
AU2004208031B2 (en) 2003-01-28 2009-10-08 Cellectis Use of meganucleases for inducing homologous recombination ex vivo and in toto in vertebrate somatic tissues and application thereof.
AU2004257373B2 (en) 2003-07-16 2011-03-24 Arbutus Biopharma Corporation Lipid encapsulated interfering RNA
US8409861B2 (en) 2003-08-08 2013-04-02 Sangamo Biosciences, Inc. Targeted deletion of cellular DNA sequences
US7888121B2 (en) 2003-08-08 2011-02-15 Sangamo Biosciences, Inc. Methods and compositions for targeted cleavage and recombination
US7803397B2 (en) 2003-09-15 2010-09-28 Protiva Biotherapeutics, Inc. Polyethyleneglycol-modified lipid compounds and uses thereof
US7972854B2 (en) 2004-02-05 2011-07-05 Sangamo Biosciences, Inc. Methods and compositions for targeted cleavage and recombination
ES2315859T3 (en) 2004-04-08 2009-04-01 Sangamo Biosciences, Inc. METHODS AND COMPOSITIONS TO TREAT NEUROPATIC AND NEURODEGENERATIVE AFFECTIONS.
EP1737498A2 (en) 2004-04-08 2007-01-03 Sangamo Biosciences Inc. Zinc finger proteins for treatment of neuropathic pain
EP1750673B1 (en) 2004-05-17 2009-12-02 Tekmira Pharmaceuticals Corporation Liposomal formulations comprising dihydrosphingomyelin and methods of use thereof
AU2005252273B2 (en) 2004-06-07 2011-04-28 Arbutus Biopharma Corporation Lipid encapsulated interfering RNA
CA2569645C (en) 2004-06-07 2014-10-28 Protiva Biotherapeutics, Inc. Cationic lipids and methods of use
WO2006033859A2 (en) 2004-09-16 2006-03-30 Sangamo Biosciences, Inc. Compositions and methods for protein production
US8192718B1 (en) 2005-01-04 2012-06-05 Gp Medical, Inc. Pharmaceutical composition of nanoparticles
EP1922300A2 (en) 2005-02-14 2008-05-21 Sirna Therapeutics Inc. Cationic lipids and formulated molecular compositions containing them
US7404969B2 (en) 2005-02-14 2008-07-29 Sirna Therapeutics, Inc. Lipid nanoparticle based compositions and methods for the delivery of biologically active molecules
WO2006121866A2 (en) 2005-05-05 2006-11-16 The Arizona Board Of Regents On Behalf Of The University Of Arizona Sequence enabled reassembly (seer) - a novel method for visualizing specific dna sequences
AU2006259415B2 (en) 2005-06-15 2012-08-30 Massachusetts Institute Of Technology Amine-containing lipids and uses thereof
CA2615532C (en) 2005-07-26 2016-06-28 Sangamo Biosciences, Inc. Targeted integration and expression of exogenous nucleic acid sequences
JP5639338B2 (en) 2005-07-27 2014-12-10 プロチバ バイオセラピューティクス インコーポレイティッド Liposome production system and production method
ES2937245T3 (en) 2005-08-23 2023-03-27 Univ Pennsylvania RNA containing modified nucleosides and methods of using the same
US7443760B2 (en) 2005-09-29 2008-10-28 Hynix Semiconductor Inc. Multi-port memory device with serial input/output interface
DK2662442T3 (en) 2005-10-18 2015-07-06 Prec Biosciences Rationally designed mechanuclease with altered dimer formation affinity
JP4681425B2 (en) 2005-11-15 2011-05-11 花王株式会社 Hair elasticity improving agent
GB2433928B (en) 2006-01-07 2009-10-14 Shane Richard Wootton Apparatus for producing material by a chemical reaction
CA2651499C (en) 2006-05-25 2015-06-30 Sangamo Biosciences, Inc. Methods and compositions for ccr-5 gene inactivation
JP5266210B2 (en) 2006-05-25 2013-08-21 サンガモ バイオサイエンシズ インコーポレイテッド Modified cleavage half-domain
ES2430206T3 (en) 2006-10-03 2013-11-19 Tekmira Pharmaceuticals Corporation Formulation containing lipids
WO2008103276A2 (en) 2007-02-16 2008-08-28 Merck & Co., Inc. Compositions and methods for potentiated activity of biologicaly active molecules
AU2008244473B2 (en) 2007-04-26 2013-06-20 Sangamo Therapeutics, Inc. Targeted integration into the PPP1R12C locus
CA2692906C (en) 2007-06-19 2016-01-19 Board Of Supervisors Of Louisiana State University And Agricultural And Mechanical College Synthesis and use of anti-reverse phosphorothioate analogs of the messenger rna cap
US8563314B2 (en) 2007-09-27 2013-10-22 Sangamo Biosciences, Inc. Methods and compositions for modulating PD1
MX2010003371A (en) 2007-09-28 2010-05-05 Intrexon Corp Therapeutic gene-switch constructs and bioreactors for the expression of biotherapeutic molecules, and uses thereof.
ES2632052T3 (en) 2007-09-28 2017-09-08 Pfizer Inc. Addressing to cancer cells using nanoparticles
US20090247608A1 (en) 2007-12-04 2009-10-01 Alnylam Pharmaceuticals, Inc. Targeting Lipids
WO2009088892A1 (en) 2008-01-02 2009-07-16 Alnylam Pharmaceuticals, Inc. Liver screening method
CA2711236A1 (en) 2008-01-02 2009-07-16 Alnylam Pharmaceuticals, Inc. Screening method for selected amino lipid-containing compositions
US20110117125A1 (en) 2008-01-02 2011-05-19 Tekmira Pharmaceuticals Corporation Compositions and methods for the delivery of nucleic acids
CA2721183C (en) 2008-04-11 2019-07-16 Alnylam Pharmaceuticals, Inc. Site-specific delivery of nucleic acids by combining targeting ligands with endosomolytic components
AU2009238629C1 (en) 2008-04-14 2015-04-30 Sangamo Therapeutics, Inc. Linear donor constructs for targeted integration
NZ588583A (en) 2008-04-15 2012-08-31 Protiva Biotherapeutics Inc Novel lipid formulations for nucleic acid delivery
US20090285881A1 (en) 2008-04-16 2009-11-19 Abbott Laboratories Cationic lipids and uses thereof
US20090263407A1 (en) 2008-04-16 2009-10-22 Abbott Laboratories Cationic Lipids and Uses Thereof
WO2009132131A1 (en) 2008-04-22 2009-10-29 Alnylam Pharmaceuticals, Inc. Amino lipid based improved lipid formulation
CA2893175C (en) 2008-06-10 2016-09-06 Sangamo Biosciences, Inc. Methods and compositions for generation of bax- and bak-deficient cell lines
HUE035770T2 (en) 2008-06-16 2018-05-28 Pfizer Methods for the preparation of targeting agent functionalized diblock copolymers for use in fabrication of therapeutic nanoparticles
CN104997732A (en) 2008-06-16 2015-10-28 佰恩德治疗股份有限公司 Drug loaded polymeric nanoparticle and method of making and using same
WO2010005726A2 (en) 2008-06-16 2010-01-14 Bind Biosciences Inc. Therapeutic polymeric nanoparticles with mtor inhibitors and methods of making and using same
JP2012501966A (en) 2008-06-16 2012-01-26 バインド バイオサイエンシズ インコーポレイテッド Vinca alkaloid-containing therapeutic polymer nanoparticles and methods for making and using the same
WO2010021865A1 (en) 2008-08-18 2010-02-25 Merck Sharp & Dohme Corp. Novel lipid nanoparticles and novel components for delivery of nucleic acids
AU2009283194B2 (en) 2008-08-22 2014-10-16 Sangamo Therapeutics, Inc. Methods and compositions for targeted single-stranded cleavage and targeted integration
US20100087337A1 (en) 2008-09-10 2010-04-08 Bind Biosciences, Inc. High Throughput Fabrication of Nanoparticles
EP3225621A1 (en) 2008-10-09 2017-10-04 Arbutus Biopharma Corporation Improved amino lipids and methods for the delivery of nucleic acids
WO2010048536A2 (en) 2008-10-23 2010-04-29 Alnylam Pharmaceuticals, Inc. Processes for preparing lipids
WO2010062322A2 (en) 2008-10-27 2010-06-03 Massachusetts Institute Of Technology Modulation of the immune response
CA3039251C (en) 2008-11-10 2024-01-09 Arbutus Biopharma Corporation Novel lipids and compositions for the delivery of therapeutics
US8722082B2 (en) 2008-11-10 2014-05-13 Tekmira Pharmaceuticals Corporation Lipids and compositions for the delivery of therapeutics
EP2362728A1 (en) 2008-11-17 2011-09-07 Enzon Pharmaceuticals, Inc. Releasable polymeric lipids for nucleic acids delivery system
CA2745031C (en) 2008-12-04 2018-08-14 Sangamo Biosciences, Inc. Genome editing in rats using zinc-finger nucleases
WO2010080724A1 (en) 2009-01-12 2010-07-15 Merck Sharp & Dohme Corp. Novel lipid nanoparticles and novel components for delivery of nucleic acids
EP2206723A1 (en) 2009-01-12 2010-07-14 Bonas, Ulla Modular DNA-binding domains
AU2010208035B2 (en) 2009-01-29 2016-06-23 Arbutus Biopharma Corporation Improved lipid formulation for the delivery of nucleic acids
CN102421417B (en) 2009-05-05 2016-03-02 阿尔尼拉姆医药品有限公司 Lipid composition
CA2760706C (en) 2009-05-05 2019-08-06 Alnylam Pharmaceuticals, Inc. Methods of delivering oligonucleotides to immune cells
EP2440183B1 (en) 2009-06-10 2018-07-18 Arbutus Biopharma Corporation Improved lipid formulation
US9051567B2 (en) 2009-06-15 2015-06-09 Tekmira Pharmaceuticals Corporation Methods for increasing efficacy of lipid formulated siRNA
US9018187B2 (en) 2009-07-01 2015-04-28 Protiva Biotherapeutics, Inc. Cationic lipids and methods for the delivery of therapeutic agents
WO2011000106A1 (en) 2009-07-01 2011-01-06 Protiva Biotherapeutics, Inc. Improved cationic lipids and methods for the delivery of therapeutic agents
EP2449114B9 (en) 2009-07-01 2017-04-19 Protiva Biotherapeutics Inc. Novel lipid formulations for delivery of therapeutic agents to solid tumors
EP2451475A2 (en) 2009-07-06 2012-05-16 Novartis AG Self replicating rna molecules and uses thereof
WO2011016840A2 (en) 2009-07-28 2011-02-10 Sangamo Biosciences, Inc. Methods and compositions for treating trinucleotide repeat disorders
BR112012002291A2 (en) 2009-07-31 2016-11-29 Ethris Gmbh "polyribonucleotide with a sequence encoding a protein or protein fragment, implant, and process for selecting nucleotide sequences"
WO2011022460A1 (en) 2009-08-20 2011-02-24 Merck Sharp & Dohme Corp. Novel cationic lipids with various head groups for oligonucleotide delivery
US20120253032A1 (en) 2009-10-08 2012-10-04 Merck Sharp & Dohme Corporation Novel cationic lipids with short lipid chains for oligonucleotide delivery
JP5823405B2 (en) 2009-11-04 2015-11-25 ザ ユニバーシティ オブ ブリティッシュ コロンビア Nucleic acid-containing lipid particles and related methods
US8956828B2 (en) 2009-11-10 2015-02-17 Sangamo Biosciences, Inc. Targeted disruption of T cell receptor genes using engineered zinc finger protein nucleases
US20130022649A1 (en) 2009-12-01 2013-01-24 Protiva Biotherapeutics, Inc. Snalp formulations containing antioxidants
US9687550B2 (en) 2009-12-07 2017-06-27 Arbutus Biopharma Corporation Compositions for nucleic acid delivery
PL2510096T5 (en) 2009-12-10 2018-06-29 Regents Of The University Of Minnesota Tal effector-mediated dna modification
EP2512459A4 (en) 2009-12-15 2013-08-07 Therapeutic polymeric nanoparticles comprising epothilone and methods of making and using same
JP5965844B2 (en) 2009-12-15 2016-08-10 バインド セラピューティックス インコーポレイテッド Therapeutic polymer nanoparticle compositions having high glass transition temperature or high molecular weight copolymers
NZ600725A (en) 2009-12-18 2015-08-28 Univ British Colombia 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
EP2526113B1 (en) 2010-01-22 2016-08-10 Sirna Therapeutics, Inc. Post-synthetic chemical modification of rna at the 2'-position of the ribose ring via "click" chemistry
MX340102B (en) 2010-01-28 2016-06-24 The Children's Hospital Of Philadelphia * A scalable manufacturing platform for viral vector purification and viral vectors so purified for use in gene therapy.
WO2011097036A1 (en) 2010-02-08 2011-08-11 Sangamo Biosciences, Inc. Engineered cleavage half-domains
EP2534163B1 (en) 2010-02-09 2015-11-04 Sangamo BioSciences, Inc. Targeted genomic modification with partially single-stranded donor molecules
US9149432B2 (en) 2010-03-19 2015-10-06 Massachusetts Institute Of Technology Lipid vesicle compositions and methods of use
WO2011127255A1 (en) 2010-04-08 2011-10-13 Merck Sharp & Dohme Corp. Preparation of lipid nanoparticles
US20110262491A1 (en) 2010-04-12 2011-10-27 Selecta Biosciences, Inc. Emulsions and methods of making nanocarriers
WO2011139335A1 (en) 2010-04-26 2011-11-10 Sangamo Biosciences, Inc. Genome editing of a rosa locus using zinc-finger nucleases
JP5902616B2 (en) 2010-04-28 2016-04-13 協和発酵キリン株式会社 Cationic lipid
JP5902617B2 (en) 2010-04-28 2016-04-13 協和発酵キリン株式会社 Cationic lipid
EP3636766A1 (en) 2010-05-03 2020-04-15 Sangamo Therapeutics, Inc. Compositions for linking zinc finger modules
US9254327B2 (en) 2010-05-10 2016-02-09 Alnylam Pharmaceuticals, Inc. Methods and compositions for delivery of active agents
WO2011141703A1 (en) 2010-05-12 2011-11-17 Protiva Biotherapeutics Inc. Compositions and methods for silencing apolipoprotein b
CA2799091A1 (en) 2010-05-12 2011-11-17 Protiva Biotherapeutics, Inc. Cationic lipids and methods of use thereof
CN103025344B (en) 2010-05-17 2016-06-29 桑格摩生物科学股份有限公司 Novel DNA-associated proteins and application thereof
EP2575895A2 (en) 2010-05-24 2013-04-10 Merck Sharp & Dohme Corp. Novel amino alcohol cationic lipids for oligonucleotide delivery
JP5893611B2 (en) 2010-06-03 2016-03-23 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 Biodegradable lipids for delivery of active agents
DK2575767T3 (en) 2010-06-04 2017-03-13 Sirna Therapeutics Inc HOWEVER UNKNOWN LOW MOLECULAR CATIONIC LIPIDS TO PROCESS OIGONUCLEOTIDES
WO2012000104A1 (en) 2010-06-30 2012-01-05 Protiva Biotherapeutics, Inc. Non-liposomal systems for nucleic acid delivery
ES2557382T3 (en) 2010-07-06 2016-01-25 Glaxosmithkline Biologicals Sa Liposomes with lipids that have an advantageous pKa value for RNA delivery
MX343410B (en) 2010-07-06 2016-11-04 Novartis Ag * Cationic oil-in-water emulsions.
WO2012012667A2 (en) 2010-07-21 2012-01-26 Sangamo Biosciences, Inc. Methods and compositions for modification of a hla locus
WO2012016184A2 (en) 2010-07-30 2012-02-02 Alnylam Pharmaceuticals, Inc. Methods and compositions for delivery of active agents
WO2012019630A1 (en) 2010-08-13 2012-02-16 Curevac Gmbh Nucleic acid comprising or coding for a histone stem-loop and a poly(a) sequence or a polyadenylation signal for increasing the expression of an encoded protein
FI4043040T3 (en) 2010-08-31 2023-04-04 Glaxosmithkline Biologicals Sa Small liposomes for delivery of immunogen-encoding rna
HUE058361T2 (en) 2010-08-31 2022-07-28 Glaxosmithkline Biologicals Sa Pegylated liposomes for delivery of immunogen-encoding rna
US20130189351A1 (en) 2010-08-31 2013-07-25 Novartis Ag Lipids suitable for liposomal delivery of protein coding rna
US8466122B2 (en) 2010-09-17 2013-06-18 Protiva Biotherapeutics, Inc. Trialkyl cationic lipids and methods of use thereof
AU2011305617A1 (en) 2010-09-20 2013-02-21 Sirna Therapeutics, Inc. Novel low molecular weight cationic lipids for oligonucleotide delivery
AU2011307277A1 (en) 2010-09-30 2013-03-07 Merck Sharp & Dohme Corp. Low molecular weight cationic lipids for oligonucleotide delivery
WO2012054743A2 (en) 2010-10-20 2012-04-26 Yongquan Xue Taste modifying compositions
CN103153347A (en) 2010-10-21 2013-06-12 默沙东公司 Novel low molecular weight cationic lipids for oligonucleotide delivery
EP2629760A4 (en) 2010-10-22 2014-04-02 Bind Therapeutics Inc Therapeutic nanoparticles with high molecular weight copolymers
WO2012061259A2 (en) 2010-11-05 2012-05-10 Merck Sharp & Dohme Corp. Novel low molecular weight cyclic amine containing cationic lipids for oligonucleotide delivery
EP2640700B1 (en) 2010-11-15 2018-10-31 Life Technologies Corporation Amine-containing transfection reagents and methods for making and using same
EP2691101A2 (en) 2011-03-31 2014-02-05 Moderna Therapeutics, Inc. Delivery and formulation of engineered nucleic acids
US8691750B2 (en) 2011-05-17 2014-04-08 Axolabs Gmbh Lipids and compositions for intracellular delivery of biologically active compounds
US20140243391A1 (en) 2011-07-22 2014-08-28 Centre National De La Recherche Scientifique Phospholipid-detergent conjugates and uses thereof
WO2013016058A1 (en) 2011-07-22 2013-01-31 Merck Sharp & Dohme Corp. Novel bis-nitrogen containing cationic lipids for oligonucleotide delivery
EP2758529B1 (en) 2011-09-21 2019-03-20 Sangamo Therapeutics, Inc. Methods and compositions for regulation of transgene expression
EP3597644B1 (en) 2011-10-18 2021-09-29 Dicerna Pharmaceuticals, Inc. Amine cationic lipids and uses thereof
CA3099582A1 (en) 2011-10-27 2013-05-02 Sangamo Biosciences, Inc. Methods and compositions for modification of the hprt locus
JP2013095755A (en) 2011-11-02 2013-05-20 Kyowa Hakko Kirin Co Ltd Cationic lipid
JP6144691B2 (en) 2011-11-16 2017-06-07 サンガモ セラピューティクス， インコーポレイテッド Modified DNA binding proteins and uses thereof
CA3170051A1 (en) 2011-12-07 2013-06-13 Alnylam Pharmaceuticals, Inc. Branched alkyl and cycloalkyl terminated biodegradable lipids for the delivery of active agents
US20140308304A1 (en) 2011-12-07 2014-10-16 Alnylam Pharmaceuticals, Inc. Lipids for the delivery of active agents
WO2013086354A1 (en) 2011-12-07 2013-06-13 Alnylam Pharmaceuticals, Inc. Biodegradable lipids for the delivery of active agents
CA3018046A1 (en) 2011-12-16 2013-06-20 Moderna Therapeutics, Inc. Modified nucleoside, nucleotide, and nucleic acid compositions
WO2013143555A1 (en) 2012-03-26 2013-10-03 Biontech Ag Rna formulation for immunotherapy
EP2830594B1 (en) 2012-03-27 2018-05-09 Sirna Therapeutics, Inc. DIETHER BASED BIODEGRADABLE CATIONIC LIPIDS FOR siRNA DELIVERY
WO2014008334A1 (en) 2012-07-06 2014-01-09 Alnylam Pharmaceuticals, Inc. Stable non-aggregating nucleic acid lipid particle formulations
US9877988B2 (en) 2012-07-11 2018-01-30 Sangamo Therapeutics, Inc. Method of treating lysosomal storage diseases using nucleases and a transgene
EP2882706A1 (en) 2012-08-13 2015-06-17 Massachusetts Institute of Technology Amine-containing lipidoids and uses thereof
EP3763810A3 (en) 2012-10-10 2021-07-14 Sangamo Therapeutics, Inc. T cell modifying compounds and uses thereof
WO2014089212A1 (en) 2012-12-05 2014-06-12 Sangamo Biosciences, Inc. Methods and compositions for regulation of metabolic disorders
WO2014089239A1 (en) 2012-12-07 2014-06-12 Alnylam Pharmaceuticals, Inc. Improved nucleic acid lipid particle formulations
US8697359B1 (en) 2012-12-12 2014-04-15 The Broad Institute, Inc. CRISPR-Cas systems and methods for altering expression of gene products
EP2968149A4 (en) 2013-03-14 2017-02-01 Dicerna Pharmaceuticals, Inc. Process for formulating an anionic agent
US20160030527A1 (en) 2013-03-14 2016-02-04 The Trustees Of The University Of Pennsylvania Compositions and Methods for Treatment of Stroke
WO2014160243A1 (en) 2013-03-14 2014-10-02 The Trustees Of The University Of Pennsylvania Purification and purity assessment of rna molecules synthesized with modified nucleosides
US9693958B2 (en) 2013-03-15 2017-07-04 Cureport, Inc. Methods and devices for preparation of lipid nanoparticles
EP2997146A4 (en) 2013-05-15 2017-04-26 Sangamo BioSciences, Inc. Methods and compositions for treatment of a genetic condition
WO2015074085A1 (en) 2013-11-18 2015-05-21 Arcturus Therapeutics, Inc. Ionizable cationic lipid for rna delivery
AU2014364051B2 (en) 2013-12-09 2018-12-20 Sangamo Therapeutics, Inc. Methods and compositions for treating hemophilia
US20170042870A1 (en) 2014-02-17 2017-02-16 The Brigham And Women's Hospital, Inc. Targeted nanoparticle compositions and methods of their use to treat obesity
WO2015130584A2 (en) 2014-02-25 2015-09-03 Merck Sharp & Dohme Corp. Lipid nanoparticle vaccine adjuvants and antigen delivery systems
US10406059B2 (en) 2014-04-21 2019-09-10 The Trustees Of Columbia University In The City Of New York Human movement research, therapeutic, and diagnostic devices, methods, and systems
HRP20220070T1 (en) 2014-04-23 2022-04-01 Modernatx, Inc. Nucleic acid vaccines
CN106413760B (en) 2014-05-08 2020-01-14 桑格摩生物科学股份有限公司 Methods and compositions for treating huntington's disease
WO2015177752A1 (en) 2014-05-22 2015-11-26 Flowserve S.R.L. Guide element for a valve actuator and actuator provided with said guide element
CA3179824A1 (en) 2014-06-25 2015-12-30 Acuitas Therapeutics Inc. Lipids and lipid nanoparticle formulations for delivery of nucleic acids
EP3169309B1 (en) 2014-07-16 2023-05-10 Novartis AG Method of encapsulating a nucleic acid in a lipid nanoparticle host
JP6339884B2 (en) 2014-07-17 2018-06-06 富士フイルム株式会社 Imidazole compound and liposome containing the same
US9816074B2 (en) 2014-07-25 2017-11-14 Sangamo Therapeutics, Inc. Methods and compositions for modulating nuclease-mediated genome engineering in hematopoietic stem cells
CN104876831B (en) 2015-04-03 2017-05-17 苏州圣诺生物医药技术有限公司 Liposome-modified spermine derivative and liposome prepared by derivative
AU2016253972B2 (en) 2015-04-27 2020-01-02 Acuitas Therapeutics Inc. Nucleoside-modified RNA for inducing an adaptive immune response
US10179918B2 (en) 2015-05-07 2019-01-15 Sangamo Therapeutics, Inc. Methods and compositions for increasing transgene activity
MX2017014446A (en) 2015-05-12 2018-06-13 Sangamo Therapeutics Inc Nuclease-mediated regulation of gene expression.
JP7072386B2 (en) 2015-06-29 2022-05-20 アクイタス セラピューティクス インコーポレイテッド Lipid and Lipid Nanoparticle Formulations for Nucleic Acid Delivery
WO2017048770A1 (en) 2015-09-15 2017-03-23 Regulus Therapeutics, Inc. Systems, compositions, and methods for formulating nucleic acid compositions
WO2017049245A2 (en) 2015-09-17 2017-03-23 Modernatx, Inc. Compounds and compositions for intracellular delivery of therapeutic agents
PL3368507T3 (en) 2015-10-28 2023-03-27 Acuitas Therapeutics Inc. Novel lipids and lipid nanoparticle formulations for delivery of nucleic acids
MX2018005272A (en) 2015-10-28 2018-09-18 Sangamo Therapeutics Inc Liver-specific constructs, factor viii expression cassettes and methods of use thereof.
KR20180088911A (en) 2015-12-18 2018-08-07 상가모 테라퓨틱스, 인코포레이티드 Targeted Collapse of MHC Cell Receptors
CN108778297B (en) 2015-12-18 2024-02-09 桑格摩生物治疗股份有限公司 Targeted disruption of T cell receptors
RS63051B1 (en) 2015-12-22 2022-04-29 Modernatx Inc Compounds and compositions for intracellular delivery of agents
WO2017117528A1 (en) * 2015-12-30 2017-07-06 Acuitas Therapeutics, Inc. Lipids and lipid nanoparticle formulations for delivery of nucleic acids
US20170266292A1 (en) 2016-03-21 2017-09-21 The Research Foundation For The State University Of New York Lipidic compound-telodendrimer hybrid nanoparticles and methods of making and uses thereof
JP7245651B2 (en) 2016-03-30 2023-03-24 インテリア セラピューティクス，インコーポレイテッド Lipid Nanoparticle Formulations for CRISPR/CAS Components
CN109152735B (en) 2016-05-09 2022-03-11 阿斯利康(瑞典)有限公司 Lipid nanoparticles comprising lipophilic anti-inflammatory agents and methods of use thereof
US20190298657A1 (en) 2016-05-18 2019-10-03 Modernatx, Inc. Polynucleotides Encoding Acyl-CoA Dehydrogenase, Very Long-Chain for the Treatment of Very Long-Chain Acyl-CoA Dehydrogenase Deficiency
US10975393B2 (en) 2016-08-24 2021-04-13 Sangamo Therapeutics, Inc. Engineered target specific nucleases
US10960085B2 (en) 2016-09-07 2021-03-30 Sangamo Therapeutics, Inc. Modulation of liver genes
CN110352071A (en) * 2016-10-26 2019-10-18 库瑞瓦格股份公司 Lipidic nanoparticles mRNA vaccine
EP3532097A1 (en) 2016-10-27 2019-09-04 The Trustees Of The University Of Pennsylvania Nucleoside-modified rna for inducing an adaptive immune response
US20180185516A1 (en) 2016-12-09 2018-07-05 Sangamo Therapeutics, Inc. Delivery of target specific nucleases
WO2018191719A1 (en) 2017-04-13 2018-10-18 Acuitas Therapeutics, Inc. Lipid delivery of therapeutic agents to adipose tissue
WO2018191657A1 (en) 2017-04-13 2018-10-18 Acuitas Therapeutics, Inc. Lipids for delivery of active agents
CA3061612A1 (en) 2017-04-28 2018-11-01 Acuitas Therapeutics, Inc. Novel carbonyl lipids and lipid nanoparticle formulations for delivery of nucleic acids
US11639329B2 (en) 2017-08-16 2023-05-02 Acuitas Therapeutics, Inc. Lipids for use in lipid nanoparticle formulations
WO2020061426A2 (en) 2018-09-21 2020-03-26 Acuitas Therapeutics, Inc. Systems and methods for manufacturing lipid nanoparticles and liposomes
EP3867225A1 (en) 2018-10-18 2021-08-25 Acuitas Therapeutics, Inc. Lipids for lipid nanoparticle delivery of active agents

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US20230123534A1 (en) 2023-04-20
BR112021013654A2 (en) 2021-09-14
CA3125485A1 (en) 2020-07-16
AU2020205717A1 (en) 2021-08-12
US20200283372A1 (en) 2020-09-10
CN113474328A (en) 2021-10-01
JP2022517783A (en) 2022-03-10
WO2020146805A1 (en) 2020-07-16
MX2021008358A (en) 2021-09-30
KR20210138569A (en) 2021-11-19

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