CA2767127A1 - Novel lipid formulations for delivery of therapeutic agents to solid tumors - Google Patents

Novel lipid formulations for delivery of therapeutic agents to solid tumors Download PDF

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Publication number
CA2767127A1
CA2767127A1 CA2767127A CA2767127A CA2767127A1 CA 2767127 A1 CA2767127 A1 CA 2767127A1 CA 2767127 A CA2767127 A CA 2767127A CA 2767127 A CA2767127 A CA 2767127A CA 2767127 A1 CA2767127 A1 CA 2767127A1
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CA
Canada
Prior art keywords
nucleic acid
lipid
nucleotides
sirna
particle
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.)
Pending
Application number
CA2767127A
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English (en)
French (fr)
Inventor
Ed Yaworski
Stephen Reid
James Heyes
Ian Maclachlan
Adam Judge
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
Protiva Biotherapeutics 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
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Application filed by Protiva Biotherapeutics Inc filed Critical Protiva Biotherapeutics Inc
Publication of CA2767127A1 publication Critical patent/CA2767127A1/en
Pending legal-status Critical Current

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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/44—Oils, fats or waxes according to two or more groups of A61K47/02-A61K47/42; Natural or modified natural oils, fats or waxes, e.g. castor oil, polyethoxylated castor oil, montan wax, lignite, shellac, rosin, beeswax or lanolin
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/54—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound
    • A61K47/543—Lipids, e.g. triglycerides; Polyamines, e.g. spermine or spermidine
    • 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
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/10—Dispersions; Emulsions
    • A61K9/127—Synthetic bilayered vehicles, e.g. liposomes or liposomes with cholesterol as the only non-phosphatidyl surfactant
    • A61K9/1271—Non-conventional liposomes, e.g. PEGylated liposomes or liposomes coated or grafted with polymers
    • A61K9/1272—Non-conventional liposomes, e.g. PEGylated liposomes or liposomes coated or grafted with polymers comprising non-phosphatidyl surfactants as bilayer-forming substances, e.g. cationic lipids or non-phosphatidyl liposomes coated or grafted with polymers
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • 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
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • 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
    • C12N15/111—General methods applicable to biologically active non-coding nucleic acids
    • C—CHEMISTRY; METALLURGY
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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C12N15/1137—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against enzymes
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/88—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation using microencapsulation, e.g. using amphiphile liposome vesicle
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/344—Position-specific modifications, e.g. on every purine, at the 3'-end
    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3521—Methyl
    • 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
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • 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
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/32—Special delivery means, e.g. tissue-specific
    • Y—GENERAL TAGGING OF NEW TECHNOLOGICAL DEVELOPMENTS; GENERAL TAGGING OF CROSS-SECTIONAL TECHNOLOGIES SPANNING OVER SEVERAL SECTIONS OF THE IPC; TECHNICAL SUBJECTS COVERED BY FORMER USPC CROSS-REFERENCE ART COLLECTIONS [XRACs] AND DIGESTS
    • Y02—TECHNOLOGIES OR APPLICATIONS FOR MITIGATION OR ADAPTATION AGAINST CLIMATE CHANGE
    • Y02A—TECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE
    • Y02A50/00—TECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE in human health protection, e.g. against extreme weather
    • Y02A50/30—Against vector-borne diseases, e.g. mosquito-borne, fly-borne, tick-borne or waterborne diseases whose impact is exacerbated by climate change

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Biophysics (AREA)
  • Epidemiology (AREA)
  • Biochemistry (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Oil, Petroleum & Natural Gas (AREA)
  • Virology (AREA)
  • Dispersion Chemistry (AREA)
  • Dermatology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
CA2767127A 2009-07-01 2010-06-30 Novel lipid formulations for delivery of therapeutic agents to solid tumors Pending CA2767127A1 (en)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US22246909P 2009-07-01 2009-07-01
US61/222,469 2009-07-01
US29513410P 2010-01-14 2010-01-14
US61/295,134 2010-01-14
PCT/CA2010/001030 WO2011000107A1 (en) 2009-07-01 2010-06-30 Novel lipid formulations for delivery of therapeutic agents to solid tumors

Publications (1)

Publication Number Publication Date
CA2767127A1 true CA2767127A1 (en) 2011-01-06

Family

ID=43410415

Family Applications (1)

Application Number Title Priority Date Filing Date
CA2767127A Pending CA2767127A1 (en) 2009-07-01 2010-06-30 Novel lipid formulations for delivery of therapeutic agents to solid tumors

Country Status (7)

Country Link
US (9) US8283333B2 (cg-RX-API-DMAC7.html)
EP (1) EP2449114B9 (cg-RX-API-DMAC7.html)
JP (1) JP5766188B2 (cg-RX-API-DMAC7.html)
CA (1) CA2767127A1 (cg-RX-API-DMAC7.html)
ES (1) ES2613498T3 (cg-RX-API-DMAC7.html)
IL (3) IL292615B2 (cg-RX-API-DMAC7.html)
WO (1) WO2011000107A1 (cg-RX-API-DMAC7.html)

Families Citing this family (416)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
ES2354607T3 (es) 2002-06-28 2011-03-16 Protiva Biotherapeutics Inc. Procedimiento y aparato para producir liposomas.
JP4951338B2 (ja) * 2003-07-16 2012-06-13 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 脂質に封入された干渉ｒｎａ
CA2628300C (en) 2005-11-02 2018-04-17 Protiva Biotherapeutics, Inc. Modified sirna molecules and uses thereof
EP2238251B1 (en) * 2007-12-27 2015-02-11 Protiva Biotherapeutics Inc. Silencing of polo-like kinase expression using interfering rna
CA2721333C (en) * 2008-04-15 2020-12-01 Protiva Biotherapeutics, Inc. Novel lipid formulations for nucleic acid delivery
JP5777519B2 (ja) 2008-10-09 2015-09-09 テクミラ ファーマシューティカルズ コーポレイション 改良されたアミノ脂質および核酸の送達方法
PL2355851T3 (pl) 2008-11-10 2018-08-31 Arbutus Biopharma Corporation Nowe lipidy i kompozycje do dostarczania środków terapeutycznych
US8283333B2 (en) 2009-07-01 2012-10-09 Protiva Biotherapeutics, Inc. Lipid formulations for nucleic acid delivery
US8236943B2 (en) * 2009-07-01 2012-08-07 Protiva Biotherapeutics, Inc. Compositions and methods for silencing apolipoprotein B
EP2512449B1 (en) * 2009-12-18 2019-08-07 The University of British Columbia Methods and compositions for delivery of nucleic acids
JP5902616B2 (ja) 2010-04-28 2016-04-13 協和発酵キリン株式会社 カチオン性脂質
JP5902617B2 (ja) * 2010-04-28 2016-04-13 協和発酵キリン株式会社 カチオン性脂質
WO2011141704A1 (en) 2010-05-12 2011-11-17 Protiva Biotherapeutics, Inc Novel cyclic cationic lipids and methods of use
EP2569276B1 (en) 2010-05-12 2021-02-24 Arbutus Biopharma Corporation Novel cationic lipids and methods of use thereof
WO2012000104A1 (en) * 2010-06-30 2012-01-05 Protiva Biotherapeutics, Inc. Non-liposomal systems for nucleic acid delivery
SI3243526T1 (sl) 2010-07-06 2020-02-28 Glaxosmithkline Biologicals S.A. Dostava RNA za sprožitev večih imunskih poti
ES2586580T3 (es) 2010-07-06 2016-10-17 Glaxosmithkline Biologicals Sa Inmunización de mamíferos grandes con dosis bajas de ARN
SI2590676T1 (sl) 2010-07-06 2016-11-30 Glaxosmithkline Biologicals, S.A. Virionu podobni delci za dajanje za samorepliciranje RNA molekul
PL2590626T3 (pl) 2010-07-06 2016-04-29 Glaxosmithkline Biologicals Sa Liposomy z lipidami o korzystnej wartości pka do dostarczania rna
EP2600901B1 (en) 2010-08-06 2019-03-27 ModernaTX, Inc. A pharmaceutical formulation comprising engineered nucleic acids and medical use thereof
SI3970742T1 (sl) 2010-08-31 2022-08-31 Glaxosmithkline Biologicals S.A. Pegilirani liposomi za dostavo RNA, ki kodira imunogen
MX2013003681A (es) 2010-10-01 2013-11-20 Moderna Therapeutics Inc Ácidos nucleicos manipulados y métodos de uso de los mismos.
BR112013008700B8 (pt) 2010-10-11 2022-10-04 Novartis Ag Molécula de rna auto-replicante, partícula de replicon de alfavírus, composição, molécula de dna recombinante, uso da molécula de rna auto-replicante
WO2012135805A2 (en) 2011-03-31 2012-10-04 modeRNA Therapeutics Delivery and formulation of engineered nucleic acids
WO2012162210A1 (en) * 2011-05-26 2012-11-29 Merck Sharp & Dohme Corp. Ring constrained cationic lipids for oligonucleotide delivery
US11896636B2 (en) 2011-07-06 2024-02-13 Glaxosmithkline Biologicals Sa Immunogenic combination compositions and uses thereof
US9464124B2 (en) 2011-09-12 2016-10-11 Moderna Therapeutics, Inc. Engineered nucleic acids and methods of use thereof
US9428535B2 (en) 2011-10-03 2016-08-30 Moderna Therapeutics, Inc. Modified nucleosides, nucleotides, and nucleic acids, and uses thereof
DE21212055T1 (de) 2011-12-07 2022-08-04 Alnylam Pharmaceuticals, Inc. Biologisch abbaubare lipide zur freisetzung von wirkstoffen
SMT202200355T1 (it) 2011-12-16 2022-11-18 Modernatx Inc Composizioni di mrna modificato
US9035039B2 (en) * 2011-12-22 2015-05-19 Protiva Biotherapeutics, Inc. Compositions and methods for silencing SMAD4
BR112014015333A2 (pt) * 2011-12-22 2017-06-13 Nuvo Res Gmbh composições lipossomais de clorito ou clorato
HUE041494T2 (hu) 2012-02-24 2019-05-28 Arbutus Biopharma Corp Trialkil-kationos lipidek és alkalmazási módszereik
ES2868174T3 (es) 2012-03-29 2021-10-21 Translate Bio Ma Inc Lípidos catiónicos ionizables
US10501512B2 (en) 2012-04-02 2019-12-10 Modernatx, Inc. Modified polynucleotides
US9283287B2 (en) 2012-04-02 2016-03-15 Moderna Therapeutics, Inc. Modified polynucleotides for the production of nuclear proteins
CN108949772A (zh) * 2012-04-02 2018-12-07 现代泰克斯公司 用于产生与人类疾病相关的生物制剂和蛋白质的修饰多核苷酸
EP2834260A4 (en) 2012-04-02 2016-08-10 Moderna Therapeutics Inc MODIFIED POLYNUCLEOTIDES FOR THE PREPARATION OF MEMBRANE PROTEINS
US10501513B2 (en) 2012-04-02 2019-12-10 Modernatx, Inc. Modified polynucleotides for the production of oncology-related proteins and peptides
US9572897B2 (en) 2012-04-02 2017-02-21 Modernatx, Inc. Modified polynucleotides for the production of cytoplasmic and cytoskeletal proteins
US10653786B2 (en) 2012-04-25 2020-05-19 Trustees Of Tufts College Silk microspheres and methods for surface lubrication
EP2922554B1 (en) 2012-11-26 2022-02-23 ModernaTX, Inc. Terminally modified rna
SG10201707569YA (en) 2012-12-12 2017-10-30 Broad Inst Inc Delivery, Engineering and Optimization of Systems, Methods and Compositions for Sequence Manipulation and Therapeutic Applications
US20160002628A1 (en) * 2013-03-11 2016-01-07 Georgia Tech Research Corporation Methods and compositions for managing vascular conditions
EP2971010B1 (en) 2013-03-14 2020-06-10 ModernaTX, Inc. Formulation and delivery of modified nucleoside, nucleotide, and nucleic acid compositions
US8980864B2 (en) 2013-03-15 2015-03-17 Moderna Therapeutics, Inc. Compositions and methods of altering cholesterol levels
CA2915795C (en) 2013-06-17 2021-07-13 The Broad Institute, Inc. Delivery, use and therapeutic applications of the crispr-cas systems and compositions for targeting disorders and diseases using viral components
EP3825406A1 (en) 2013-06-17 2021-05-26 The Broad Institute Inc. Delivery and use of the crispr-cas systems, vectors and compositions for hepatic targeting and therapy
SG11201510327TA (en) 2013-06-17 2016-01-28 Broad Inst Inc Delivery, engineering and optimization of systems, methods and compositions for targeting and modeling diseases and disorders of post mitotic cells
CN105555757A (zh) 2013-07-23 2016-05-04 普洛体维生物治疗公司 用于递送信使rna的组合物和方法
EP3041938A1 (en) 2013-09-03 2016-07-13 Moderna Therapeutics, Inc. Circular polynucleotides
CA2923029A1 (en) 2013-09-03 2015-03-12 Moderna Therapeutics, Inc. Chimeric polynucleotides
EP3052106A4 (en) 2013-09-30 2017-07-19 ModernaTX, Inc. Polynucleotides encoding immune modulating polypeptides
JP2016538829A (ja) 2013-10-03 2016-12-15 モデルナ セラピューティクス インコーポレイテッドＭｏｄｅｒｎａ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ． 低密度リポタンパク質受容体をコードするポリヌクレオチド
EP3071696B1 (en) 2013-11-22 2019-08-07 Mina Therapeutics Limited C/ebp alpha short activating rna compositions and methods of use
AU2014362248A1 (en) 2013-12-12 2016-06-16 Massachusetts Institute Of Technology Compositions and methods of use of CRISPR-Cas systems in nucleotide repeat disorders
CN106536729A (zh) 2013-12-12 2017-03-22 布罗德研究所有限公司 使用粒子递送组分靶向障碍和疾病的crispr‑cas系统和组合物的递送、用途和治疗应用
AU2014361781B2 (en) 2013-12-12 2021-04-01 Massachusetts Institute Of Technology Delivery, use and therapeutic applications of the CRISPR -Cas systems and compositions for genome editing
WO2015089465A1 (en) 2013-12-12 2015-06-18 The Broad Institute Inc. Delivery, use and therapeutic applications of the crispr-cas systems and compositions for hbv and viral diseases and disorders
EP4219699A1 (en) 2013-12-12 2023-08-02 The Broad Institute, Inc. Engineering of systems, methods and optimized guide compositions with new architectures for sequence manipulation
EP3080271B1 (en) 2013-12-12 2020-02-12 The Broad Institute, Inc. Systems, methods and compositions for sequence manipulation with optimized functional crispr-cas systems
CN106536056B (zh) 2014-06-13 2021-07-16 儿童医学中心公司 分离线粒体的产品和方法
IL298516B2 (en) 2014-06-25 2025-03-01 Acuitas Therapeutics Inc Novel lipids and lipid nanoparticle compositions for nucleic acid delivery
JP2017520549A (ja) * 2014-06-26 2017-07-27 ラモット アット テル アビブ ユニバーシティ， リミテッド 核酸の送達のためのリポソーム製剤
US20170204152A1 (en) 2014-07-16 2017-07-20 Moderna Therapeutics, Inc. Chimeric polynucleotides
EP3171895A1 (en) 2014-07-23 2017-05-31 Modernatx, Inc. Modified polynucleotides for the production of intrabodies
WO2016028682A1 (en) 2014-08-17 2016-02-25 The Broad Institute Inc. Genome editing using cas9 nickases
WO2016049163A2 (en) 2014-09-24 2016-03-31 The Broad Institute Inc. Use and production of chd8+/- transgenic animals with behavioral phenotypes characteristic of autism spectrum disorder
WO2016049258A2 (en) 2014-09-25 2016-03-31 The Broad Institute Inc. Functional screening with optimized functional crispr-cas systems
BR112017006679A2 (pt) 2014-10-02 2017-12-26 Protiva Biotherapeutics Inc moléculas, composição, partícula, composição farmacêutica, métodos para silenciar a expressão de um gene, usos de uma partícula, métodos para melhorar um ou mais sintomas, métodos para tratar uma infecção, usos de uma composição, método para inibir a replicação do vírus da hepatite d
EP3230452B1 (en) 2014-12-12 2025-06-11 The Broad Institute, Inc. Dead guides for crispr transcription factors
WO2016094880A1 (en) 2014-12-12 2016-06-16 The Broad Institute Inc. Delivery, use and therapeutic applications of crispr systems and compositions for genome editing as to hematopoietic stem cells (hscs)
EP3230451B1 (en) 2014-12-12 2021-04-07 The Broad Institute, Inc. Protected guide rnas (pgrnas)
WO2016094874A1 (en) 2014-12-12 2016-06-16 The Broad Institute Inc. Escorted and functionalized guides for crispr-cas systems
EP3234192B1 (en) 2014-12-19 2021-07-14 The Broad Institute, Inc. Unbiased identification of double-strand breaks and genomic rearrangement by genome-wide insert capture sequencing
WO2016106236A1 (en) 2014-12-23 2016-06-30 The Broad Institute Inc. Rna-targeting system
WO2016106244A1 (en) 2014-12-24 2016-06-30 The Broad Institute Inc. Crispr having or associated with destabilization domains
US20180245074A1 (en) 2015-06-04 2018-08-30 Protiva Biotherapeutics, Inc. Treating hepatitis b virus infection using crispr
WO2016205745A2 (en) 2015-06-18 2016-12-22 The Broad Institute Inc. Cell sorting
US9790490B2 (en) 2015-06-18 2017-10-17 The Broad Institute Inc. CRISPR enzymes and systems
KR102840885B1 (ko) 2015-06-18 2025-07-30 더 브로드 인스티튜트, 인코퍼레이티드 표적외 효과를 감소시키는 crispr 효소 돌연변이
AU2016279062A1 (en) 2015-06-18 2019-03-28 Omar O. Abudayyeh Novel CRISPR enzymes and systems
FI3430134T3 (fi) 2015-06-18 2023-01-13 Uusia CRISPR-entsyymejä ja järjestelmiä
CA2990202A1 (en) 2015-06-29 2017-01-05 Acuitas Therapeutics Inc. Lipids and lipid nanoparticle formulations for delivery of nucleic acids
WO2017019891A2 (en) * 2015-07-29 2017-02-02 Protiva Biotherapeutics, Inc. Compositions and methods for silencing hepatitis b virus gene expression
US11214800B2 (en) 2015-08-18 2022-01-04 The Broad Institute, Inc. Methods and compositions for altering function and structure of chromatin loops and/or domains
ES2910425T3 (es) 2015-09-17 2022-05-12 Modernatx Inc Compuestos y composiciones para la administración intracelular de agentes terapéuticos
WO2017069958A2 (en) 2015-10-09 2017-04-27 The Brigham And Women's Hospital, Inc. Modulation of novel immune checkpoint targets
JP6921833B2 (ja) 2015-10-22 2021-08-18 モデルナティーエックス， インコーポレイテッド ヒトサイトメガロウイルスワクチン
CN116814590A (zh) 2015-10-22 2023-09-29 布罗德研究所有限公司 Vi-b型crispr酶和系统
EP4349405A3 (en) 2015-10-22 2024-06-19 ModernaTX, Inc. Respiratory virus vaccines
WO2017074788A1 (en) 2015-10-27 2017-05-04 The Broad Institute Inc. Compositions and methods for targeting cancer-specific sequence variations
WO2018081480A1 (en) 2016-10-26 2018-05-03 Acuitas Therapeutics, Inc. Lipid nanoparticle formulations
WO2017075451A1 (en) 2015-10-28 2017-05-04 The Broad Institute Inc. Compositions and methods for evaluating and modulating immune responses by detecting and targeting pou2af1
SMT202300052T1 (it) 2015-10-28 2023-05-12 Acuitas Therapeutics Inc Nuovi lipidi e for­mulazioni di nanoparticelle lipidiche per il rilascio di acidi nucleici
WO2017075465A1 (en) 2015-10-28 2017-05-04 The Broad Institute Inc. Compositions and methods for evaluating and modulating immune responses by detecting and targeting gata3
WO2017075478A2 (en) 2015-10-28 2017-05-04 The Broad Institute Inc. Compositions and methods for evaluating and modulating immune responses by use of immune cell gene signatures
WO2017095940A1 (en) 2015-11-30 2017-06-08 Flagship Pioneering, Inc. Methods and compositions relating to chondrisomes from cultured cells
WO2017099823A1 (en) 2015-12-10 2017-06-15 Modernatx, Inc. Compositions and methods for delivery of therapeutic agents
WO2017106657A1 (en) 2015-12-18 2017-06-22 The Broad Institute Inc. Novel crispr enzymes and systems
EP4036079B1 (en) 2015-12-22 2025-11-19 ModernaTX, Inc. Compounds and compositions for intracellular delivery of agents
LT3394093T (lt) 2015-12-23 2022-04-25 Modernatx, Inc. Ox40 ligandus koduojančių polinukleotidų naudojimo būdai
WO2017120612A1 (en) 2016-01-10 2017-07-13 Modernatx, Inc. Therapeutic mrnas encoding anti ctla-4 antibodies
FI4272834T3 (fi) 2016-01-15 2025-12-05 Childrens Medical Ct Corp Mitokondrioiden ja yhdistettyjen mitokondriaalisten aineiden terapeuttinen käyttö
WO2017184768A1 (en) 2016-04-19 2017-10-26 The Broad Institute Inc. Novel crispr enzymes and systems
CA3026110A1 (en) 2016-04-19 2017-11-02 The Broad Institute, Inc. Novel crispr enzymes and systems
US11286478B2 (en) 2016-04-19 2022-03-29 The Broad Institute, Inc. Cpf1 complexes with reduced indel activity
WO2017219027A1 (en) 2016-06-17 2017-12-21 The Broad Institute Inc. Type vi crispr orthologs and systems
US20210222164A1 (en) 2016-06-29 2021-07-22 The Broad Institute, Inc. Crispr-cas systems having destabilization domain
CA3034681A1 (en) * 2016-06-30 2018-01-04 Arbutus Biopharma Corporation Compositions and methods for delivering messenger rna
US20200283743A1 (en) 2016-08-17 2020-09-10 The Broad Institute, Inc. Novel crispr enzymes and systems
US11352647B2 (en) 2016-08-17 2022-06-07 The Broad Institute, Inc. Crispr enzymes and systems
WO2018039131A1 (en) 2016-08-22 2018-03-01 Protiva Biotherapeutics, Inc. Anti-pd-1 antibodies, or fragments thereof, for treating hepatitis b
WO2018049025A2 (en) 2016-09-07 2018-03-15 The Broad Institute Inc. Compositions and methods for evaluating and modulating immune responses
WO2018064208A1 (en) 2016-09-28 2018-04-05 The Broad Institute, Inc. Systematic screening and mapping of regulatory elements in non-coding genomic regions, methods, compositions, and applications thereof
WO2018067991A1 (en) 2016-10-07 2018-04-12 The Brigham And Women's Hospital, Inc. Modulation of novel immune checkpoint targets
US12491261B2 (en) 2016-10-26 2025-12-09 Acuitas Therapeutics, Inc. Lipid nanoparticle formulations
EP3538067A1 (en) 2016-11-08 2019-09-18 Modernatx, Inc. Stabilized formulations of lipid nanoparticles
RS63953B1 (sr) 2017-03-15 2023-02-28 Modernatx Inc Jedinjenje i kompozicije za intracelularnu isporuku terapeutskih sredstava
CN110959039B (zh) 2017-03-15 2025-03-14 博德研究所 新型cas13b直向同源物crispr酶和系统
AU2018234814B2 (en) 2017-03-15 2022-06-30 Modernatx, Inc. Crystal forms of amino lipids
WO2018170336A1 (en) 2017-03-15 2018-09-20 Modernatx, Inc. Lipid nanoparticle formulation
JP7162021B2 (ja) 2017-03-17 2022-10-27 ニューカッスル ユニバーシティ 筋ジストロフィーを治療するためのマイクロジストロフィン断片のアデノ随伴ウイルスベクター送達
KR20200006054A (ko) 2017-04-12 2020-01-17 더 브로드 인스티튜트, 인코퍼레이티드 신규 타입 vi crispr 오르소로그 및 시스템
WO2018191719A1 (en) 2017-04-13 2018-10-18 Acuitas Therapeutics, Inc. Lipid delivery of therapeutic agents to adipose tissue
US11357856B2 (en) 2017-04-13 2022-06-14 Acuitas Therapeutics, Inc. Lipids for delivery of active agents
WO2018191750A2 (en) 2017-04-14 2018-10-18 The Broad Institute Inc. Novel delivery of large payloads
EP3615510B1 (en) 2017-04-28 2024-03-27 Acuitas Therapeutics, Inc. Novel carbonyl lipids and lipid nanoparticle formulations for delivery of nucleic acids
WO2018204777A2 (en) 2017-05-05 2018-11-08 The Broad Institute, Inc. Methods for identification and modification of lncrna associated with target genotypes and phenotypes
EP3622079B1 (en) 2017-05-08 2025-08-20 Flagship Pioneering Innovations V, Inc. Compositions for facilitating membrane fusion and uses thereof
US12297436B2 (en) 2017-05-18 2025-05-13 The Broad Institute, Inc. Systems, methods, and compositions for targeted nucleic acid editing
AU2018270111B2 (en) 2017-05-18 2022-07-14 Modernatx, Inc. Polynucleotides encoding tethered interleukin-12 (IL12) polypeptides and uses thereof
CA3066750A1 (en) 2017-06-13 2018-12-20 Flagship Pioneering Innovations V, Inc. Compositions comprising curons and uses thereof
WO2018232120A1 (en) 2017-06-14 2018-12-20 Modernatx, Inc. Compounds and compositions for intracellular delivery of agents
MA49395A (fr) 2017-06-14 2020-04-22 Modernatx Inc Polynucléotides codant pour le facteur viii de coagulation
EP3638215A4 (en) 2017-06-15 2021-03-24 Modernatx, Inc. RNA FORMULATIONS
EP3668833A1 (en) 2017-08-16 2020-06-24 Acuitas Therapeutics, Inc. Lipids for use in lipid nanoparticle formulations
JP7461872B2 (ja) 2017-08-17 2024-04-04 アクイタス セラピューティクス インコーポレイテッド 脂質ナノ粒子製剤における使用のための脂質
WO2019036028A1 (en) 2017-08-17 2019-02-21 Acuitas Therapeutics, Inc. LIPIDS FOR USE IN LIPID NANOPARTICULAR FORMULATIONS
US11524932B2 (en) 2017-08-17 2022-12-13 Acuitas Therapeutics, Inc. Lipids for use in lipid nanoparticle formulations
CA3073211A1 (en) 2017-08-31 2019-03-07 Modernatx, Inc. Methods of making lipid nanoparticles
EP3679139B1 (en) 2017-09-08 2022-11-02 MiNA Therapeutics Limited Stabilized hnf4a sarna compositions and methods of use
EP3679140B1 (en) 2017-09-08 2022-11-16 MiNA Therapeutics Limited Stabilized cebpa sarna compositions and methods of use
KR102338449B1 (ko) 2017-09-21 2021-12-10 더 브로드 인스티튜트, 인코퍼레이티드 표적화된 핵산 편집을 위한 시스템, 방법, 및 조성물
WO2019071054A1 (en) 2017-10-04 2019-04-11 The Broad Institute, Inc. METHODS AND COMPOSITIONS FOR MODIFYING THE FUNCTION AND STRUCTURE OF BUCKLES AND / OR CHROMATIN DOMAINS
WO2019135816A2 (en) 2017-10-23 2019-07-11 The Broad Institute, Inc. Novel nucleic acid modifiers
WO2019089803A1 (en) 2017-10-31 2019-05-09 The Broad Institute, Inc. Methods and compositions for studying cell evolution
WO2019089828A1 (en) 2017-10-31 2019-05-09 Acuitas Therapeutics, Inc. Lamellar lipid nanoparticles
US20190231690A1 (en) 2017-11-08 2019-08-01 L.E.A.F. Holdings Group Llc Platinum complexes and uses thereof
WO2019094983A1 (en) 2017-11-13 2019-05-16 The Broad Institute, Inc. Methods and compositions for treating cancer by targeting the clec2d-klrb1 pathway
WO2019126709A1 (en) 2017-12-22 2019-06-27 The Broad Institute, Inc. Cas12b systems, methods, and compositions for targeted dna base editing
WO2019157138A1 (en) 2018-02-07 2019-08-15 L.E.A.F. Holdings Group Llc Alpha polyglutamated pemetrexed and uses thereof
JP7490239B2 (ja) 2018-02-07 2024-05-27 エル．イー．エー．エフ． ホールディングス グループ エルエルシー ガンマポリグルタミン酸化ペメトレキセドおよびその使用
US11566246B2 (en) 2018-04-12 2023-01-31 Mina Therapeutics Limited SIRT1-saRNA compositions and methods of use
KR20250159271A (ko) 2018-05-03 2025-11-10 엘.이.에이.에프. 홀딩스 그룹 엘엘씨. 카로티노이드 조성물 및 이의 용도
PT3807413T (pt) 2018-06-18 2025-08-19 Res Inst Nationwide Childrens Hospital Administração por vetor de vírus adenoassociado de microdistrofina específica do músculo para tratar a distrofia muscular
CN113286884A (zh) 2018-08-07 2021-08-20 博德研究所 新型cas12b酶和系统
US20210317461A1 (en) 2018-08-09 2021-10-14 Verseau Therapeutics, Inc. Oligonucleotide compositions for targeting ccr2 and csf1r and uses thereof
US12421507B2 (en) 2018-08-20 2025-09-23 The Broad Institute, Inc. Methods and compositions for optochemical control of CRISPR-CAS9
EP3853202A1 (en) 2018-09-19 2021-07-28 ModernaTX, Inc. Compounds and compositions for intracellular delivery of therapeutic agents
CN112996854B (zh) 2018-09-19 2024-08-30 摩登纳特斯有限公司 高纯度peg脂质和其用途
EP3852732A1 (en) 2018-09-19 2021-07-28 ModernaTX, Inc. Peg lipids and uses thereof
WO2020061457A1 (en) 2018-09-20 2020-03-26 Modernatx, Inc. Preparation of lipid nanoparticles and methods of administration thereof
CA3113449A1 (en) 2018-09-21 2020-03-26 Acuitas Therapeutics, Inc. Systems and methods for manufacturing lipid nanoparticles and liposomes
IL281135B2 (en) 2018-09-28 2024-12-01 Nutcracker Therapeutics Inc Tertiary amino lipidated cationic peptides for nucleic acid delivery
WO2020077007A1 (en) 2018-10-09 2020-04-16 The University Of British Columbia Compositions and systems comprising transfection-competent vesicles free of organic-solvents and detergents and methods related thereto
CN113286882A (zh) 2018-11-09 2021-08-20 阿布特斯生物制药公司 脂质纳米颗粒制剂
US11166996B2 (en) 2018-12-12 2021-11-09 Flagship Pioneering Innovations V, Inc. Anellovirus compositions and methods of use
CA3118034A1 (en) 2018-12-21 2020-06-25 Curevac Ag Rna for malaria vaccines
IL284535B2 (en) 2019-01-11 2025-03-01 Acuitas Therapeutics Inc Lipids for lipid nanoparticle delivery of active ingredients
WO2020161342A1 (en) 2019-02-08 2020-08-13 Curevac Ag Coding rna administered into the suprachoroidal space in the treatment of ophtalmic diseases
US20220177863A1 (en) 2019-03-18 2022-06-09 The Broad Institute, Inc. Type vii crispr proteins and systems
US20220211740A1 (en) 2019-04-12 2022-07-07 Mina Therapeutics Limited Sirt1-sarna compositions and methods of use
WO2020236972A2 (en) 2019-05-20 2020-11-26 The Broad Institute, Inc. Non-class i multi-component nucleic acid targeting systems
CA3142883A1 (en) 2019-06-07 2020-12-10 Benjamin OAKES Engineered casx systems
WO2020254535A1 (en) 2019-06-18 2020-12-24 Curevac Ag Rotavirus mrna vaccine
ES2982157T3 (es) 2019-07-18 2024-10-14 Institut Nat De La Sante Et De Larecherche Medicale Inserm Mutante de RAC2 para su uso en la ablación de la hematopoyesis y en el tratamiento de neoplasias malignas hematopoyéticas
CA3147875A1 (en) 2019-07-19 2021-01-28 Flagship Pioneering Innovations Vi, Llc Recombinase compositions and methods of use
CA3149914A1 (en) 2019-08-06 2021-02-11 L.E.A.F. Holdings Group Llc Processes of preparing polyglutamated antifolates and uses of their compositions
EP4454640A3 (en) 2019-08-14 2025-02-12 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
CN110564729A (zh) * 2019-09-17 2019-12-13 四川大学 一种对PLK1基因具有沉默活性且具有高血清稳定性的siRNA及其应用
AR120080A1 (es) 2019-09-19 2022-02-02 Modernatx Inc Compuestos lipídicos de cola ramificada y composiciones para la administración intracelular de agentes terapéuticos
AU2020355000A1 (en) 2019-09-23 2022-03-17 Omega Therapeutics, Inc. Compositions and methods for modulating apolipoprotein B (APOB) gene expression
US11987791B2 (en) 2019-09-23 2024-05-21 Omega Therapeutics, Inc. Compositions and methods for modulating hepatocyte nuclear factor 4-alpha (HNF4α) gene expression
EP3886897A1 (en) 2020-02-04 2021-10-06 CureVac AG Coronavirus vaccine
CN115485385A (zh) 2020-03-04 2022-12-16 瑞泽恩制药公司 用于使肿瘤细胞对免疫疗法敏感的方法和组合物
EP4118207A1 (en) 2020-03-11 2023-01-18 Omega Therapeutics, Inc. Compositions and methods for modulating forkhead box p3 (foxp3) gene expression
MX2022011806A (es) 2020-03-24 2023-01-11 Generation Bio Co Vectores de adn no virales y usos de estos para expresar agentes terapéuticos contra gaucher.
IL296660A (en) 2020-03-24 2022-11-01 Generation Bio Co Non-viral dna vectors and uses thereof for expressing factor ix therapeutics
WO2021198157A1 (en) 2020-03-30 2021-10-07 BioNTech SE Rna compositions targeting claudin-18.2
US20230193311A1 (en) 2020-05-20 2023-06-22 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
CN116322758A (zh) 2020-05-29 2023-06-23 库尔维科欧洲股份公司 基于核酸的组合疫苗
EP4158031A1 (en) 2020-05-29 2023-04-05 Flagship Pioneering Innovations VI, LLC Trem compositions and methods relating thereto
CA3182026A1 (en) 2020-05-29 2021-12-02 Flagship Pioneering Innovations Vi, Llc. Trem compositions and methods relating thereto
WO2021257595A1 (en) 2020-06-15 2021-12-23 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
KR20230051172A (ko) 2020-07-16 2023-04-17 아퀴타스 테라퓨틱스 인크. 지질 나노 입자에 사용하기 위한 양이온성 지질
CA3189740A1 (en) 2020-07-27 2022-02-03 Anjarium Biosciences Ag Compositions of dna molecules, methods of making therefor, and methods of use thereof
WO2022023559A1 (en) 2020-07-31 2022-02-03 Curevac Ag Nucleic acid encoded antibody mixtures
MX2023001461A (es) 2020-08-06 2023-04-26 Modernatx Inc Composiciones para la administracion de moleculas de carga activa al epitelio de las vias respiratorias.
WO2022043551A2 (en) 2020-08-31 2022-03-03 Curevac Ag Multivalent nucleic acid based coronavirus vaccines
JP2023542492A (ja) 2020-09-03 2023-10-10 フラッグシップ パイオニアリング イノベーションズ シックス，エルエルシー 免疫原性組成物及びその使用
EP4213882A4 (en) 2020-09-15 2025-02-26 Verve Therapeutics, Inc. LIPID FORMULATIONS FOR GENE EDITING
MX2023004371A (es) 2020-10-14 2023-07-26 George Mason Res Foundation Inc Lípidos ionizables, y métodos de fabricación y uso de los mismos.
TW202233232A (zh) 2020-11-06 2022-09-01 法商賽諾菲公司 遞送mRNA疫苗的脂質奈米顆粒
US11591544B2 (en) 2020-11-25 2023-02-28 Akagera Medicines, Inc. Ionizable cationic lipids
KR20230128289A (ko) 2020-12-03 2023-09-04 스크라이브 테라퓨틱스 인크. 조작된 클래스 2 유형 v crispr 시스템
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
US20240299309A1 (en) 2020-12-22 2024-09-12 CureVac SE Pharmaceutical composition comprising lipid-based carriers encapsulating rna for multidose administration
WO2022137133A1 (en) 2020-12-22 2022-06-30 Curevac Ag Rna vaccine against sars-cov-2 variants
CA3206285A1 (en) 2020-12-23 2022-06-30 Flagship Pioneering, Inc. Compositions of modified trems and uses thereof
EP4087938A2 (en) 2021-01-27 2022-11-16 CureVac AG Method of reducing the immunostimulatory properties of in vitro transcribed rna
US11524023B2 (en) 2021-02-19 2022-12-13 Modernatx, Inc. Lipid nanoparticle compositions and methods of formulating the same
US20240181038A1 (en) 2021-03-26 2024-06-06 Glaxosmithkline Biologicals Sa Immunogenic compositions
TW202305133A (zh) 2021-03-26 2023-02-01 英商米納治療有限公司 Tmem173 sarna組合物及其使用方法
CA3214085A1 (en) 2021-03-31 2022-10-06 Darby Rye Schmidt Thanotransmission polypeptides and their use in treating cancer
CA3171429A1 (en) 2021-03-31 2022-09-30 Alexander SCHWENGER Syringes containing pharmaceutical compositions comprising rna
EP4319803B1 (en) 2021-04-08 2025-11-26 Vaxthera SAS Coronavirus vaccine comprising a mosaic protein
EP4326860A1 (en) 2021-04-20 2024-02-28 Anjarium Biosciences AG Compositions of dna molecules encoding amylo-alpha-1, 6-glucosidase, 4-alpha-glucanotransferase, methods of making thereof, and methods of use thereof
WO2022232286A1 (en) 2021-04-27 2022-11-03 Generation Bio Co. Non-viral dna vectors expressing anti-coronavirus antibodies and uses thereof
IL308404A (en) 2021-04-27 2024-01-01 Generation Bio Co Non-viral dna vectors expressing therapeutic antibodies and uses thereof
CA3171589A1 (en) 2021-05-03 2022-11-03 Moritz THRAN Improved nucleic acid sequence for cell type specific expression
AU2022290278A1 (en) 2021-06-11 2024-01-04 LifeEDIT Therapeutics, Inc. Rna polymerase iii promoters and methods of use
WO2023283359A2 (en) 2021-07-07 2023-01-12 Omega Therapeutics, Inc. Compositions and methods for modulating secreted frizzled receptor protein 1 (sfrp1) gene expression
WO2023009547A1 (en) 2021-07-26 2023-02-02 Flagship Pioneering Innovations Vi, Llc Trem compositions and uses thereof
WO2023014974A1 (en) 2021-08-06 2023-02-09 University Of Iowa Research Foundation Double stranded mrna vaccines
WO2023023055A1 (en) 2021-08-16 2023-02-23 Renagade Therapeutics Management Inc. Compositions and methods for optimizing tropism of delivery systems for rna
CN117940158A (zh) 2021-09-03 2024-04-26 库瑞瓦格欧洲公司 用于核酸递送的包含磷脂酰丝氨酸的新型脂质纳米颗粒
US20240398940A1 (en) 2021-09-03 2024-12-05 CureVac SE Novel lipid nanoparticles for delivery of nucleic acids
CA3232386A1 (en) 2021-09-14 2023-03-23 Renagade Therapeutics Management Inc. Cyclic lipids and methods of use thereof
JP2024534697A (ja) 2021-09-14 2024-09-20 レナゲード セラピューティクス マネージメント インコーポレイテッド 非環状脂質及びその使用方法
IL311450A (en) 2021-09-17 2024-05-01 Flagship Pioneering Innovations Vi Llc Compositions and methods for producing circular polyribonucleotides
US20240392271A1 (en) 2021-09-21 2024-11-28 Scribe Therapeutics Inc. Engineered casx repressor systems
WO2023069397A1 (en) 2021-10-18 2023-04-27 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
EP4426832A1 (en) 2021-11-03 2024-09-11 The J. David Gladstone Institutes, A Testamentary Trust Established under The Will of J. David Gladstone Precise genome editing using retrons
EP4430024A1 (en) 2021-11-08 2024-09-18 Orna Therapeutics, Inc. Lipid nanoparticle compositions for delivering circular polynucleotides
WO2023086465A1 (en) 2021-11-12 2023-05-19 Modernatx, Inc. Compositions for the delivery of payload molecules to airway epithelium
JP2024542188A (ja) 2021-11-16 2024-11-13 セイル バイオメディシンズ インコーポレイテッド 新規イオン化可能な脂質および脂質ナノ粒子ならびにそれらを使用する方法
MX2024006134A (es) 2021-11-22 2024-07-29 Sail Biomedicines Inc Lipidos ionizables novedosos y nanoparticulas de lipido y metodos para usar los mismos.
CA3239266A1 (en) 2021-11-24 2023-06-01 Flagship Pioneering Innovations Vi, Llc Coronavirus immunogen compositions and their uses
MX2024006440A (es) 2021-11-24 2024-08-19 Flagship Pioneering Innovations Vi Llc Composiciones inmunogenicas y sus usos.
US20250368688A1 (en) 2021-11-24 2025-12-04 Flagship Pioneering Innovations Vi, Llc Varicella-zoster virus 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
KR20240123832A (ko) 2021-12-16 2024-08-14 아퀴타스 테라퓨틱스 인크. 지질 나노입자 제형에 사용하기 위한 지질
JP2024546855A (ja) 2021-12-17 2024-12-26 フラッグシップ パイオニアリング イノベーションズ シックス，エルエルシー 変性条件下での環状ｒｎａの濃縮方法
US20250051386A1 (en) 2021-12-22 2025-02-13 Flagship Pioneering Innovations Vi, Llc Compositions and methods for purifying polyribonucleotides
CN118922211A (zh) 2021-12-23 2024-11-08 旗舰创业创新六公司 编码抗融合多肽的环状多核糖核苷酸
CA3243054A1 (en) 2021-12-23 2025-04-08 Renagade Therapeutics Management Inc. Constrained Lipids and Associated Utilization Processes
EP4463475A2 (en) 2022-01-14 2024-11-20 Anjarium Biosciences AG Compositions of dna molecules encoding factor viii, methods of making thereof, and methods of use thereof
JP2025502257A (ja) 2022-01-17 2025-01-24 アンスティチュ ナショナル ドゥ ラ サンテ エ ドゥ ラ ルシェルシュ メディカル 固形腫瘍細胞の集団の細胞死を誘導する方法
WO2023141602A2 (en) 2022-01-21 2023-07-27 Renagade Therapeutics Management Inc. Engineered retrons and methods of use
US20250345416A1 (en) 2022-01-27 2025-11-13 BioNTech SE Pharmaceutical compositions for delivery of herpes simplex virus antigens and related methods
EP4469091A1 (en) 2022-01-28 2024-12-04 CureVac SE Nucleic acid encoded transcription factor inhibitors
EP4475882A1 (en) 2022-02-09 2024-12-18 ModernaTX, Inc. Mucosal administration methods and formulations
US20250064899A1 (en) 2022-02-14 2025-02-27 INSERM (Institut National de la Santé et de la Recherche Médicale) Use of the 15-lipoxygenase for the treatment of lymphedema
JP2025508467A (ja) 2022-02-24 2025-03-26 アイオー バイオテック エーピーエス 癌治療のヌクレオチド送達
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
AU2023238223A1 (en) 2022-03-23 2024-10-10 Nanovation Therapeutics Inc. High sterol-containing lipid nanoparticles
JP2025510229A (ja) 2022-03-25 2025-04-14 セイル バイオメディシンズ インコーポレイテッド 新規のイオン化脂質および脂質ナノ粒子ならびにそれらを使用する方法
CN119654166A (zh) 2022-04-01 2025-03-18 内诺维什疗法股份有限公司 mRNA递送方法及其组合物
CN119562762A (zh) 2022-04-04 2025-03-04 加利福尼亚大学董事会 遗传互补组合物和方法
AU2023251104A1 (en) 2022-04-07 2024-10-17 Renagade Therapeutics Management Inc. Cyclic lipids and lipid nanoparticles (lnp) for the delivery of nucleic acids or peptides for use in vaccinating against infectious agents
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
US20250288659A1 (en) 2022-04-27 2025-09-18 Shanghai Regenelead Therapies Co., Ltd. Nucleic acid construct and use thereof
AU2023269030A1 (en) 2022-05-09 2024-11-14 Flagship Pioneering Innovations Vi, Llc Trem compositions and methods of use for treating proliferative disorders
EP4522753A2 (en) 2022-05-13 2025-03-19 Flagship Pioneering Innovations VII, LLC Double stranded dna compositions and related methods
CA3253047A1 (en) 2022-05-13 2025-07-04 Shanghai Regenelead Therapies Co., Ltd CONSTRUCTION OF NUCLEIC ACIDS INCLUDING A UTR AND ITS USE
CA3252451A1 (en) 2022-05-13 2023-11-16 BioNTech SE HIV-TARGETING RNA COMPOSITIONS
AU2023277551A1 (en) 2022-05-25 2024-12-05 BioNTech SE Rna compositions for delivery of monkeypox antigens and related methods
US20250345407A1 (en) 2022-05-25 2025-11-13 CureVac SE Nucleic acid based vaccine encoding an escherichia coli fimh antigenic polypeptide
WO2023230587A2 (en) 2022-05-25 2023-11-30 Akagera Medicines, Inc. Lipid nanoparticles for delivery of nucleic acids and methods of use thereof
EP4518845A1 (en) 2022-05-30 2025-03-12 BioNTech SE Complexes for delivery of nucleic acids
AU2023278164A1 (en) 2022-06-02 2024-12-05 Scribe Therapeutics Inc. Engineered class 2 type v crispr systems
JP2025524360A (ja) 2022-06-07 2025-07-30 スクライブ・セラピューティクス・インコーポレイテッド Ｐｃｓｋ９の標的化のための組成物及び方法
EP4536819A1 (en) 2022-06-07 2025-04-16 Scribe Therapeutics Inc. Compositions and methods for the targeting of pcsk9
EP4536197A1 (en) 2022-06-07 2025-04-16 Generation Bio Co. Lipid nanoparticle compositions and uses thereof
WO2023250112A1 (en) 2022-06-22 2023-12-28 Flagship Pioneering Innovations Vi, Llc Compositions of modified trems and uses thereof
WO2024023034A1 (en) 2022-07-25 2024-02-01 Institut National de la Santé et de la Recherche Médicale Use of apelin for the treatment of lymphedema
CA3261283A1 (en) 2022-08-01 2024-02-08 Flagship Pioneering Innovations Vii, Llc Immunomodulatory Proteins and Associated Methods
KR20250058785A (ko) 2022-08-12 2025-04-30 라이프 에디트 테라퓨틱스, 인크. Rna-가이드된 뉴클레아제 및 그의 활성 단편 및 변이체 및 사용 방법
WO2024035952A1 (en) 2022-08-12 2024-02-15 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
CN120239693A (zh) 2022-08-31 2025-07-01 赛欧生物医药股份有限公司 新型可电离脂质和脂质纳米颗粒以及其使用方法
WO2024047247A1 (en) 2022-09-02 2024-03-07 Institut National de la Santé et de la Recherche Médicale Base editing approaches for the treatment of amyotrophic lateral sclerosis
EP4342460A1 (en) 2022-09-21 2024-03-27 NovoArc GmbH Lipid nanoparticle with nucleic acid cargo
JP2025532177A (ja) 2022-09-23 2025-09-29 エヌクロマ・バイオ，インコーポレーテッド Ｈｂｖ遺伝子発現のエピジェネティック調節のための組成物および方法
CN120152736A (zh) 2022-09-23 2025-06-13 生物技术欧洲股份公司 用于递送疟原虫csp抗原的组合物及相关方法
WO2024064931A1 (en) 2022-09-23 2024-03-28 BioNTech SE Compositions for delivery of liver stage antigens and related methods
WO2024063789A1 (en) 2022-09-23 2024-03-28 BioNTech SE Compositions for delivery of malaria antigens and related methods
WO2024063788A1 (en) 2022-09-23 2024-03-28 BioNTech SE Compositions for delivery of malaria antigens and related methods
KR20250075664A (ko) 2022-09-26 2025-05-28 글락소스미스클라인 바이오로지칼즈 에스.에이. 인플루엔자 바이러스 백신
US20240174732A1 (en) 2022-10-05 2024-05-30 Flagship Pioneering Innovations V, Inc. Nucleic acid molecules encoding trif and additional polypeptides and their use in treating cancer
WO2024074211A1 (en) 2022-10-06 2024-04-11 BioNTech SE Rna compositions targeting claudin-18.2
EP4598954A1 (en) 2022-10-06 2025-08-13 BioNTech SE Rna compositions targeting claudin-18.2
WO2024089638A1 (en) 2022-10-28 2024-05-02 Glaxosmithkline Biologicals Sa Nucleic acid based vaccine
KR20250099195A (ko) 2022-10-31 2025-07-01 플래그쉽 파이어니어링 이노베이션스 브이아이, 엘엘씨 폴리리보뉴클레오티드를 정제하기 위한 조성물 및 방법
CN120435298A (zh) 2022-11-08 2025-08-05 旗舰创业创新第六有限责任公司 用于产生环状多核糖核苷酸的组合物和方法
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
WO2024102762A1 (en) 2022-11-08 2024-05-16 Orna Therapeutics, Inc. Lipids and lipid 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
EP4626444A2 (en) 2022-12-01 2025-10-08 Generation Bio Co. Lipid nanoparticles comprising nucleic acids, ionizable lipids, sterols, lipid anchored polymers and helper lipids, their uses
WO2024119074A1 (en) 2022-12-01 2024-06-06 Generation Bio Co. Stealth lipid nanoparticle compositions for cell targeting
WO2024119051A1 (en) 2022-12-01 2024-06-06 Generation Bio Co. Novel polyglycerol-conjugated lipids and lipid nanoparticle compositions comprising the same
CN120693181A (zh) 2022-12-08 2025-09-23 瑞科德治疗公司 脂质纳米颗粒组合物及其用途
WO2024121378A1 (en) 2022-12-09 2024-06-13 Institut National de la Santé et de la Recherche Médicale Novel human antiviral genes related to the eleos and lamassu prokaryotic systems
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
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
WO2024134199A1 (en) 2022-12-22 2024-06-27 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
EP4648789A1 (en) 2023-01-09 2025-11-19 Institut National de la Santé et de la Recherche Médicale Use of the recombinant fibrinogen-like domain of angiopoietin-like 4 for treating adverse post-ischemic cardiac remodeling in a patient who experienced a myocardial infarction
US20240252520A1 (en) 2023-01-09 2024-08-01 Beth Israel Deaconess Medical Center, Inc. Therapeutic agents and their use for treating chronic wounds
US20240269251A1 (en) 2023-01-09 2024-08-15 Flagship Pioneering Innovations V, Inc. Genetic switches and their use in treating cancer
WO2024151673A2 (en) 2023-01-09 2024-07-18 President And Fellows Of Harvard College Recombinant nucleic acid molecules and their use in wound healing
WO2024151583A2 (en) 2023-01-09 2024-07-18 Flagship Pioneering Innovations Vii, Llc Vaccines and related methods
WO2024153636A1 (en) 2023-01-17 2024-07-25 Institut National de la Santé et de la Recherche Médicale Vasorin as a biomarker and biotarget in nephrology
EP4654984A1 (en) 2023-01-27 2025-12-03 Institut National de la Santé et de la Recherche Médicale Use of amphiregulin (areg) in methods of treating vascular hyperpermeability
EP4658239A1 (en) 2023-02-03 2025-12-10 GlaxoSmithKline Biologicals S.A. 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
IL322468A (en) 2023-02-13 2025-09-01 Flagship Pioneering Innovations Vii Llc Ionizable lipids containing degradable crosslinkers and lipid carriers for therapeutic compositions
GB202302092D0 (en) 2023-02-14 2023-03-29 Glaxosmithkline Biologicals Sa Analytical method
WO2024173836A2 (en) 2023-02-17 2024-08-22 Flagship Pioneering Innovations Vii, Llc Dna compositions comprising modified cytosine
IL322201A (en) 2023-02-17 2025-09-01 Flagship Pioneering Innovations Vii Llc DNA structures containing modified uracil
AU2024233180A1 (en) 2023-03-08 2025-09-25 CureVac SE Novel lipid nanoparticle formulations for delivery of nucleic acids
KR20250162610A (ko) 2023-03-15 2025-11-18 플래그쉽 파이어니어링 이노베이션스 브이아이, 엘엘씨 폴리리보뉴클레오티드를 포함하는 조성물 및 이의 용도
WO2024192422A1 (en) 2023-03-15 2024-09-19 Flagship Pioneering Innovations Vi, Llc Immunogenic compositions and uses thereof
WO2024194484A1 (en) 2023-03-23 2024-09-26 Institut National de la Santé et de la Recherche Médicale Modulating the expression and/or activity of gas7 for modulating viral replication
WO2024205657A2 (en) 2023-03-29 2024-10-03 Orna Therapeutics, Inc. Lipids and lipid nanoparticle compositions for delivering polynucleotides
AU2024242151A1 (en) 2023-03-29 2025-09-25 Scribe Therapeutics Inc. Compositions and methods for the targeting of lpa
AU2024248139A1 (en) 2023-03-29 2025-09-25 Scribe Therapeutics Inc. Compositions and methods for the targeting of pcsk9
TW202444910A (zh) 2023-03-29 2024-11-16 美商斯奎柏治療公司 編碼casx之信使rna
AU2024246046A1 (en) 2023-03-29 2025-10-02 Scribe Therapeutics Inc. Repressor fusion protein systems
WO2024216128A1 (en) 2023-04-12 2024-10-17 Flagship Pioneering Innovations Vi, Llc Trems for use in correction of missense mutations
WO2024216191A1 (en) 2023-04-12 2024-10-17 Flagship Pioneering Innovations Vi, Llc Modified trems, compositions, and related methods thereof
WO2024220746A2 (en) 2023-04-21 2024-10-24 Flagship Pioneering Innovations Vii, Llc Rnai agents targeting fatty acid synthase and related methods
WO2024223724A1 (en) 2023-04-27 2024-10-31 Glaxosmithkline Biologicals Sa Influenza virus vaccines
CN121001738A (zh) 2023-04-27 2025-11-21 葛兰素史克生物有限公司 流感病毒疫苗
WO2024228044A1 (en) 2023-05-03 2024-11-07 BioNTech SE Optimized csp variants and related methods
AU2024266138A1 (en) 2023-05-03 2025-11-13 Manifold Biotechnologies, Inc. Methods and compositions for high-throughput protein delivery, screening, and detection
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
WO2024238700A1 (en) 2023-05-15 2024-11-21 Chroma Medicine, Inc. Compositions and methods for epigenetic regulation of hbv gene expression
AU2024270764A1 (en) 2023-05-15 2025-12-04 Nchroma Bio, Inc. Compositions and methods for epigenetic regulation of hbv gene expression
WO2024243438A2 (en) 2023-05-23 2024-11-28 Omega Therapeutics, Inc. Compositions and methods for reducing cxcl9, cxcl10, and cxcl11 gene expression
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
WO2025011529A2 (en) 2023-07-07 2025-01-16 Shanghai Circode Biomed Co., Ltd. Circular rna vaccines for seasonal flu and methods of uses
WO2025024335A2 (en) 2023-07-21 2025-01-30 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
WO2025021083A1 (en) * 2023-07-24 2025-01-30 National Institute Of Biological Sciences, Beijing Lipid nanoparticle compositions for localized treatment of skin diseases
WO2025024337A1 (en) 2023-07-24 2025-01-30 BioNTech SE Compositions for delivery of plasmodium antigens and related methods
US20250092426A1 (en) 2023-07-25 2025-03-20 Flagship Pioneering Innovations Vii, Llc Cas endonucleases and related methods
WO2025022367A2 (en) 2023-07-27 2025-01-30 Life Edit Therapeutics, Inc. Rna-guided nucleases and active fragments and variants thereof and methods of use
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
WO2025027579A2 (en) 2023-08-03 2025-02-06 BioNTech SE Rna compositions targeting hiv
WO2025027576A2 (en) 2023-08-03 2025-02-06 BioNTech SE Rna compositions targeting hiv
WO2025030097A2 (en) 2023-08-03 2025-02-06 BioNTech SE Pharmaceutical compositions for delivery of herpes simplex virus antigens and related methods
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
WO2025045142A1 (en) 2023-08-29 2025-03-06 Shanghai Circode Biomed Co., Ltd. Circular rna encoding vegf polypeptides, formulations, and methods of uses
WO2025046121A1 (en) 2023-09-01 2025-03-06 Novoarc Gmbh Lipid nanoparticle with nucleic acid cargo and ionizable lipid
EP4520345A1 (en) 2023-09-06 2025-03-12 Myneo Nv Product
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
WO2025057088A1 (en) 2023-09-11 2025-03-20 BioNTech SE Rna compositions for delivery of incretin agents
TW202525266A (zh) 2023-09-18 2025-07-01 美商旗艦先鋒創新有限責任（Ｖｉｉ）公司 可電離類脂質組成物及其治療用途
WO2025072383A1 (en) 2023-09-25 2025-04-03 The Broad Institute, Inc. Viral open reading frames, uses thereof, and methods of detecting the same
WO2025072331A1 (en) 2023-09-26 2025-04-03 Flagship Pioneering Innovations Vii, Llc Cas nucleases and related methods
WO2025072293A1 (en) 2023-09-27 2025-04-03 Scribe Therapeutics Inc. Optimized mrnas encoding casx proteins
WO2025081042A1 (en) 2023-10-12 2025-04-17 Renagade Therapeutics Management Inc. Nickase-retron template-based precision editing system and methods of use
WO2025083619A1 (en) 2023-10-18 2025-04-24 Life Edit Therapeutics, Inc. Rna-guided nucleases and acive fragments and variants thereof and methods of use
WO2025083211A1 (en) 2023-10-20 2025-04-24 Institut National de la Santé et de la Recherche Médicale Use of factor h for the treatment of dementia
WO2025096807A2 (en) 2023-10-31 2025-05-08 Flagship Pioneering Innovations Vii, Llc Novel therapeutic dna forms
WO2025097055A2 (en) 2023-11-02 2025-05-08 The Broad Institute, Inc. Compositions and methods of use of t cells in immunotherapy
WO2025101501A1 (en) 2023-11-07 2025-05-15 Orna Therapeutics, Inc. Circular rna compositions
WO2025106670A1 (en) 2023-11-14 2025-05-22 Flagship Pioneering Innovations Vii, Llc Ionizable lipidoid compositions and therapeutic uses thereof
US20250161347A1 (en) 2023-11-22 2025-05-22 Flagship Pioneering Innovations Vii, Llc Methods and compositions for treating non-alcoholic fatty liver disease
US12364773B2 (en) 2023-12-01 2025-07-22 Recode Therapeutics, Inc. Lipid nanoparticle compositions and uses thereof
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
WO2025129128A1 (en) 2023-12-15 2025-06-19 Vivasor, Inc. Compositions and methods for non-viral delivery of therapeutic compounds
WO2025129158A1 (en) 2023-12-15 2025-06-19 The Broad Institute, Inc. Engineered arc delivery vesicles and uses thereof
WO2025132839A1 (en) 2023-12-21 2025-06-26 Glaxosmithkline Biologicals Sa Influenza virus vaccines
TW202525287A (zh) 2023-12-21 2025-07-01 德商拜恩技術運輸科技有限責任公司 可離子化脂質
WO2025134066A1 (en) 2023-12-21 2025-06-26 Biontech Delivery Technologies Gmbh Ionizable lipids
WO2025133105A1 (en) 2023-12-21 2025-06-26 Biontech Delivery Technologies Gmbh Compositions and methods
WO2025137646A1 (en) 2023-12-22 2025-06-26 Recode Therapeutics, Inc. Gene editing methods and compositions for treating cystic fibrosis
WO2025155753A2 (en) 2024-01-17 2025-07-24 Renagade Therapeutics Management Inc. Improved gene editing system, guides, and methods
WO2025160334A1 (en) 2024-01-26 2025-07-31 Flagship Pioneering Innovations Vii, Llc Immunoreceptor inhibitory proteins and related methods
WO2025166325A1 (en) 2024-02-02 2025-08-07 Editas Medicine, Inc. MODIFIED GUIDE RNAs
WO2025174908A1 (en) 2024-02-12 2025-08-21 Life Edit Therapeutics, Inc. Novel rna-guided nucleases and proteins for polymerase editing
WO2025184508A1 (en) 2024-03-01 2025-09-04 Acuitas Therapeutics, Inc. Materials and methods for encapsulating therapeutics in lipid nanoparticles
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
WO2025213138A1 (en) 2024-04-05 2025-10-09 Editas Medicine, Inc. Crispr/rna-guided nuclease related methods and compositions for treating primary open angle glaucoma
WO2025217275A2 (en) 2024-04-10 2025-10-16 Flagship Pioneering Innovations Vii, Llc Immune cell targeted compositions and related methods
WO2025215072A1 (en) 2024-04-10 2025-10-16 Institut National de la Santé et de la Recherche Médicale Class i lanthipeptides with anti-viral function
US20250353881A1 (en) 2024-05-16 2025-11-20 Flagship Pioneering Innovations Vii, Llc Immunoreceptor inhibitory proteins and related methods
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
WO2025250808A1 (en) 2024-05-29 2025-12-04 The Brigham And Women’S Hospital, Inc. Anti-crispr delivery compositions and methods
WO2025248096A1 (en) 2024-05-31 2025-12-04 Institut National de la Santé et de la Recherche Médicale Antimicrobial peptides having liquid-liquid phase separation activities
WO2025259931A1 (en) 2024-06-14 2025-12-18 Orbital Therapeutics, Inc. Compositions and methods for rna circularization

Family Cites Families (244)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
NL154598B (nl) 1970-11-10 1977-09-15 Organon Nv Werkwijze voor het aantonen en bepalen van laagmoleculire verbindingen en van eiwitten die deze verbindingen specifiek kunnen binden, alsmede testverpakking.
US3901654A (en) 1971-06-21 1975-08-26 Biological Developments Receptor assays of biologically active compounds employing biologically specific receptors
US3817827A (en) 1972-03-30 1974-06-18 Scott Paper Co Soft absorbent fibrous webs containing elastomeric bonding material and formed by creping and embossing
US3935074A (en) 1973-12-17 1976-01-27 Syva Company Antibody steric hindrance immunoassay with two antibodies
US3996345A (en) 1974-08-12 1976-12-07 Syva Company Fluorescence quenching with immunological pairs in immunoassays
US4034074A (en) 1974-09-19 1977-07-05 The Board Of Trustees Of Leland Stanford Junior University Universal reagent 2-site immunoradiometric assay using labelled anti (IgG)
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
US3984533A (en) 1975-11-13 1976-10-05 General Electric Company Electrophoretic method of detecting antigen-antibody reaction
US4086257A (en) 1976-10-12 1978-04-25 Sears Barry D Phosphatidyl quaternary ammonium compounds
US4098876A (en) 1976-10-26 1978-07-04 Corning Glass Works Reverse sandwich immunoassay
CH624011A5 (cg-RX-API-DMAC7.html) 1977-08-05 1981-07-15 Battelle Memorial Institute
US4394448A (en) 1978-02-24 1983-07-19 Szoka Jr Francis C Method of inserting DNA into living cells
US4235871A (en) 1978-02-24 1980-11-25 Papahadjopoulos Demetrios P Method of encapsulating biologically active materials in lipid vesicles
DE3068743D1 (en) 1980-01-16 1984-08-30 Weder Hans G Process and dialysis-installation for the preparation of bilayer-vesicles and their use
US4598051A (en) 1980-03-12 1986-07-01 The Regents Of The University Of California Liposome conjugates and diagnostic methods therewith
US4452901A (en) 1980-03-20 1984-06-05 Ciba-Geigy Corporation Electrophoretically transferring electropherograms to nitrocellulose sheets for immuno-assays
US4376110A (en) 1980-08-04 1983-03-08 Hybritech, Incorporated Immunometric assays using monoclonal antibodies
US4522803A (en) 1983-02-04 1985-06-11 The Liposome Company, Inc. Stable plurilamellar vesicles, their preparation and use
US4588578A (en) 1983-08-08 1986-05-13 The Liposome Company, Inc. Lipid vesicles prepared in a monophase
US4515736A (en) 1983-05-12 1985-05-07 The Regents Of The University Of California Method for encapsulating materials into liposomes
US5545412A (en) 1985-01-07 1996-08-13 Syntex (U.S.A.) Inc. N-[1, (1-1)-dialkyloxy]-and N-[1, (1-1)-dialkenyloxy]-alk-1-yl-n,n,n-tetrasubstituted ammonium lipids and uses therefor
US5208036A (en) 1985-01-07 1993-05-04 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
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
US4683195A (en) 1986-01-30 1987-07-28 Cetus Corporation Process for amplifying, detecting, and/or-cloning nucleic acid sequences
US4683202A (en) 1985-03-28 1987-07-28 Cetus Corporation Process for amplifying nucleic acid sequences
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
US5320906A (en) 1986-12-15 1994-06-14 Vestar, Inc. Delivery vehicles with amphiphile-associated active ingredient
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
US5703055A (en) 1989-03-21 1997-12-30 Wisconsin Alumni Research Foundation Generation of antibodies through lipid mediated DNA delivery
FR2645866B1 (fr) 1989-04-17 1991-07-05 Centre Nat Rech Scient Nouvelles lipopolyamines, leur preparation et leur emploi
US5725871A (en) 1989-08-18 1998-03-10 Danbiosyst Uk Limited Drug delivery compositions comprising lysophosphoglycerolipid
CA2039718C (en) 1989-08-31 2003-02-25 John J. Rossi Chimeric dna-rna catalytic sequences
US5286634A (en) 1989-09-28 1994-02-15 Stadler Joan K Synergistic method for host cell transformation
JPH03126211A (ja) 1989-10-12 1991-05-29 Nippon Chemicon Corp 電解コンデンサ用電解液
US5225212A (en) 1989-10-20 1993-07-06 Liposome Technology, Inc. Microreservoir liposome composition and method
US5013556A (en) 1989-10-20 1991-05-07 Liposome Technology, Inc. Liposomes with enhanced circulation time
US5707644A (en) 1989-11-04 1998-01-13 Danbiosyst Uk Limited Small particle compositions for intranasal drug delivery
US6753423B1 (en) 1990-01-11 2004-06-22 Isis Pharmaceuticals, Inc. Compositions and methods for enhanced biostability and altered biodistribution of oligonucleotides in mammals
US5279833A (en) 1990-04-04 1994-01-18 Yale University Liposomal transfection of nucleic acids into animal cells
US5264618A (en) 1990-04-19 1993-11-23 Vical, Inc. Cationic lipids for intracellular delivery of biologically active molecules
US6465188B1 (en) 1990-06-11 2002-10-15 Gilead Sciences, Inc. Nucleic acid ligand complexes
US6365730B1 (en) 1990-06-19 2002-04-02 Gene Shears Pty. Limited DNA-Armed ribozymes and minizymes
US5789573A (en) 1990-08-14 1998-08-04 Isis Pharmaceuticals, Inc. Antisense inhibition of ICAM-1, E-selectin, and CMV IE1/IE2
DE552178T1 (de) 1990-10-12 1994-02-03 Max Planck Gesellschaft Abgeänderte ribozyme.
DE4114491A1 (de) * 1991-05-03 1992-11-05 Henkel Kgaa Fluessigwaschmittel
DE4216134A1 (de) 1991-06-20 1992-12-24 Europ Lab Molekularbiolog Synthetische katalytische oligonukleotidstrukturen
JP2774417B2 (ja) 1991-08-07 1998-07-09 株式会社ディ・ディ・エス研究所 ペプチド骨格を有する分枝鎖型糖複合体及び微粒子キャリヤー
US5283185A (en) 1991-08-28 1994-02-01 University Of Tennessee Research Corporation Method for delivering nucleic acids into cells
US6831166B2 (en) 1992-10-23 2004-12-14 Isis Pharmaceuticals, Inc. Derivatized oligonucleotides having improved uptake and other properties
US5756353A (en) 1991-12-17 1998-05-26 The Regents Of The University Of California Expression of cloned genes in the lung by aerosol-and liposome-based delivery
EP0625207A1 (en) 1991-12-17 1994-11-23 The Regents Of The University Of California Gene therapy for cystic fibrosis transmembrane conductance regulator activity (cftr)
US5858784A (en) 1991-12-17 1999-01-12 The Regents Of The University Of California Expression of cloned genes in the lung by aerosol- and liposome-based delivery
US5652094A (en) 1992-01-31 1997-07-29 University Of Montreal Nucleozymes
CA2130264A1 (en) 1992-02-19 1993-09-02 Harris Busch Oligonucleotide modulation of cell growth
AU687736B2 (en) 1992-05-11 1998-03-05 Ribozyme Pharmaceuticals, Inc. Method and reagent for inhibiting viral replication
ES2105262T3 (es) 1992-05-18 1997-10-16 Minnesota Mining & Mfg Dispositivo de suministro de farmaco a traves de las mucosas.
AU4528493A (en) 1992-06-04 1994-01-04 Regents Of The University Of California, The In vivo gene therapy with intron-free sequence of interest
US5792451A (en) 1994-03-02 1998-08-11 Emisphere Technologies, Inc. Oral drug delivery compositions and methods
AU4769893A (en) 1992-07-17 1994-02-14 Ribozyme Pharmaceuticals, Inc. Method and reagent for treatment of animal diseases
DE69334134T2 (de) 1992-07-31 2007-12-20 Genentech, Inc., South San Francisco Wässrige Formulierung enthaltend Menschliches Wachstumshormon
US5334761A (en) 1992-08-28 1994-08-02 Life Technologies, Inc. Cationic lipids
JP2854203B2 (ja) 1992-09-03 1999-02-03 株式会社ディ・ディ・エス研究所 リポソームの製造法
IL108367A0 (en) 1993-01-27 1994-04-12 Hektoen Inst For Medical Resea Antisense polynzcleotide inhibition of human growth factor-sensitive cancer cells
WO1995002698A1 (en) 1993-07-12 1995-01-26 Life Technologies, Inc. Composition and methods for transfecting eukaryotic cells
US5426039A (en) 1993-09-08 1995-06-20 Bio-Rad Laboratories, Inc. Direct molecular cloning of primer extended DNA containing an alkane diol
US5801154A (en) 1993-10-18 1998-09-01 Isis Pharmaceuticals, Inc. Antisense oligonucleotide modulation of multidrug resistance-associated protein
US5674908A (en) 1993-12-20 1997-10-07 Life Technologies, Inc. Highly packed polycationic ammonium, sulfonium and phosphonium lipids
FR2714830B1 (fr) 1994-01-10 1996-03-22 Rhone Poulenc Rorer Sa Composition contenant des acides nucléiques, préparation et utilisations.
US6075012A (en) 1994-02-11 2000-06-13 Life Technologies, Inc. Reagents for intracellular delivery of macromolecules
US6989434B1 (en) 1994-02-11 2006-01-24 Invitrogen Corporation 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
AU696881B2 (en) 1994-06-22 1998-09-24 Megabios Corporation Cationic amphiphiles
FR2722506B1 (fr) 1994-07-13 1996-08-14 Rhone Poulenc Rorer Sa Composition contenant des acides nucleiques, preparation et utilisations
US5820873A (en) 1994-09-30 1998-10-13 The University Of British Columbia Polyethylene glycol modified ceramide lipids and liposome uses thereof
US5885613A (en) 1994-09-30 1999-03-23 The University Of British Columbia Bilayer stabilizing components and their use in forming programmable fusogenic liposomes
US5753613A (en) 1994-09-30 1998-05-19 Inex Pharmaceuticals Corporation Compositions for the introduction of polyanionic materials into cells
EP1179340A3 (en) 1994-09-30 2003-05-07 INEX Pharmaceutical Corp. Compositions for the introduction of polyanionic materials into cells
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
US5580579A (en) 1995-02-15 1996-12-03 Nano Systems L.L.C. Site-specific adhesion within the GI tract using nanoparticles stabilized by high molecular weight, linear poly (ethylene oxide) polymers
IE80468B1 (en) 1995-04-04 1998-07-29 Elan Corp Plc Controlled release biodegradable nanoparticles containing insulin
US5747470A (en) 1995-06-07 1998-05-05 Gen-Probe Incorporated Method for inhibiting cellular proliferation using antisense oligonucleotides to gp130 mRNA
US6051429A (en) 1995-06-07 2000-04-18 Life Technologies, Inc. Peptide-enhanced cationic lipid transfections
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
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
JP4338106B2 (ja) 1995-06-07 2009-10-07 ライフ テクノロジーズ コーポレーション ペプチド増強カチオン脂質トランスフェクション
DE69634084T2 (de) 1995-06-07 2005-12-08 Inex Pharmaceuticals Corp. Herstellung von lipid-nukleinsäure partikeln duch ein hydrophobische lipid-nukleinsäuree komplexe zwischenprodukt und zur verwendung in der gentransfer
AU701106B2 (en) 1995-06-07 1999-01-21 Promega Biosciences, Inc. Novel carbamate-based cationic lipids
US20030069173A1 (en) 1998-03-16 2003-04-10 Life Technologies, Inc. Peptide-enhanced transfections
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
US5981501A (en) 1995-06-07 1999-11-09 Inex Pharmaceuticals Corp. Methods for encapsulating plasmids in lipid bilayers
US5811406A (en) 1995-06-07 1998-09-22 Regents Of The University Of California Dry powder formulations of polynucleotide complexes
US6020526A (en) 1995-07-21 2000-02-01 Genta, Incorporated Amide-based cationic lipids
US6339173B1 (en) 1996-07-22 2002-01-15 Promega Biosciences, Inc. Amide-based cationic lipids
US20040142895A1 (en) 1995-10-26 2004-07-22 Sirna Therapeutics, Inc. Nucleic acid-based modulation of gene expression in the vascular endothelial growth factor pathway
US20030216335A1 (en) 2001-11-30 2003-11-20 Jennifer Lockridge Method and reagent for the modulation of female reproductive diseases and conditions
US6330349B1 (en) 1995-11-30 2001-12-11 Chromavision Medical Systems, Inc. Automated method for image analysis of residual protein
US5817856A (en) 1995-12-11 1998-10-06 Yissum Research Development Company Of The Hebrew University Of Jerusalem Radiation-protective phospholipid and method
US5998203A (en) 1996-04-16 1999-12-07 Ribozyme Pharmaceuticals, Inc. Enzymatic nucleic acids containing 5'-and/or 3'-cap structures
US6713069B1 (en) 1996-04-16 2004-03-30 Board Of Supervisors Of Louisiana State University And Agricultural And Mechanical College Compositions and methods for detecting, preventing, and treating African Hemorrhagic Fever
US20050119470A1 (en) 1996-06-06 2005-06-02 Muthiah Manoharan Conjugated oligomeric compounds and their use in gene modulation
US6284267B1 (en) 1996-08-14 2001-09-04 Nutrimed Biotech Amphiphilic materials and liposome formulations thereof
US6111085A (en) 1996-09-13 2000-08-29 Isis Pharmaceuticals, Inc. Carbamate-derivatized nucleosides and oligonucleosides
US6210707B1 (en) 1996-11-12 2001-04-03 The Regents Of The University Of California Methods of forming protein-linked lipidic microparticles, and compositions thereof
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
DE69841002D1 (de) 1997-05-14 2009-09-03 Univ British Columbia Hochwirksame verkapselung von nukleinsäuren in lipidvesikeln
AU7490098A (en) 1997-05-15 1998-12-08 Genzyme Corporation Cationic amphiphile formulations
US20030073640A1 (en) 1997-07-23 2003-04-17 Ribozyme Pharmaceuticals, Inc. Novel compositions for the delivery of negatively charged molecules
US6110745A (en) 1997-07-24 2000-08-29 Inex Pharmaceuticals Corp. Preparation of lipid-nucleic acid particles using a solvent extraction and direct hydration method
CA2309727C (en) 1997-10-10 2012-12-04 Inex Pharmaceuticals Corporation Methods for encapsulating nucleic acids in lipid bilayers
US6320017B1 (en) 1997-12-23 2001-11-20 Inex Pharmaceuticals Corp. Polyamide oligomers
US6410328B1 (en) 1998-02-03 2002-06-25 Protiva Biotherapeutics Inc. Sensitizing cells to compounds using lipid-mediated gene and compound delivery
CA2271582A1 (en) 1998-05-14 1999-11-14 Sean C. Semple Method for administration of therapeutic agents, including antisense, with repeat dosing
US6429200B1 (en) 1998-07-17 2002-08-06 Mirus Corporation Reverse micelles for delivery of nucleic acids
ATE237312T1 (de) 1998-07-20 2003-05-15 Protiva Biotherapeutics Inc In liposomen verkapselte nukleinsäurekomplexe
US6900049B2 (en) 1998-09-10 2005-05-31 Cell Genesys, Inc. Adenovirus vectors containing cell status-specific response elements and methods of use thereof
DE69937964T2 (de) 1998-11-12 2009-01-02 Invitrogen Corp., Carlsbad Transportreagentien
US6649780B1 (en) 1998-12-22 2003-11-18 Valentis, Inc. Cationic lipids
CN1349402A (zh) 1999-03-02 2002-05-15 利普索姆公司 生物活性络合物于脂质体中囊封工艺
CA2370690A1 (en) 1999-04-20 2000-10-26 The University Of British Columbia Cationic peg-lipids and methods of use
US6852334B1 (en) 1999-04-20 2005-02-08 The University Of British Columbia Cationic peg-lipids and methods of use
US6696424B1 (en) 1999-05-28 2004-02-24 Vical Incorporated Cytofectin dimers and methods of use thereof
US20010048940A1 (en) 1999-06-18 2001-12-06 Jennifer D. Tousignant Cationic amphiphile micellar complexes
RU2250911C2 (ru) 1999-07-14 2005-04-27 Элзэ Копэрейшн Липосомальная композиция, нейтральный липополимер и способ увеличения времени циркуляции липосомы
AU769357B2 (en) 1999-07-15 2004-01-22 University Of British Columbia, The Methods for preparation of lipid-encapsulated therapeutic agents
HUP0202327A3 (en) 1999-08-27 2004-07-28 Inex Pharmaceuticals Corp Burn Compositions for stimulating cytokine secretion and inducing an immune response
US6458382B1 (en) 1999-11-12 2002-10-01 Mirus Corporation Nucleic acid transfer complexes
JP4782966B2 (ja) 2000-01-10 2011-09-28 イッサム・リサーチ・ディベロップメント・カンパニー・オブ・ザ・ヘブルー・ユニバーシティ・オブ・エルサレム・リミテッド 疾患の治療における脂質コンジュゲートの使用
US8202979B2 (en) 2002-02-20 2012-06-19 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid
US7491805B2 (en) 2001-05-18 2009-02-17 Sirna Therapeutics, Inc. Conjugates and compositions for cellular delivery
US7833992B2 (en) 2001-05-18 2010-11-16 Merck Sharpe & Dohme Conjugates and compositions for cellular delivery
DE60140676D1 (de) 2000-03-30 2010-01-14 Massachusetts Inst Technology Mediatoren von rns-interferenz, die rns-sequenzspezifisch sind
US7189705B2 (en) 2000-04-20 2007-03-13 The University Of British Columbia Methods of enhancing SPLP-mediated transfection using endosomal membrane destabilizers
AU2001275423B2 (en) 2000-06-09 2007-01-11 Regulon, Inc. Encapsulation of polynucleotides and drugs into targeted liposomes
US20030072794A1 (en) 2000-06-09 2003-04-17 Teni Boulikas Encapsulation of plasmid DNA (lipogenes™) and therapeutic agents with nuclear localization signal/fusogenic peptide conjugates into targeted liposome complexes
US20030082103A1 (en) 2000-10-11 2003-05-01 Targesome, Inc. Targeted therapeutic lipid constructs having cell surface targets
EP1328254B1 (en) 2000-10-25 2007-02-21 The University Of British Columbia Lipid formulations for target delivery
AU2002232387A1 (en) 2000-10-27 2002-05-06 Invitrogen Corporation Method for introducing antisense oligonucleotides into eucaryotic cells
US20020081736A1 (en) 2000-11-03 2002-06-27 Conroy Susan E. Nucleic acid delivery
CZ308053B6 (cs) 2000-12-01 2019-11-27 Max Planck Gesellschaft Izolovaná molekula dvouřetězcové RNA, způsob její výroby a její použití
WO2002069369A2 (en) 2000-12-08 2002-09-06 Coley Pharmaceutical Gmbh Cpg-like nucleic acids and methods of use thereof
TW593427B (en) 2000-12-18 2004-06-21 Nektar Therapeutics Al Corp Synthesis of high molecular weight non-peptidic polymer derivatives
US7053150B2 (en) 2000-12-18 2006-05-30 Nektar Therapeutics Al, Corporation Segmented polymers and their conjugates
EP1386004A4 (en) 2001-04-05 2005-02-16 Ribozyme Pharm Inc MODULATION OF GENE EXPRESSION ASSOCIATED WITH INFLAMMATORY PROLIFERATION AND GROWTH OF NEURITIES BY METHODS INVOLVING NUCLEIC ACID
US20030026831A1 (en) 2001-04-20 2003-02-06 Aparna Lakkaraju Anionic liposomes for delivery of bioactive agents
US20040142892A1 (en) 2001-04-30 2004-07-22 The University Of British Columbia Autogene nucleic acids encoding a secretable RNA polymerase
US20030077829A1 (en) 2001-04-30 2003-04-24 Protiva Biotherapeutics Inc.. Lipid-based formulations
US20070173473A1 (en) 2001-05-18 2007-07-26 Sirna Therapeutics, Inc. RNA interference mediated inhibition of proprotein convertase subtilisin Kexin 9 (PCSK9) gene expression using short interfering nucleic acid (siNA)
US20030130186A1 (en) 2001-07-20 2003-07-10 Chandra Vargeese Conjugates and compositions for cellular delivery
US7109165B2 (en) 2001-05-18 2006-09-19 Sirna Therapeutics, Inc. Conjugates and compositions for cellular delivery
US20050282188A1 (en) 2001-05-18 2005-12-22 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using short interfering nucleic acid (siNA)
DE10163098B4 (de) 2001-10-12 2005-06-02 Alnylam Europe Ag Verfahren zur Hemmung der Replikation von Viren
US20040063654A1 (en) 2001-11-02 2004-04-01 Davis Mark E. Methods and compositions for therapeutic use of RNA interference
AU2003207708A1 (en) 2002-02-20 2003-09-09 Sirna Therapeutics, Inc. Rna interference mediated inhibition of map kinase genes
CA2515243A1 (en) 2002-02-22 2003-08-28 Klaus Strebhardt Agent for inhibiting development or progress of proliferative diseases and especially cancer diseases and pharmaceutical composition containing said agent
EP1509203B1 (en) 2002-05-15 2016-04-13 California Pacific Medical Center Delivery of nucleic acid-like compounds
ES2354607T3 (es) 2002-06-28 2011-03-16 Protiva Biotherapeutics Inc. Procedimiento y aparato para producir liposomas.
DE60326961D1 (de) 2002-07-26 2009-05-14 Novartis Vaccines & Diagnostic Modifizierte kleine irns moleküle und methoden zu deren anwendung
US8090542B2 (en) 2002-11-14 2012-01-03 Dharmacon Inc. Functional and hyperfunctional siRNA
WO2006006948A2 (en) 2002-11-14 2006-01-19 Dharmacon, Inc. METHODS AND COMPOSITIONS FOR SELECTING siRNA OF IMPROVED FUNCTIONALITY
MXPA05007651A (es) 2003-01-16 2005-10-26 Univ Pennsylvania Composiciones y metodos para la inhibicion por arnsi de la molecula de adhesion intercelular-1.
US20040167090A1 (en) 2003-02-21 2004-08-26 Monahan Sean D. Covalent modification of RNA for in vitro and in vivo delivery
US7803781B2 (en) 2003-02-28 2010-09-28 Isis Pharmaceuticals, Inc. Modulation of growth hormone receptor expression and insulin-like growth factor expression
US20050164212A1 (en) 2003-03-06 2005-07-28 Todd Hauser Modulation of gene expression using DNA-RNA hybrids
US20040185559A1 (en) 2003-03-21 2004-09-23 Isis Pharmaceuticals Inc. Modulation of diacylglycerol acyltransferase 1 expression
JP2006522158A (ja) 2003-04-03 2006-09-28 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 ｉＲＮＡ複合体
WO2004091515A2 (en) 2003-04-09 2004-10-28 Alnylam Pharmaceuticals, Inc. iRNA CONJUGATES
JP4597976B2 (ja) 2003-04-17 2010-12-15 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 修飾ｉＲＮＡ剤
WO2004104199A2 (en) 2003-05-15 2004-12-02 Oligo Engine, Inc. Modulation of gene expression using dna-dna hybrids
EP1638919B1 (en) 2003-06-18 2012-09-26 Biolab Ltd. Sphingolipids polyalkylamines conjugates
JP4951338B2 (ja) 2003-07-16 2012-06-13 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 脂質に封入された干渉ｒｎａ
US7825235B2 (en) 2003-08-18 2010-11-02 Isis Pharmaceuticals, Inc. Modulation of diacylglycerol acyltransferase 2 expression
CA2551022C (en) 2003-09-15 2013-06-04 Protiva Biotherapeutics, Inc. Polyethyleneglycol-modified lipid compounds and uses thereof
CN100368423C (zh) * 2003-11-21 2008-02-13 杭州新瑞佳生物医药技术开发有限公司 有效杀伤肿瘤细胞的针对PLK1 mRNA的小干扰核糖核酸分子（SiRNA）、其混合物及用途
US20070265220A1 (en) 2004-03-15 2007-11-15 City Of Hope Methods and compositions for the specific inhibition of gene expression by double-stranded RNA
US20050277610A1 (en) 2004-03-15 2005-12-15 City Of Hope Methods and compositions for the specific inhibition of gene expression by double-stranded RNA
EP1766035B1 (en) * 2004-06-07 2011-12-07 Protiva Biotherapeutics Inc. Lipid encapsulated interfering rna
JP4764426B2 (ja) * 2004-06-07 2011-09-07 プロチバ バイオセラピューティクス インコーポレイティッド カチオン性脂質および使用方法
AU2005259799A1 (en) 2004-07-02 2006-01-12 Protiva Biotherapeutics, Inc. Immunostimulatory siRNA molecules and uses therefor
EP1793835A4 (en) 2004-09-10 2010-12-01 Somagenics Inc SMALL INTERFERING RNA EFFECTIVELY INHIBITING VIRAL GENE EXPRESSION AND METHODS OF USE THEREOF
US7528118B2 (en) 2004-09-24 2009-05-05 Alnylam Pharmaceuticals, Inc. RNAi modulation of ApoB and uses thereof
JPWO2006035515A1 (ja) 2004-09-28 2008-05-15 平 前川 膀胱表在性癌の治療又は予防用医薬組成物、及びその利用
CA2587411A1 (en) 2004-11-17 2006-05-26 Protiva Biotherapeutics, Inc. Sirna silencing of apolipoprotein b
WO2006074108A2 (en) 2004-12-30 2006-07-13 Hauser Todd M Compositions and methods for modulating gene expression using self-protected oligonucleotides
WO2007086881A2 (en) 2005-02-14 2007-08-02 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
WO2006102163A2 (en) 2005-03-17 2006-09-28 Invitrogen Corporation Transfection reagents for non-adherent suspension cells
CN101267805A (zh) 2005-07-27 2008-09-17 普洛体维生物治疗公司 制造脂质体的系统和方法
JP2007052863A (ja) * 2005-08-18 2007-03-01 Toshiba Corp 光ディスク、光ディスク作製方法、光ディスク装置
US20070054873A1 (en) 2005-08-26 2007-03-08 Protiva Biotherapeutics, Inc. Glucocorticoid modulation of nucleic acid-mediated immune stimulation
US7838658B2 (en) * 2005-10-20 2010-11-23 Ian Maclachlan siRNA silencing of filovirus gene expression
CA2628300C (en) 2005-11-02 2018-04-17 Protiva Biotherapeutics, Inc. Modified sirna molecules and uses thereof
US20070218122A1 (en) 2005-11-18 2007-09-20 Protiva Biotherapeutics, Inc. siRNA silencing of influenza virus gene expression
US20070231392A1 (en) 2006-01-23 2007-10-04 Ernst Wagner CHEMICALLY MODIFIED POLYCATION POLYMER FOR siRNA DELIVERY
EA015676B1 (ru) 2006-05-11 2011-10-31 Элнилэм Фармасьютикалз, Инк. Композиции и способы ингибирования экспрессии гена pcsk9
US8598333B2 (en) * 2006-05-26 2013-12-03 Alnylam Pharmaceuticals, Inc. SiRNA silencing of genes expressed in cancer
US7915399B2 (en) 2006-06-09 2011-03-29 Protiva Biotherapeutics, Inc. Modified siRNA molecules and uses thereof
US20080311040A1 (en) 2007-03-06 2008-12-18 Flagship Ventures METHODS AND COMPOSITIONS FOR IMPROVED THERAPEUTIC EFFECTS WITH siRNA
SI2494993T1 (sl) 2007-05-04 2019-01-31 Marina Biotech, Inc. Aminokislinski lipidi in njihove uporabe
US20110159586A1 (en) 2007-12-07 2011-06-30 Halo-Bio Rnai Therapeutics, Inc. Compositions and methods for modulating gene expression using asymmetrically-active precursor polynucleotides
WO2009076400A2 (en) 2007-12-10 2009-06-18 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of factor vii gene
EP2238251B1 (en) 2007-12-27 2015-02-11 Protiva Biotherapeutics Inc. Silencing of polo-like kinase expression using interfering rna
JP5749494B2 (ja) 2008-01-02 2015-07-15 テクミラ ファーマシューティカルズ コーポレイション 核酸の送達のための改善された組成物および方法
MX2010009611A (es) 2008-03-05 2010-12-15 Alnylam Pharmaceuticals Inc Composiciones y metodos para inhibir la expresion de genes eg5 y vegf.
EP2281041B1 (en) * 2008-04-15 2014-07-02 Protiva Biotherapeutics Inc. Silencing of csn5 gene expression using interfering rna
CA2721333C (en) 2008-04-15 2020-12-01 Protiva Biotherapeutics, Inc. Novel lipid formulations for nucleic acid delivery
US9006193B2 (en) * 2008-05-13 2015-04-14 University Of Washington Polymeric carrier
WO2010006282A2 (en) 2008-07-10 2010-01-14 Serina Therapeutics, Inc. Polyoxazolines with inert terminating groups, polyoxazolines prepared from protected initiating groups and related compounds
JP5777519B2 (ja) 2008-10-09 2015-09-09 テクミラ ファーマシューティカルズ コーポレイション 改良されたアミノ脂質および核酸の送達方法
PL2937418T3 (pl) 2008-10-20 2018-04-30 Alnylam Pharmaceuticals, Inc. Kompozycje i sposoby hamowania ekspresji transtyretyny
PL2355851T3 (pl) 2008-11-10 2018-08-31 Arbutus Biopharma Corporation Nowe lipidy i kompozycje do dostarczania środków terapeutycznych
US8722082B2 (en) 2008-11-10 2014-05-13 Tekmira Pharmaceuticals Corporation Lipids and compositions for the delivery of therapeutics
EP3243504A1 (en) 2009-01-29 2017-11-15 Arbutus Biopharma Corporation Improved lipid formulation
CA2754043A1 (en) 2009-03-12 2010-09-16 Alnylam Pharmaceuticals, Inc. Lipid formulated compositions and methods for inhibiting expression of eg5 and vegf genes
LT2440183T (lt) 2009-06-10 2018-08-10 Arbutus Biopharma Corporation Patobulinta lipido kompozicija
WO2010147992A1 (en) 2009-06-15 2010-12-23 Alnylam Pharmaceuticals, Inc. Methods for increasing efficacy of lipid formulated sirna
US8236943B2 (en) * 2009-07-01 2012-08-07 Protiva Biotherapeutics, Inc. Compositions and methods for silencing apolipoprotein B
US8283333B2 (en) 2009-07-01 2012-10-09 Protiva Biotherapeutics, Inc. Lipid formulations for nucleic acid delivery
US8569256B2 (en) * 2009-07-01 2013-10-29 Protiva Biotherapeutics, Inc. Cationic lipids and methods for the delivery of therapeutic agents
US9018187B2 (en) * 2009-07-01 2015-04-28 Protiva Biotherapeutics, Inc. Cationic lipids and methods for the delivery of therapeutic agents
WO2011011447A1 (en) 2009-07-20 2011-01-27 Protiva Biotherapeutics, Inc. Compositions and methods for silencing ebola virus gene expression
EP2496714B1 (en) * 2009-11-04 2016-08-31 DiamiR, LLC Methods of using micro-rna from bodily fluids for diagnosis and monitoring of mild cognitive impairment
US8455455B1 (en) * 2010-03-31 2013-06-04 Protiva Biotherapeutics, Inc. Compositions and methods for silencing genes involved in hemorrhagic fever
WO2011141703A1 (en) 2010-05-12 2011-11-17 Protiva Biotherapeutics Inc. Compositions and methods for silencing apolipoprotein b
WO2011141704A1 (en) 2010-05-12 2011-11-17 Protiva Biotherapeutics, Inc Novel cyclic cationic lipids and methods of use
WO2012000104A1 (en) 2010-06-30 2012-01-05 Protiva Biotherapeutics, Inc. Non-liposomal systems for nucleic acid delivery
US8466122B2 (en) * 2010-09-17 2013-06-18 Protiva Biotherapeutics, Inc. Trialkyl cationic lipids and methods of use thereof
US20120136073A1 (en) 2010-11-15 2012-05-31 Life Technologies Corporation Amine-Containing Transfection Reagents and methods for making and using same
US9126966B2 (en) 2011-08-31 2015-09-08 Protiva Biotherapeutics, Inc. Cationic lipids and methods of use thereof
EP2764007A4 (en) 2011-10-05 2015-05-06 Protiva Biotherapeutics Inc COMPOSITIONS AND METHODS FOR DISABLING ALDEHYDE DEHYDROGENASE
SMT202200355T1 (it) 2011-12-16 2022-11-18 Modernatx Inc Composizioni di mrna modificato
US9035039B2 (en) 2011-12-22 2015-05-19 Protiva Biotherapeutics, Inc. Compositions and methods for silencing SMAD4
KR102410072B1 (ko) 2014-08-29 2022-06-20 알닐람 파마슈티칼스 인코포레이티드 트랜스타이레틴(ttr) 매개 아밀로이드증의 치료 방법
BR112017006679A2 (pt) 2014-10-02 2017-12-26 Protiva Biotherapeutics Inc moléculas, composição, partícula, composição farmacêutica, métodos para silenciar a expressão de um gene, usos de uma partícula, métodos para melhorar um ou mais sintomas, métodos para tratar uma infecção, usos de uma composição, método para inibir a replicação do vírus da hepatite d
US20200315967A1 (en) 2016-06-24 2020-10-08 Modernatx, Inc. Lipid nanoparticles
EP3638215A4 (en) 2017-06-15 2021-03-24 Modernatx, Inc. RNA FORMULATIONS

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