KR20220045089A - 트리알킬 양이온성 지질 및 그의 사용 방법 - Google Patents

트리알킬 양이온성 지질 및 그의 사용 방법 Download PDF

Info

Publication number
KR20220045089A
KR20220045089A KR1020227011265A KR20227011265A KR20220045089A KR 20220045089 A KR20220045089 A KR 20220045089A KR 1020227011265 A KR1020227011265 A KR 1020227011265A KR 20227011265 A KR20227011265 A KR 20227011265A KR 20220045089 A KR20220045089 A KR 20220045089A
Authority
KR
South Korea
Prior art keywords
lipid
mol
sirna
nucleotides
nucleic acid
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Ceased
Application number
KR1020227011265A
Other languages
English (en)
Korean (ko)
Inventor
제임스 헤이즈
마크 우드
알란 마틴
Original Assignee
아뷰터스 바이오파마 코포레이션
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by 아뷰터스 바이오파마 코포레이션 filed Critical 아뷰터스 바이오파마 코포레이션
Publication of KR20220045089A publication Critical patent/KR20220045089A/ko
Ceased legal-status Critical Current

Links

Classifications

    • 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
    • A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/16—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite containing nitrogen, e.g. nitro-, nitroso-, azo-compounds, nitriles, cyanates
    • A61K47/18—Amines; Amides; Ureas; Quaternary ammonium compounds; Amino acids; Oligopeptides having up to five amino acids
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07C—ACYCLIC OR CARBOCYCLIC COMPOUNDS
    • C07C219/00—Compounds containing amino and esterified hydroxy groups bound to the same carbon skeleton
    • C07C219/02—Compounds containing amino and esterified hydroxy groups bound to the same carbon skeleton having esterified hydroxy groups and amino groups bound to acyclic carbon atoms of the same carbon skeleton
    • C07C219/20—Compounds containing amino and esterified hydroxy groups bound to the same carbon skeleton having esterified hydroxy groups and amino groups bound to acyclic carbon atoms of the same carbon skeleton the carbon skeleton being unsaturated
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • 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
    • A61K38/00—Medicinal preparations containing peptides
    • 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
    • A61K9/14—Particulate form, e.g. powders, Processes for size reducing of pure drugs or the resulting products, Pure drug nanoparticles
    • A61K9/141—Intimate drug-carrier mixtures characterised by the carrier, e.g. ordered mixtures, adsorbates, solid solutions, eutectica, co-dried, co-solubilised, co-kneaded, co-milled, co-ground products, co-precipitates, co-evaporates, co-extrudates, co-melts; Drug nanoparticles with adsorbed surface modifiers
    • A61K9/145—Intimate drug-carrier mixtures characterised by the carrier, e.g. ordered mixtures, adsorbates, solid solutions, eutectica, co-dried, co-solubilised, co-kneaded, co-milled, co-ground products, co-precipitates, co-evaporates, co-extrudates, co-melts; Drug nanoparticles with adsorbed surface modifiers with organic compounds
    • 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
    • A61K9/48—Preparations in capsules, e.g. of gelatin, of chocolate
    • A61K9/50—Microcapsules having a gas, liquid or semi-solid filling; Solid microparticles or pellets surrounded by a distinct coating layer, e.g. coated microspheres, coated drug crystals
    • A61K9/51—Nanocapsules; Nanoparticles
    • A61K9/5107—Excipients; Inactive ingredients
    • A61K9/5123—Organic compounds, e.g. fats, sugars
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12—Antivirals
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07C—ACYCLIC OR CARBOCYCLIC COMPOUNDS
    • C07C217/00—Compounds containing amino and etherified hydroxy groups bound to the same carbon skeleton
    • C07C217/02—Compounds containing amino and etherified hydroxy groups bound to the same carbon skeleton having etherified hydroxy groups and amino groups bound to acyclic carbon atoms of the same carbon skeleton
    • C07C217/04—Compounds containing amino and etherified hydroxy groups bound to the same carbon skeleton having etherified hydroxy groups and amino groups bound to acyclic carbon atoms of the same carbon skeleton the carbon skeleton being acyclic and saturated
    • C07C217/06—Compounds containing amino and etherified hydroxy groups bound to the same carbon skeleton having etherified hydroxy groups and amino groups bound to acyclic carbon atoms of the same carbon skeleton the carbon skeleton being acyclic and saturated having only one etherified hydroxy group and one amino group bound to the carbon skeleton, which is not further substituted
    • C07C217/08—Compounds containing amino and etherified hydroxy groups bound to the same carbon skeleton having etherified hydroxy groups and amino groups bound to acyclic carbon atoms of the same carbon skeleton the carbon skeleton being acyclic and saturated having only one etherified hydroxy group and one amino group bound to the carbon skeleton, which is not further substituted the oxygen atom of the etherified hydroxy group being further bound to an acyclic carbon atom
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07C—ACYCLIC OR CARBOCYCLIC COMPOUNDS
    • C07C217/00—Compounds containing amino and etherified hydroxy groups bound to the same carbon skeleton
    • C07C217/02—Compounds containing amino and etherified hydroxy groups bound to the same carbon skeleton having etherified hydroxy groups and amino groups bound to acyclic carbon atoms of the same carbon skeleton
    • C07C217/46—Compounds containing amino and etherified hydroxy groups bound to the same carbon skeleton having etherified hydroxy groups and amino groups bound to acyclic carbon atoms of the same carbon skeleton the carbon skeleton being acyclic and unsaturated
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07C—ACYCLIC OR CARBOCYCLIC COMPOUNDS
    • C07C219/00—Compounds containing amino and esterified hydroxy groups bound to the same carbon skeleton
    • C07C219/02—Compounds containing amino and esterified hydroxy groups bound to the same carbon skeleton having esterified hydroxy groups and amino groups bound to acyclic carbon atoms of the same carbon skeleton
    • C07C219/04—Compounds containing amino and esterified hydroxy groups bound to the same carbon skeleton having esterified hydroxy groups and amino groups bound to acyclic carbon atoms of the same carbon skeleton the carbon skeleton being acyclic and saturated
    • C07C219/06—Compounds containing amino and esterified hydroxy groups bound to the same carbon skeleton having esterified hydroxy groups and amino groups bound to acyclic carbon atoms of the same carbon skeleton the carbon skeleton being acyclic and saturated having the hydroxy groups esterified by carboxylic acids having the esterifying carboxyl groups bound to hydrogen atoms or to acyclic carbon atoms of an acyclic saturated carbon skeleton
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07C—ACYCLIC OR CARBOCYCLIC COMPOUNDS
    • C07C229/00—Compounds containing amino and carboxyl groups bound to the same carbon skeleton
    • C07C229/02—Compounds containing amino and carboxyl groups bound to the same carbon skeleton having amino and carboxyl groups bound to acyclic carbon atoms of the same carbon skeleton
    • C07C229/04—Compounds containing amino and carboxyl groups bound to the same carbon skeleton having amino and carboxyl groups bound to acyclic carbon atoms of the same carbon skeleton the carbon skeleton being acyclic and saturated
    • C07C229/06—Compounds containing amino and carboxyl groups bound to the same carbon skeleton having amino and carboxyl groups bound to acyclic carbon atoms of the same carbon skeleton the carbon skeleton being acyclic and saturated having only one amino and one carboxyl group bound to the carbon skeleton
    • C07C229/10—Compounds containing amino and carboxyl groups bound to the same carbon skeleton having amino and carboxyl groups bound to acyclic carbon atoms of the same carbon skeleton the carbon skeleton being acyclic and saturated having only one amino and one carboxyl group bound to the carbon skeleton the nitrogen atom of the amino group being further bound to acyclic carbon atoms or to carbon atoms of rings other than six-membered aromatic rings
    • C07C229/12—Compounds containing amino and carboxyl groups bound to the same carbon skeleton having amino and carboxyl groups bound to acyclic carbon atoms of the same carbon skeleton the carbon skeleton being acyclic and saturated having only one amino and one carboxyl group bound to the carbon skeleton the nitrogen atom of the amino group being further bound to acyclic carbon atoms or to carbon atoms of rings other than six-membered aromatic rings to carbon atoms of acyclic carbon skeletons
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07C—ACYCLIC OR CARBOCYCLIC COMPOUNDS
    • C07C271/00—Derivatives of carbamic acids, i.e. compounds containing any of the groups, the nitrogen atom not being part of nitro or nitroso groups
    • C07C271/06—Esters of carbamic acids
    • C07C271/08—Esters of carbamic acids having oxygen atoms of carbamate groups bound to acyclic carbon atoms
    • C07C271/10—Esters of carbamic acids having oxygen atoms of carbamate groups bound to acyclic carbon atoms with the nitrogen atoms of the carbamate groups bound to hydrogen atoms or to acyclic carbon atoms
    • C07C271/20—Esters of carbamic acids having oxygen atoms of carbamate groups bound to acyclic carbon atoms with the nitrogen atoms of the carbamate groups bound to hydrogen atoms or to acyclic carbon atoms to carbon atoms of hydrocarbon radicals substituted by nitrogen atoms not being part of nitro or nitroso groups
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07C—ACYCLIC OR CARBOCYCLIC COMPOUNDS
    • C07C2601/00—Systems containing only non-condensed rings
    • C07C2601/02—Systems containing only non-condensed rings with a three-membered ring
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering 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
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/32—Special delivery means, e.g. tissue-specific

Landscapes

  • Chemical & Material Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Epidemiology (AREA)
  • Molecular Biology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Biochemistry (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • Oil, Petroleum & Natural Gas (AREA)
  • Physics & Mathematics (AREA)
  • Immunology (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Oncology (AREA)
  • Communicable Diseases (AREA)
  • Virology (AREA)
  • Nanotechnology (AREA)
  • Optics & Photonics (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
KR1020227011265A 2012-02-24 2013-02-22 트리알킬 양이온성 지질 및 그의 사용 방법 Ceased KR20220045089A (ko)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201261602990P 2012-02-24 2012-02-24
US61/602,990 2012-02-24
KR1020217010379A KR102384791B1 (ko) 2012-02-24 2013-02-22 트리알킬 양이온성 지질 및 그의 사용 방법
PCT/US2013/027469 WO2013126803A1 (en) 2012-02-24 2013-02-22 Trialkyl cationic lipids and methods of use thereof

Related Parent Applications (1)

Application Number Title Priority Date Filing Date
KR1020217010379A Division KR102384791B1 (ko) 2012-02-24 2013-02-22 트리알킬 양이온성 지질 및 그의 사용 방법

Publications (1)

Publication Number Publication Date
KR20220045089A true KR20220045089A (ko) 2022-04-12

Family

ID=47884528

Family Applications (4)

Application Number Title Priority Date Filing Date
KR1020227011265A Ceased KR20220045089A (ko) 2012-02-24 2013-02-22 트리알킬 양이온성 지질 및 그의 사용 방법
KR1020207003227A Active KR102239887B1 (ko) 2012-02-24 2013-02-22 트리알킬 양이온성 지질 및 그의 사용 방법
KR1020217010379A Active KR102384791B1 (ko) 2012-02-24 2013-02-22 트리알킬 양이온성 지질 및 그의 사용 방법
KR1020147026598A Active KR102075810B1 (ko) 2012-02-24 2013-02-22 트리알킬 양이온성 지질 및 그의 사용 방법

Family Applications After (3)

Application Number Title Priority Date Filing Date
KR1020207003227A Active KR102239887B1 (ko) 2012-02-24 2013-02-22 트리알킬 양이온성 지질 및 그의 사용 방법
KR1020217010379A Active KR102384791B1 (ko) 2012-02-24 2013-02-22 트리알킬 양이온성 지질 및 그의 사용 방법
KR1020147026598A Active KR102075810B1 (ko) 2012-02-24 2013-02-22 트리알킬 양이온성 지질 및 그의 사용 방법

Country Status (27)

Country Link
US (4) US9352042B2 (enExample)
EP (3) EP2817287B1 (enExample)
JP (4) JP6275655B2 (enExample)
KR (4) KR20220045089A (enExample)
CN (2) CN104321304A (enExample)
AU (1) AU2013222179B2 (enExample)
BR (1) BR112014020824B1 (enExample)
CA (1) CA2865412C (enExample)
CY (1) CY1121232T1 (enExample)
DK (1) DK2817287T3 (enExample)
ES (2) ES2702874T3 (enExample)
HR (1) HRP20182156T1 (enExample)
HU (1) HUE041494T2 (enExample)
IL (3) IL234266B (enExample)
LT (1) LT2817287T (enExample)
MX (3) MX392812B (enExample)
NZ (1) NZ700075A (enExample)
PH (1) PH12014501901B1 (enExample)
PL (1) PL2817287T3 (enExample)
PT (1) PT2817287T (enExample)
RS (1) RS58077B1 (enExample)
RU (2) RU2718053C2 (enExample)
SG (1) SG11201405157PA (enExample)
SI (1) SI2817287T1 (enExample)
UA (1) UA120026C2 (enExample)
WO (1) WO2013126803A1 (enExample)
ZA (1) ZA201406896B (enExample)

Families Citing this family (232)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3403647A1 (en) 2009-12-01 2018-11-21 Translate Bio, Inc. Delivery of mrna for the augmentation of proteins and enzymes in human genetic diseases
US8853377B2 (en) 2010-11-30 2014-10-07 Shire Human Genetic Therapies, Inc. mRNA for use in treatment of human genetic diseases
CA3198966A1 (en) 2011-06-08 2012-12-13 Translate Bio, Inc. Cleavable lipids
US20140206753A1 (en) 2011-06-08 2014-07-24 Shire Human Genetic Therapies, Inc. Lipid nanoparticle compositions and methods for mrna delivery
WO2013126803A1 (en) 2012-02-24 2013-08-29 Protiva Biotherapeutics Inc. Trialkyl cationic lipids and methods of use thereof
EP2830596B1 (en) 2012-03-29 2020-12-30 Translate Bio, Inc. Lipid-derived neutral nanoparticles
ES2762873T3 (es) 2012-03-29 2020-05-26 Translate Bio Inc Lípidos catiónicos ionizables
EP2859102A4 (en) 2012-06-08 2016-05-11 Shire Human Genetic Therapies NUCLEASE RESISTANT POLYNUCLEOTIDES AND USES THEREOF
MX2014015041A (es) 2012-06-08 2015-06-17 Shire Human Genetic Therapies Administración pulmonar de arnm a células objetivo no pulmonares.
WO2014152211A1 (en) 2013-03-14 2014-09-25 Moderna Therapeutics, Inc. Formulation and delivery of modified nucleoside, nucleotide, and nucleic acid compositions
LT2970456T (lt) 2013-03-14 2021-08-10 Translate Bio, Inc. Būdai ir kompozicijos, skirti mrnr koduojamų antikūnų pristatymui
ES2981185T3 (es) 2013-03-14 2024-10-07 Translate Bio Inc Métodos para la purificación de ARN mensajero
CA2904151C (en) 2013-03-14 2023-09-12 Shire Human Genetic Therapies, Inc. Cftr mrna compositions and related methods and uses
PL4332576T3 (pl) 2013-03-15 2025-09-08 Translate Bio, Inc. Synergistyczne wzmocnienie dostarczania kwasów nukleinowych poprzez formulacje zmieszane
JP6620093B2 (ja) * 2013-07-23 2019-12-11 アービュートゥス バイオファーマ コーポレイションＡｒｂｕｔｕｓ Ｂｉｏｐｈａｒｍａ Ｃｏｒｐｏｒａｔｉｏｎ メッセンジャーｒｎａを送達するための組成物及び方法
US20160194368A1 (en) 2013-09-03 2016-07-07 Moderna Therapeutics, Inc. Circular polynucleotides
EA201690675A1 (ru) 2013-10-03 2016-08-31 Модерна Терапьютикс, Инк. Полинуклеотиды, кодирующие рецептор липопротеинов низкой плотности
BR112016009014B1 (pt) 2013-10-22 2024-02-06 Translate Bio, Inc USO DE COMPOSIÇÃO COMPREENDENDO mRNA PARA DEFICIÊNCIA DE ARGININOSSUCINATO SINTETASE
US20150110857A1 (en) 2013-10-22 2015-04-23 Shire Human Genetic Therapies, Inc. Cns delivery of mrna and uses thereof
MX2016005238A (es) 2013-10-22 2016-08-12 Shire Human Genetic Therapies Formulaciones de lipidos para la administracion de acido ribonucleico mensajero.
WO2015061491A1 (en) 2013-10-22 2015-04-30 Shire Human Genetic Therapies, Inc. Mrna therapy for phenylketonuria
MX373952B (es) 2014-04-25 2020-07-13 Shire Human Genetic Therapies Métodos de purificación de arn mensajero.
CN106659731A (zh) 2014-05-30 2017-05-10 夏尔人类遗传性治疗公司 用于递送核酸的可生物降解脂质
UA121863C2 (uk) 2014-06-24 2020-08-10 Транслейт Байо, Інк. Стереохімічно збагачені композиції для доставки нуклеїнових кислот
HRP20221536T1 (hr) 2014-06-25 2023-02-17 Acuitas Therapeutics Inc. Novi lipidi i formulacije lipidnih nanočestica za isporuku nukleinskih kiselina
EP3164112A1 (en) 2014-07-02 2017-05-10 Shire Human Genetic Therapies, Inc. Encapsulation of messenger rna
WO2016011226A1 (en) 2014-07-16 2016-01-21 Moderna Therapeutics, Inc. Chimeric polynucleotides
US20170210788A1 (en) 2014-07-23 2017-07-27 Modernatx, Inc. Modified polynucleotides for the production of intrabodies
PL3178807T3 (pl) 2014-08-07 2020-08-24 Takeda Pharmaceutical Company Limited Lipid kationowy
US9339029B2 (en) * 2014-09-04 2016-05-17 Preceres Inc. Hydrazinyl lipidoids and uses thereof
WO2016090262A1 (en) 2014-12-05 2016-06-09 Shire Human Genetic Therapies, Inc. Messenger rna therapy for treatment of articular disease
WO2016142449A1 (en) * 2015-03-11 2016-09-15 INSERM (Institut National de la Santé et de la Recherche Médicale) Methods and pharmaceutical compositions for the treatment of filovirus infections
CA2979695C (en) 2015-03-19 2025-02-06 Translate Bio Inc RNA THERAPY FOR POMPE DISEASE
EP3289083A4 (en) 2015-04-27 2018-12-19 The Trustees Of The University Of Pennsylvania Nucleoside-modified rna for inducing an adaptive immune response
CA2990202A1 (en) 2015-06-29 2017-01-05 Acuitas Therapeutics Inc. Lipids and lipid nanoparticle formulations for delivery of nucleic acids
DK3350157T3 (da) 2015-09-17 2022-02-14 Modernatx Inc Forbindelser og sammensætninger til intracellulær afgivelse af terapeutiske midler
CA3001852A1 (en) 2015-10-14 2017-04-20 Translate Bio, Inc. Modification of rna-related enzymes for enhanced production
MA46316A (fr) 2015-10-22 2021-03-24 Modernatx Inc Vaccin contre le cytomégalovirus humain
PT3368507T (pt) 2015-10-28 2023-02-07 Acuitas Therapeutics Inc Novos lípidos e formulações de nanopartículas lipídicas para distribuição de ácidos nucleicos
AU2016377681B2 (en) 2015-12-22 2021-05-13 Modernatx, Inc. Compounds and compositions for intracellular delivery of agents
EP4039699A1 (en) 2015-12-23 2022-08-10 ModernaTX, Inc. Methods of using ox40 ligand encoding polynucleotides
US20190241658A1 (en) 2016-01-10 2019-08-08 Modernatx, Inc. Therapeutic mRNAs encoding anti CTLA-4 antibodies
JP7150608B6 (ja) 2016-04-08 2022-11-11 トランスレイト バイオ， インコーポレイテッド 多量体コード核酸及びその使用
WO2017201342A1 (en) 2016-05-18 2017-11-23 Modernatx, Inc. Polynucleotides encoding jagged1 for the treatment of alagille syndrome
JP2019522047A (ja) 2016-06-13 2019-08-08 トランスレイト バイオ， インコーポレイテッド オルニチントランスカルバミラーゼ欠損症治療のためのメッセンジャーｒｎａ療法
EP3478838A1 (en) 2016-06-30 2019-05-08 Arbutus Biopharma Corporation Compositions and methods for delivering messenger rna
US12491261B2 (en) 2016-10-26 2025-12-09 Acuitas Therapeutics, Inc. Lipid nanoparticle formulations
US11583504B2 (en) 2016-11-08 2023-02-21 Modernatx, Inc. Stabilized formulations of lipid nanoparticles
US11241490B2 (en) 2017-01-11 2022-02-08 The Trustees Of The University Of Pennsylvania Nucleoside-modified RNA for inducing an immune response against zika virus
MX2019010155A (es) 2017-02-27 2020-12-10 Translate Bio Inc Arnm de cftr optimizado por codón novedoso.
CA3055653A1 (en) 2017-03-15 2018-09-20 Modernatx, Inc. Lipid nanoparticle formulation
AU2018234692B2 (en) 2017-03-15 2022-06-02 Modernatx, Inc. Compounds and compositions for intracellular delivery of therapeutic agents
US11752206B2 (en) 2017-03-15 2023-09-12 Modernatx, Inc. Herpes simplex virus vaccine
US11739335B2 (en) 2017-03-24 2023-08-29 CureVac SE Nucleic acids encoding CRISPR-associated proteins and uses thereof
US11357856B2 (en) 2017-04-13 2022-06-14 Acuitas Therapeutics, Inc. Lipids for delivery of active agents
US11820728B2 (en) 2017-04-28 2023-11-21 Acuitas Therapeutics, Inc. Carbonyl lipids and lipid nanoparticle formulations for delivery of nucleic acids
FR3066115B1 (fr) * 2017-05-10 2019-06-28 Universite de Bordeaux Comprimes de vecteurs d'acides nucleiques
MA49138A (fr) 2017-05-16 2020-03-25 Translate Bio Inc Traitement de la fibrose kystique par administration d'arnm à codons optimisés codant pour la cftr
WO2018213731A1 (en) 2017-05-18 2018-11-22 Modernatx, Inc. Polynucleotides encoding tethered interleukin-12 (il12) polypeptides and uses thereof
EP3638292A1 (en) 2017-06-14 2020-04-22 ModernaTX, Inc. Polynucleotides encoding coagulation factor viii
WO2018232120A1 (en) 2017-06-14 2018-12-20 Modernatx, Inc. Compounds and compositions for intracellular delivery of agents
US11786607B2 (en) 2017-06-15 2023-10-17 Modernatx, Inc. RNA formulations
US20210228738A1 (en) 2017-07-17 2021-07-29 INSERM (Institut National de la Santé et de la Recherche Médicale) Compositions and methods for increasing or enhancing transduction of gene therapy vectors and for removing or reducing immunoglobulins
EP3668833A1 (en) 2017-08-16 2020-06-24 Acuitas Therapeutics, Inc. Lipids for use in lipid nanoparticle formulations
WO2019036000A1 (en) 2017-08-17 2019-02-21 Acuitas Therapeutics, Inc. LIPIDS FOR USE IN LIPID NANOPARTICLE FORMULATIONS
WO2019036030A1 (en) 2017-08-17 2019-02-21 Acuitas Therapeutics, Inc. LIPIDS FOR USE IN LIPID NANOPARTICLE FORMULATIONS
US11542225B2 (en) 2017-08-17 2023-01-03 Acuitas Therapeutics, Inc. Lipids for use in lipid nanoparticle formulations
US11866696B2 (en) 2017-08-18 2024-01-09 Modernatx, Inc. Analytical HPLC methods
MX2020002348A (es) 2017-08-31 2020-10-08 Modernatx Inc Métodos de elaboración de nanopartículas lipídicas.
CA3084061A1 (en) 2017-12-20 2019-06-27 Translate Bio, Inc. Improved composition and methods for treatment of ornithine transcarbamylase deficiency
TWI894123B (zh) * 2017-12-27 2025-08-21 日商武田藥品工業有限公司 含核酸之脂質奈米粒子及其用途
TWI837161B (zh) 2018-08-10 2024-04-01 日商武田藥品工業股份有限公司 陽離子性脂質
WO2020041793A1 (en) 2018-08-24 2020-02-27 Translate Bio, Inc. Methods for purification of messenger rna
CA3113025A1 (en) 2018-09-19 2020-03-26 Modernatx, Inc. Peg lipids and uses thereof
EP4509118A3 (en) 2018-09-19 2025-05-14 ModernaTX, Inc. High-purity peg lipids and uses thereof
US12263248B2 (en) 2018-09-19 2025-04-01 Modernatx, Inc. Compounds and compositions for intracellular delivery of therapeutic agents
US12090235B2 (en) 2018-09-20 2024-09-17 Modernatx, Inc. Preparation of lipid nanoparticles and methods of administration thereof
EP3852911B1 (en) 2018-09-21 2025-01-22 Acuitas Therapeutics, Inc. Systems and methods for manufacturing lipid nanoparticles and liposomes
KR20210093232A (ko) 2018-10-09 2021-07-27 더 유니버시티 오브 브리티시 콜롬비아 유기용매와 세제가 없는 형질감염 적격 소포를 포함하는 조성물과 시스템 및 관련 방법
MX2021004357A (es) 2018-10-18 2021-05-31 Takeda Pharmaceuticals Co Metodo para la activacion y proliferacion de las celulas t.
EP3877521A4 (en) * 2018-11-09 2022-07-27 Arbutus Biopharma Corporation Lipid nanoparticle formulations
CN113728101A (zh) * 2018-11-09 2021-11-30 阿布特斯生物制药公司 脂质纳米颗粒制剂
AU2019384557B2 (en) 2018-11-21 2025-07-17 Translate Bio, Inc. Treatment of cystic fibrosis by delivery of nebulized mRNA encoding CFTR
MA55321A (fr) 2019-03-15 2022-01-19 Modernatx Inc Vaccins à base d'arn contre le vih
EP3986915A1 (en) 2019-06-18 2022-04-27 Janssen Sciences Ireland Unlimited Company Recombinant interleukin 12 construct and uses thereof
WO2020255011A1 (en) 2019-06-18 2020-12-24 Janssen Sciences Ireland Unlimited Company Combination of hepatitis b virus (hbv) vaccines and anti-pd-1 or anti-pd-l1 antibody
JP2022537324A (ja) 2019-06-18 2022-08-25 ヤンセン・サイエンシズ・アイルランド・アンリミテッド・カンパニー Ｂ型肝炎ウイルス（ｈｂｖ）ワクチンおよび抗ｐｄ－１抗体の組合せ
WO2020255010A1 (en) 2019-06-18 2020-12-24 Janssen Sciences Ireland Unlimited Company Combination of recombinant interleukin 12 construct and hepatitis b virus (hbv) vaccines
WO2020255007A1 (en) 2019-06-18 2020-12-24 Janssen Sciences Ireland Unlimited Company Combination of hepatitis b virus (hbv) vaccines and hbv-targeting rnai
US20220305108A1 (en) 2019-06-20 2022-09-29 Janssen Sciences Ireland Unlimited Company Lipid nanoparticle or liposome delivery of hepatitis b virus (hbv) vaccines
CN115279743B (zh) * 2019-06-29 2024-05-14 精密纳米系统无限责任公司 用于核酸递送的可离子化脂质
EP3999642A1 (en) 2019-07-19 2022-05-25 Flagship Pioneering Innovations VI, LLC Recombinase compositions and methods of use
KR20220103923A (ko) * 2019-09-19 2022-07-25 모더나티엑스, 인크. 치료제의 세포내 전달을 위한 신규한 헤드기 지질 화합물 및 조성물
CA3154618A1 (en) 2019-09-19 2021-03-25 Modernatx, Inc. Branched tail lipid compounds and compositions for intracellular delivery of therapeutic agents
CN114391040A (zh) 2019-09-23 2022-04-22 欧米茄治疗公司 用于调节载脂蛋白b(apob)基因表达的组合物和方法
US11987791B2 (en) 2019-09-23 2024-05-21 Omega Therapeutics, Inc. Compositions and methods for modulating hepatocyte nuclear factor 4-alpha (HNF4α) gene expression
AU2020371697A1 (en) * 2019-10-22 2022-05-26 Genevant Sciences Gmbh Ornithine transcarbamylase (OTC) constructs and methods of using the same
CN116096886A (zh) 2020-03-11 2023-05-09 欧米茄治疗公司 用于调节叉头框p3(foxp3)基因表达的组合物和方法
JP7709981B2 (ja) * 2020-03-17 2025-07-17 ジェネヴァント サイエンシズ ゲーエムベーハー 肝星細胞への治療薬の脂質ナノ粒子送達のためのカチオン性脂質
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.
JP2023520764A (ja) 2020-03-24 2023-05-19 ジェネレーション バイオ カンパニー 第ｉｘ因子治療薬を発現するための非ウイルス性ｄｎａベクター及びその使用
IL298362A (en) 2020-05-20 2023-01-01 Flagship Pioneering Innovations Vi Llc Coronavirus antigen compositions and their uses
AU2021275213A1 (en) 2020-05-20 2023-02-02 Flagship Pioneering Innovations Vi, Llc Immunogenic compositions and uses thereof
AU2021281453A1 (en) 2020-05-29 2022-11-17 Flagship Pioneering Innovations Vi, Llc. Trem compositions and methods relating thereto
CA3180101A1 (en) 2020-05-29 2021-12-02 Flagship Pioneering Innovations Vi, Llc Trem compositions and methods relating thereto
PH12023550030A1 (en) 2020-07-08 2024-03-11 Janssen Sciences Ireland Unlimited Co Rna replicon vaccines against hbv
MX2023000614A (es) 2020-07-16 2023-02-13 Acuitas Therapeutics Inc Lipidos cationicos para usarse en nanoparticulas lipidicas.
WO2022023284A1 (en) 2020-07-27 2022-02-03 Anjarium Biosciences Ag Compositions of dna molecules, methods of making therefor, and methods of use thereof
CA3189854A1 (en) 2020-08-06 2022-02-10 Modernatx, Inc. Compositions for the delivery of payload molecules to airway epithelium
WO2022051629A1 (en) 2020-09-03 2022-03-10 Flagship Pioneering Innovations Vi, Llc Immunogenic compositions and uses thereof
JP2023545420A (ja) * 2020-10-12 2023-10-30 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア 高分子を細胞に送達するためのエンドソームエスケープドメイン
US20220218622A1 (en) 2020-10-14 2022-07-14 George Mason Research Foundation, Inc. Ionizable lipids and methods of manufacture and use thereof
US20240148663A1 (en) 2020-12-18 2024-05-09 Genevant Sciences Gmbh Peg lipids and lipid nanoparticles
TW202245809A (zh) 2020-12-18 2022-12-01 美商詹森藥物公司 用於治療b型肝炎病毒感染之組合療法
JP2024501288A (ja) 2020-12-23 2024-01-11 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 修飾ｔｒｅｍの組成物及びその使用
EP4267740A1 (en) 2020-12-28 2023-11-01 Arcturus Therapeutics, Inc. Transcription activator-like effector nucleases (talens) targeting hbv
EP4297722A1 (en) 2021-02-26 2024-01-03 Ethris GmbH Formulations for aerosol formation and aerosols for the delivery of nucleic acid
US11952461B2 (en) 2021-03-22 2024-04-09 Sunbio, Inc. Siloxy polyethylene glycol and derivatives thereof
CA3214085A1 (en) 2021-03-31 2022-10-06 Darby Rye Schmidt Thanotransmission polypeptides and their use in treating cancer
CN115197079A (zh) * 2021-04-08 2022-10-18 厦门赛诺邦格生物科技股份有限公司 一种聚乙二醇化脂质及其修饰的脂质体、含该脂质体的药物组合物及其制剂和应用
AU2022260111A1 (en) 2021-04-20 2023-11-30 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
AU2022264509A1 (en) 2021-04-27 2023-12-14 Generation Bio Co. Non-viral dna vectors expressing therapeutic antibodies and uses thereof
EP4329884A1 (en) 2021-04-27 2024-03-06 Generation Bio Co. Non-viral dna vectors expressing anti-coronavirus antibodies and uses thereof
WO2022229903A1 (en) 2021-04-28 2022-11-03 Genevant Sciences Gmbh Mrna delivery constructs and methods of using the same
CN112979483B (zh) * 2021-05-14 2021-08-06 苏州艾博生物科技有限公司 一种阳离子脂质化合物、包含其的组合物及应用
WO2023283359A2 (en) 2021-07-07 2023-01-12 Omega Therapeutics, Inc. Compositions and methods for modulating secreted frizzled receptor protein 1 (sfrp1) gene expression
JP2024528697A (ja) 2021-07-20 2024-07-30 エイジーエス・セラピューティクス・ソシエテ・パール・アクシオン・サンプリフィエ 微細藻類由来の細胞外小胞、その調製および使用
EP4377457A1 (en) 2021-07-26 2024-06-05 Flagship Pioneering Innovations VI, LLC Trem compositions and uses thereof
EP4271818B1 (en) 2021-09-17 2024-07-31 Flagship Pioneering Innovations VI, LLC Compositions and methods for producing circular polyribonucleotides
TW202322826A (zh) 2021-10-18 2023-06-16 美商旗艦先鋒創新有限責任公司 用於純化多核糖核苷酸之組成物及方法
WO2023081526A1 (en) 2021-11-08 2023-05-11 Orna Therapeutics, Inc. Lipid nanoparticle compositions for delivering circular polynucleotides
US20250049727A1 (en) 2021-11-12 2025-02-13 Modernatx, Inc. Compositions for the delivery of payload molecules to airway epithelium
CA3238370A1 (en) 2021-11-24 2023-06-01 Flagship Pioneering Innovations Vi, Llc Varicella-zoster virus immunogen compositions and their uses
MX2024006440A (es) 2021-11-24 2024-08-19 Flagship Pioneering Innovations Vi Llc Composiciones inmunogenicas y sus usos.
US20250352638A1 (en) 2021-11-24 2025-11-20 Flagship Pioneering Innovations Vi, Llc Coronavirus immunogen compositions and their uses
IL313486A (en) 2021-12-16 2024-08-01 Acuitas Therapeutics Inc Lipids for use in lipid nanoparticle formulations
WO2023115013A1 (en) 2021-12-17 2023-06-22 Flagship Pioneering Innovations Vi, Llc Methods for enrichment of circular rna under denaturing conditions
JP2025500360A (ja) 2021-12-22 2025-01-09 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 ポリリボヌクレオチドを精製するための組成物及び方法
IL313714A (en) 2021-12-23 2024-08-01 Flagship Pioneering Innovations Vi Llc Cyclic polyribonucleotides encoding antifusogenic polypeptides
CN114044741B (zh) * 2022-01-13 2022-04-15 北京悦康科创医药科技股份有限公司 一种阳离子脂质化合物、包含其的组合物及用途
WO2023135273A2 (en) 2022-01-14 2023-07-20 Anjarium Biosciences Ag Compositions of dna molecules encoding factor viii, methods of making thereof, and methods of use thereof
WO2023144127A1 (en) 2022-01-31 2023-08-03 Ags Therapeutics Sas Extracellular vesicles from microalgae, their biodistribution upon administration, and uses
EP4475882A1 (en) 2022-02-09 2024-12-18 ModernaTX, Inc. Mucosal administration methods and formulations
US20250327041A1 (en) 2022-02-24 2025-10-23 Io Biotech Aps Nucleotide delivery of cancer therapy
US20250250227A1 (en) * 2022-02-28 2025-08-07 Shenzhen Shenxin Biotechnology Co., Ltd. Amino lipid compound, preparation method therefor, composition thereof and use thereof
KR20240161965A (ko) 2022-03-14 2024-11-13 제너레이션 바이오 컴퍼니 이종 프라임 부스트 백신 조성물 및 사용 방법
CN114380724B (zh) * 2022-03-23 2022-05-24 深圳市瑞吉生物科技有限公司 用于递送核酸的阳离子脂质化合物和组合物及用途
CN119072464A (zh) 2022-03-25 2024-12-03 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
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
CN114685784B (zh) * 2022-04-26 2023-09-15 北京清科胜因生物科技有限公司 一种用于核酸递送的聚(2-噁唑啉)脂质与脂质纳米颗粒及应用
CN115626983A (zh) * 2022-04-27 2023-01-20 北京清科胜因生物科技有限公司 一种聚(2-噁唑啉)脂质及脂质纳米颗粒
WO2023220083A1 (en) 2022-05-09 2023-11-16 Flagship Pioneering Innovations Vi, Llc Trem compositions and methods of use for treating proliferative disorders
WO2023218420A1 (en) 2022-05-13 2023-11-16 Janssen Pharmaceuticals, Inc. Mrna compositions for inducing latent hiv-1 reversal
EP4522753A2 (en) 2022-05-13 2025-03-19 Flagship Pioneering Innovations VII, LLC Double stranded dna compositions and related methods
WO2023233290A1 (en) 2022-05-31 2023-12-07 Janssen Sciences Ireland Unlimited Company Rnai agents targeting pd-l1
CN114940756B (zh) * 2022-06-02 2023-11-07 北京清科胜因生物科技有限公司 一种聚（2-噁唑啉）脂质与脂质纳米颗粒及应用
WO2023232976A1 (en) 2022-06-03 2023-12-07 Ags Therapeutics Sas Extracellular vesicles from genetically-modified microalgae containing endogenously-loaded cargo, their preparation, and uses
EP4536197A1 (en) 2022-06-07 2025-04-16 Generation Bio Co. Lipid nanoparticle compositions and uses thereof
CN117263818A (zh) * 2022-06-14 2023-12-22 杭州高田生物医药有限公司 阳离子脂质化合物及其制备方法和应用
EP4544043A1 (en) 2022-06-22 2025-04-30 Flagship Pioneering Innovations VI, LLC Compositions of modified trems and uses thereof
CN120129693A (zh) 2022-08-01 2025-06-10 旗舰创业创新第七有限责任公司 免疫调节蛋白及相关方法
EP4569112A1 (en) 2022-08-12 2025-06-18 Remix Therapeutics Inc. Methods and compositions for modulating splicing at alternative splice sites
WO2024040222A1 (en) 2022-08-19 2024-02-22 Generation Bio Co. Cleavable closed-ended dna (cedna) and methods of use thereof
JP2025529925A (ja) 2022-08-26 2025-09-09 エスリス ゲーエムベーハー 安定な脂質またはリピドイドナノ粒子懸濁液
EP4327829A1 (en) 2022-08-26 2024-02-28 Ethris GmbH Stabilization of lipid or lipidoid nanoparticle suspensions
EP4581012A2 (en) 2022-08-31 2025-07-09 Sail Biomedicines, Inc. Novel ionizable lipids and lipid nanoparticles and methods of using the same
WO2024077191A1 (en) 2022-10-05 2024-04-11 Flagship Pioneering Innovations V, Inc. Nucleic acid molecules encoding trif and additionalpolypeptides and their use in treating cancer
WO2024088808A1 (en) 2022-10-24 2024-05-02 Ags Therapeutics Sas Extracellular vesicles from microalgae, their biodistribution upon intranasal administration, and uses thereof
AU2023372397A1 (en) 2022-10-31 2025-05-08 Flagship Pioneering Innovations Vi, Llc Compositions and methods for purifying polyribonucleotides
EP4615465A1 (en) 2022-11-08 2025-09-17 Flagship Pioneering Innovations VI, LLC Compositions and methods for producing circular polyribonucleotides
TW202425959A (zh) 2022-11-08 2024-07-01 美商歐納醫療公司 遞送多核苷酸的脂質及奈米顆粒組合物
WO2024102762A1 (en) 2022-11-08 2024-05-16 Orna Therapeutics, Inc. Lipids and lipid nanoparticle compositions for delivering polynucleotides
TW202428289A (zh) 2022-11-08 2024-07-16 美商歐納醫療公司 環狀rna組合物
AU2023406303A1 (en) 2022-12-01 2025-05-29 Generation Bio Co. Lipid nanoparticles comprising nucleic acids, ionizable lipids, sterols, lipid anchored polymers and helper lipids, their uses
EP4626401A1 (en) 2022-12-01 2025-10-08 Generation Bio Co. Stealth lipid nanoparticle compositions for cell targeting
EP4626402A1 (en) 2022-12-01 2025-10-08 Generation Bio Co. Lipid nanoparticles comprising nucleic acids and lipid-anchored polymers
AU2023406321A1 (en) 2022-12-01 2025-05-29 Generation Bio Co. Novel polyglycerol-conjugated lipids and lipid nanoparticle compositions comprising the same
EP4630561A1 (en) 2022-12-09 2025-10-15 Takeda Pharmaceutical Company Limited Modified immunomodulators
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
WO2024151673A2 (en) 2023-01-09 2024-07-18 President And Fellows Of Harvard College Recombinant nucleic acid molecules and their use in wound healing
TW202438673A (zh) 2023-01-09 2024-10-01 美商旗艦先鋒創新有限責任（Ｖｉｉ）公司 疫苗及相關方法
US20240252520A1 (en) 2023-01-09 2024-08-01 Beth Israel Deaconess Medical Center, Inc. Therapeutic agents and their use for treating chronic wounds
WO2024151687A1 (en) 2023-01-09 2024-07-18 Flagship Pioneering Innovations V, Inc. Genetic switches and their use in treating cancer
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
US20240293318A1 (en) 2023-02-13 2024-09-05 Flagship Pioneering Innovations Vii, Llc Cleavable linker-containing ionizable lipids and lipid carriers for therapeutic compositions
IL322201A (en) 2023-02-17 2025-09-01 Flagship Pioneering Innovations Vii Llc DNA structures containing modified uracil
US20240293582A1 (en) 2023-02-17 2024-09-05 Flagship Pioneering Innovations Vii, Llc Dna compositions comprising modified cytosine
WO2024192422A1 (en) 2023-03-15 2024-09-19 Flagship Pioneering Innovations Vi, Llc Immunogenic compositions and uses thereof
KR20250162610A (ko) 2023-03-15 2025-11-18 플래그쉽 파이어니어링 이노베이션스 브이아이, 엘엘씨 폴리리보뉴클레오티드를 포함하는 조성물 및 이의 용도
WO2024205657A2 (en) 2023-03-29 2024-10-03 Orna Therapeutics, Inc. Lipids and lipid nanoparticle compositions for delivering polynucleotides
WO2024200826A1 (en) 2023-03-30 2024-10-03 Ose Immunotherapeutics Lipid-based nanoparticle targeted at activated immune cells for the expression of immune cell inhibiting molecule and use thereof
WO2024200823A1 (en) 2023-03-30 2024-10-03 Ose Immunotherapeutics Lipid-based nanoparticle targeted at activated immune cells for the expression of immune cell enhancing molecule and use thereof
AR132276A1 (es) 2023-04-07 2025-06-11 Takeda Pharmaceuticals Co Complejo de conjugación
AU2024252590A1 (en) 2023-04-12 2025-10-23 Flagship Pioneering Innovations Vi, Llc Trems for use in correction of missense mutations
AU2024255972A1 (en) 2023-04-12 2025-10-23 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
WO2024233308A2 (en) 2023-05-05 2024-11-14 Orna Therapeutics, Inc. Circular rna compositions and methods
WO2024236361A1 (en) 2023-05-15 2024-11-21 Takeda Pharmaceutical Company Limited Compositions and methods for delivery of nucleic acids to cells
WO2024236504A1 (en) 2023-05-15 2024-11-21 Takeda Pharmaceutical Company Limited Sequences and methods for delivery of dna and rna
WO2024243438A2 (en) 2023-05-23 2024-11-28 Omega Therapeutics, Inc. Compositions and methods for reducing cxcl9, cxcl10, and cxcl11 gene expression
WO2024240962A1 (en) 2023-05-25 2024-11-28 Ethris Gmbh Gm-csf-encoding nucleic acids, pharmaceutical compositions, methods and uses thereof
WO2024251108A1 (zh) * 2023-06-07 2024-12-12 北京因诺惟康医药科技有限公司 可电离脂质化合物、包含其的脂质载体及应用
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
WO2025024486A2 (en) 2023-07-25 2025-01-30 Flagship Pioneering Innovations Vii, Llc Cas endonucleases and related methods
WO2025027116A1 (en) 2023-08-01 2025-02-06 Institut Curie Nanoparticles comprising nucleic acid sequences encoding cyclic gmp-amp synthase
WO2025042786A1 (en) 2023-08-18 2025-02-27 Flagship Pioneering Innovations Vi, Llc Compositions comprising circular polyribonucleotides and uses thereof
WO2025045767A1 (en) 2023-08-25 2025-03-06 Ethris Gmbh Stabilized lipid and lipidoid nanoparticle formulations with specific surfactant properties for enhanced pharmaceutical applications
WO2025049690A1 (en) 2023-08-29 2025-03-06 Orna Therapeutics, Inc. Circular polyethylene glycol lipids
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
WO2025052180A2 (en) 2023-09-07 2025-03-13 Axelyf ehf. Lipids and lipid nanoparticles
WO2025064475A2 (en) 2023-09-18 2025-03-27 Flagship Pioneering Innovations Vii, Llc Ionizable lipidoid compositions and therapeutic uses thereof
WO2025072331A1 (en) 2023-09-26 2025-04-03 Flagship Pioneering Innovations Vii, Llc Cas nucleases and related methods
WO2025096807A2 (en) 2023-10-31 2025-05-08 Flagship Pioneering Innovations Vii, Llc Novel therapeutic dna forms
CN117482066B (zh) * 2023-11-02 2024-08-13 深圳市易瑞生物技术股份有限公司 脂质组合物和用于脂质组合物的化合物
WO2025101501A1 (en) 2023-11-07 2025-05-15 Orna Therapeutics, Inc. Circular rna compositions
TW202535835A (zh) 2023-11-14 2025-09-16 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
TW202523695A (zh) 2023-11-22 2025-06-16 美商旗艦先鋒創新有限責任（Ｖｉｉ）公司 用於治療非酒精性脂肪性肝病之方法及組成物
WO2025117877A2 (en) 2023-12-01 2025-06-05 Flagship Pioneering Innovations Vii, Llc Cas nucleases and related methods
WO2025129128A1 (en) 2023-12-15 2025-06-19 Vivasor, Inc. Compositions and methods for non-viral delivery of therapeutic compounds
WO2025133115A1 (en) 2023-12-21 2025-06-26 Ose Immunotherapeutics Lipid-based nanoparticles comprising il-35
WO2025133951A1 (en) 2023-12-21 2025-06-26 Genevant Sciences Gmbh Ionizable lipids suitable for lipid nanoparticles
US20250375499A1 (en) 2024-01-26 2025-12-11 Flagship Pioneering Innovations Vii, Llc Immunoreceptor inhibitory proteins and related methods
WO2025176843A1 (en) 2024-02-21 2025-08-28 Ags Therapeutics Sas Microalgae extracellular vesicle based gene therapy vectors (mev-gtvs), their preparation, and uses thereof
WO2025194019A1 (en) 2024-03-14 2025-09-18 Flagship Pioneering Innovations Vii, Llc Methods for treating liver fibrosis and non-alcoholic fatty liver disease
WO2025210521A1 (en) 2024-04-04 2025-10-09 Genevant Sciences Gmbh Lipid nanoparticles for stimulating t cells
WO2025210520A1 (en) 2024-04-04 2025-10-09 Genevant Sciences Gmbh Lipid nanoparticles for inducing an immunological response
WO2025217275A2 (en) 2024-04-10 2025-10-16 Flagship Pioneering Innovations Vii, Llc Immune cell targeted compositions and related methods
US20250353881A1 (en) 2024-05-16 2025-11-20 Flagship Pioneering Innovations Vii, Llc Immunoreceptor inhibitory proteins and related methods
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

Family Cites Families (147)

* 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 (enExample) 1977-08-05 1981-07-15 Battelle Memorial Institute
US4235871A (en) 1978-02-24 1980-11-25 Papahadjopoulos Demetrios P Method of encapsulating biologically active materials in lipid vesicles
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
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
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
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
US5725871A (en) 1989-08-18 1998-03-10 Danbiosyst Uk Limited Drug delivery compositions comprising lysophosphoglycerolipid
WO1991003162A1 (en) 1989-08-31 1991-03-21 City Of Hope Chimeric dna-rna catalytic sequences
US5286634A (en) 1989-09-28 1994-02-15 Stadler Joan K Synergistic method for host cell transformation
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
US6365730B1 (en) 1990-06-19 2002-04-02 Gene Shears Pty. Limited DNA-Armed ribozymes and minizymes
US5068120A (en) * 1990-08-01 1991-11-26 Nabisco Brands, Inc. Amine ester derivatives as low calorie fat mimetics
US5789573A (en) 1990-08-14 1998-08-04 Isis Pharmaceuticals, Inc. Antisense inhibition of ICAM-1, E-selectin, and CMV IE1/IE2
DE69123979T2 (de) 1990-10-12 1997-04-30 Max Planck Gesellschaft Abgeänderte ribozyme
DE4216134A1 (de) 1991-06-20 1992-12-24 Europ Lab Molekularbiolog Synthetische katalytische oligonukleotidstrukturen
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
US5652094A (en) 1992-01-31 1997-07-29 University Of Montreal Nucleozymes
AU687736B2 (en) 1992-05-11 1998-03-05 Ribozyme Pharmaceuticals, Inc. Method and reagent for inhibiting viral replication
DE69312487T2 (de) 1992-05-18 1997-11-27 Minnesota Mining & Mfg Einrichtung zur transmucosalen wirkstoffabgabe
US5792451A (en) 1994-03-02 1998-08-11 Emisphere Technologies, Inc. Oral drug delivery compositions and methods
EP0786522A2 (en) 1992-07-17 1997-07-30 Ribozyme Pharmaceuticals, Inc. Enzymatic RNA molecules for treatment of stenotic conditions
IL108367A0 (en) 1993-01-27 1994-04-12 Hektoen Inst For Medical Resea Antisense polynzcleotide inhibition of human growth factor-sensitive cancer 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
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
DE69527206T2 (de) 1994-09-30 2003-02-27 Inex Pharmaceuticals Corp., Vancouver Mittel zum einbringen polyanionischer materialien in zellen
US5753613A (en) 1994-09-30 1998-05-19 Inex Pharmaceuticals Corporation Compositions for the introduction of polyanionic materials into cells
US5885613A (en) 1994-09-30 1999-03-23 The University Of British Columbia Bilayer stabilizing components and their use in forming programmable fusogenic liposomes
AU3889595A (en) 1994-10-05 1996-05-02 Amgen, Inc. Method for inhibiting smooth muscle cell proliferation and oligonucleotides for use therein
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
US5646241A (en) * 1995-05-12 1997-07-08 Quantum Materials, Inc. Bleed resistant cyanate ester-containing compositions
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
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
US5981501A (en) 1995-06-07 1999-11-09 Inex Pharmaceuticals Corp. Methods for encapsulating plasmids in lipid bilayers
US5747470A (en) 1995-06-07 1998-05-05 Gen-Probe Incorporated Method for inhibiting cellular proliferation using antisense oligonucleotides to gp130 mRNA
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
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
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
US6111085A (en) 1996-09-13 2000-08-29 Isis Pharmaceuticals, Inc. Carbamate-derivatized nucleosides and oligonucleosides
US5739119A (en) 1996-11-15 1998-04-14 Galli; Rachel L. Antisense oligonucleotides specific for the muscarinic type 2 acetylcholine receptor MRNA
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
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
US6320017B1 (en) 1997-12-23 2001-11-20 Inex Pharmaceuticals Corp. Polyamide oligomers
EP1096921B1 (en) 1998-07-20 2003-04-16 Protiva Biotherapeutics Inc. Liposomal encapsulated nucleic acid-complexes
US6852334B1 (en) 1999-04-20 2005-02-08 The University Of British Columbia Cationic peg-lipids and methods of use
AU783647B2 (en) 1999-04-20 2005-11-17 University Of British Columbia, The Cationic peg-lipids and methods of use
MXPA02002107A (es) 1999-08-27 2003-08-20 Inex Pharmaceuticals Corp Composiciones para estimular la secrecion de citoquina e inducir una respuesta inmune.
US7491805B2 (en) 2001-05-18 2009-02-17 Sirna Therapeutics, Inc. Conjugates and compositions for cellular delivery
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
US7833992B2 (en) 2001-05-18 2010-11-16 Merck Sharpe & Dohme Conjugates and compositions for cellular delivery
US7189705B2 (en) 2000-04-20 2007-03-13 The University Of British Columbia Methods of enhancing SPLP-mediated transfection using endosomal membrane destabilizers
WO2002069369A2 (en) 2000-12-08 2002-09-06 Coley Pharmaceutical Gmbh Cpg-like nucleic acids and methods of use thereof
US7053150B2 (en) 2000-12-18 2006-05-30 Nektar Therapeutics Al, Corporation Segmented polymers and their conjugates
TW593427B (en) 2000-12-18 2004-06-21 Nektar Therapeutics Al Corp Synthesis of high molecular weight non-peptidic polymer derivatives
WO2002087541A1 (en) 2001-04-30 2002-11-07 Protiva Biotherapeutics Inc. Lipid-based formulations for gene transfer
US20030130186A1 (en) 2001-07-20 2003-07-10 Chandra Vargeese 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)
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)
US7109165B2 (en) 2001-05-18 2006-09-19 Sirna Therapeutics, Inc. Conjugates and compositions for cellular delivery
GB0118517D0 (en) * 2001-07-30 2001-09-19 Mitsubishi Tokyo Pharm Inc Compound
DE10163098B4 (de) 2001-10-12 2005-06-02 Alnylam Europe Ag Verfahren zur Hemmung der Replikation von Viren
AU2003207708A1 (en) 2002-02-20 2003-09-09 Sirna Therapeutics, Inc. Rna interference mediated inhibition of map kinase genes
US20050107316A1 (en) 2002-02-22 2005-05-19 Klaus Strebhardt Agent for inhibiting development or progress of proliferative diseases and especially cancer diseases and pharmaceutical composition containing said agent
US7901708B2 (en) 2002-06-28 2011-03-08 Protiva Biotherapeutics, Inc. Liposomal apparatus and manufacturing methods
CA2884658A1 (en) 2002-07-26 2004-02-05 Novartis Vaccines And Diagnostics, Inc. Modified small interfering rna molecules and methods of use
WO2006006948A2 (en) 2002-11-14 2006-01-19 Dharmacon, Inc. METHODS AND COMPOSITIONS FOR SELECTING siRNA OF IMPROVED FUNCTIONALITY
DK2284266T3 (da) 2002-11-14 2014-01-13 Thermo Fisher Scient Biosciences Inc sIRNA-MOLEKYLE MOD TP53
US20040167090A1 (en) 2003-02-21 2004-08-26 Monahan Sean D. Covalent modification of RNA for in vitro and in vivo delivery
US20040185559A1 (en) 2003-03-21 2004-09-23 Isis Pharmaceuticals Inc. Modulation of diacylglycerol acyltransferase 1 expression
EP1608735A4 (en) 2003-04-03 2008-11-05 Alnylam Pharmaceuticals RNAI CONJUGATES
CA2521464C (en) 2003-04-09 2013-02-05 Alnylam Pharmaceuticals, Inc. Irna conjugates
ES2702942T3 (es) 2003-04-17 2019-03-06 Alnylam Pharmaceuticals Inc Agentes de ARNi modificados
SG190613A1 (en) 2003-07-16 2013-06-28 Protiva Biotherapeutics Inc Lipid encapsulated interfering rna
US7825235B2 (en) 2003-08-18 2010-11-02 Isis Pharmaceuticals, Inc. Modulation of diacylglycerol acyltransferase 2 expression
EP1664316B1 (en) * 2003-09-15 2012-08-29 Protiva Biotherapeutics Inc. Polyethyleneglycol-modified lipid compounds and uses thereof
JP5243789B2 (ja) 2004-03-15 2013-07-24 シティ・オブ・ホープ 二本鎖ｒｎａによる遺伝子発現の特異的阻害のための方法及び組成物
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
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
AU2005252273B2 (en) 2004-06-07 2011-04-28 Arbutus Biopharma Corporation Lipid encapsulated interfering RNA
JP2008512500A (ja) 2004-09-10 2008-04-24 ソマジェニックス インコーポレーティッド ウィルス遺伝子発現を効率的に阻害する低分子干渉ｒｎａおよびその使用方法
AU2005289588B2 (en) 2004-09-24 2011-12-22 Alnylam Pharmaceuticals, Inc. RNAi modulation of ApoB and uses thereof
WO2006053430A1 (en) 2004-11-17 2006-05-26 Protiva Biotherapeutics, Inc. Sirna silencing of apolipoprotein b
WO2007086883A2 (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
JP5639338B2 (ja) 2005-07-27 2014-12-10 プロチバ バイオセラピューティクス インコーポレイティッド リポソームの製造システムおよび製造方法
US7838658B2 (en) 2005-10-20 2010-11-23 Ian Maclachlan siRNA silencing of filovirus gene expression
US8101741B2 (en) 2005-11-02 2012-01-24 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
GB0605900D0 (en) * 2006-03-23 2006-05-03 Lipigen As Modulators of nuclear receptors
SG171676A1 (en) 2006-05-11 2011-06-29 Alnylam Pharmaceuticals Inc Compositions and methods for inhibiting expression of the pcsk9 gene
US8598333B2 (en) 2006-05-26 2013-12-03 Alnylam Pharmaceuticals, Inc. SiRNA silencing of genes expressed in cancer
CA2927045A1 (en) 2006-10-03 2008-04-10 Muthiah Manoharan Lipid containing formulations
KR100996975B1 (ko) * 2007-08-24 2010-11-29 한국화학연구원 혈류 내 순환시간 증가를 위한 단백질로 수식된 리포솜 및이의 제조방법
CA2710713C (en) 2007-12-27 2017-09-19 Protiva Biotherapeutics, Inc. Silencing of polo-like kinase expression using interfering rna
US20110117125A1 (en) * 2008-01-02 2011-05-19 Tekmira Pharmaceuticals Corporation Compositions and methods for the delivery of nucleic acids
CA3252166A1 (en) * 2008-01-02 2025-11-29 Arbutus Biopharma Corp Screening method for selected amino lipid-containing compositions
CA2721380A1 (en) 2008-04-15 2009-10-22 Protiva Biotherapeutics, Inc. Silencing of csn5 gene expression using interfering rna
US8058069B2 (en) 2008-04-15 2011-11-15 Protiva Biotherapeutics, Inc. Lipid formulations for nucleic acid delivery
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
KR100996976B1 (ko) 2008-08-27 2010-11-29 현대엔지니어링 주식회사 장수명 ｍｔｏ 반응용 촉매 및 이의 제조방법
WO2010042877A1 (en) 2008-10-09 2010-04-15 Tekmira Pharmaceuticals Corporation Improved amino lipids and methods for the delivery of nucleic acids
CN105152939A (zh) * 2008-11-10 2015-12-16 阿尔尼拉姆医药品有限公司 用于递送治疗剂的脂质和组合物
KR20230098713A (ko) * 2009-06-10 2023-07-04 알닐람 파마슈티칼스 인코포레이티드 향상된 지질 조성물
EP2449114B9 (en) 2009-07-01 2017-04-19 Protiva Biotherapeutics Inc. Novel lipid formulations for delivery of therapeutic agents to solid tumors
US8569256B2 (en) * 2009-07-01 2013-10-29 Protiva Biotherapeutics, Inc. Cationic lipids and methods for the delivery of therapeutic agents
CA2784568A1 (en) * 2009-12-18 2011-06-23 Martin A. Maier Lipid particles for delivery of nucleic acids
WO2011143230A1 (en) * 2010-05-10 2011-11-17 Alnylam Pharmaceuticals Methods and compositions for delivery of active agents
US10077232B2 (en) * 2010-05-12 2018-09-18 Arbutus Biopharma Corporation Cyclic cationic lipids and methods of use
US20130123338A1 (en) * 2010-05-12 2013-05-16 Protiva Biotherapeutics, Inc. Novel cationic lipids and methods of use thereof
US8865675B2 (en) * 2010-05-12 2014-10-21 Protiva Biotherapeutics, Inc. Compositions and methods for silencing apolipoprotein B
EP2575767B1 (en) * 2010-06-04 2017-01-04 Sirna Therapeutics, Inc. Novel low molecular weight cationic lipids for oligonucleotide delivery
US8466122B2 (en) 2010-09-17 2013-06-18 Protiva Biotherapeutics, Inc. Trialkyl cationic lipids and methods of use thereof
JP2013545727A (ja) * 2010-10-21 2013-12-26 メルク・シャープ・アンド・ドーム・コーポレーション オリゴヌクレオチド送達用の新規低分子量カチオン性脂質
WO2013126803A1 (en) * 2012-02-24 2013-08-29 Protiva Biotherapeutics Inc. Trialkyl cationic lipids and methods of use thereof
US9744103B1 (en) 2013-03-08 2017-08-29 Chad Ricker Infant teething apparatus
JP6620093B2 (ja) 2013-07-23 2019-12-11 アービュートゥス バイオファーマ コーポレイションＡｒｂｕｔｕｓ Ｂｉｏｐｈａｒｍａ Ｃｏｒｐｏｒａｔｉｏｎ メッセンジャーｒｎａを送達するための組成物及び方法
WO2017015504A1 (en) 2015-07-21 2017-01-26 Scheibel Steven Frederick Treatment and prevention of anal conditions

Also Published As

Publication number Publication date
DK2817287T3 (da) 2019-01-02
BR112014020824B1 (pt) 2022-10-04
RS58077B1 (sr) 2019-02-28
RU2014138476A (ru) 2016-04-10
HRP20182156T1 (hr) 2019-02-22
CA2865412A1 (en) 2013-08-29
US20200306378A1 (en) 2020-10-01
BR112014020824A2 (enExample) 2017-06-20
HK1202855A1 (en) 2015-10-09
MX392812B (es) 2025-03-24
IL234266B (en) 2020-09-30
HUE041494T2 (hu) 2019-05-28
JP2018039844A (ja) 2018-03-15
EP2817287B1 (en) 2018-10-03
JP2021014462A (ja) 2021-02-12
RU2020111326A (ru) 2020-04-29
MX356904B (es) 2018-06-07
NZ700075A (en) 2016-05-27
KR20140129263A (ko) 2014-11-06
US11395854B2 (en) 2022-07-26
KR102384791B1 (ko) 2022-04-08
US20170007702A1 (en) 2017-01-12
US10561732B2 (en) 2020-02-18
AU2013222179A1 (en) 2014-10-09
JP2015509505A (ja) 2015-03-30
IL234266A0 (en) 2014-11-02
IL290931A (en) 2022-04-01
AU2013222179B2 (en) 2017-08-24
ZA201406896B (en) 2016-05-25
ES2898912T3 (es) 2022-03-09
EP3473611B1 (en) 2021-10-20
CA2865412C (en) 2022-07-19
JP2022140533A (ja) 2022-09-26
PH12014501901A1 (en) 2014-11-24
CN104321304A (zh) 2015-01-28
PH12014501901B1 (en) 2014-11-24
US20150064242A1 (en) 2015-03-05
KR20200016396A (ko) 2020-02-14
CY1121232T1 (el) 2020-05-29
EP3473611A1 (en) 2019-04-24
JP7110297B2 (ja) 2022-08-01
IL276924A (en) 2020-10-29
LT2817287T (lt) 2018-12-27
KR102075810B1 (ko) 2020-02-10
MX2014010211A (es) 2015-08-10
PL2817287T3 (pl) 2019-10-31
UA120026C2 (uk) 2019-09-25
PT2817287T (pt) 2018-12-28
KR102239887B1 (ko) 2021-04-13
SI2817287T1 (sl) 2019-02-28
CN110003030A (zh) 2019-07-12
JP6275655B2 (ja) 2018-02-07
JP6789918B2 (ja) 2020-11-25
RU2718053C2 (ru) 2020-03-30
US9352042B2 (en) 2016-05-31
EP3988104A1 (en) 2022-04-27
KR20210041135A (ko) 2021-04-14
MX2022006512A (es) 2022-07-11
SG11201405157PA (en) 2014-10-30
WO2013126803A1 (en) 2013-08-29
EP2817287A1 (en) 2014-12-31
US20230109183A1 (en) 2023-04-06
ES2702874T3 (es) 2019-03-06

Similar Documents

Publication Publication Date Title
US12129467B2 (en) Non-liposomal systems for nucleic acid delivery
JP7110297B2 (ja) トリアルキルカチオン性脂質およびその使用方法
US9126966B2 (en) Cationic lipids and methods of use thereof
US8466122B2 (en) Trialkyl cationic lipids and methods of use thereof
HK40063335A (en) Trialkyl cationic lipids and methods of use thereof
HK40008127A (en) Trialkyl cationic lipids and methods of use thereof
HK40008127B (en) Trialkyl cationic lipids and methods of use thereof
HK1202855B (en) Trialkyl cationic lipids and methods of use thereof

Legal Events

Date Code Title Description
A107 Divisional application of patent
PA0104 Divisional application for international application

Comment text: Divisional Application for International Patent

Patent event code: PA01041R01D

Patent event date: 20220405

Application number text: 1020217010379

Filing date: 20210407

PG1501 Laying open of application
PA0201 Request for examination

Patent event code: PA02012R01D

Patent event date: 20220504

Comment text: Request for Examination of Application

E902 Notification of reason for refusal
PE0902 Notice of grounds for rejection

Comment text: Notification of reason for refusal

Patent event date: 20220620

Patent event code: PE09021S01D

E601 Decision to refuse application
PE0601 Decision on rejection of patent

Patent event date: 20230103

Comment text: Decision to Refuse Application

Patent event code: PE06012S01D

Patent event date: 20220620

Comment text: Notification of reason for refusal

Patent event code: PE06011S01I