HK1202855A1 - Trialkyl cationic lipids and methods of use thereof - Google Patents

Trialkyl cationic lipids and methods of use thereof

Info

Publication number
HK1202855A1
HK1202855A1 HK15103551.8A HK15103551A HK1202855A1 HK 1202855 A1 HK1202855 A1 HK 1202855A1 HK 15103551 A HK15103551 A HK 15103551A HK 1202855 A1 HK1202855 A1 HK 1202855A1
Authority
HK
Hong Kong
Prior art keywords
methods
cationic lipids
trialkyl cationic
trialkyl
lipids
Prior art date
Application number
HK15103551.8A
Other languages
English (en)
Chinese (zh)
Inventor
Heyes James
Wood Mark
Martin Alan
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
Publication date
Application filed by Protiva Biotherapeutics Inc filed Critical Protiva Biotherapeutics Inc
Publication of HK1202855A1 publication Critical patent/HK1202855A1/xx

Links

Classifications

    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07C—ACYCLIC OR CARBOCYCLIC COMPOUNDS
    • C07C219/00—Compounds containing amino and esterified hydroxy groups bound to the same carbon skeleton
    • C07C219/02—Compounds containing amino and esterified hydroxy groups bound to the same carbon skeleton having esterified hydroxy groups and amino groups bound to acyclic carbon atoms of the same carbon skeleton
    • C07C219/20—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
    • 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
    • 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 N.A.
    • 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)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (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)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Oil, Petroleum & Natural Gas (AREA)
  • Physics & Mathematics (AREA)
  • Immunology (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Oncology (AREA)
  • Communicable Diseases (AREA)
  • Virology (AREA)
  • Gastroenterology & Hepatology (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)
HK15103551.8A 2012-02-24 2015-04-13 Trialkyl cationic lipids and methods of use thereof HK1202855A1 (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201261602990P 2012-02-24 2012-02-24
PCT/US2013/027469 WO2013126803A1 (fr) 2012-02-24 2013-02-22 Lipides cationiques trialkylés et leurs procédés d'utilisation

Publications (1)

Publication Number Publication Date
HK1202855A1 true HK1202855A1 (en) 2015-10-09

Family

ID=47884528

Family Applications (1)

Application Number Title Priority Date Filing Date
HK15103551.8A HK1202855A1 (en) 2012-02-24 2015-04-13 Trialkyl cationic lipids and methods of use thereof

Country Status (28)

Country Link
US (4) US9352042B2 (fr)
EP (3) EP3988104A1 (fr)
JP (4) JP6275655B2 (fr)
KR (4) KR102384791B1 (fr)
CN (2) CN104321304A (fr)
AU (1) AU2013222179B2 (fr)
BR (1) BR112014020824B1 (fr)
CA (1) CA2865412C (fr)
CY (1) CY1121232T1 (fr)
DK (1) DK2817287T3 (fr)
ES (2) ES2898912T3 (fr)
HK (1) HK1202855A1 (fr)
HR (1) HRP20182156T1 (fr)
HU (1) HUE041494T2 (fr)
IL (3) IL234266B (fr)
LT (1) LT2817287T (fr)
MX (2) MX356904B (fr)
NZ (1) NZ700075A (fr)
PH (1) PH12014501901B1 (fr)
PL (1) PL2817287T3 (fr)
PT (1) PT2817287T (fr)
RS (1) RS58077B1 (fr)
RU (2) RU2020111326A (fr)
SG (1) SG11201405157PA (fr)
SI (1) SI2817287T1 (fr)
UA (1) UA120026C2 (fr)
WO (1) WO2013126803A1 (fr)
ZA (1) ZA201406896B (fr)

Families Citing this family (159)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
NZ600616A (en) 2009-12-01 2014-11-28 Shire Human Genetic Therapies Delivery of mrna for the augmentation of proteins and enzymes in human genetic diseases
WO2012075040A2 (fr) 2010-11-30 2012-06-07 Shire Human Genetic Therapies, Inc. Arnm pour l'utilisation dans le traitement de maladies génétiques humaines
DK2718269T3 (en) 2011-06-08 2018-04-09 Translate Bio Inc SPLITLY LIPIDS
KR102128248B1 (ko) 2011-06-08 2020-07-01 샤이어 휴먼 지네틱 테라피즈 인크. Mrna 전달을 위한 지질 나노입자 조성물 및 방법
KR102384791B1 (ko) * 2012-02-24 2022-04-08 아뷰터스 바이오파마 코포레이션 트리알킬 양이온성 지질 및 그의 사용 방법
EP3620447B1 (fr) 2012-03-29 2021-02-17 Translate Bio, Inc. Lipides cationiques ionisables
JP6211054B2 (ja) 2012-03-29 2017-10-11 シャイアー ヒューマン ジェネティック セラピーズ インコーポレイテッド 脂質誘導される中性ナノ粒子
EA201492055A1 (ru) 2012-06-08 2015-11-30 Шир Хьюман Дженетик Терапис, Инк. ИНГАЛЯЦИОННАЯ ДОСТАВКА мРНК В НЕЛЕГОЧНЫЕ КЛЕТКИ-МИШЕНИ
US20150267192A1 (en) 2012-06-08 2015-09-24 Shire Human Genetic Therapies, Inc. Nuclease resistant polynucleotides and uses thereof
AU2014239184B2 (en) 2013-03-14 2018-11-08 Translate Bio, Inc. Methods and compositions for delivering mRNA coded antibodies
WO2014152211A1 (fr) 2013-03-14 2014-09-25 Moderna Therapeutics, Inc. Formulation et administration de compositions de nucléosides, de nucléotides, et d'acides nucléiques modifiés
DK2968586T3 (en) 2013-03-14 2018-10-08 Translate Bio Inc CFTR MRNA COMPOSITIONS AND RELATED PROCEDURES AND APPLICATIONS
AU2014236396A1 (en) 2013-03-14 2015-08-13 Shire Human Genetic Therapies, Inc. Methods for purification of messenger RNA
EP3757570B1 (fr) 2013-03-15 2023-10-11 Translate Bio, Inc. Amélioration synergique de l'administration d'acides nucléiques par l'intermédiaire de formulations mélangées
CN110974981A (zh) * 2013-07-23 2020-04-10 野草莓树生物制药公司 用于递送信使rna的组合物和方法
US20160194368A1 (en) 2013-09-03 2016-07-07 Moderna Therapeutics, Inc. Circular polynucleotides
US10323076B2 (en) 2013-10-03 2019-06-18 Modernatx, Inc. Polynucleotides encoding low density lipoprotein receptor
BR112016008832A2 (pt) 2013-10-22 2017-10-03 Shire Human Genetic Therapies Distribuição de mrna no snc e suas utilizações
WO2015061500A1 (fr) 2013-10-22 2015-04-30 Shire Human Genetic Therapies, Inc. Thérapie arnm pour déficience en argininosuccinate synthétase
MX2016005239A (es) 2013-10-22 2016-08-12 Shire Human Genetic Therapies Tratamiento con acido ribonucleico mensajero para la fenilcetonuria.
EA201690576A1 (ru) 2013-10-22 2016-10-31 Шир Хьюман Дженетик Терапис, Инк. Липидные композиции для доставки матричной рнк
JP6571679B2 (ja) 2014-04-25 2019-09-04 トランスレイト バイオ， インコーポレイテッド メッセンジャーｒｎａの精製方法
EP3587409B8 (fr) 2014-05-30 2022-07-13 Translate Bio, Inc. Lipides biodégradables pour l'administration d'acides nucléiques
WO2015200465A1 (fr) 2014-06-24 2015-12-30 Shire Human Genetic Therapies, Inc. Compositions enrichies stéréochimiquement pour l'administration d'acides nucléiques
ES2834556T3 (es) 2014-06-25 2021-06-17 Acuitas Therapeutics Inc Lípidos y formulaciones de nanopartículas lipídicas novedosos para la entrega de ácidos nucleicos
JP6782171B2 (ja) 2014-07-02 2020-11-11 シャイアー ヒューマン ジェネティック セラピーズ インコーポレイテッド メッセンジャーｒｎａのカプセル化
CA2955250A1 (fr) 2014-07-16 2016-01-21 Moderna Therapeutics, Inc. Polynucleotides chimeriques
US20170210788A1 (en) 2014-07-23 2017-07-27 Modernatx, Inc. Modified polynucleotides for the production of intrabodies
CA2956554C (fr) * 2014-08-07 2022-03-01 Takeda Pharmaceutical Company Limited Preparation de composes lipidiques cationiques et leurs sels comme supports d'acide nucleique
WO2016036966A1 (fr) * 2014-09-04 2016-03-10 Preceres Inc. Lipidoïdes d'hydrazinyle et utilisations de ceux-ci
EP3226912B1 (fr) 2014-12-05 2021-01-20 Translate Bio, Inc. Thérapie par l'arn messager pour le traitement des maladies articulaires
EP3268473B1 (fr) * 2015-03-11 2020-05-13 INSERM - Institut National de la Santé et de la Recherche Médicale Procédés et compositions pharmaceutiques pour le traitement d'infections par filovirus
CA2979695A1 (fr) 2015-03-19 2016-09-22 Translate Bio, Inc. Therapie d'arnm pour maladie de pompe
JP7072386B2 (ja) * 2015-06-29 2022-05-20 アクイタス セラピューティクス インコーポレイテッド 核酸の送達のための脂質および脂質ナノ粒子製剤
ES2862412T3 (es) 2015-10-14 2021-10-07 Translate Bio Inc Modificación de enzimas relacionados con el ARN para producción mejorada
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
CN108368028B (zh) 2015-10-28 2021-09-03 爱康泰生治疗公司 用于递送核酸的新型脂质和脂质纳米颗粒制剂
ES2919552T3 (es) 2015-12-23 2022-07-27 Modernatx Inc Procedimientos de utilización de polinucleotidos codificadores de ligando ox40
EP3400023A1 (fr) 2016-01-10 2018-11-14 ModernaTX, Inc. Arnm thérapeutiques codant pour des anticorps anti-ctla-4
KR102475301B1 (ko) 2016-04-08 2022-12-09 트랜슬레이트 바이오 인코포레이티드 다량체 코딩 핵산 및 그 용도
EP3458107B1 (fr) 2016-05-18 2024-03-13 ModernaTX, Inc. Polynucléotides codant pour jagged1 pour le traitement du syndrome d'alagille
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
MX2018016389A (es) 2016-06-30 2019-08-16 Arbutus Biopharma Corp Composiciones y metodos para suministro de arn mensajero.
AU2018224326B2 (en) 2017-02-27 2024-01-04 Translate Bio, Inc. Novel codon-optimized CFTR mRNA
AU2018234828A1 (en) 2017-03-15 2019-09-19 Modernatx, Inc. Lipid nanoparticle formulation
US11752206B2 (en) 2017-03-15 2023-09-12 Modernatx, Inc. Herpes simplex virus vaccine
SG11201906297QA (en) 2017-03-24 2019-10-30 Curevac Ag 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
IL301115A (en) 2017-04-28 2023-05-01 Acuitas Therapeutics Inc New lipid carbonyl and lipid nanoparticle formulations for delivery of nucleic acids
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
CA3063723A1 (fr) 2017-05-18 2018-11-22 Modernatx, Inc. Polynucleotides codant pour des polypeptides d'interleukine-12 (il12) ancres et leurs utilisations
US20200268666A1 (en) 2017-06-14 2020-08-27 Modernatx, Inc. Polynucleotides encoding coagulation factor viii
MA49421A (fr) 2017-06-15 2020-04-22 Modernatx Inc Formulations d'arn
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
CA3073020A1 (fr) 2017-08-16 2019-02-21 Acuitas Therapeutics, Inc. Lipides destines a etre utilises dans des formulations nanoparticulaires lipidiques
WO2019036030A1 (fr) 2017-08-17 2019-02-21 Acuitas Therapeutics, Inc. Lipides destinés à être utilisés dans des formulations de nanoparticules lipidiques
WO2019036028A1 (fr) 2017-08-17 2019-02-21 Acuitas Therapeutics, Inc. Lipides destinés à être utilisés dans des formulations nanoparticulaires lipidiques
WO2019036683A1 (fr) 2017-08-18 2019-02-21 Modernatx, Inc. Procédés analytiques par hplc
WO2019046809A1 (fr) 2017-08-31 2019-03-07 Modernatx, Inc. Procédés de fabrication de nanoparticules lipidiques
EP3727428A1 (fr) 2017-12-20 2020-10-28 Translate Bio, Inc. Compositions et procédés améliorés pour le traitement du déficit en ornithine transcarbamylase
US20210052646A1 (en) * 2017-12-27 2021-02-25 Takeda Pharmaceutical Company Limited Nucleic-acid containing lipid nano-particle and use thereof
AU2019325702A1 (en) 2018-08-24 2021-02-25 Translate Bio, Inc. Methods for purification of messenger RNA
MA53652A (fr) 2018-09-19 2021-07-28 Modernatx Inc Lipides peg de haute pureté et leurs utilisations
MA53650A (fr) 2018-09-19 2021-07-28 Modernatx Inc Lipides peg et leurs utilisations
JP2022512578A (ja) 2018-10-09 2022-02-07 ザ ユニヴァーシティ オブ ブリティッシュ コロンビア 有機溶媒不含かつ劣化剤不含のトランスフェクション・コンピテント・ベシクルを含む組成物及びシステム並びにそれらに関連する方法
BR112021007360A2 (pt) 2018-10-18 2021-07-20 Takeda Pharmaceutical Company Limited métodos para ativar/proliferar células t, para distribuir um ácido nucleico em células t e para produzir um medicamento, célula t, medicamento, cultura de células, composição, e, kit para distribuir um ácido nucleico em células t
CA3119071A1 (fr) * 2018-11-09 2020-05-14 Arbutus Biopharma Corporation Formulations de nanoparticules lipidiques
WO2020097548A1 (fr) * 2018-11-09 2020-05-14 Arbutus Biopharma Corporation Formulations de nanoparticules lipidiques
CA3141323A1 (fr) 2019-06-18 2020-12-24 Helen Horton Construction de l'interleukine 12 recombinante et ses utilisations
EP3986460A2 (fr) 2019-06-18 2022-04-27 Janssen Sciences Ireland Unlimited Company Combinaison de vaccins contre le virus de l'hépatite b (vhb) et d'anticorps anti-pd-1
WO2020255010A1 (fr) 2019-06-18 2020-12-24 Janssen Sciences Ireland Unlimited Company Combinaison d'une construction d'interleukine 12 recombinante et de vaccins contre le virus de l'hépatite b (vhb)
KR20220041080A (ko) 2019-06-18 2022-03-31 얀센 사이언시즈 아일랜드 언리미티드 컴퍼니 B형 간염 바이러스(hbv) 백신 및 항-pd-1 또는 항-pc-l1 항체의 조합
EP3986562A1 (fr) 2019-06-18 2022-04-27 Janssen Sciences Ireland Unlimited Company Combinaison de vaccins contre le virus de l'hépatite b (vhb) et d'arni ciblant le virus de l'hépatite b
US20220305108A1 (en) 2019-06-20 2022-09-29 Janssen Sciences Ireland Unlimited Company Lipid nanoparticle or liposome delivery of hepatitis b virus (hbv) vaccines
WO2021000041A1 (fr) * 2019-06-29 2021-01-07 Precision Nanosystems Inc. Lipides ionisables pour l'administration d'acides nucléiques
WO2021016075A1 (fr) 2019-07-19 2021-01-28 Flagship Pioneering Innovations Vi, Llc Compositions à recombinase et leurs méthodes d'utilisation
MX2022003402A (es) * 2019-09-19 2022-06-14 Modernatx Inc Grupos principales de compuestos lipídicos y composiciones para la administración intracelular de agentes terapéuticos.
EP4041894A1 (fr) 2019-09-23 2022-08-17 Omega Therapeutics, Inc. COMPOSITIONS ET PROCÉDÉS DE MODULATION DE L'EXPRESSION GÉNIQUE DU FACTEUR NUCLÉAIRE HÉPATOCYTAIRE 4-ALPHA (HNF4a)
AU2020355000A1 (en) 2019-09-23 2022-03-17 Omega Therapeutics, Inc. Compositions and methods for modulating apolipoprotein B (APOB) gene expression
EP4118207A1 (fr) 2020-03-11 2023-01-18 Omega Therapeutics, Inc. Compositions et procédés de modulation de l'expression génique de forkhead box p3 (foxp3)
KR20220154740A (ko) * 2020-03-17 2022-11-22 제네반트 사이언시즈 게엠베하 간 성상 세포로 치료제의 지질 나노입자 전달을 위한 양이온성 지질
CN115667530A (zh) 2020-03-24 2023-01-31 世代生物公司 非病毒dna载体和其用于表达因子ix治疗剂的用途
KR20230003478A (ko) 2020-03-24 2023-01-06 제너레이션 바이오 컴퍼니 비-바이러스성 dna 벡터 및 고셰 치료제 발현을 위한 이의 용도
JP2023526423A (ja) 2020-05-20 2023-06-21 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
CA3179420A1 (fr) 2020-05-20 2021-11-25 Avak Kahvejian Compositions d'antigenes de coronavirus et leurs utilisations
WO2021243301A2 (fr) 2020-05-29 2021-12-02 Flagship Pioneering Innovations Vi, Llc. Compositions à base de trem et procédés associés
JP2023527413A (ja) 2020-05-29 2023-06-28 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 Ｔｒｅｍ組成物及びそれに関連する方法
WO2022008613A1 (fr) 2020-07-08 2022-01-13 Janssen Sciences Ireland Unlimited Company Vaccins à base de réplicon d'arn contre le vhb
WO2022016070A1 (fr) 2020-07-16 2022-01-20 Acuitas Therapeutics, Inc. Lipides cationiques destinés à être utilisés dans des nanoparticules lipidiques
JP2023535632A (ja) 2020-07-27 2023-08-18 アンジャリウム バイオサイエンシズ エージー Ｄｎａ分子の組成物、その作製方法、及びその使用方法
CA3189854A1 (fr) 2020-08-06 2022-02-10 Modernatx, Inc. Compositions pour l'administration de molecules de charge utile a l'epithelium des voies respiratoires
WO2022051629A1 (fr) 2020-09-03 2022-03-10 Flagship Pioneering Innovations Vi, Llc Compositions immunogènes et leurs utilisations
WO2022081589A1 (fr) * 2020-10-12 2022-04-21 The Regents Of The University Of California Domaines d'échappement endosomal pour introduire des macromolécules dans des cellules
WO2022133230A1 (fr) 2020-12-18 2022-06-23 Janssen Pharmaceuticals, Inc. Polythérapie pour le traitement d'une infection par le virus de l'hépatite b
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
JP2024501022A (ja) 2020-12-28 2024-01-10 アークトゥルス セラピューティクス， インコーポレイテッド Ｈｂｖを標的とする転写活性化因子様エフェクターヌクレアーゼ（ｔａｌｅｎ）
CA3209032A1 (fr) 2021-02-26 2022-09-01 Ethris Gmbh Formulations destinees a la formation d'aerosol et aerosols destines a l'administration d'acide nucleique
US11952461B2 (en) 2021-03-22 2024-04-09 Sunbio, Inc. Siloxy polyethylene glycol and derivatives thereof
US20220325287A1 (en) 2021-03-31 2022-10-13 Flagship Pioneering Innovations V, Inc. Thanotransmission polypeptides and their use in treating cancer
CN115197078B (zh) * 2021-04-08 2024-06-04 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
EP4326860A1 (fr) 2021-04-20 2024-02-28 Anjarium Biosciences AG Compositions de molécules d'adn codant pour la beta-alpha-1, 6-glucosidase, 4-alpha-glucanotransférase, leurs procédés de fabrication et leurs procédés d'utilisation
WO2022232286A1 (fr) 2021-04-27 2022-11-03 Generation Bio Co. Vecteurs d'adn non viraux exprimant des anticorps anti-coronavirus et leurs utilisations
KR20240011714A (ko) 2021-04-27 2024-01-26 제너레이션 바이오 컴퍼니 치료용 항체를 발현하는 비바이러스성 dna 벡터 및 이의 용도
WO2022229903A1 (fr) 2021-04-28 2022-11-03 Genevant Sciences Gmbh Constructions d'administration d'arnm et leurs procédés d'utilisation
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
EP4367242A2 (fr) 2021-07-07 2024-05-15 Omega Therapeutics, Inc. Compositions et procédés de modulation de l'expression génique de la protéine 1 du récepteur frizzled secrété (sfrp1)
CA3226019A1 (fr) 2021-07-20 2023-01-26 Ags Therapeutics Sas Vesicules extracellulaires provenant de microalgues, leur preparation et leurs utilisations
EP4377457A1 (fr) 2021-07-26 2024-06-05 Flagship Pioneering Innovations VI, LLC Compositions de trem et leurs utilisations
TW202330916A (zh) 2021-09-17 2023-08-01 美商旗艦先鋒創新有限責任公司 用於產生環狀多核糖核苷酸之組成物和方法
AU2022370530A1 (en) 2021-10-18 2024-05-02 Flagship Pioneering Innovations Vi, Llc Compositions and methods for purifying polyribonucleotides
TW202334080A (zh) 2021-11-08 2023-09-01 美商歐納醫療公司 用於遞送環狀聚核苷酸之脂質奈米粒子組合物
WO2023086465A1 (fr) 2021-11-12 2023-05-19 Modernatx, Inc. Compositions pour l'administration de molécules de charge utile à l'épithélium des voies respiratoires
CA3238735A1 (fr) 2021-11-24 2023-06-01 Jennifer A. Nelson Compositions immunogenes et leurs utilisations
AU2022397292A1 (en) 2021-11-24 2024-05-30 Flagship Pioneering Innovations Vi, Llc Varicella-zoster virus immunogen compositions and their uses
CA3239266A1 (fr) 2021-11-24 2023-06-01 Flagship Pioneering Innovations Vi, Llc Compositions immunogenes de coronavirus et leurs utilisations
AR128002A1 (es) 2021-12-17 2024-03-20 Flagship Pioneering Innovations Vi Llc Métodos de enriquecimiento de rna circular en condiciones desnaturalizantes
WO2023122745A1 (fr) 2021-12-22 2023-06-29 Flagship Pioneering Innovations Vi, Llc Compositions et procédés de purification de polyribonucléotides
WO2023122789A1 (fr) 2021-12-23 2023-06-29 Flagship Pioneering Innovations Vi, Llc Polyribonucléotides circulaires codant pour des polypeptides antifusogènes
CN114957027B (zh) * 2022-01-13 2023-05-19 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 (fr) 2022-01-14 2023-07-20 Anjarium Biosciences Ag Compositions de molécules d'adn codant pour le facteur viii, leurs procédés de préparation et leurs méthodes d'utilisation
WO2023144127A1 (fr) 2022-01-31 2023-08-03 Ags Therapeutics Sas Vésicules extracellulaires provenant de microalgues, leur biodistribution suite à leur administration, et leurs utilisations
WO2023154818A1 (fr) 2022-02-09 2023-08-17 Modernatx, Inc. Méthodes et formulations d'administration par voie muqueuse
WO2023161350A1 (fr) 2022-02-24 2023-08-31 Io Biotech Aps Administration nucléotidique d'une thérapie anticancéreuse
WO2023160702A1 (fr) * 2022-02-28 2023-08-31 深圳深信生物科技有限公司 Composé amino-lipide, son procédé de préparation, composition et utilisation de celui-ci
WO2023177655A1 (fr) 2022-03-14 2023-09-21 Generation Bio Co. Compositions vaccinales prime-boost hétérologues et méthodes d'utilisation
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
WO2023183616A1 (fr) 2022-03-25 2023-09-28 Senda Biosciences, Inc. Nouveaux lipides et nanoparticules lipidiques ionisables et leurs procédés d'utilisation
WO2023196634A2 (fr) 2022-04-08 2023-10-12 Flagship Pioneering Innovations Vii, Llc Vaccins et procédés associés
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 (fr) 2022-05-09 2023-11-16 Flagship Pioneering Innovations Vi, Llc Compositions de trem et procédés d'utilisation pour traiter des troubles prolifératifs
WO2023218420A1 (fr) 2022-05-13 2023-11-16 Janssen Pharmaceuticals, Inc. Compositions d'arnm pour induire une inversion latente du vih-1
WO2023220729A2 (fr) 2022-05-13 2023-11-16 Flagship Pioneering Innovations Vii, Llc Compositions d'adn à double brin et procédés associés
WO2023233290A1 (fr) 2022-05-31 2023-12-07 Janssen Sciences Ireland Unlimited Company Agents d'arni ciblant 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 (fr) 2022-06-03 2023-12-07 Ags Therapeutics Sas Vésicules extracellulaires provenant de microalgues génétiquement modifiées contenant une cargaison chargée de manière endogène, leur préparation et utilisations
WO2023239756A1 (fr) 2022-06-07 2023-12-14 Generation Bio Co. Compositions de nanoparticules lipidiques et leurs utilisations
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
WO2023250112A1 (fr) 2022-06-22 2023-12-28 Flagship Pioneering Innovations Vi, Llc Compositions de trem modifiées et leurs utilisations
US20240042021A1 (en) 2022-08-01 2024-02-08 Flagship Pioneering Innovations Vii, Llc Immunomodulatory proteins and related methods
WO2024035952A1 (fr) 2022-08-12 2024-02-15 Remix Therapeutics Inc. Procédés et compositions pour moduler l'épissage au niveau de sites d'épissage alternatifs
WO2024040222A1 (fr) 2022-08-19 2024-02-22 Generation Bio Co. Adn à extrémités fermées clivable (adnce) et ses procédés d'utilisation
WO2024042236A1 (fr) 2022-08-26 2024-02-29 Ethris Gmbh Suspensions de nanoparticules lipidiques ou lipidoïdes stables
EP4327829A1 (fr) 2022-08-26 2024-02-28 Ethris GmbH Stabilisation de suspensions de nanoparticules lipidiques ou lipidoïdes
WO2024049979A2 (fr) 2022-08-31 2024-03-07 Senda Biosciences, Inc. Nouveaux lipides ionisables et nanoparticules lipidiques, ainsi que procédés d'utilisation associés
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
WO2024088808A1 (fr) 2022-10-24 2024-05-02 Ags Therapeutics Sas Vésicules extracellulaires provenant de microalgues, leur biodistribution lors d'une administration intranasale, et leurs utilisations
WO2024097664A1 (fr) 2022-10-31 2024-05-10 Flagship Pioneering Innovations Vi, Llc Compositions et procédés de purification de polyribonucléotides
WO2024102762A1 (fr) 2022-11-08 2024-05-16 Orna Therapeutics, Inc. Lipides et compositions de nanoparticules lipidiques pour administration de polynucléotides
WO2024102730A1 (fr) 2022-11-08 2024-05-16 Orna Therapeutics, Inc. Lipides et compositions de nanoparticules pour l'administration de polynucléotides
WO2024102677A1 (fr) 2022-11-08 2024-05-16 Orna Therapeutics, Inc. Compositions d'arn circulaire
WO2024102799A1 (fr) 2022-11-08 2024-05-16 Flagship Pioneering Innovations Vi, Llc Compositions et procédés de production de polyribonucléotides circulaires
WO2024119103A1 (fr) 2022-12-01 2024-06-06 Generation Bio Co. Nanoparticules lipidiques comprenant des acides nucléiques et des polymères à ancrage lipidique
WO2024119039A2 (fr) 2022-12-01 2024-06-06 Generation Bio Co. Nanoparticules lipidiques furtives et leurs utilisations
WO2024119074A1 (fr) 2022-12-01 2024-06-06 Generation Bio Co. Compositions de nanoparticules lipidiques furtives pour le ciblage cellulaire
WO2024119051A1 (fr) 2022-12-01 2024-06-06 Generation Bio Co. Nouveaux lipides conjugués à un polyglycérol et compositions de nanoparticules lipidiques les comprenant
WO2024121814A1 (fr) 2022-12-09 2024-06-13 Takeda Pharmaceutical Company Limited Immunomodulateurs modifiés
WO2024129988A1 (fr) 2022-12-14 2024-06-20 Flagship Pioneering Innovations Vii, Llc Compositions et procédés d'administration d'agents thérapeutiques à un os
CN117482066A (zh) * 2023-11-02 2024-02-02 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

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 (fr) 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 (fr) 1988-09-20 1999-01-19 Arnold E. Hampel Catalyseur d'arn pour le clivage de sequences specifiques d'arn
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
JP3058686B2 (ja) 1989-08-31 2000-07-04 シティ・オブ・ホープ キメラｄｎａ―ｒｎａ触媒活性配列
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
CA2093664C (fr) 1990-10-12 2003-07-29 Fritz Eckstein Ribozymes modifies
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
CA2135646A1 (fr) 1992-05-11 1993-11-25 Kenneth G. Draper Methode et reagent permettant l'inhibition de la replication virale
WO1993023011A1 (fr) 1992-05-18 1993-11-25 Minnesota Mining And Manufacturing Company Dispositif d'apport de medicament a travers des muqueuses
US5792451A (en) 1994-03-02 1998-08-11 Emisphere Technologies, Inc. Oral drug delivery compositions and methods
EP0786522A2 (fr) 1992-07-17 1997-07-30 Ribozyme Pharmaceuticals, Inc. Molécules de RNA enzymatiques pour le traitement de conditions sténotiques
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
US5785992A (en) 1994-09-30 1998-07-28 Inex Pharmaceuticals Corp. Compositions for the introduction of polyanionic materials into cells
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
US5747470A (en) 1995-06-07 1998-05-05 Gen-Probe Incorporated Method for inhibiting cellular proliferation using antisense oligonucleotides to gp130 mRNA
ATE285477T1 (de) 1995-06-07 2005-01-15 Inex Pharmaceutical Corp Herstellung von lipid-nukleinsäure partikeln duch ein hydrophobische lipid-nukleinsäuree komplexe zwischenprodukt und zur verwendung in der gentransfer
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
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
US6287591B1 (en) 1997-05-14 2001-09-11 Inex Pharmaceuticals Corp. Charged therapeutic agents encapsulated in lipid particles containing four lipid components
WO1999005303A1 (fr) 1997-07-24 1999-02-04 Inex Pharmaceuticals Corporation Preparation de particules de lipides-acides nucleiques grace a un procede d'extraction par solvant et d'hydratation directe
US6320017B1 (en) 1997-12-23 2001-11-20 Inex Pharmaceuticals Corp. Polyamide oligomers
DE69906977T2 (de) 1998-07-20 2004-05-19 Protiva Biotherapeutics Inc., Burnaby In liposomen verkapselte nukleinsäurekomplexe
EP1173600A2 (fr) 1999-04-20 2002-01-23 The University Of British Columbia Lipides peg cationiques et m thodes d'utilisation
US6852334B1 (en) 1999-04-20 2005-02-08 The University Of British Columbia 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
US7833992B2 (en) 2001-05-18 2010-11-16 Merck Sharpe & Dohme 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
US7189705B2 (en) 2000-04-20 2007-03-13 The University Of British Columbia Methods of enhancing SPLP-mediated transfection using endosomal membrane destabilizers
EP1985702A3 (fr) 2000-12-08 2010-08-18 Coley Pharmaceutical GmbH Acides nucléiques de type CPG et leurs procédés d'utilisation
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
US20030077829A1 (en) 2001-04-30 2003-04-24 Protiva Biotherapeutics Inc.. Lipid-based formulations
US20050282188A1 (en) 2001-05-18 2005-12-22 Sirna Therapeutics, Inc. RNA interference mediated inhibition of 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
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
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
EP1432724A4 (fr) 2002-02-20 2006-02-01 Sirna Therapeutics Inc Inhibition a mediation par interference d'arn de genes de map kinase
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
WO2004002453A1 (fr) 2002-06-28 2004-01-08 Protiva Biotherapeutics Ltd. Appareil liposomal et procedes de fabrication
DE60326961D1 (de) 2002-07-26 2009-05-14 Novartis Vaccines & Diagnostic Modifizierte kleine irns moleküle und methoden zu deren anwendung
WO2006006948A2 (fr) 2002-11-14 2006-01-19 Dharmacon, Inc. Methodes et compositions permettant de selectionner des arnsi presentant une fonctionnalite amelioree
JP2006507841A (ja) 2002-11-14 2006-03-09 ダーマコン， インコーポレイテッド 機能的ｓｉＲＮＡおよび超機能的ｓｉＲＮＡ
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
AU2004227414A1 (en) 2003-04-03 2004-10-21 Alnylam Pharmaceuticals iRNA conjugates
CA2521464C (fr) 2003-04-09 2013-02-05 Alnylam Pharmaceuticals, Inc. Conjugues arni
ES2702942T3 (es) 2003-04-17 2019-03-06 Alnylam Pharmaceuticals Inc Agentes de ARNi modificados
EP1648519B1 (fr) 2003-07-16 2014-10-08 Protiva Biotherapeutics Inc. Arn interferant encapsule dans un lipide
US7825235B2 (en) 2003-08-18 2010-11-02 Isis Pharmaceuticals, Inc. Modulation of diacylglycerol acyltransferase 2 expression
AU2004272646B2 (en) 2003-09-15 2011-11-24 Arbutus Biopharma Corporation Polyethyleneglycol-modified lipid compounds and uses thereof
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
US8084599B2 (en) 2004-03-15 2011-12-27 City Of Hope Methods and compositions for the specific inhibition of gene expression by double-stranded RNA
EP1781593B1 (fr) 2004-06-07 2011-12-14 Protiva Biotherapeutics Inc. Lipides cationiques et leurs procédés d'utilisation
CA2569664C (fr) 2004-06-07 2013-07-16 Protiva Biotherapeutics, Inc. Arn interferant encapsule dans des lipides
WO2006031901A2 (fr) 2004-09-10 2006-03-23 Somagenics, Inc. Petits arn interferents inhibant efficacement l'expression genique virale et methodes d'utilisation associees
NZ554214A (en) 2004-09-24 2009-10-30 Alnylam Pharmaceuticals Inc Rnai modulation of apob and uses thereof
AU2005306533B2 (en) 2004-11-17 2012-05-31 Arbutus Biopharma Corporation siRNA silencing of apolipoprotein B
WO2007086883A2 (fr) 2005-02-14 2007-08-02 Sirna Therapeutics, Inc. Compositions à base de nanoparticules lipidiques et méthodes pour l'administration de molécules biologiquement actives
US7404969B2 (en) 2005-02-14 2008-07-29 Sirna Therapeutics, Inc. Lipid nanoparticle based compositions and methods for the delivery of biologically active molecules
AU2006274413B2 (en) 2005-07-27 2013-01-10 Arbutus Biopharma Corporation Systems and methods for manufacturing liposomes
US7838658B2 (en) 2005-10-20 2010-11-23 Ian Maclachlan siRNA silencing of filovirus gene expression
WO2007051303A1 (fr) 2005-11-02 2007-05-10 Protiva Biotherapeutics, Inc. Molecules d'arnsi modifiees et utilisations de celles-ci
WO2007056861A1 (fr) 2005-11-18 2007-05-24 Protiva Biotherapeutics, Inc. Attenuation de l'expression du gene du virus de la grippe par arnsi
GB0605900D0 (en) * 2006-03-23 2006-05-03 Lipigen As Modulators of nuclear receptors
JP5570806B2 (ja) 2006-05-11 2014-08-13 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 Ｐｃｓｋ９遺伝子の発現を阻害するための組成物および方法
US8598333B2 (en) 2006-05-26 2013-12-03 Alnylam Pharmaceuticals, Inc. SiRNA silencing of genes expressed in cancer
AU2007303205A1 (en) 2006-10-03 2008-04-10 Tekmira Pharmaceuticals Corporation Lipid containing formulations
KR100996975B1 (ko) * 2007-08-24 2010-11-29 한국화학연구원 혈류 내 순환시간 증가를 위한 단백질로 수식된 리포솜 및이의 제조방법
CA2710713C (fr) 2007-12-27 2017-09-19 Protiva Biotherapeutics, Inc. Silencage de l'expression de la polo-like kinase a l'aide d'un arn interferent
WO2009088891A1 (fr) * 2008-01-02 2009-07-16 Alnylam Pharmaceuticals, Inc. Procédé de criblage pour des compositions contenant des aminolipides sélectionnés
WO2009086558A1 (fr) 2008-01-02 2009-07-09 Tekmira Pharmaceuticals Corporation Compositions et procédés améliorés pour la délivrance d'acides nucléiques
CN102119217B (zh) 2008-04-15 2015-06-03 普洛体维生物治疗公司 用于核酸递送的新型制剂
EP2770057A1 (fr) 2008-04-15 2014-08-27 Protiva Biotherapeutics Inc. Silençage de l'expression du gène CSN5 au moyen d'ARN interférant
KR101647334B1 (ko) 2008-07-10 2016-08-10 세리나 쎄라퓨틱스, 인코포레이티드 불활성 말단기를 가진 폴리옥사졸린, 보호된 개시기로부터 제조된 폴리옥사졸린 및 그와 관련된 화합물
KR100996976B1 (ko) 2008-08-27 2010-11-29 현대엔지니어링 주식회사 장수명 ｍｔｏ 반응용 촉매 및 이의 제조방법
ES2475065T3 (es) 2008-10-09 2014-07-10 Tekmira Pharmaceuticals Corporation Aminol�pidos mejorados y métodos para la administración de ácidos nucleicos
KR20220150995A (ko) * 2008-11-10 2022-11-11 알닐람 파마슈티칼스 인코포레이티드 치료제 운반용 신규 지질 및 조성물
TR201811076T4 (tr) * 2009-06-10 2018-08-27 Arbutus Biopharma Corp Geliştirilmiş lipit formulasyonu.
IL292615B2 (en) * 2009-07-01 2023-11-01 Protiva Biotherapeutics Inc Nucleic acid-lipid particles, preparations containing them and their uses
WO2011000106A1 (fr) * 2009-07-01 2011-01-06 Protiva Biotherapeutics, Inc. Lipides cationiques et procédés améliorés pour l'administration d'agents thérapeutiques
CA2784568A1 (fr) * 2009-12-18 2011-06-23 Martin A. Maier Particules de lipide destinees a la distribution d'acides nucleiques
WO2011143230A1 (fr) * 2010-05-10 2011-11-17 Alnylam Pharmaceuticals Procédés et compositions pour la distribution d'agents actifs
WO2011141703A1 (fr) * 2010-05-12 2011-11-17 Protiva Biotherapeutics Inc. Compositions et procédés pour réduire au silence l'apolipoprotéine b
WO2011141704A1 (fr) * 2010-05-12 2011-11-17 Protiva Biotherapeutics, Inc Nouveaux lipides cationiques cycliques et procédés d'utilisation
JP2013527856A (ja) 2010-05-12 2013-07-04 プロチバ バイオセラピューティクス インコーポレイティッド 陽イオン性脂質およびその使用方法
JP5957646B2 (ja) * 2010-06-04 2016-07-27 サーナ・セラピューティクス・インコーポレイテッドＳｉｒｎａ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ． オリゴヌクレオチド送達のための新規な低分子量カチオン性脂質
US8466122B2 (en) 2010-09-17 2013-06-18 Protiva Biotherapeutics, Inc. Trialkyl cationic lipids and methods of use thereof
CA2813024A1 (fr) 2010-10-21 2012-04-26 Merck Sharp & Dohme Corp. Nouveaux lipides cationiques a faible poids moleculaire destines a une administration d'oligonucleotides
KR102384791B1 (ko) 2012-02-24 2022-04-08 아뷰터스 바이오파마 코포레이션 트리알킬 양이온성 지질 및 그의 사용 방법
US9744103B1 (en) 2013-03-08 2017-08-29 Chad Ricker Infant teething apparatus
CN110974981A (zh) 2013-07-23 2020-04-10 野草莓树生物制药公司 用于递送信使rna的组合物和方法
WO2017015504A1 (fr) 2015-07-21 2017-01-26 Scheibel Steven Frederick Traitement et prévention d'affections anales

Also Published As

Publication number Publication date
ES2898912T3 (es) 2022-03-09
KR20140129263A (ko) 2014-11-06
PL2817287T3 (pl) 2019-10-31
BR112014020824A2 (fr) 2017-06-20
JP2022140533A (ja) 2022-09-26
RU2020111326A (ru) 2020-04-29
WO2013126803A1 (fr) 2013-08-29
MX2022006512A (es) 2022-07-11
AU2013222179A1 (en) 2014-10-09
HRP20182156T1 (hr) 2019-02-22
IL276924A (en) 2020-10-29
ZA201406896B (en) 2016-05-25
RS58077B1 (sr) 2019-02-28
IL234266B (en) 2020-09-30
JP2018039844A (ja) 2018-03-15
IL234266A0 (en) 2014-11-02
KR102384791B1 (ko) 2022-04-08
JP6275655B2 (ja) 2018-02-07
EP2817287B1 (fr) 2018-10-03
CA2865412A1 (fr) 2013-08-29
MX356904B (es) 2018-06-07
EP3988104A1 (fr) 2022-04-27
SG11201405157PA (en) 2014-10-30
US10561732B2 (en) 2020-02-18
SI2817287T1 (sl) 2019-02-28
JP2021014462A (ja) 2021-02-12
PH12014501901A1 (en) 2014-11-24
EP3473611B1 (fr) 2021-10-20
CY1121232T1 (el) 2020-05-29
EP2817287A1 (fr) 2014-12-31
KR20210041135A (ko) 2021-04-14
KR102075810B1 (ko) 2020-02-10
RU2718053C2 (ru) 2020-03-30
DK2817287T3 (da) 2019-01-02
MX2014010211A (es) 2015-08-10
IL290931A (en) 2022-04-01
AU2013222179B2 (en) 2017-08-24
PH12014501901B1 (en) 2014-11-24
LT2817287T (lt) 2018-12-27
CN104321304A (zh) 2015-01-28
CN110003030A (zh) 2019-07-12
KR20200016396A (ko) 2020-02-14
UA120026C2 (uk) 2019-09-25
RU2014138476A (ru) 2016-04-10
CA2865412C (fr) 2022-07-19
PT2817287T (pt) 2018-12-28
US20150064242A1 (en) 2015-03-05
KR102239887B1 (ko) 2021-04-13
HUE041494T2 (hu) 2019-05-28
US9352042B2 (en) 2016-05-31
JP7110297B2 (ja) 2022-08-01
BR112014020824B1 (pt) 2022-10-04
US20200306378A1 (en) 2020-10-01
US20170007702A1 (en) 2017-01-12
KR20220045089A (ko) 2022-04-12
EP3473611A1 (fr) 2019-04-24
JP6789918B2 (ja) 2020-11-25
US11395854B2 (en) 2022-07-26
JP2015509505A (ja) 2015-03-30
NZ700075A (en) 2016-05-27
ES2702874T3 (es) 2019-03-06
US20230109183A1 (en) 2023-04-06

Similar Documents

Publication Publication Date Title
IL290931A (en) Trialkyl cationic lipids and methods of using them
IL262110A (en) dll3 modulators and methods to use
HRP20181628T1 (hr) Spojevi i postupci za njihovu upotrebu
HK1205719A1 (zh) 陽離子可聚合組合物及其使用方法
ZA201502503B (en) Hppd variants and methods of use
ZA201405540B (en) Packaging and method of packaging
EP2912178A4 (fr) Super-activateurs et procédés d'utilisation de ceux-ci
IL236270B (en) Cationic lipid vaccine compositions and methods of use
EP2852397A4 (fr) Variants d'huwentoxine iv et procédés d'utilisation
EP2906576A4 (fr) Glycosphingolipides et leurs procédés d'utilisation
ZA201408128B (en) Etract of greyia radlkoferi and use thereof