MA47824A - Administration polyanionique d'acides nucléiques - Google Patents

Administration polyanionique d'acides nucléiques

Info

Publication number
MA47824A
MA47824A MA047824A MA47824A MA47824A MA 47824 A MA47824 A MA 47824A MA 047824 A MA047824 A MA 047824A MA 47824 A MA47824 A MA 47824A MA 47824 A MA47824 A MA 47824A
Authority
MA
Morocco
Prior art keywords
polyanionic
administration
nucleic acids
nucleic
acids
Prior art date
Application number
MA047824A
Other languages
English (en)
Inventor
Frank Derosa
Michael Heartlein
Shrirang Karve
Original Assignee
Translate Bio 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 Translate Bio Inc filed Critical Translate Bio Inc
Publication of MA47824A publication Critical patent/MA47824A/fr

Links

Classifications

    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07CACYCLIC OR CARBOCYCLIC COMPOUNDS
    • C07C305/00Esters of sulfuric acids
    • C07C305/02Esters of sulfuric acids having oxygen atoms of sulfate groups bound to acyclic carbon atoms of a carbon skeleton
    • C07C305/04Esters of sulfuric acids having oxygen atoms of sulfate groups bound to acyclic carbon atoms of a carbon skeleton being acyclic and saturated
    • C07C305/06Hydrogenosulfates
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/7105Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/54Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound
    • A61K47/543Lipids, e.g. triglycerides; Polyamines, e.g. spermine or spermidine
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/56Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic macromolecular compound, e.g. an oligomeric, polymeric or dendrimeric molecule
    • A61K47/58Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic macromolecular compound, e.g. an oligomeric, polymeric or dendrimeric molecule obtained by reactions only involving carbon-to-carbon unsaturated bonds, e.g. poly[meth]acrylate, polyacrylamide, polystyrene, polyvinylpyrrolidone, polyvinylalcohol or polystyrene sulfonic acid resin
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/10Dispersions; Emulsions
    • A61K9/127Synthetic bilayered vehicles, e.g. liposomes or liposomes with cholesterol as the only non-phosphatidyl surfactant
    • A61K9/1271Non-conventional liposomes, e.g. PEGylated liposomes or liposomes coated or grafted with polymers
    • A61K9/1272Non-conventional liposomes, e.g. PEGylated liposomes or liposomes coated or grafted with polymers comprising non-phosphatidyl surfactants as bilayer-forming substances, e.g. cationic lipids or non-phosphatidyl liposomes coated or grafted with polymers
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07CACYCLIC OR CARBOCYCLIC COMPOUNDS
    • C07C317/00Sulfones; Sulfoxides
    • C07C317/16Sulfones; Sulfoxides having sulfone or sulfoxide groups and singly-bound oxygen atoms bound to the same carbon skeleton
    • C07C317/18Sulfones; Sulfoxides having sulfone or sulfoxide groups and singly-bound oxygen atoms bound to the same carbon skeleton with sulfone or sulfoxide groups bound to acyclic carbon atoms of the carbon skeleton

Landscapes

  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Organic Chemistry (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biophysics (AREA)
  • Dispersion Chemistry (AREA)
  • Biochemistry (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
MA047824A 2017-03-07 2018-03-07 Administration polyanionique d'acides nucléiques MA47824A (fr)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US201762468244P 2017-03-07 2017-03-07

Publications (1)

Publication Number Publication Date
MA47824A true MA47824A (fr) 2020-01-15

Family

ID=61764150

Family Applications (1)

Application Number Title Priority Date Filing Date
MA047824A MA47824A (fr) 2017-03-07 2018-03-07 Administration polyanionique d'acides nucléiques

Country Status (4)

Country Link
US (2) US10961184B2 (fr)
EP (1) EP3592728A1 (fr)
MA (1) MA47824A (fr)
WO (1) WO2018165257A1 (fr)

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US8853377B2 (en) * 2010-11-30 2014-10-07 Shire Human Genetic Therapies, Inc. mRNA for use in treatment of human genetic diseases
JP7423522B2 (ja) 2017-11-22 2024-01-29 モダーナティエックス・インコーポレイテッド 尿素サイクル異常症の治療のためのオルニチントランスカルバミラーゼをコードするポリヌクレオチド
EP3793689A1 (fr) * 2018-05-15 2021-03-24 Translate Bio, Inc. Administration sous-cutanée d'arn messager
CN112888451A (zh) 2018-10-19 2021-06-01 川斯勒佰尔公司 信使rna的无泵包封
US12280147B2 (en) 2018-11-09 2025-04-22 Translate Bio, Inc. PEG lipidoid compounds
US12357705B2 (en) * 2018-11-09 2025-07-15 Arbutus Biopharma Corporation Negatively charged peg-lipid conjugates
US20220016265A1 (en) 2018-11-09 2022-01-20 Translate Bio, Inc. Messenger rna therapy for treatment of ocular diseases
JP2022516356A (ja) * 2019-01-07 2022-02-25 トランスレイト バイオ, インコーポレイテッド 原発性線毛機能不全症の治療のための組成物および方法
JP7586886B2 (ja) 2019-07-08 2024-11-19 トランスレイト バイオ, インコーポレイテッド 改善されたmRNA装填脂質ナノ粒子、およびそれを作製するプロセス
US12514932B2 (en) 2020-05-14 2026-01-06 Translate Bio, Inc. PEG lipidoid compounds
EP4229208A4 (fr) 2020-10-14 2024-10-30 George Mason Research Foundation, Inc. Méthodes de fabrication de nanoparticules lipidiques et compositions dérivées de celles-ci
CN115300609A (zh) * 2021-05-08 2022-11-08 西湖大学 缺氧诱导脂滴相关蛋白(hilpda)在预防或治疗高血脂相关疾病中的应用
WO2022241045A1 (fr) 2021-05-12 2022-11-17 The Broad Institute, Inc. Arnm modifié, arn non codant modifié, et leurs utilisations
EP4395748A1 (fr) 2021-09-03 2024-07-10 CureVac SE Nouvelles nanoparticules lipidiques pour l'administration d'acides nucléiques
US20250027108A1 (en) 2021-10-29 2025-01-23 CureVac SE Improved circular rna for expressing therapeutic proteins
EP4469091A1 (fr) 2022-01-28 2024-12-04 CureVac SE Inhibiteurs de facteurs de transcription codés par un acide nucleique
US20250345407A1 (en) 2022-05-25 2025-11-13 CureVac SE Nucleic acid based vaccine encoding an escherichia coli fimh antigenic polypeptide
US12186389B2 (en) 2022-10-28 2025-01-07 Glaxosmithkline Biologicals Sa Nucleic acid base vaccine against emerging SARS-CoV-2 variants
AU2024233180A1 (en) 2023-03-08 2025-09-25 CureVac SE Novel lipid nanoparticle formulations for delivery of nucleic acids
WO2024230934A1 (fr) 2023-05-11 2024-11-14 CureVac SE Acide nucléique thérapeutique pour le traitement de maladies ophtalmiques

Family Cites Families (31)

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US4458066A (en) 1980-02-29 1984-07-03 University Patents, Inc. Process for preparing polynucleotides
US4500707A (en) 1980-02-29 1985-02-19 University Patents, Inc. Nucleosides useful in the preparation of polynucleotides
US5132418A (en) 1980-02-29 1992-07-21 University Patents, Inc. Process for preparing polynucleotides
US4973679A (en) 1981-03-27 1990-11-27 University Patents, Inc. Process for oligonucleo tide synthesis using phosphormidite intermediates
US4668777A (en) 1981-03-27 1987-05-26 University Patents, Inc. Phosphoramidite nucleoside compounds
US4415732A (en) 1981-03-27 1983-11-15 University Patents, Inc. Phosphoramidite compounds and processes
US4401796A (en) 1981-04-30 1983-08-30 City Of Hope Research Institute Solid-phase synthesis of polynucleotides
US4373071A (en) 1981-04-30 1983-02-08 City Of Hope Research Institute Solid-phase synthesis of polynucleotides
US4897355A (en) 1985-01-07 1990-01-30 Syntex (U.S.A.) Inc. N[ω,(ω-1)-dialkyloxy]- and N-[ω,(ω-1)-dialkenyloxy]-alk-1-yl-N,N,N-tetrasubstituted ammonium lipids and uses therefor
US5153319A (en) 1986-03-31 1992-10-06 University Patents, Inc. Process for preparing polynucleotides
US5262530A (en) 1988-12-21 1993-11-16 Applied Biosystems, Inc. Automated system for polynucleotide synthesis and purification
US5047524A (en) 1988-12-21 1991-09-10 Applied Biosystems, Inc. Automated system for polynucleotide synthesis and purification
FR2645866B1 (fr) 1989-04-17 1991-07-05 Centre Nat Rech Scient Nouvelles lipopolyamines, leur preparation et leur emploi
US5334761A (en) 1992-08-28 1994-08-02 Life Technologies, Inc. Cationic lipids
US5885613A (en) 1994-09-30 1999-03-23 The University Of British Columbia Bilayer stabilizing components and their use in forming programmable fusogenic liposomes
US5700642A (en) 1995-05-22 1997-12-23 Sri International Oligonucleotide sizing using immobilized cleavable primers
US5744335A (en) 1995-09-19 1998-04-28 Mirus Corporation Process of transfecting a cell with a polynucleotide mixed with an amphipathic compound and a DNA-binding protein
EP1766035B1 (fr) 2004-06-07 2011-12-07 Protiva Biotherapeutics Inc. Arn interferant encapsule dans des lipides
WO2008016473A2 (fr) 2006-07-28 2008-02-07 Applera Corporation ANALOGUES DE COIFFES DE NUCLÉOTIDE D'ARNm
WO2009058911A2 (fr) 2007-10-31 2009-05-07 Applied Biosystems Inc. Préparation et isolement d'un arnm coiffé en 5'
JP5777519B2 (ja) 2008-10-09 2015-09-09 テクミラ ファーマシューティカルズ コーポレイション 改良されたアミノ脂質および核酸の送達方法
WO2010053572A2 (fr) 2008-11-07 2010-05-14 Massachusetts Institute Of Technology Lipidoïdes aminoalcool et leurs utilisations
JP4857373B2 (ja) * 2009-09-09 2012-01-18 国立大学法人東京農工大学 デンドリマー被覆磁気微粒子並びにその製造方法及び用途
ME03091B (fr) 2009-12-01 2019-01-20 Translate Bio Inc Administration d'arnm pour l'augmentation des protéines et des enzymes dans des maladies génétiques humaines
EP3998064A1 (fr) 2011-06-08 2022-05-18 Translate Bio, Inc. Lipides clivables
PT2717893T (pt) 2011-06-08 2019-08-20 Translate Bio Inc Composições de nanopartículas lipídicas e métodos para transferência de arnm
EP2535326A1 (fr) * 2011-06-16 2012-12-19 Koninklijke Philips Electronics N.V. Nouveaux composés lipidomimétiques et leurs utilisations
MX363734B (es) 2011-10-27 2019-03-29 Massachusetts Inst Technology Derivados de aminoacidos funcionalizados en la terminal n capaces de formar microesferas encapsuladoras de farmaco.
KR20150127582A (ko) 2013-03-14 2015-11-17 샤이어 휴먼 지네틱 테라피즈 인크. 4''-티오 개질된 뉴클레오티드를 갖는 리보핵산 및 관련 방법
ES3032013T3 (en) * 2013-03-15 2025-07-14 Translate Bio Inc Synergistic enhancement of the delivery of nucleic acids via blended formulations
DK3145984T3 (da) * 2014-05-21 2022-04-11 Icd Therapeutics Gmbh Terapeutiske konjugater med sulfaterede dendrimerer til intracellulær målretning

Also Published As

Publication number Publication date
US10961184B2 (en) 2021-03-30
EP3592728A1 (fr) 2020-01-15
US11427535B2 (en) 2022-08-30
US20200017440A1 (en) 2020-01-16
US20210261502A1 (en) 2021-08-26
WO2018165257A1 (fr) 2018-09-13

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