CL2016003263A1 - Composiciones enriquecidas esteroquímicamente para la liberación de ácidos nucléicos. - Google Patents

Composiciones enriquecidas esteroquímicamente para la liberación de ácidos nucléicos.

Info

Publication number
CL2016003263A1
CL2016003263A1 CL2016003263A CL2016003263A CL2016003263A1 CL 2016003263 A1 CL2016003263 A1 CL 2016003263A1 CL 2016003263 A CL2016003263 A CL 2016003263A CL 2016003263 A CL2016003263 A CL 2016003263A CL 2016003263 A1 CL2016003263 A1 CL 2016003263A1
Authority
CL
Chile
Prior art keywords
sterochemically
release
nucleic acids
enriched compositions
solvate
Prior art date
Application number
CL2016003263A
Other languages
English (en)
Inventor
Michael Heartlein
Frank Derosa
Shrirang Karve
Original Assignee
Shire Human Genetic Therapies
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 Shire Human Genetic Therapies filed Critical Shire Human Genetic Therapies
Publication of CL2016003263A1 publication Critical patent/CL2016003263A1/es

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Classifications

    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07DHETEROCYCLIC COMPOUNDS
    • C07D241/00Heterocyclic compounds containing 1,4-diazine or hydrogenated 1,4-diazine rings
    • C07D241/02Heterocyclic compounds containing 1,4-diazine or hydrogenated 1,4-diazine rings not condensed with other rings
    • C07D241/06Heterocyclic compounds containing 1,4-diazine or hydrogenated 1,4-diazine rings not condensed with other rings having one or two double bonds between ring members or between ring members and non-ring members
    • C07D241/08Heterocyclic compounds containing 1,4-diazine or hydrogenated 1,4-diazine rings not condensed with other rings having one or two double bonds between ring members or between ring members and non-ring members with oxygen atoms directly attached to ring carbon atoms
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/33Heterocyclic compounds
    • A61K31/395Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/495Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with two or more nitrogen atoms as the only ring heteroatoms, e.g. piperazine or tetrazines
    • 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
    • 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
    • A61K47/06Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/22Heterocyclic compounds, e.g. ascorbic acid, tocopherol or pyrrolidones
    • 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
    • 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00

Landscapes

  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • General Health & Medical Sciences (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Epidemiology (AREA)
  • Organic Chemistry (AREA)
  • Molecular Biology (AREA)
  • Dispersion Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Biochemistry (AREA)
  • Oil, Petroleum & Natural Gas (AREA)
  • Biophysics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Nanotechnology (AREA)
  • Optics & Photonics (AREA)
  • Physics & Mathematics (AREA)
  • Biotechnology (AREA)
  • Peptides Or Proteins (AREA)
  • Agricultural Chemicals And Associated Chemicals (AREA)

Abstract

<p>COMPOSICIÓN QUE COMPRENDE UNA O MÁS ENTIDADES QUÍMICAS DERIVADAS DE DIOXOPIPERAZINA O UNA SAL FARMACÉUTICAMENTE ACEPTABLE DE ESTE, UN SOLVATO DE ESTE O UN SOLVATO DE UNA SAL FARMACÉUTICAMENTE ACEPTABLE DE ESTE.</p>
CL2016003263A 2014-06-24 2016-12-20 Composiciones enriquecidas esteroquímicamente para la liberación de ácidos nucléicos. CL2016003263A1 (es)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US201462016512P 2014-06-24 2014-06-24

Publications (1)

Publication Number Publication Date
CL2016003263A1 true CL2016003263A1 (es) 2017-06-02

Family

ID=54929772

Family Applications (1)

Application Number Title Priority Date Filing Date
CL2016003263A CL2016003263A1 (es) 2014-06-24 2016-12-20 Composiciones enriquecidas esteroquímicamente para la liberación de ácidos nucléicos.

Country Status (17)

Country Link
US (4) US10138213B2 (es)
EP (1) EP3160959B1 (es)
JP (1) JP6599373B2 (es)
KR (3) KR102559979B1 (es)
CN (3) CN106795142B (es)
AU (4) AU2015279968B2 (es)
CA (1) CA2952824C (es)
CL (1) CL2016003263A1 (es)
EA (1) EA033966B1 (es)
ES (1) ES2964588T3 (es)
IL (1) IL249615A0 (es)
MA (1) MA40241A (es)
MX (3) MX389802B (es)
PE (1) PE20171238A1 (es)
SG (1) SG11201610670WA (es)
UA (1) UA121863C2 (es)
WO (1) WO2015200465A1 (es)

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ES3032935T3 (en) * 2013-10-22 2025-07-29 Translate Bio Inc Lipid formulations for delivery of messenger rna
JP6557722B2 (ja) 2014-05-30 2019-08-07 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 核酸の送達のための生分解性脂質
WO2018144709A2 (en) * 2017-02-01 2018-08-09 The Trustees Of The University Of Pennsylvania Gene therapy for treating citrullenemia
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EP3585892B8 (en) 2017-02-27 2022-07-13 Translate Bio, Inc. Methods for purification of messenger rna
EP3746496A1 (en) 2018-02-02 2020-12-09 Translate Bio, Inc. Cationic polymers
AU2019271132B2 (en) * 2018-05-15 2026-02-05 Translate Bio, Inc. Subcutaneous delivery of messenger RNA
KR102950141B1 (ko) 2018-08-24 2026-04-07 트랜슬레이트 바이오 인코포레이티드 전령 rna의 정제 방법
HRP20240586T1 (hr) 2018-10-09 2024-07-19 The University Of British Columbia Sastavi i sustavi koji sadrže transfekcijske sposobne vezikle bez organskih otapala i deterdženta i s njim povezanim postupcima
KR20210089648A (ko) 2018-11-08 2021-07-16 트랜슬레이트 바이오 인코포레이티드 메신저 rna 정제를 위한 방법 및 조성물
US20220233444A1 (en) * 2019-04-22 2022-07-28 Translate Bio, Inc. Thioester cationic lipids
CN114901360A (zh) 2019-12-20 2022-08-12 库瑞瓦格股份公司 用于递送核酸的新型脂质纳米颗粒
CN111658782A (zh) * 2020-06-10 2020-09-15 深圳近邻生物科技有限公司 mRNA疫苗递送载体及制备方法、mRNA疫苗及制备方法
IL301536A (en) * 2020-09-23 2023-05-01 Translate Bio Inc Cationic lipids based on piperazine
CZ310613B6 (cs) 2020-09-23 2026-01-28 Ústav organické chemie a biochemie AV ČR, v. v. i. Lipidoidy pro transfekci nukleových kyselin a jejich použití
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WO2022099003A1 (en) 2020-11-06 2022-05-12 Sanofi Lipid nanoparticles for delivering mrna vaccines
CZ310443B6 (cs) 2021-07-19 2025-06-25 Ústav organické chemie a biochemie AV ČR, v. v. i. Cyklohexanové lipidoidy pro transfekci nukleových kyselin a jejich použití
US20240398940A1 (en) 2021-09-03 2024-12-05 CureVac SE Novel lipid nanoparticles for delivery of nucleic acids
EP4422698A1 (en) 2021-10-29 2024-09-04 CureVac SE Improved circular rna for expressing therapeutic proteins
US20250099614A1 (en) 2022-01-28 2025-03-27 CureVac SE Nucleic acid encoded transcription factor inhibitors
US20250345407A1 (en) 2022-05-25 2025-11-13 CureVac SE Nucleic acid based vaccine encoding an escherichia coli fimh antigenic polypeptide
EP4608442A1 (en) 2022-10-28 2025-09-03 GlaxoSmithKline Biologicals S.A. Nucleic acid based vaccine
WO2024184500A1 (en) 2023-03-08 2024-09-12 CureVac SE Novel lipid nanoparticle formulations for delivery of nucleic acids
WO2024230934A1 (en) 2023-05-11 2024-11-14 CureVac SE Therapeutic nucleic acid for the treatment of ophthalmic diseases
CN118852328A (zh) * 2024-07-02 2024-10-29 北京大学 肽基可离子化脂质合成及其应用

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