MX2023003344A - Lipidos cationicos a base de piperazina. - Google Patents

Lipidos cationicos a base de piperazina.

Info

Publication number
MX2023003344A
MX2023003344A MX2023003344A MX2023003344A MX2023003344A MX 2023003344 A MX2023003344 A MX 2023003344A MX 2023003344 A MX2023003344 A MX 2023003344A MX 2023003344 A MX2023003344 A MX 2023003344A MX 2023003344 A MX2023003344 A MX 2023003344A
Authority
MX
Mexico
Prior art keywords
piperazine
cationic lipids
based cationic
useful
mrna
Prior art date
Application number
MX2023003344A
Other languages
English (en)
Spanish (es)
Inventor
Ryan Landis
Saswata Karmakar
Ramesh Dasari
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 MX2023003344A publication Critical patent/MX2023003344A/es

Links

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
    • C07D241/04Heterocyclic compounds containing 1,4-diazine or hydrogenated 1,4-diazine rings not condensed with other rings having no double bonds between ring members or between ring members and non-ring members
    • 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
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0008Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • A61K48/0025Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid
    • A61K48/0033Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid the non-active part being non-polymeric
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/48Preparations in capsules, e.g. of gelatin, of chocolate
    • A61K9/50Microcapsules 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
    • A61K9/51Nanocapsules; Nanoparticles
    • A61K9/5107Excipients; Inactive ingredients
    • A61K9/5123Organic compounds, e.g. fats, sugars
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/87Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/88Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation using microencapsulation, e.g. using amphiphile liposome vesicle
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0091Purification or manufacturing processes for gene therapy compositions

Landscapes

  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Medicinal Chemistry (AREA)
  • Molecular Biology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Biophysics (AREA)
  • Zoology (AREA)
  • Physics & Mathematics (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Dispersion Chemistry (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Nanotechnology (AREA)
  • Optics & Photonics (AREA)
  • Manufacturing & Machinery (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Detergent Compositions (AREA)
  • Agricultural Chemicals And Associated Chemicals (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
MX2023003344A 2020-09-23 2021-09-23 Lipidos cationicos a base de piperazina. MX2023003344A (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US202063082101P 2020-09-23 2020-09-23
PCT/US2021/051763 WO2022066916A1 (en) 2020-09-23 2021-09-23 Piperazine-based cationic lipids

Publications (1)

Publication Number Publication Date
MX2023003344A true MX2023003344A (es) 2023-06-05

Family

ID=78414070

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2023003344A MX2023003344A (es) 2020-09-23 2021-09-23 Lipidos cationicos a base de piperazina.

Country Status (12)

Country Link
US (1) US20230357166A1 (https=)
EP (1) EP4216933B1 (https=)
JP (1) JP7791182B2 (https=)
KR (1) KR20230074526A (https=)
CN (1) CN116457027A (https=)
AU (1) AU2021349262A1 (https=)
BR (1) BR112023005207A2 (https=)
CA (1) CA3193347A1 (https=)
ES (1) ES2994955T3 (https=)
IL (1) IL301536A (https=)
MX (1) MX2023003344A (https=)
WO (1) WO2022066916A1 (https=)

Families Citing this family (16)

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WO2022099003A1 (en) 2020-11-06 2022-05-12 Sanofi Lipid nanoparticles for delivering mrna vaccines
BR112023020027A2 (pt) * 2021-04-15 2023-11-14 Translate Bio Inc Lipídeos catiônicos baseados em tampão "good"
WO2023178167A1 (en) * 2022-03-16 2023-09-21 Translate Bio, Inc. Asymmetric piperazine-based cationic lipids
US20250263385A1 (en) 2022-04-13 2025-08-21 Sanofi "Good" buffer-based cationic lipids
US20240150306A1 (en) * 2022-09-27 2024-05-09 Reinvigoron Theratech, Inc. Compounds with cleavable disulfide moieties
EP4637813A1 (en) 2022-12-20 2025-10-29 Sanofi Pasteur Rhinovirus mrna vaccine
CN116082275B (zh) * 2023-03-13 2023-07-14 北京悦康科创医药科技股份有限公司 一种脾脏中高表达的阳离子脂质化合物、包含其的组合物及用途
EP4734960A1 (en) 2023-06-28 2026-05-06 Sanofi Pasteur Inc. Sterol analogs in lipid nanoparticle formulations
US12599560B2 (en) 2023-11-02 2026-04-14 Longuide Biopharma Corporation Lipids and lipid nanoparticle formulations
CN121219273A (zh) * 2023-11-02 2025-12-26 深圳厚存纳米药业有限公司 脂质和脂质纳米颗粒制剂
US12539279B2 (en) 2023-12-11 2026-02-03 The Governing Council Of The University Of Toronto Supramolecular ionizable lipid molecules with heteroatomic tuning for nucleic acid delivery
WO2025141521A1 (en) 2023-12-29 2025-07-03 Sanofi Lipids having dendritic moieties
WO2025160118A1 (en) * 2024-01-22 2025-07-31 The Trustees Of The University Of Pennsylvania Compositions and methods for hematopoietic stem cell (hsc) targeted delivery of therapeutic agents
WO2025255534A1 (en) * 2024-06-07 2025-12-11 Innovac Therapeutics Hk Limited Diol-based novel ionizable lipids and their uses thereof
WO2026008743A1 (en) 2024-07-02 2026-01-08 Sanofi Pasteur Inc. Water-soluble polyanionic polymer as adjuvant for carrier-formulated nucleic acid
WO2026053147A1 (en) 2024-09-05 2026-03-12 Sanofi Pasteur Inc. Helper lipids and lnp compositions comprising same

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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
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
US4973679A (en) 1981-03-27 1990-11-27 University Patents, Inc. Process for oligonucleo tide synthesis using phosphormidite intermediates
US4373071A (en) 1981-04-30 1983-02-08 City Of Hope Research Institute Solid-phase synthesis of polynucleotides
US4401796A (en) 1981-04-30 1983-08-30 City Of Hope Research Institute Solid-phase synthesis of polynucleotides
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
US5885613A (en) 1994-09-30 1999-03-23 The University Of British Columbia Bilayer stabilizing components and their use in forming programmable fusogenic liposomes
US5780014A (en) 1995-04-14 1998-07-14 Inhale Therapeutic Systems Method and apparatus for pulmonary administration of dry powder alpha 1-antitrypsin
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
US7101995B2 (en) * 2001-08-27 2006-09-05 Mirus Bio Corporation Compositions and processes using siRNA, amphipathic compounds and polycations
KR101766408B1 (ko) 2009-06-10 2017-08-10 알닐람 파마슈티칼스 인코포레이티드 향상된 지질 조성물
WO2014028487A1 (en) 2012-08-13 2014-02-20 Massachusetts Institute Of Technology Amine-containing lipidoids and uses thereof
ES3032935T3 (en) * 2013-10-22 2025-07-29 Translate Bio Inc Lipid formulations for delivery of messenger rna
KR102559979B1 (ko) * 2014-06-24 2023-07-25 샤이어 휴먼 지네틱 테라피즈 인크. 핵산의 전달용 입체화학적으로 풍부한 조성물
WO2016205691A1 (en) * 2015-06-19 2016-12-22 Massachusetts Institute Of Technology Alkenyl substituted 2,5-piperazinediones and their use in compositions for delivering an agent to a subject or cell
AU2017356699B2 (en) * 2016-11-08 2021-11-11 Ramot At Tel-Aviv University Ltd. Cationic lipids for nucleic acid delivery and preparation thereof
AU2017357758B2 (en) 2016-11-10 2023-11-16 Translate Bio, Inc. Improved process of preparing mRNA-loaded lipid nanoparticles
JP7422977B2 (ja) * 2018-07-23 2024-01-29 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用乾燥粉末製剤
AU2019376660B2 (en) 2018-11-09 2025-06-26 Translate Bio, Inc. 2,5-dioxopiperazine lipids with intercalated ester, thioester, disulfide and anhydride moieities
EP4146680A1 (en) 2020-05-07 2023-03-15 Translate Bio, Inc. Composition and methods for treatment of primary ciliary dyskinesia
WO2021226468A1 (en) 2020-05-07 2021-11-11 Translate Bio, Inc. Improved compositions for cftr mrna therapy

Also Published As

Publication number Publication date
AU2021349262A1 (en) 2023-06-08
US20230357166A1 (en) 2023-11-09
KR20230074526A (ko) 2023-05-30
CN116457027A (zh) 2023-07-18
AU2021349262A9 (en) 2025-03-13
ES2994955T3 (en) 2025-02-04
JP7791182B2 (ja) 2025-12-23
EP4216933B1 (en) 2024-09-18
WO2022066916A1 (en) 2022-03-31
IL301536A (en) 2023-05-01
JP2023542944A (ja) 2023-10-12
EP4216933A1 (en) 2023-08-02
CA3193347A1 (en) 2022-03-31
EP4216933C0 (en) 2024-09-18
BR112023005207A2 (pt) 2023-04-25

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