CO2023014991A2 - Cationic lipids based on good buffers - Google Patents

Cationic lipids based on good buffers

Info

Publication number
CO2023014991A2
CO2023014991A2 CONC2023/0014991A CO2023014991A CO2023014991A2 CO 2023014991 A2 CO2023014991 A2 CO 2023014991A2 CO 2023014991 A CO2023014991 A CO 2023014991A CO 2023014991 A2 CO2023014991 A2 CO 2023014991A2
Authority
CO
Colombia
Prior art keywords
cationic lipids
lipids based
good buffers
compounds
cationic
Prior art date
Application number
CONC2023/0014991A
Other languages
Spanish (es)
Inventor
Shrirang Karve
Frank Derosa
Ryan Landis
Ramesh Dasari
Saswata Karmakar
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
Priority claimed from PCT/US2022/025067 external-priority patent/WO2022221688A1/en
Application filed by Translate Bio Inc filed Critical Translate Bio Inc
Publication of CO2023014991A2 publication Critical patent/CO2023014991A2/en

Links

Classifications

    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07DHETEROCYCLIC COMPOUNDS
    • C07D295/00Heterocyclic compounds containing polymethylene-imine rings with at least five ring members, 3-azabicyclo [3.2.2] nonane, piperazine, morpholine or thiomorpholine rings, having only hydrogen atoms directly attached to the ring carbon atoms
    • C07D295/04Heterocyclic compounds containing polymethylene-imine rings with at least five ring members, 3-azabicyclo [3.2.2] nonane, piperazine, morpholine or thiomorpholine rings, having only hydrogen atoms directly attached to the ring carbon atoms with substituted hydrocarbon radicals attached to ring nitrogen atoms
    • C07D295/08Heterocyclic compounds containing polymethylene-imine rings with at least five ring members, 3-azabicyclo [3.2.2] nonane, piperazine, morpholine or thiomorpholine rings, having only hydrogen atoms directly attached to the ring carbon atoms with substituted hydrocarbon radicals attached to ring nitrogen atoms substituted by singly bound oxygen or sulfur atoms
    • C07D295/084Heterocyclic compounds containing polymethylene-imine rings with at least five ring members, 3-azabicyclo [3.2.2] nonane, piperazine, morpholine or thiomorpholine rings, having only hydrogen atoms directly attached to the ring carbon atoms with substituted hydrocarbon radicals attached to ring nitrogen atoms substituted by singly bound oxygen or sulfur atoms with the ring nitrogen atoms and the oxygen or sulfur atoms attached to the same carbon chain, which is not interrupted by carbocyclic rings
    • C07D295/088Heterocyclic compounds containing polymethylene-imine rings with at least five ring members, 3-azabicyclo [3.2.2] nonane, piperazine, morpholine or thiomorpholine rings, having only hydrogen atoms directly attached to the ring carbon atoms with substituted hydrocarbon radicals attached to ring nitrogen atoms substituted by singly bound oxygen or sulfur atoms with the ring nitrogen atoms and the oxygen or sulfur atoms attached to the same carbon chain, which is not interrupted by carbocyclic rings to an acyclic saturated chain
    • 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
    • A61K9/127Liposomes
    • A61K9/1271Non-conventional liposomes, e.g. PEGylated liposomes, liposomes coated with polymers
    • A61K9/1272Non-conventional liposomes, e.g. PEGylated liposomes, liposomes coated with polymers with substantial amounts of non-phosphatidyl, i.e. non-acylglycerophosphate, surfactants as bilayer-forming substances, e.g. cationic lipids
    • 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

Landscapes

  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Public Health (AREA)
  • General Health & Medical Sciences (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Molecular Biology (AREA)
  • Dispersion Chemistry (AREA)
  • Biophysics (AREA)
  • Organic Low-Molecular-Weight Compounds And Preparation Thereof (AREA)
  • Engineering & Computer Science (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Medicinal Preparation (AREA)
  • Biochemistry (AREA)
  • Biomedical Technology (AREA)
  • Nanotechnology (AREA)
  • Optics & Photonics (AREA)
  • Physics & Mathematics (AREA)
  • Genetics & Genomics (AREA)
  • Magnetic Resonance Imaging Apparatus (AREA)
  • Biotechnology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)

Abstract

La presente invención proporciona, entre otras cosas, una nueva clase de compuestos lipídicos catiónicos (por ejemplo, lípidos catiónicos que tienen una estructura de acuerdo con la fórmula (I)) para la administración in vivo de agentes terapéuticos, tales como los ácidos nucleicos. Se contempla que estos compuestos son capaces de un suministro in vivo altamente efectiva mientras mantienen un perfil de toxicidad favorable.The present invention provides, among other things, a new class of cationic lipid compounds (for example, cationic lipids having a structure according to formula (I)) for the in vivo administration of therapeutic agents, such as nucleic acids. It is contemplated that these compounds are capable of highly effective in vivo delivery while maintaining a favorable toxicity profile.

CONC2023/0014991A 2021-04-15 2023-11-03 Cationic lipids based on good buffers CO2023014991A2 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202163175429P 2021-04-15 2021-04-15
US202263313578P 2022-02-24 2022-02-24
PCT/US2022/025067 WO2022221688A1 (en) 2021-04-15 2022-04-15 "good"buffer-based cationic lipids

Publications (1)

Publication Number Publication Date
CO2023014991A2 true CO2023014991A2 (en) 2024-02-05

Family

ID=88412572

Family Applications (1)

Application Number Title Priority Date Filing Date
CONC2023/0014991A CO2023014991A2 (en) 2021-04-15 2023-11-03 Cationic lipids based on good buffers

Country Status (9)

Country Link
EP (1) EP4323346A1 (en)
JP (1) JP2024514326A (en)
KR (1) KR20230171974A (en)
AU (1) AU2022259686A1 (en)
BR (1) BR112023020027A2 (en)
CA (1) CA3215137A1 (en)
CO (1) CO2023014991A2 (en)
IL (1) IL307652A (en)
MX (1) MX2023012195A (en)

Also Published As

Publication number Publication date
EP4323346A1 (en) 2024-02-21
KR20230171974A (en) 2023-12-21
MX2023012195A (en) 2023-12-15
BR112023020027A2 (en) 2023-11-14
CA3215137A1 (en) 2022-10-20
AU2022259686A1 (en) 2023-11-30
JP2024514326A (en) 2024-04-01
IL307652A (en) 2023-12-01

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