BR112022004771A2 - Compostos e composições de lipídio de grupo de cabeça para entrega intracelular de agentes terapêuticos - Google Patents

Compostos e composições de lipídio de grupo de cabeça para entrega intracelular de agentes terapêuticos

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Publication number
BR112022004771A2
BR112022004771A2 BR112022004771A BR112022004771A BR112022004771A2 BR 112022004771 A2 BR112022004771 A2 BR 112022004771A2 BR 112022004771 A BR112022004771 A BR 112022004771A BR 112022004771 A BR112022004771 A BR 112022004771A BR 112022004771 A2 BR112022004771 A2 BR 112022004771A2
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BR
Brazil
Prior art keywords
compositions
lipids
lnps
intracellular delivery
therapeutic agents
Prior art date
Application number
BR112022004771A
Other languages
English (en)
Inventor
Andrew Mckenzie
Edward Hennessy
E Benenato Kerry
Mark Cornebise
Original Assignee
Modernatx Inc
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Publication date
Application filed by Modernatx Inc filed Critical Modernatx Inc
Publication of BR112022004771A2 publication Critical patent/BR112022004771A2/pt

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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/10—Dispersions; Emulsions
    • A61K9/127—Liposomes
    • A61K9/1271—Non-conventional liposomes, e.g. PEGylated liposomes, liposomes coated with polymers
    • A61K9/1272—Non-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
    • 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
    • A61K31/7042—Compounds having saccharide radicals and heterocyclic rings
    • A61K31/7052—Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
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    • A61K47/18—Amines; Amides; Ureas; Quaternary ammonium compounds; Amino acids; Oligopeptides having up to five amino acids
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    • A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/22—Heterocyclic compounds, e.g. ascorbic acid, tocopherol or pyrrolidones
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    • C07C311/36—Sulfonamides, the carbon skeleton of the acid part being further substituted by singly-bound nitrogen atoms, not being part of nitro or nitroso groups having the sulfur atom of at least one of the sulfonamide groups bound to a carbon atom of a ring other than a six-membered aromatic ring
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    • C07D207/444—Heterocyclic compounds containing five-membered rings not condensed with other rings, with one nitrogen atom as the only ring hetero atom with only hydrogen or carbon atoms directly attached to the ring nitrogen atom having three double bonds between ring members or between ring members and non-ring members having two doubly-bound oxygen atoms directly attached in positions 2 and 5
    • C07D207/456—Heterocyclic compounds containing five-membered rings not condensed with other rings, with one nitrogen atom as the only ring hetero atom with only hydrogen or carbon atoms directly attached to the ring nitrogen atom having three double bonds between ring members or between ring members and non-ring members having two doubly-bound oxygen atoms directly attached in positions 2 and 5 with hetero atoms or with carbon atoms having three bonds to hetero atoms with at the most one bond to halogen, e.g. ester or nitrile radicals, directly attached to other ring carbon atoms
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Abstract

compostos e composições de lipídio de grupo de cabeça para entrega intracelular de agentes terapêuticos. a presente invenção refere-se a lipídios da fórmula (a-1) e a composições que envolvem os mesmos. as nanopartículas de lipídio (por exemplo, lnps vazias ou lnps carregadas) incluem tal lipídio, assim como lipídios adicionais, como fosfolipídios, lipídios estruturais e lipídios de peg. as nanopartículas de lipídio (por exemplo, lnps vazias ou lnps carregadas) que incluem, ainda, agentes terapêuticos e/ou profiláticos, como o rna, são úteis na entrega de agentes terapêuticos e/ou profiláticos às células ou órgãos de mamíferos para, por exemplo, regular a expressão de polipeptídeos, proteínas ou genes.
BR112022004771A 2019-09-19 2020-09-18 Compostos e composições de lipídio de grupo de cabeça para entrega intracelular de agentes terapêuticos BR112022004771A2 (pt)

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