AR125204A1 - Compuestos de lípidos de cola ramificada y composiciones para la administración intracelular de agentes terapéuticos - Google Patents

Compuestos de lípidos de cola ramificada y composiciones para la administración intracelular de agentes terapéuticos

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Publication number
AR125204A1
AR125204A1 ARP220100685A ARP220100685A AR125204A1 AR 125204 A1 AR125204 A1 AR 125204A1 AR P220100685 A ARP220100685 A AR P220100685A AR P220100685 A ARP220100685 A AR P220100685A AR 125204 A1 AR125204 A1 AR 125204A1
Authority
AR
Argentina
Prior art keywords
alkenyl
alkyl
independently selected
group
carbocycle
Prior art date
Application number
ARP220100685A
Other languages
English (en)
Inventor
Kerry E Benenato
Mark Cornebise
Edward Hennessy
Mohindra Seepersaud
Michael W Danneman
Original Assignee
Modernatx Inc
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Filing date
Publication date
Application filed by Modernatx Inc filed Critical Modernatx Inc
Publication of AR125204A1 publication Critical patent/AR125204A1/es

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    • C07C311/22—Sulfonamides, the carbon skeleton of the acid part being further substituted by singly-bound oxygen atoms
    • C07C311/23—Sulfonamides, the carbon skeleton of the acid part being further substituted by singly-bound oxygen atoms having the sulfur atoms of the sulfonamide groups bound to acyclic carbon atoms
    • C07C311/24—Sulfonamides, the carbon skeleton of the acid part being further substituted by singly-bound oxygen atoms having the sulfur atoms of the sulfonamide groups bound to acyclic carbon atoms of an acyclic saturated carbon skeleton
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/48—Preparations in capsules, e.g. of gelatin, of chocolate
    • A61K9/50—Microcapsules 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
    • A61K9/51—Nanocapsules; Nanoparticles
    • A61K9/5107—Excipients; Inactive ingredients
    • A61K9/5123—Organic compounds, e.g. fats, sugars
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/48—Preparations in capsules, e.g. of gelatin, of chocolate
    • A61K9/50—Microcapsules 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
    • A61K9/51—Nanocapsules; Nanoparticles
    • A61K9/5107—Excipients; Inactive ingredients
    • A61K9/513—Organic macromolecular compounds; Dendrimers
    • A61K9/5146—Organic macromolecular compounds; Dendrimers obtained otherwise than by reactions only involving carbon-to-carbon unsaturated bonds, e.g. polyethylene glycol, polyamines, polyanhydrides
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    • C07C233/00—Carboxylic acid amides
    • C07C233/01—Carboxylic acid amides having carbon atoms of carboxamide groups bound to hydrogen atoms or to acyclic carbon atoms
    • C07C233/34—Carboxylic acid amides having carbon atoms of carboxamide groups bound to hydrogen atoms or to acyclic carbon atoms having the nitrogen atom of at least one of the carboxamide groups bound to a carbon atom of a hydrocarbon radical substituted by amino groups
    • C07C233/35—Carboxylic acid amides having carbon atoms of carboxamide groups bound to hydrogen atoms or to acyclic carbon atoms having the nitrogen atom of at least one of the carboxamide groups bound to a carbon atom of a hydrocarbon radical substituted by amino groups with the substituted hydrocarbon radical bound to the nitrogen atom of the carboxamide group by an acyclic carbon atom
    • C07C233/36—Carboxylic acid amides having carbon atoms of carboxamide groups bound to hydrogen atoms or to acyclic carbon atoms having the nitrogen atom of at least one of the carboxamide groups bound to a carbon atom of a hydrocarbon radical substituted by amino groups with the substituted hydrocarbon radical bound to the nitrogen atom of the carboxamide group by an acyclic carbon atom having the carbon atom of the carboxamide group bound to a hydrogen atom or to a carbon atom of an acyclic saturated carbon skeleton
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    • C07C233/00—Carboxylic acid amides
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    • C07C233/34—Carboxylic acid amides having carbon atoms of carboxamide groups bound to hydrogen atoms or to acyclic carbon atoms having the nitrogen atom of at least one of the carboxamide groups bound to a carbon atom of a hydrocarbon radical substituted by amino groups
    • C07C233/35—Carboxylic acid amides having carbon atoms of carboxamide groups bound to hydrogen atoms or to acyclic carbon atoms having the nitrogen atom of at least one of the carboxamide groups bound to a carbon atom of a hydrocarbon radical substituted by amino groups with the substituted hydrocarbon radical bound to the nitrogen atom of the carboxamide group by an acyclic carbon atom
    • C07C233/38—Carboxylic acid amides having carbon atoms of carboxamide groups bound to hydrogen atoms or to acyclic carbon atoms having the nitrogen atom of at least one of the carboxamide groups bound to a carbon atom of a hydrocarbon radical substituted by amino groups with the substituted hydrocarbon radical bound to the nitrogen atom of the carboxamide group by an acyclic carbon atom having the carbon atom of the carboxamide group bound to a carbon atom of an acyclic unsaturated carbon skeleton
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    • C07C233/35—Carboxylic acid amides having carbon atoms of carboxamide groups bound to hydrogen atoms or to acyclic carbon atoms having the nitrogen atom of at least one of the carboxamide groups bound to a carbon atom of a hydrocarbon radical substituted by amino groups with the substituted hydrocarbon radical bound to the nitrogen atom of the carboxamide group by an acyclic carbon atom
    • C07C233/40—Carboxylic acid amides having carbon atoms of carboxamide groups bound to hydrogen atoms or to acyclic carbon atoms having the nitrogen atom of at least one of the carboxamide groups bound to a carbon atom of a hydrocarbon radical substituted by amino groups with the substituted hydrocarbon radical bound to the nitrogen atom of the carboxamide group by an acyclic carbon atom having the carbon atom of the carboxamide group bound to an acyclic carbon atom of a carbon skeleton containing six-membered aromatic rings
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    • C07C233/00—Carboxylic acid amides
    • C07C233/57—Carboxylic acid amides having carbon atoms of carboxamide groups bound to carbon atoms of rings other than six-membered aromatic rings
    • C07C233/58—Carboxylic acid amides having carbon atoms of carboxamide groups bound to carbon atoms of rings other than six-membered aromatic rings having the nitrogen atoms of the carboxamide groups bound to hydrogen atoms or to carbon atoms of unsubstituted hydrocarbon radicals
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    • C07C233/64—Carboxylic acid amides having carbon atoms of carboxamide groups bound to carbon atoms of six-membered aromatic rings
    • C07C233/77—Carboxylic acid amides having carbon atoms of carboxamide groups bound to carbon atoms of six-membered aromatic rings having the nitrogen atom of at least one of the carboxamide groups bound to a carbon atom of a hydrocarbon radical substituted by amino groups
    • C07C233/78—Carboxylic acid amides having carbon atoms of carboxamide groups bound to carbon atoms of six-membered aromatic rings having the nitrogen atom of at least one of the carboxamide groups bound to a carbon atom of a hydrocarbon radical substituted by amino groups with the substituted hydrocarbon radical bound to the nitrogen atom of the carboxamide group by an acyclic carbon atom
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    • C07C235/00—Carboxylic acid amides, the carbon skeleton of the acid part being further substituted by oxygen atoms
    • C07C235/02—Carboxylic acid amides, the carbon skeleton of the acid part being further substituted by oxygen atoms having carbon atoms of carboxamide groups bound to acyclic carbon atoms and singly-bound oxygen atoms bound to the same carbon skeleton
    • C07C235/04—Carboxylic acid amides, the carbon skeleton of the acid part being further substituted by oxygen atoms having carbon atoms of carboxamide groups bound to acyclic carbon atoms and singly-bound oxygen atoms bound to the same carbon skeleton the carbon skeleton being acyclic and saturated
    • C07C235/10—Carboxylic acid amides, the carbon skeleton of the acid part being further substituted by oxygen atoms having carbon atoms of carboxamide groups bound to acyclic carbon atoms and singly-bound oxygen atoms bound to the same carbon skeleton the carbon skeleton being acyclic and saturated having the nitrogen atom of at least one of the carboxamide groups bound to an acyclic carbon atom of a hydrocarbon radical substituted by nitrogen atoms not being part of nitro or nitroso groups
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    • C07C235/00—Carboxylic acid amides, the carbon skeleton of the acid part being further substituted by oxygen atoms
    • C07C235/40—Carboxylic acid amides, the carbon skeleton of the acid part being further substituted by oxygen atoms having carbon atoms of carboxamide groups bound to carbon atoms of rings other than six-membered aromatic rings and singly-bound oxygen atoms bound to the same carbon skeleton
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    • C07C237/00—Carboxylic acid amides, the carbon skeleton of the acid part being further substituted by amino groups
    • C07C237/02—Carboxylic acid amides, the carbon skeleton of the acid part being further substituted by amino groups having the carbon atoms of the carboxamide groups bound to acyclic carbon atoms of the carbon skeleton
    • C07C237/22—Carboxylic acid amides, the carbon skeleton of the acid part being further substituted by amino groups having the carbon atoms of the carboxamide groups bound to acyclic carbon atoms of the carbon skeleton having nitrogen atoms of amino groups bound to the carbon skeleton of the acid part, further acylated
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    • C07C311/00—Amides of sulfonic acids, i.e. compounds having singly-bound oxygen atoms of sulfo groups replaced by nitrogen atoms, not being part of nitro or nitroso groups
    • C07C311/01—Sulfonamides having sulfur atoms of sulfonamide groups bound to acyclic carbon atoms
    • C07C311/02—Sulfonamides having sulfur atoms of sulfonamide groups bound to acyclic carbon atoms of an acyclic saturated carbon skeleton
    • C07C311/03—Sulfonamides having sulfur atoms of sulfonamide groups bound to acyclic carbon atoms of an acyclic saturated carbon skeleton having the nitrogen atoms of the sulfonamide groups bound to hydrogen atoms or to acyclic carbon atoms
    • C07C311/05—Sulfonamides having sulfur atoms of sulfonamide groups bound to acyclic carbon atoms of an acyclic saturated carbon skeleton having the nitrogen atoms of the sulfonamide groups bound to hydrogen atoms or to acyclic carbon atoms to acyclic carbon atoms of hydrocarbon radicals substituted by nitrogen atoms, not being part of nitro or nitroso groups
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    • C07D205/00—Heterocyclic compounds containing four-membered rings with one nitrogen atom as the only ring hetero atom
    • C07D205/02—Heterocyclic compounds containing four-membered rings with one nitrogen atom as the only ring hetero atom not condensed with other rings
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    • C07D207/02—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
    • C07D207/04—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 no double bonds between ring members or between ring members and non-ring members
    • C07D207/10—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 no double bonds between ring members or between ring members and non-ring members 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 ring carbon atoms
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    • C07D213/04—Heterocyclic compounds containing six-membered rings, not condensed with other rings, with one nitrogen atom as the only ring hetero atom and three or more double bonds between ring members or between ring members and non-ring members having three double bonds between ring members or between ring members and non-ring members having no bond between the ring nitrogen atom and a non-ring member or having only hydrogen or carbon atoms directly attached to the ring nitrogen atom
    • C07D213/60—Heterocyclic compounds containing six-membered rings, not condensed with other rings, with one nitrogen atom as the only ring hetero atom and three or more double bonds between ring members or between ring members and non-ring members having three double bonds between ring members or between ring members and non-ring members having no bond between the ring nitrogen atom and a non-ring member or having only hydrogen or carbon atoms directly attached to the ring nitrogen atom 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 ring carbon atoms
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    • C07D307/04—Heterocyclic compounds containing five-membered rings having one oxygen atom as the only ring hetero atom not condensed with other rings having no double bonds between ring members or between ring members and non-ring members
    • C07D307/18—Heterocyclic compounds containing five-membered rings having one oxygen atom as the only ring hetero atom not condensed with other rings having no double bonds between ring members or between ring members and non-ring members 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 ring carbon atoms
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    • C07D309/08—Heterocyclic compounds containing six-membered rings having one oxygen atom as the only ring hetero atom, not condensed with other rings having no double bonds between ring members or between ring members and non-ring members 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 ring carbon atoms
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    • C07C2602/44—Systems containing two condensed rings the rings having more than two atoms in common the bicyclo ring system containing eight carbon atoms

Abstract

La descripción presenta composiciones y lípidos nuevos que involucran a los mismos. Las nanopartículas de lípidos (p. ej., LNP vacías o LNP cargadas) incluyen un lípido nuevo y lípidos adicionales como fosfolípidos, lípidos estructurales y lípidos PEG. Las nanopartículas de lípidos (p. ej., LNP vacías o LNP cargadas) que incluyen además productos terapéuticos y/o profilácticos como el ARN son útiles en la administración de productos terapéuticos y/o profilácticos a células u órganos de mamíferos para, por ejemplo, regular la expresión de polipéptidos, proteínas o genes. Reivindicación 1: Un lípido de fórmula (I-1) o su N-óxido, o una sal o isómero del mismo, en donde R’ᵃ es: un compuesto de fórmula (2) y R’ᵇ es: un compuesto de fórmula (3) o (4); en donde ⁻⁻⁻⁻⁻|⁻⁻⁻⁻⁻ denota un punto de unión; Rᵃᵇ, Rᵃᵍ y Rᵃᵈ se seleccionan cada uno independientemente del grupo que consiste en H, alquilo C₁₋₁₂ y alquenilo C₂₋₁₂, en donde al menos uno de Rᵃᵇ, Rᵃᵍ y Rᵃᵈ se selecciona del grupo que consiste en alquilo C₁₋₁₂ y alquenilo C₂₋₁₂; Rᵇᵇ, Rᵇᵍ y Rᵇᵈ se seleccionan cada uno independientemente del grupo que consiste en H, alquilo C₁₋₁₂ y alquenilo C₂₋₁₂, en donde al menos uno de Rᵇᵇ, Rᵇᵍ y Rᵇᵈ se selecciona del grupo que consiste en alquilo C₁₋₁₂ y alquenilo C₂₋₁₂; cada uno de R² y R³ se selecciona independientemente del grupo que consiste en alquilo C₁₋₁₄ y alquenilo C₂₋₁₄; R⁴ se selecciona de -(CH₂)ₙNRTQ, -(CH₂)ₙNRS(O)₂TQ, -(CH₂)ₙNRC(O)H y -(CH₂)ₙNRC(O)TQ en donde n se selecciona de 1, 2, 3, 4, y 5; T es un enlace o un enlazador de alquilo C₁₋₃, enlazador de alquenilo C₂₋₃ o enlazador de alquinilo C₂₋₃; Q se selecciona de un heterociclo de 3 - 14 miembros que contiene de 1 - 5 heteroátomos seleccionados de N, O y S, carbociclo C₃₋₁₀, alquilo C₁₋₆, alcoxi C₁₋₆ y alquenilo C₂₋₆, en donde el alquilo, alcoxi, alquenilo, heterociclo, y carbociclo están cada uno opcionalmente sustituidos con uno o más RQ; cada RQ se selecciona independientemente del grupo que consiste en oxo, hidroxilo, ciano, amino, alquilamino C₁₋₆, di-alquilamino C₁₋₆, alquilo C₁₋₆, alcoxi C₁₋₆, alquenilo C₂₋₆, alcanolilo C₁₋₆, carbociclo C₃₋₁₀, -C(O)alquilo C₁₋₆ y -NRC(O)alquilo C₁₋₆; cada R se selecciona independientemente de H, alquilo C₁₋₆ y alquenilo C₂₋₆; cada R’ se selecciona independientemente de alquilo C₁₋₁₂ y alquenilo C₂₋₁₂; m se selecciona de 1, 2, 3, 4, 5, 6, 7, 8 y 9; y l se selecciona de 1, 2, 3, 4, 5, 6, 7, 8 y 9. Reivindicación 2: Un lípido de fórmula (1) o su N-óxido, o una sal o isómero del mismo, en donde R’ᵃ es: un compuesto de fórmula (5) y R’ᵇ es: un compuesto de fórmula (3) o (6); en donde ⁻⁻⁻⁻⁻|⁻⁻⁻⁻⁻ denota un punto de unión; Rᵃᵍ y Rᵇᵍ son cada uno independientemente alquilo C₁₋₁₂ o alquenilo C₁₋₁₂; cada uno de R² y R³ se selecciona independientemente del grupo que consiste en alquilo C₁₋₁₄ y alquenilo C₂₋₁₄; R⁴ se selecciona de -(CH₂)ₙNRTQ, -(CH₂)ₙNRS(O)₂TQ, -(CH₂)ₙNRC(O)H y -(CH₂)ₙNRC(O)TQ en donde n se selecciona de 1, 2, 3, 4, y 5; T es un enlace o un enlazador de alquilo C₁₋₃, enlazador de alquenilo C₂₋₃ o enlazador de alquinilo C₂₋₃; Q se selecciona de un heterociclo de 3 - 14 miembros que contiene de 1 - 5 heteroátomos seleccionados de N, O y S, carbociclo C₃₋₁₀, alquilo C₁₋₆, alcoxi C₁₋₆ y alquenilo C₂₋₆, en donde el alquilo, alcoxi, alquenilo, heterociclo, y carbociclo están cada uno opcionalmente sustituidos con uno o más RQ; cada RQ se selecciona independientemente del grupo que consiste en oxo, hidroxilo, ciano, amino, alquilamino C₁₋₆, di-alquilamino C₁₋₆, alquilo C₁₋₆, alcoxi C₁₋₆, alquenilo C₂₋₆, alcanolilo C₁₋₆, carbociclo C₃₋₁₀, -C(O)alquilo C₁₋₆ y -NRC(O)alquilo C₁₋₆; cada R se selecciona independientemente de H, alquilo C₁₋₆ y alquenilo C₂₋₆; cada R’ se selecciona independientemente de alquilo C₁₋₁₂ y alquenilo C₂₋₁₂; m se selecciona de 3, 4, 5, 6 y 7; y l se selecciona de 3, 4, 5, 6 y 7.
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