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AR127659A1 - Lípidos catiónicos ionizables para la administración de arn - Google Patents

Lípidos catiónicos ionizables para la administración de arn

Info

Publication number
AR127659A1
AR127659A1 ARP220103119A ARP220103119A AR127659A1 AR 127659 A1 AR127659 A1 AR 127659A1 AR P220103119 A ARP220103119 A AR P220103119A AR P220103119 A ARP220103119 A AR P220103119A AR 127659 A1 AR127659 A1 AR 127659A1
Authority
AR
Argentina
Prior art keywords
alkyl
group
optionally substituted
alkoxy
substituents selected
Prior art date
Application number
ARP220103119A
Other languages
English (en)
Inventor
Kumar Rajappan
Steven Tanis
Amit Sagi
Priya Prakash Karmali
Padmanabh Chivukula
Original Assignee
Arcturus Therapeutics 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 Arcturus Therapeutics Inc filed Critical Arcturus Therapeutics Inc
Publication of AR127659A1 publication Critical patent/AR127659A1/es

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    • C07C—ACYCLIC OR CARBOCYCLIC COMPOUNDS
    • C07C333/00—Derivatives of thiocarbamic acids, i.e. compounds containing any of the groups, the nitrogen atom not being part of nitro or nitroso groups
    • C07C333/02—Monothiocarbamic acids; Derivatives thereof
    • C07C333/04—Monothiocarbamic acids; Derivatives thereof having nitrogen atoms of thiocarbamic groups bound to hydrogen atoms or to acyclic carbon atoms
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    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/54—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound
    • A61K47/543—Lipids, e.g. triglycerides; Polyamines, e.g. spermine or spermidine
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    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
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    • A61K47/08—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite containing oxygen, e.g. ethers, acetals, ketones, quinones, aldehydes, peroxides
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    • A61K47/30—Macromolecular organic or inorganic compounds, e.g. inorganic polyphosphates
    • A61K47/34—Macromolecular compounds obtained otherwise than by reactions only involving carbon-to-carbon unsaturated bonds, e.g. polyesters, polyamino acids, polysiloxanes, polyphosphazines, copolymers of polyalkylene glycol or poloxamers
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    • A61K47/69—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the conjugate being characterised by physical or galenical forms, e.g. emulsion, particle, inclusion complex, stent or kit
    • A61K47/6905—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the conjugate being characterised by physical or galenical forms, e.g. emulsion, particle, inclusion complex, stent or kit the form being a colloid or an emulsion
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    • C07C229/00—Compounds containing amino and carboxyl groups bound to the same carbon skeleton
    • C07C229/02—Compounds containing amino and carboxyl groups bound to the same carbon skeleton having amino and carboxyl groups bound to acyclic carbon atoms of the same carbon skeleton
    • C07C229/04—Compounds containing amino and carboxyl groups bound to the same carbon skeleton having amino and carboxyl groups bound to acyclic carbon atoms of the same carbon skeleton the carbon skeleton being acyclic and saturated
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    • C07C229/06—Compounds containing amino and carboxyl groups bound to the same carbon skeleton having amino and carboxyl groups bound to acyclic carbon atoms of the same carbon skeleton the carbon skeleton being acyclic and saturated having only one amino and one carboxyl group bound to the carbon skeleton
    • C07C229/10—Compounds containing amino and carboxyl groups bound to the same carbon skeleton having amino and carboxyl groups bound to acyclic carbon atoms of the same carbon skeleton the carbon skeleton being acyclic and saturated having only one amino and one carboxyl group bound to the carbon skeleton the nitrogen atom of the amino group being further bound to acyclic carbon atoms or to carbon atoms of rings other than six-membered aromatic rings
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    • C07C229/06—Compounds containing amino and carboxyl groups bound to the same carbon skeleton having amino and carboxyl groups bound to acyclic carbon atoms of the same carbon skeleton the carbon skeleton being acyclic and saturated having only one amino and one carboxyl group bound to the carbon skeleton
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    • C07C229/16—Compounds containing amino and carboxyl groups bound to the same carbon skeleton having amino and carboxyl groups bound to acyclic carbon atoms of the same carbon skeleton the carbon skeleton being acyclic and saturated having only one amino and one carboxyl group bound to the carbon skeleton the nitrogen atom of the amino group being further bound to acyclic carbon atoms or to carbon atoms of rings other than six-membered aromatic rings to carbon atoms of hydrocarbon radicals substituted by amino or carboxyl groups, e.g. ethylenediamine-tetra-acetic acid, iminodiacetic acids
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    • C07C—ACYCLIC OR CARBOCYCLIC COMPOUNDS
    • C07C271/00—Derivatives of carbamic acids, i.e. compounds containing any of the groups, the nitrogen atom not being part of nitro or nitroso groups
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    • C07C271/20—Esters of carbamic acids having oxygen atoms of carbamate groups bound to acyclic carbon atoms with the nitrogen atoms of the carbamate groups bound to hydrogen atoms or to acyclic carbon atoms to carbon atoms of hydrocarbon radicals substituted by nitrogen atoms not being part of nitro or nitroso groups
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    • C07C275/16—Derivatives of urea, i.e. compounds containing any of the groups, the nitrogen atoms not being part of nitro or nitroso groups having nitrogen atoms of urea groups bound to acyclic carbon atoms of an acyclic and saturated carbon skeleton being further substituted by carboxyl groups
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Abstract

Reivindicación 1: Un compuesto de fórmula (1) o una sal farmacéuticamente aceptable de este, en donde: cada uno de R¹ y R² es independientemente H o alquilo C₁₋₆; o R¹ y R² se unen para formar un anillo heterocíclico saturado, en donde: R¹ es un alquileno C₁₋₄ lineal; y R² es -(CH₂)ₘ(X)ₙ-, en donde X es O, S o NR⁹, en donde R⁹ es H o alquilo C₁₋₆; m es 1, 2, 3 o 4, y n es 0 o 1; L¹ es un alquileno C₁₋₆ lineal opcionalmente sustituido con uno a tres grupos metilo; Y se selecciona del grupo que consiste en: los compuestos del grupo de fórmulas (2); en donde: cada asterisco (*) indica el átomo unido a L² y L³; y R¹⁰ es H o alquilo C₁₋₆; L² y L³ son cada uno independientemente un alquileno C₁₋₈ lineal; L⁴, L⁵, L⁶, L⁷, L⁸ y L⁹ están cada uno independientemente ausentes o son -CH₂-, siempre que: al menos dos de L⁴, L⁶ y L⁸ sean -CH₂-; y al menos dos de L⁵, L⁷ y L⁹ sean -CH₂-; cada uno de R³ y R⁴ es independientemente H, metilo o etilo; y R⁵, R⁶, R⁷ y R⁸ se seleccionan cada uno independientemente del grupo que consiste en: alquilo C₁₋₂₀ lineal, en donde cada alquilo C₁₋₂₀ lineal está opcionalmente sustituido con uno o más sustituyentes seleccionados del grupo que consiste en: alquilo C₁₋₆, alcoxi C₁₋₆ y -F, en donde cada sustituyente alquilo C₁₋₆ está opcionalmente sustituido con uno o más grupos seleccionados del grupo que consiste en alcoxi C₁₋₃ y -F; monocicloalquilo C₃₋₈, en donde cada monocicloalquilo C₃₋₈ está opcionalmente sustituido con uno o más sustituyentes seleccionados del grupo que consiste en alquilo C₁₋₆, alcoxi C₁₋₆ y -F; bicicloalquilo C₇₋₁₂, en donde cada bicicloalquilo C₇₋₁₂ está opcionalmente sustituido con uno o más sustituyentes seleccionados del grupo que consiste en alquilo C₁₋₆, alcoxi C₁₋₆ y -F; y arilo C₆₋₁₀, en donde cada arilo C₆₋₁₀ es un hidrocarburo aromático monocíclico o bicíclico opcionalmente sustituido con uno o más sustituyentes seleccionados del grupo que consiste en alquilo C₁₋₆, alcoxi C₁₋₆ y -F; monocicloalquilo C₃₋₈, en donde cada monocicloalquilo C₃₋₈ está opcionalmente sustituido con uno o más sustituyentes seleccionados del grupo que consiste en alquilo C₁₋₆, alcoxi C₁₋₆ y -F; bicicloalquilo C₇₋₁₂, en donde cada bicicloalquilo C₇₋₁₂ está opcionalmente sustituido con uno o más sustituyentes seleccionados del grupo que consiste en alquilo C₁₋₆, alcoxi C₁₋₆ y -F; y arilo C₆₋₁₀, en donde cada arilo C₆₋₁₀ es un hidrocarburo aromático monocíclico o bicíclico opcionalmente sustituido con uno o más sustituyentes seleccionados del grupo que consiste en alquilo C₁₋₆, alcoxi C₁₋₆ y -F.
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