BR112018068230A2 - ligantes de direcionamento para compostos terapêuticos - Google Patents

ligantes de direcionamento para compostos terapêuticos

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Publication number
BR112018068230A2
BR112018068230A2 BR112018068230A BR112018068230A BR112018068230A2 BR 112018068230 A2 BR112018068230 A2 BR 112018068230A2 BR 112018068230 A BR112018068230 A BR 112018068230A BR 112018068230 A BR112018068230 A BR 112018068230A BR 112018068230 A2 BR112018068230 A2 BR 112018068230A2
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BR
Brazil
Prior art keywords
compounds
targeting
expression
ligands disclosed
inhibiting oligomeric
Prior art date
Application number
BR112018068230A
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English (en)
Other versions
BR112018068230B1 (pt
Inventor
V Blokhin Andrei
H Wakefield Darren
B Rozema David
Fleitz Fred
D Benson Jonathan
Pei Tao
Li Zhen
Original Assignee
Arrowhead Pharmaceuticals 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.)
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Publication date
Application filed by Arrowhead Pharmaceuticals Inc filed Critical Arrowhead Pharmaceuticals Inc
Publication of BR112018068230A2 publication Critical patent/BR112018068230A2/pt
Publication of BR112018068230B1 publication Critical patent/BR112018068230B1/pt

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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • 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
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—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
    • 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
    • A61K47/549—Sugars, nucleosides, nucleotides or nucleic acids
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0008—Medicinal 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
    • A61K48/0025—Medicinal 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
    • A61K48/0033—Medicinal 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H15/00—Compounds containing hydrocarbon or substituted hydrocarbon radicals directly attached to hetero atoms of saccharide radicals
    • C07H15/02—Acyclic radicals, not substituted by cyclic structures
    • C07H15/04—Acyclic radicals, not substituted by cyclic structures attached to an oxygen atom of the saccharide radical
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/317—Chemical structure of the backbone with an inverted bond, e.g. a cap structure
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/3521—Methyl
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    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
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    • C12N2310/3525—MOE, methoxyethoxy
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    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/353—Nature of the modification linked to the nucleic acid via an atom other than carbon
    • C12N2310/3533—Halogen

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  • Health & Medical Sciences (AREA)
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  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Biochemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Zoology (AREA)
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  • Crystallography & Structural Chemistry (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
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  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
  • Medicinal Preparation (AREA)
  • Organic Low-Molecular-Weight Compounds And Preparation Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Saccharide Compounds (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)

Abstract

são descritos novos ligantes de direcionamento que podem ser ligados a compostos, tais compostos terapêuticos, que são úteis em direcionar os compostos ao alvo in vivo. os ligantes de direcionamento aqui divulgados podem servir para direcionar compostos oligoméricos inibidores de expressão, tais como agentes rnai, às células do fígado para modular a expressão genética. os ligantes de direcionamento aqui divulgados, quando conjugados a um composto oligomérico inibidor de expressão, podem ser utilizados em uma variedade de aplicações, incluindo o uso em aplicações terapêuticas, diagnósticas, validação de alvo e descoberta genômica. composições incluindo os ligantes de direcionamento aqui divulgados quando ligados a compostos oligoméricos inibidores de expressão são capazes de mediar a expressão de sequências de ácido nucleico alvo em células do fígado, tais como hepatócitos, que podem ser úteis no tratamento de doenças ou condições que respondem a inibição de expressão genética ou atividade em uma célula, tecido ou organismo.
BR112018068230-6A 2016-03-07 2017-03-07 Ligantes de direcionamento, compostos, composição que compreende os ditos ligantes, uso terapêutico dos compostos, da composição e dos ligantes de direcionamento e ainda método que utiliza os ditos ligantes BR112018068230B1 (pt)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US201662304652P 2016-03-07 2016-03-07
US62/304,652 2016-03-07
US201662370754P 2016-08-04 2016-08-04
US62/370,754 2016-08-04
US201662426916P 2016-11-28 2016-11-28
US62/426,916 2016-11-28
PCT/US2017/021175 WO2017156012A1 (en) 2016-03-07 2017-03-07 Targeting ligands for therapeutic compounds

Publications (2)

Publication Number Publication Date
BR112018068230A2 true BR112018068230A2 (pt) 2019-07-30
BR112018068230B1 BR112018068230B1 (pt) 2024-02-27

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BR112018068230-6A BR112018068230B1 (pt) 2016-03-07 2017-03-07 Ligantes de direcionamento, compostos, composição que compreende os ditos ligantes, uso terapêutico dos compostos, da composição e dos ligantes de direcionamento e ainda método que utiliza os ditos ligantes

Country Status (14)

Country Link
US (3) US10246709B2 (pt)
EP (1) EP3426261A4 (pt)
JP (2) JP6983797B2 (pt)
KR (3) KR20230048446A (pt)
CN (2) CN109069529B (pt)
AU (2) AU2017229469B2 (pt)
BR (1) BR112018068230B1 (pt)
CA (1) CA3011946A1 (pt)
IL (3) IL296377A (pt)
JO (2) JOP20170057B1 (pt)
MX (3) MX2018009854A (pt)
TW (2) TWI783434B (pt)
UY (1) UY37146A (pt)
WO (1) WO2017156012A1 (pt)

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