BR112013030432A2 - método para preparar um peptídeo conjugado alvo para uma enzima lisossomal recombinante, método para preparar uma molécula para terapia de reposição de enzima, conjugado, método para tratar um indivíduo sofrendo de uma doença de armazenamento lisossomal, método para tratar um indivíduo sofrendo de uma doença de armazenamento lisossomal, método para tratar um paciente sofrendo da doença de pompe, doença de fabry, e doença de gaucher, mps i, mps ii, mps vii, tay sachs, sandhoff, alfa-manosidose, e wohlman, sequência de dna e sequência de aminoácidos - Google Patents

método para preparar um peptídeo conjugado alvo para uma enzima lisossomal recombinante, método para preparar uma molécula para terapia de reposição de enzima, conjugado, método para tratar um indivíduo sofrendo de uma doença de armazenamento lisossomal, método para tratar um indivíduo sofrendo de uma doença de armazenamento lisossomal, método para tratar um paciente sofrendo da doença de pompe, doença de fabry, e doença de gaucher, mps i, mps ii, mps vii, tay sachs, sandhoff, alfa-manosidose, e wohlman, sequência de dna e sequência de aminoácidos

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Publication number
BR112013030432A2
BR112013030432A2 BR112013030432A BR112013030432A BR112013030432A2 BR 112013030432 A2 BR112013030432 A2 BR 112013030432A2 BR 112013030432 A BR112013030432 A BR 112013030432A BR 112013030432 A BR112013030432 A BR 112013030432A BR 112013030432 A2 BR112013030432 A2 BR 112013030432A2
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Prior art keywords
disease
treating
mps
preparing
lysosomal
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BR112013030432A
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English (en)
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Hung Do
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Callidus Biopharma Inc
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    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K1/00—General methods for the preparation of peptides, i.e. processes for the organic chemical preparation of peptides or proteins of any length
    • C07K1/107—General methods for the preparation of peptides, i.e. processes for the organic chemical preparation of peptides or proteins of any length by chemical modification of precursor peptides
    • 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
    • A61K47/68—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 antibody, an immunoglobulin or a fragment thereof, e.g. an Fc-fragment
    • A61K47/6835—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 antibody, an immunoglobulin or a fragment thereof, e.g. an Fc-fragment the modifying agent being an antibody or an immunoglobulin bearing at least one antigen-binding site
    • A61K47/6847—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 antibody, an immunoglobulin or a fragment thereof, e.g. an Fc-fragment the modifying agent being an antibody or an immunoglobulin bearing at least one antigen-binding site the antibody targeting a hormone or a hormone-releasing or -inhibiting factor
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A61K38/47—Hydrolases (3) acting on glycosyl compounds (3.2), e.g. cellulases, lactases
    • 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
    • A61K47/62—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 a protein, peptide or polyamino acid
    • A61K47/64—Drug-peptide, drug-protein or drug-polyamino acid conjugates, i.e. the modifying agent being a peptide, protein or polyamino acid which is covalently bonded or complexed to a therapeutically active agent
    • A61K47/642—Drug-peptide, drug-protein or drug-polyamino acid conjugates, i.e. the modifying agent being a peptide, protein or polyamino acid which is covalently bonded or complexed to a therapeutically active agent the peptide or protein in the drug conjugate being a cytokine, e.g. IL2, chemokine, growth factors or interferons being the inactive part of the conjugate
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/08—Drugs for disorders of the metabolism for glucose homeostasis
    • 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
    • C07K—PEPTIDES
    • C07K1/00—General methods for the preparation of peptides, i.e. processes for the organic chemical preparation of peptides or proteins of any length
    • C07K1/14—Extraction; Separation; Purification
    • C07K1/16—Extraction; Separation; Purification by chromatography
    • C07K1/18—Ion-exchange chromatography
    • C—CHEMISTRY; METALLURGY
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    • C07K1/00—General methods for the preparation of peptides, i.e. processes for the organic chemical preparation of peptides or proteins of any length
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    • C07K1/16—Extraction; Separation; Purification by chromatography
    • C07K1/22—Affinity chromatography or related techniques based upon selective absorption processes
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
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    • C07K1/00—General methods for the preparation of peptides, i.e. processes for the organic chemical preparation of peptides or proteins of any length
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    • C07K1/30—Extraction; Separation; Purification by precipitation
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    • C07K1/00—General methods for the preparation of peptides, i.e. processes for the organic chemical preparation of peptides or proteins of any length
    • C07K1/14—Extraction; Separation; Purification
    • C07K1/30—Extraction; Separation; Purification by precipitation
    • C07K1/306—Extraction; Separation; Purification by precipitation by crystallization
    • C—CHEMISTRY; METALLURGY
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    • C07K1/00—General methods for the preparation of peptides, i.e. processes for the organic chemical preparation of peptides or proteins of any length
    • C07K1/14—Extraction; Separation; Purification
    • C07K1/36—Extraction; Separation; Purification by a combination of two or more processes of different types
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
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    • C07—ORGANIC CHEMISTRY
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    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/575—Hormones
    • C07K14/65—Insulin-like growth factors, i.e. somatomedins, e.g. IGF-1, IGF-2
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/24—Hydrolases (3) acting on glycosyl compounds (3.2)
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/96—Stabilising an enzyme by forming an adduct or a composition; Forming enzyme conjugates
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    • C07K2319/00—Fusion polypeptide
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    • C07K2319/21—Fusion polypeptide containing a tag with affinity for a non-protein ligand containing a His-tag
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
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    • C07K2319/00—Fusion polypeptide
    • C07K2319/50—Fusion polypeptide containing protease site

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Abstract

resumo “método para preparar um peptídeo conjugado alvo para uma enzima lisossomal recombinante, método para preparar uma molécula para terapia de reposição de enzima, conjugado, método para tratar um indivíduo sofrendo de uma doença de armazenamento lisossomal, método para tratar um indivíduo sofrendo de uma doença de armazenamento lisossomal, método para tratar um paciente sofrendo da doença de pompe, doença de fabry, e doença de gaucher, mps i, mps ii, mps vii, tay sachs, sandhoff, ?-manosidose, e wohlman, sequência de dna e sequência de aminoácidos” são descritos aqui métodos para preparar peptídeos conjugados alvos para enzimas lisossomais recombinantes através da modificação do (n)-terminal amino e um ou mais resíduos de lisina nas enzimas lisossomais humanas recombinantes usando um primeiro agente reticulante para obter enzimas lisossomal humana recombinante modificada pelo primeiro agente reticulante, modificando o primeiro aminoácido dentro de um ligante curto no (n)-terminal amino em um peptídeo igf-2 variante usando um segundo agente reticulante para obter um peptídeo igf-2 variante modificado pelo segundo agente reticulante, e então conjugar a enzima lisossomal humana recombinante modificada pelo primeiro agente reticulante ao peptídeo igf-2 variante modificado pelo segundo agente reticulante contendo um ligante curto. também são descritos aqui os conjugados sintetizados caracterizados como tendo afinidade maior para o receptor igf2/ci-mpr e absorção celular usando os métodos descritos aqui. são descritos ainda, métodos para tratamento usando os conjugados descritos. 1
BR112013030432A 2011-05-27 2012-05-25 método para preparar um peptídeo conjugado alvo para uma enzima lisossomal recombinante, método para preparar uma molécula para terapia de reposição de enzima, conjugado, método para tratar um indivíduo sofrendo de uma doença de armazenamento lisossomal, método para tratar um indivíduo sofrendo de uma doença de armazenamento lisossomal, método para tratar um paciente sofrendo da doença de pompe, doença de fabry, e doença de gaucher, mps i, mps ii, mps vii, tay sachs, sandhoff, alfa-manosidose, e wohlman, sequência de dna e sequência de aminoácidos BR112013030432A2 (pt)

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US201161490957P 2011-05-27 2011-05-27
PCT/US2012/039705 WO2012166653A2 (en) 2011-05-27 2012-05-25 Methods for coupling targeting peptides onto recombinant lysosomal enzymes for improved treatments of lysosomal storage diseases

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BR112013030432A2 true BR112013030432A2 (pt) 2016-12-13

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BR112013030432A BR112013030432A2 (pt) 2011-05-27 2012-05-25 método para preparar um peptídeo conjugado alvo para uma enzima lisossomal recombinante, método para preparar uma molécula para terapia de reposição de enzima, conjugado, método para tratar um indivíduo sofrendo de uma doença de armazenamento lisossomal, método para tratar um indivíduo sofrendo de uma doença de armazenamento lisossomal, método para tratar um paciente sofrendo da doença de pompe, doença de fabry, e doença de gaucher, mps i, mps ii, mps vii, tay sachs, sandhoff, alfa-manosidose, e wohlman, sequência de dna e sequência de aminoácidos

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JP (2) JP6300720B2 (pt)
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BR (1) BR112013030432A2 (pt)
CA (1) CA2836318C (pt)
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