ES2630057T3 - Métodos y composiciones para el tratamiento de la miopatía miotubular usando polipéptidos quiméricos que comprenden polipéptidos de miotubularina 1 (mtm1) - Google Patents

Métodos y composiciones para el tratamiento de la miopatía miotubular usando polipéptidos quiméricos que comprenden polipéptidos de miotubularina 1 (mtm1) Download PDF

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Publication number
ES2630057T3
ES2630057T3 ES10790070.6T ES10790070T ES2630057T3 ES 2630057 T3 ES2630057 T3 ES 2630057T3 ES 10790070 T ES10790070 T ES 10790070T ES 2630057 T3 ES2630057 T3 ES 2630057T3
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mtm1
polypeptide
amino acid
internalizing
seq
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ES10790070.6T
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Spanish (es)
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Dustin D. Armstrong
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Valerion Therapeutics LLC
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Valerion Therapeutics LLC
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    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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)
    • 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
    • A61K47/6801—Drug-antibody or immunoglobulin conjugates defined by the pharmacologically or therapeutically active agent
    • A61K47/6803—Drugs conjugated to an antibody or immunoglobulin, e.g. cisplatin-antibody conjugates
    • A61K47/6811—Drugs conjugated to an antibody or immunoglobulin, e.g. cisplatin-antibody conjugates the drug being a protein or peptide, e.g. transferrin or bleomycin
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K16/00—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies
    • C07K16/18—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans
    • C07K16/28—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K16/00—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies
    • C07K16/44—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material not provided for elsewhere, e.g. haptens, metals, DNA, RNA, amino acids
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K2039/505—Medicinal preparations containing antigens or antibodies comprising antibodies
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2317/00—Immunoglobulins specific features
    • C07K2317/20—Immunoglobulins specific features characterized by taxonomic origin
    • C07K2317/24—Immunoglobulins specific features characterized by taxonomic origin containing regions, domains or residues from different species, e.g. chimeric, humanized or veneered
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2317/00—Immunoglobulins specific features
    • C07K2317/50—Immunoglobulins specific features characterized by immunoglobulin fragments
    • C07K2317/54—F(ab')2
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2317/00—Immunoglobulins specific features
    • C07K2317/50—Immunoglobulins specific features characterized by immunoglobulin fragments
    • C07K2317/55—Fab or Fab'
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2317/00—Immunoglobulins specific features
    • C07K2317/50—Immunoglobulins specific features characterized by immunoglobulin fragments
    • C07K2317/56—Immunoglobulins specific features characterized by immunoglobulin fragments variable (Fv) region, i.e. VH and/or VL
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2317/00—Immunoglobulins specific features
    • C07K2317/70—Immunoglobulins specific features characterized by effect upon binding to a cell or to an antigen
    • C07K2317/77—Internalization into the cell
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/33—Fusion polypeptide fusions for targeting to specific cell types, e.g. tissue specific targeting, targeting of a bacterial subspecies
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y301/00—Hydrolases acting on ester bonds (3.1)
    • C12Y301/03—Phosphoric monoester hydrolases (3.1.3)
    • C12Y301/03064—Phosphatidylinositol-3-phosphatase (3.1.3.64)

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  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Immunology (AREA)
  • Genetics & Genomics (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Epidemiology (AREA)
  • Biophysics (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Biotechnology (AREA)
  • Microbiology (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Neurology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Peptides Or Proteins (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
ES10790070.6T 2009-06-15 2010-06-15 Métodos y composiciones para el tratamiento de la miopatía miotubular usando polipéptidos quiméricos que comprenden polipéptidos de miotubularina 1 (mtm1) Active ES2630057T3 (es)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US26873209P 2009-06-15 2009-06-15
US268732P 2009-06-15
PCT/US2010/038703 WO2010148010A1 (en) 2009-06-15 2010-06-15 Methods and compositions for treatment of myotubular myopathy using chimeric polypeptides comprising myotubularih 1 (mtm1) polypeptides

Publications (1)

Publication Number Publication Date
ES2630057T3 true ES2630057T3 (es) 2017-08-17

Family

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Family Applications (1)

Application Number Title Priority Date Filing Date
ES10790070.6T Active ES2630057T3 (es) 2009-06-15 2010-06-15 Métodos y composiciones para el tratamiento de la miopatía miotubular usando polipéptidos quiméricos que comprenden polipéptidos de miotubularina 1 (mtm1)

Country Status (8)

Country Link
US (2) US8834866B2 (enExample)
EP (1) EP2443155B1 (enExample)
JP (1) JP6166041B2 (enExample)
AU (1) AU2010260145B2 (enExample)
BR (1) BRPI1011236A2 (enExample)
CA (1) CA2797480A1 (enExample)
ES (1) ES2630057T3 (enExample)
WO (1) WO2010148010A1 (enExample)

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AU2010260145B2 (en) 2009-06-15 2015-08-20 Valerion Therapeutics, Llc Methods and compositions for treatment of Myotubular Myopathy using chimeric polypeptides comprising myotubularin 1 (MTM1) polypeptides
JP6178785B2 (ja) 2011-04-01 2017-08-09 イェール ユニバーシティーＹａｌｅ Ｕｎｉｖｅｒｓｉｔｙ 細胞浸透性抗ｄｎａ抗体およびｄｎａ修復を阻害するためのその使用
US9610362B2 (en) 2012-03-16 2017-04-04 Valerion Therapeutics, Llc Antisense conjugates for decreasing expression of DMPK
US9732146B2 (en) * 2012-03-30 2017-08-15 The United States Of America As Represented By The Department Of Veterans Affairs Antibody-mediated transduction of heat shock proteins into living cells
US20150152170A1 (en) * 2012-05-23 2015-06-04 Valerion Therapeutics, Llc Methods for increasing muscle contractility
JP2016514096A (ja) 2013-02-20 2016-05-19 バレリオン セラピューティクス， エルエルシー ポンぺ病の処置のための方法および組成物
US8957044B2 (en) 2013-03-01 2015-02-17 Wake Forest University Health Sciences Systemic gene replacement therapy for treatment of X-linked myotubular myopathy (XLMTM)
FR3004463A1 (fr) 2013-04-11 2014-10-17 Genethon Systeme d'expression pour une therapie genique selective
WO2015106290A1 (en) 2014-01-13 2015-07-16 Valerion Therapeutics, Llc Internalizing moieties
WO2015128846A1 (en) * 2014-02-28 2015-09-03 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Broadly neutralizing monoclonal antibodies against hiv-1 v1v2 env region
EP3154570A4 (en) 2014-06-13 2017-12-27 Valerion Therapeutics, LLC Methods and compositions for treatment of glycogen storage diseases and glycogen metabolism disorders
CA2995673A1 (en) 2014-08-27 2016-03-03 Valerion Therapeutics, Llc Internalizing moieties for treatment of cancer
US10383945B2 (en) 2015-02-18 2019-08-20 The United States of America as Represented by the Department of Verterans Affairs Methods for DNA-dependent targeting of a cell permeant antibody
US12486318B2 (en) 2016-06-15 2025-12-02 Yale University Anti-guanosine antibody as a molecular delivery vehicle
EP3471777A1 (en) 2016-06-15 2019-04-24 Yale University Antibody-mediated autocatalytic, targeted delivery of nanocarriers to tumors
CA3036524A1 (en) * 2016-09-09 2018-03-15 Valerion Therapeutics, Llc Methods and compositions for treatment of lafora disease
JP2020529836A (ja) * 2017-07-03 2020-10-15 ユニヴェルシテ・ドゥ・ストラスブール ミオパチーの処置における使用のためのｍｔｍｒ２−ｓポリペプチド
KR20220101073A (ko) * 2019-08-30 2022-07-19 예일 유니버시티 핵산을 세포로 전달하기 위한 조성물 및 방법
KR20230154921A (ko) * 2021-03-03 2023-11-09 예일 유니버시티 골격근 질환을 치료하기 위한 조성물 및 방법
GB202201713D0 (en) * 2022-02-10 2022-03-30 Univ Dundee An affinity directed phosphatase system for targeted protein dephosphorylation
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Also Published As

Publication number Publication date
US20150218540A1 (en) 2015-08-06
EP2443155B1 (en) 2017-03-29
US20120213760A1 (en) 2012-08-23
US8834866B2 (en) 2014-09-16
US9447394B2 (en) 2016-09-20
WO2010148010A1 (en) 2010-12-23
EP2443155A1 (en) 2012-04-25
EP2443155A4 (en) 2012-12-26
JP6166041B2 (ja) 2017-07-19
AU2010260145A1 (en) 2012-02-02
BRPI1011236A2 (pt) 2019-07-09
CA2797480A1 (en) 2010-12-23
JP2012529891A (ja) 2012-11-29
AU2010260145B2 (en) 2015-08-20

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