AU2010260145B2 - Methods and compositions for treatment of Myotubular Myopathy using chimeric polypeptides comprising myotubularin 1 (MTM1) polypeptides - Google Patents

Methods and compositions for treatment of Myotubular Myopathy using chimeric polypeptides comprising myotubularin 1 (MTM1) polypeptides Download PDF

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Publication number
AU2010260145B2
AU2010260145B2 AU2010260145A AU2010260145A AU2010260145B2 AU 2010260145 B2 AU2010260145 B2 AU 2010260145B2 AU 2010260145 A AU2010260145 A AU 2010260145A AU 2010260145 A AU2010260145 A AU 2010260145A AU 2010260145 B2 AU2010260145 B2 AU 2010260145B2
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polypeptide
chimeric polypeptide
antibody
mtm1
antigen
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AU2010260145A
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AU2010260145A1 (en
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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 [IGs], e.g. monoclonal or polyclonal antibodies
    • C07K16/18—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans
    • C07K16/28—Immunoglobulins [IGs], 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 [IGs], e.g. monoclonal or polyclonal antibodies
    • C07K16/44—Immunoglobulins [IGs], 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)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Epidemiology (AREA)
  • Biophysics (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • Microbiology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Physical Education & Sports Medicine (AREA)
  • General Chemical & Material Sciences (AREA)
  • Neurology (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)
AU2010260145A 2009-06-15 2010-06-15 Methods and compositions for treatment of Myotubular Myopathy using chimeric polypeptides comprising myotubularin 1 (MTM1) polypeptides Ceased AU2010260145B2 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US26873209P 2009-06-15 2009-06-15
US61/268,732 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 (2)

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AU2010260145A1 AU2010260145A1 (en) 2012-02-02
AU2010260145B2 true AU2010260145B2 (en) 2015-08-20

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AU2010260145A Ceased AU2010260145B2 (en) 2009-06-15 2010-06-15 Methods and compositions for treatment of Myotubular Myopathy using chimeric polypeptides comprising myotubularin 1 (MTM1) polypeptides

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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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ES2744840T3 (es) 2011-04-01 2020-02-26 Univ Yale Anticuerpos anti-ADN que penetran en las células y usos de los mismos para inhibir la reparación del ADN
CA2907072A1 (en) * 2012-03-16 2013-09-19 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
AU2014218854B2 (en) 2013-02-20 2019-01-24 Valerion Therapeutics, Llc Methods and compositions for treatment of Pompe disease
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
JP6709733B2 (ja) * 2014-01-13 2020-06-17 バレリオン セラピューティクス， エルエルシー 内在化部分
EP3110844B1 (en) * 2014-02-28 2020-02-12 The U.S.A. as represented by the Secretary, Department of Health and Human Services Broadly neutralizing monoclonal antibodies against hiv-1 v1v2 env region
WO2015192092A1 (en) * 2014-06-13 2015-12-17 Valerion Therapeutics, Llc Methods and compositions for treatment of glycogen storage diseases and glycogen metabolism disorders
AU2015308894A1 (en) 2014-08-27 2017-03-23 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
CA3027960C (en) 2016-06-15 2022-06-14 Yale University Antibody-mediated autocatalytic, targeted delivery of nanocarriers to tumors
WO2017218824A1 (en) 2016-06-15 2017-12-21 Yale University Anti-guanosine antibody as a molecular delivery vehicle
JP2019535645A (ja) 2016-09-09 2019-12-12 バレリオン セラピューティクス， エルエルシー ラフォラ病の処置のための方法及び組成物
CA3067497A1 (en) * 2017-07-03 2019-01-10 Universite De Strasbourg Mtmr2-s polypeptide for use in the treatment of myopathies
KR20220101073A (ko) * 2019-08-30 2022-07-19 예일 유니버시티 핵산을 세포로 전달하기 위한 조성물 및 방법
JP2024508309A (ja) * 2021-03-03 2024-02-26 イエール ユニバーシティ 骨格筋疾患を治療するための組成物及び方法
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
JP2012529891A (ja) 2012-11-29
AU2010260145A1 (en) 2012-02-02
US20150218540A1 (en) 2015-08-06
ES2630057T3 (es) 2017-08-17
EP2443155A1 (en) 2012-04-25
CA2797480A1 (en) 2010-12-23
US20120213760A1 (en) 2012-08-23
BRPI1011236A2 (pt) 2019-07-09
US9447394B2 (en) 2016-09-20
EP2443155B1 (en) 2017-03-29
WO2010148010A1 (en) 2010-12-23
US8834866B2 (en) 2014-09-16
EP2443155A4 (en) 2012-12-26
JP6166041B2 (ja) 2017-07-19

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