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

Info

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
Authority
ES
Spain
Prior art keywords
mtm1
polypeptide
amino acid
internalizing
seq
Prior art date
Legal status (The legal status 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 status listed.)
Active
Application number
ES10790070.6T
Other languages
English (en)
Spanish (es)
Inventor
Dustin D. Armstrong
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Valerion Therapeutics LLC
Original Assignee
Valerion Therapeutics LLC
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 Valerion Therapeutics LLC filed Critical Valerion Therapeutics LLC
Application granted granted Critical
Publication of ES2630057T3 publication Critical patent/ES2630057T3/es
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Classifications

    • 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)

Landscapes

  • 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)
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

ID=43356726

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)

Families Citing this family (21)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
BRPI1011236A2 (pt) 2009-06-15 2019-07-09 4S3 Bioscience Inc métodos e composições para tratamento de miopatia miotubular usando polipeptídeos quiméricos compreendendo polipeptídeos miotubularesih 1 (mtm1)
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
WO2024238807A2 (en) 2023-05-16 2024-11-21 Affinia Therapeutics Inc. Recombinant aavs with improved tropism and specificity

Family Cites Families (34)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4179337A (en) 1973-07-20 1979-12-18 Davis Frank F Non-immunogenic polypeptides
JPS6023084B2 (ja) 1979-07-11 1985-06-05 味の素株式会社 代用血液
US4640835A (en) 1981-10-30 1987-02-03 Nippon Chemiphar Company, Ltd. Plasminogen activator derivatives
US4496689A (en) 1983-12-27 1985-01-29 Miles Laboratories, Inc. Covalently attached complex of alpha-1-proteinase inhibitor with a water soluble polymer
EP0206448B1 (en) 1985-06-19 1990-11-14 Ajinomoto Co., Inc. Hemoglobin combined with a poly(alkylene oxide)
US4791192A (en) 1986-06-26 1988-12-13 Takeda Chemical Industries, Ltd. Chemically modified protein with polyethyleneglycol
US5223409A (en) 1988-09-02 1993-06-29 Protein Engineering Corp. Directed evolution of novel binding proteins
US5198346A (en) 1989-01-06 1993-03-30 Protein Engineering Corp. Generation and selection of novel DNA-binding proteins and polypeptides
US5096815A (en) 1989-01-06 1992-03-17 Protein Engineering Corporation Generation and selection of novel dna-binding proteins and polypeptides
US5264618A (en) 1990-04-19 1993-11-23 Vical, Inc. Cationic lipids for intracellular delivery of biologically active molecules
AU7979491A (en) 1990-05-03 1991-11-27 Vical, Inc. Intracellular delivery of biologically active substances by means of self-assembling lipid complexes
US5173414A (en) 1990-10-30 1992-12-22 Applied Immune Sciences, Inc. Production of recombinant adeno-associated virus vectors
US5541087A (en) 1994-09-14 1996-07-30 Fuji Immunopharmaceuticals Corporation Expression and export technology of proteins as immunofusins
US5622699A (en) 1995-09-11 1997-04-22 La Jolla Cancer Research Foundation Method of identifying molecules that home to a selected organ in vivo
US6068829A (en) 1995-09-11 2000-05-30 The Burnham Institute Method of identifying molecules that home to a selected organ in vivo
JP2000507225A (ja) 1996-03-08 2000-06-13 ザ リージェンツ オブ ザ ユニバーシティー オブ カリフォルニア ｍａｂ ３Ｅ１０ ならびにその突然変異体および／または機能性フラグメントを使用する送達システム
US6329501B1 (en) 1997-05-29 2001-12-11 Auburn University Methods and compositions for targeting compounds to muscle
US6180084B1 (en) 1998-08-25 2001-01-30 The Burnham Institute NGR receptor and methods of identifying tumor homing molecules that home to angiogenic vasculature using same
JPH11151088A (ja) 1997-11-20 1999-06-08 Univ Tokyo 脱リン酸化反応を触媒する新規酵素
US6232287B1 (en) 1998-03-13 2001-05-15 The Burnham Institute Molecules that home to various selected organs or tissues
US6174687B1 (en) 1999-02-26 2001-01-16 The Burnham Institute Methods of identifying lung homing molecules using membrane dipeptidase
US6303573B1 (en) 1999-06-07 2001-10-16 The Burnham Institute Heart homing peptides and methods of using same
US6551809B2 (en) 2001-03-20 2003-04-22 Applera Corporation Isolated human phosphatase proteins, nucleic acid molecules encoding human phophatase proteins, and uses thereof
US6992174B2 (en) 2001-03-30 2006-01-31 Emd Lexigen Research Center Corp. Reducing the immunogenicity of fusion proteins
US7316998B2 (en) * 2004-05-27 2008-01-08 Acceleron Pharma Inc. Cerberus/Coco derivatives and uses thereof
JP2010516708A (ja) * 2007-01-22 2010-05-20 アメリカ合衆国 抗体結合体の使用
EP2164875A4 (en) 2007-05-24 2011-11-30 Us Dept Veterans Affairs INTRANUCLEAR PROTEIN TRANSDUCTION THROUGH A NUCLEOSIDE SALVAGE PATHWAY
WO2010044894A1 (en) 2008-10-15 2010-04-22 4S3 Bioscience Inc. Methods and compositions for treatment of myotonic dystrophy
CA2799608C (en) 2009-05-28 2023-02-28 Richard H. Weisbart Amino acid sequences which enhance peptide conjugate solubility
BRPI1011236A2 (pt) 2009-06-15 2019-07-09 4S3 Bioscience Inc métodos e composições para tratamento de miopatia miotubular usando polipeptídeos quiméricos compreendendo polipeptídeos miotubularesih 1 (mtm1)
GB2476671B (en) 2010-01-04 2014-11-26 Plastic Logic Ltd Touch-sensing systems
JP5240316B2 (ja) 2010-10-26 2013-07-17 株式会社デンソー 車両乗員非操作運転システム
KR20140035357A (ko) 2011-04-05 2014-03-21 셰브런 오로나이트 컴퍼니 엘엘씨 저점도 선박 실린더 윤활유 조성물
US20150152170A1 (en) 2012-05-23 2015-06-04 Valerion Therapeutics, Llc Methods for increasing muscle contractility

Also Published As

Publication number Publication date
JP2012529891A (ja) 2012-11-29
AU2010260145A1 (en) 2012-02-02
US20150218540A1 (en) 2015-08-06
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
AU2010260145B2 (en) 2015-08-20

Similar Documents

Publication Publication Date Title
ES2630057T3 (es) Métodos y composiciones para el tratamiento de la miopatía miotubular usando polipéptidos quiméricos que comprenden polipéptidos de miotubularina 1 (mtm1)
US10017581B2 (en) Methods and compositions for treatment of Pompe disease
US9114178B2 (en) Methods and compositions for treatment of myotonic dystrophy
US20180028676A1 (en) Methods and compositions for treatment of forbes-cori disease
US10202591B2 (en) Methods and compositions for treatment of glycogen storage diseases and glycogen metabolism disorders
US20130053322A1 (en) Hsa-related compositions and methods of use
US20150152170A1 (en) Methods for increasing muscle contractility
JP2014510518A (ja) Ｈｓａ関連組成物および使用方法
US20210040464A1 (en) Methods and compositions for treatment of polyglucosan disorders
HK1169663B (en) Methods and compositions for treatment of myotubular myopathy using chimeric polypeptides comprising myotubularin 1 (mtm1) polypeptides
HK1169663A (en) Methods and compositions for treatment of myotubular myopathy using chimeric polypeptides comprising myotubularin 1 (mtm1) polypeptides
TW201618809A (zh) 用於治療肝醣儲積症及肝醣代謝病症之方法及組合物