BRPI1011236A2 - métodos e composições para tratamento de miopatia miotubular usando polipeptídeos quiméricos compreendendo polipeptídeos miotubularesih 1 (mtm1) - Google Patents
métodos e composições para tratamento de miopatia miotubular usando polipeptídeos quiméricos compreendendo polipeptídeos miotubularesih 1 (mtm1)Info
- Publication number
- BRPI1011236A2 BRPI1011236A2 BRPI1011236A BRPI1011236A BRPI1011236A2 BR PI1011236 A2 BRPI1011236 A2 BR PI1011236A2 BR PI1011236 A BRPI1011236 A BR PI1011236A BR PI1011236 A BRPI1011236 A BR PI1011236A BR PI1011236 A2 BRPI1011236 A2 BR PI1011236A2
- Authority
- BR
- Brazil
- Prior art keywords
- polypeptides
- compositions
- mtm1
- methods
- myotubular myopathy
- Prior art date
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 [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)
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)
- 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)
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US26873209P | 2009-06-15 | 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 |
|---|---|
| BRPI1011236A2 true BRPI1011236A2 (pt) | 2019-07-09 |
Family
ID=43356726
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| BRPI1011236A BRPI1011236A2 (pt) | 2009-06-15 | 2010-06-15 | métodos e composições para tratamento de miopatia miotubular usando polipeptídeos quiméricos compreendendo polipeptídeos miotubularesih 1 (mtm1) |
Country Status (8)
| Country | Link |
|---|---|
| US (2) | US8834866B2 (https=) |
| EP (1) | EP2443155B1 (https=) |
| JP (1) | JP6166041B2 (https=) |
| AU (1) | AU2010260145B2 (https=) |
| BR (1) | BRPI1011236A2 (https=) |
| CA (1) | CA2797480A1 (https=) |
| ES (1) | ES2630057T3 (https=) |
| WO (1) | WO2010148010A1 (https=) |
Families Citing this family (21)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| 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 |
| 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 |
| WO2013177428A1 (en) * | 2012-05-23 | 2013-11-28 | Valerion Therapeutics, Inc. | 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 |
| AU2015204446A1 (en) * | 2014-01-13 | 2016-07-14 | Valerion Therapeutics, Llc | Internalizing moieties |
| EP3778630A1 (en) * | 2014-02-28 | 2021-02-17 | 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 |
| 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 |
| WO2017218824A1 (en) | 2016-06-15 | 2017-12-21 | 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 |
| 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 |
| WO2019007991A1 (en) * | 2017-07-03 | 2019-01-10 | Universite De Strasbourg | MTMR2-S POLYPEPTIDE FOR USE IN THE TREATMENT OF MYOPATHIES |
| WO2021042060A1 (en) | 2019-08-30 | 2021-03-04 | Yale University | Compositions and methods for delivery of nucleic acids to cells |
| AU2022228255A1 (en) * | 2021-03-03 | 2023-09-28 | Yale University | Compositions and methods for treating skeletal muscle disease |
| 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)
| 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 |
| WO1991017424A1 (en) | 1990-05-03 | 1991-11-14 | 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 |
| US6068829A (en) | 1995-09-11 | 2000-05-30 | The Burnham Institute | Method of identifying molecules that home to a selected organ in vivo |
| 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 |
| 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 |
| WO2002079415A2 (en) | 2001-03-30 | 2002-10-10 | Lexigen Pharmaceuticals Corp. | Reducing the immunogenicity of fusion proteins |
| JP2008500373A (ja) | 2004-05-27 | 2008-01-10 | アクセルロン ファーマ インコーポレーテッド | ケルベロス（Ｃｅｒｂｅｒｕｓ）／ココ（Ｃｏｃｏ）誘導体およびそれらの使用 |
| AU2008207948A1 (en) | 2007-01-22 | 2008-07-31 | The United States Government As Represented By The Department Of Veterans Affairs | Use of antibody conjugates |
| EP3486653A1 (en) | 2007-05-24 | 2019-05-22 | The United States Government as represented by The Department of Veterans Affairs | Treatment of skeletal muscle disorder using ent2 |
| WO2010044894A1 (en) | 2008-10-15 | 2010-04-22 | 4S3 Bioscience Inc. | Methods and compositions for treatment of myotonic dystrophy |
| US9155801B2 (en) | 2009-05-28 | 2015-10-13 | The United States Government As Represented By The Department Of Veterans Affairs | Amino acid sequences which enhance peptide conjugate solubility |
| 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 |
| 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 |
| WO2012138733A2 (en) | 2011-04-05 | 2012-10-11 | Chevron Oronite Company Llc | Low viscosity marine cylinder lubricating oil compositions |
| WO2013177428A1 (en) | 2012-05-23 | 2013-11-28 | Valerion Therapeutics, Inc. | Methods for increasing muscle contractility |
-
2010
- 2010-06-15 AU AU2010260145A patent/AU2010260145B2/en not_active Ceased
- 2010-06-15 ES ES10790070.6T patent/ES2630057T3/es active Active
- 2010-06-15 US US13/378,283 patent/US8834866B2/en not_active Expired - Fee Related
- 2010-06-15 WO PCT/US2010/038703 patent/WO2010148010A1/en not_active Ceased
- 2010-06-15 JP JP2012515229A patent/JP6166041B2/ja not_active Expired - Fee Related
- 2010-06-15 CA CA2797480A patent/CA2797480A1/en not_active Abandoned
- 2010-06-15 EP EP10790070.6A patent/EP2443155B1/en not_active Not-in-force
- 2010-06-15 BR BRPI1011236A patent/BRPI1011236A2/pt not_active Application Discontinuation
-
2014
- 2014-09-12 US US14/485,524 patent/US9447394B2/en not_active Expired - Fee Related
Also Published As
| Publication number | Publication date |
|---|---|
| US9447394B2 (en) | 2016-09-20 |
| JP2012529891A (ja) | 2012-11-29 |
| ES2630057T3 (es) | 2017-08-17 |
| US20120213760A1 (en) | 2012-08-23 |
| EP2443155A4 (en) | 2012-12-26 |
| JP6166041B2 (ja) | 2017-07-19 |
| EP2443155B1 (en) | 2017-03-29 |
| CA2797480A1 (en) | 2010-12-23 |
| US20150218540A1 (en) | 2015-08-06 |
| EP2443155A1 (en) | 2012-04-25 |
| WO2010148010A1 (en) | 2010-12-23 |
| AU2010260145B2 (en) | 2015-08-20 |
| US8834866B2 (en) | 2014-09-16 |
| AU2010260145A1 (en) | 2012-02-02 |
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