ES2644468T3 - Transgén sintético de la metilmalonil-CoA mutasa para el tratamiento de la acidemia metilmalónica (MMA) de clase MUT - Google Patents

Transgén sintético de la metilmalonil-CoA mutasa para el tratamiento de la acidemia metilmalónica (MMA) de clase MUT Download PDF

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Publication number
ES2644468T3
ES2644468T3 ES14729502.6T ES14729502T ES2644468T3 ES 2644468 T3 ES2644468 T3 ES 2644468T3 ES 14729502 T ES14729502 T ES 14729502T ES 2644468 T3 ES2644468 T3 ES 2644468T3
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ES
Spain
Prior art keywords
methylmalonyl
mma
treatment
coa mutase
methylmalonic acidemia
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
ES14729502.6T
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English (en)
Inventor
Charles P. Venditti
Randy J. CHANDLER
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.)
US Department of Health and Human Services
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US Department of Health and Human Services
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Publication date
Application filed by US Department of Health and Human Services filed Critical US Department of Health and Human Services
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Publication of ES2644468T3 publication Critical patent/ES2644468T3/es
Active legal-status Critical Current
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Classifications

    • 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
    • A61K38/52—Isomerases (5)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
    • 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
    • C12N9/90—Isomerases (5.)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y504/00—Intramolecular transferases (5.4)
    • C12Y504/99—Intramolecular transferases (5.4) transferring other groups (5.4.99)
    • C12Y504/99002—Methylmalonyl-CoA mutase (5.4.99.2)

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Medicinal Chemistry (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Microbiology (AREA)
  • Biomedical Technology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Immunology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Apparatus For Radiation Diagnosis (AREA)
  • Pyrane Compounds (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)

Description

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Claims (1)

  1. imagen1
ES14729502.6T 2013-03-15 2014-03-14 Transgén sintético de la metilmalonil-CoA mutasa para el tratamiento de la acidemia metilmalónica (MMA) de clase MUT Active ES2644468T3 (es)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201361792081P 2013-03-15 2013-03-15
US201361792081P 2013-03-15
PCT/US2014/028045 WO2014143884A2 (en) 2013-03-15 2014-03-14 Synthetic methylmalonyl-coa mutase transgene for the treatment of mut class methylmalonic acidemia (mma)

Publications (1)

Publication Number Publication Date
ES2644468T3 true ES2644468T3 (es) 2017-11-29

Family

ID=50928242

Family Applications (1)

Application Number Title Priority Date Filing Date
ES14729502.6T Active ES2644468T3 (es) 2013-03-15 2014-03-14 Transgén sintético de la metilmalonil-CoA mutasa para el tratamiento de la acidemia metilmalónica (MMA) de clase MUT

Country Status (5)

Country Link
US (3) US9719080B2 (es)
EP (1) EP2968602B1 (es)
DK (1) DK2968602T3 (es)
ES (1) ES2644468T3 (es)
WO (1) WO2014143884A2 (es)

Families Citing this family (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US9944918B2 (en) * 2013-03-15 2018-04-17 The United States Of America, As Represented By The Secretary, Dept. Of Health & Human Services Synthetic methylmalonyl-CoA mutase transgene for the treatment of MUT class methylmalonic acidemia (MMA)
ES2644468T3 (es) * 2013-03-15 2017-11-29 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Transgén sintético de la metilmalonil-CoA mutasa para el tratamiento de la acidemia metilmalónica (MMA) de clase MUT
US20180087041A1 (en) * 2015-04-24 2018-03-29 The United States Of America, As Represented By The Secretary, Dept. Of Health And Human Services Tagged form of mut enzyme, genetic constructs incorporating it, and its use in gene thereapy
EP3294894B8 (en) 2015-05-12 2019-09-25 The U.S.A. as represented by the Secretary, Department of Health and Human Services Aav isolate and fusion protein comprising nerve growth factor signal peptide and parathyroid hormone
WO2017099838A1 (en) * 2015-12-11 2017-06-15 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Gene therapy for combined methylmalonic acidemia/aciduria and hyperhomocysteinemia/homocystinuria, cobalamin c type, and deficiency of mmachc
AU2016369612B2 (en) 2015-12-17 2023-06-01 Modernatx, Inc. Polynucleotides encoding methylmalonyl-CoA mutase
US20180126003A1 (en) * 2016-05-04 2018-05-10 Curevac Ag New targets for rna therapeutics
WO2018237066A1 (en) * 2017-06-20 2018-12-27 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services CODON OPTIMIZED HUMAN NPC1 GENES FOR THE TREATMENT OF C1-TYPE NIEMANN-PICK DISEASE AND ASSOCIATED STATES
EP3833755A4 (en) 2018-08-10 2022-05-25 Logicbio Therapeutics, Inc. DISRUPTIVE GENE THERAPY FOR THE TREATMENT OF MMA
US20230145188A1 (en) 2018-09-14 2023-05-11 Translate Bio, Inc. Composition and methods for treatment of methylmalonic acidemia
US20230383278A1 (en) * 2020-09-18 2023-11-30 The United States Of America,As Represented By The Secretary,Department Of Health And Human Services Novel adeno-associated viral (aav) vectors to treat hereditary methylmalonic acidemia (mma) caused by methylmalonyl-coa mutase (mmut) deficiency
WO2022246020A1 (en) * 2021-05-19 2022-11-24 Modernatx, Inc. Polynucleotides encoding methylmalonyl-coa mutase for the treatment of methylmalonic acidemia

Family Cites Families (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2001283062A1 (en) * 2000-08-02 2002-02-13 The Johns Hopkins University Endothelial cell expression patterns
US20040142325A1 (en) 2001-09-14 2004-07-22 Liat Mintz Methods and systems for annotating biomolecular sequences
CA2868391A1 (en) 2012-04-02 2013-10-10 Stephane Bancel Polynucleotides comprising n1-methyl-pseudouridine and methods for preparing the same
WO2013151664A1 (en) * 2012-04-02 2013-10-10 modeRNA Therapeutics Modified polynucleotides for the production of proteins
US9944918B2 (en) 2013-03-15 2018-04-17 The United States Of America, As Represented By The Secretary, Dept. Of Health & Human Services Synthetic methylmalonyl-CoA mutase transgene for the treatment of MUT class methylmalonic acidemia (MMA)
ES2644468T3 (es) * 2013-03-15 2017-11-29 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Transgén sintético de la metilmalonil-CoA mutasa para el tratamiento de la acidemia metilmalónica (MMA) de clase MUT

Also Published As

Publication number Publication date
WO2014143884A2 (en) 2014-09-18
EP2968602B1 (en) 2017-08-30
US20160040150A1 (en) 2016-02-11
US20190298814A1 (en) 2019-10-03
WO2014143884A3 (en) 2014-11-06
US9719080B2 (en) 2017-08-01
EP2968602A2 (en) 2016-01-20
DK2968602T3 (en) 2017-10-30
US10307469B2 (en) 2019-06-04
US20170326212A1 (en) 2017-11-16
US10894077B2 (en) 2021-01-19

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