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
Original Assignee
US Department of Health and Human Services
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.)
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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)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Microbiology (AREA)
  • Biomedical Technology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Apparatus For Radiation Diagnosis (AREA)
  • Pyrane Compounds (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 (15)

* 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
WO2016172574A2 (en) * 2015-04-24 2016-10-27 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Tagged form of mut enzyme, genetic constructs incorporating it, and its use in gene therapy
JP6805174B2 (ja) * 2015-05-12 2020-12-23 アメリカ合衆国 神経成長因子シグナルペプチド及び副甲状腺ホルモンを含むａａｖ分離株及び融合タンパク質
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
MA43568A (fr) 2015-12-17 2018-11-14 Modernatx Inc Polynucléotides codant pour la méthylmalonyl-coa mutase
US20180126003A1 (en) * 2016-05-04 2018-05-10 Curevac Ag New targets for rna therapeutics
CA3068010A1 (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 niemann-pick type c1 deficiency and related conditions
MA53252A (fr) 2018-08-10 2021-09-15 Logicbio Therapeutics Inc Thérapie génique non perturbatrice pour le traitement d'un mma
EP3849617A1 (en) 2018-09-14 2021-07-21 Translate Bio, Inc. Composition and methods for treatment of methylmalonic acidemia
WO2020106946A1 (en) 2018-11-21 2020-05-28 Translate Bio, Inc. TREATMENT OF CYSTIC FIBROSIS BY DELIVERY OF NEBULIZED mRNA ENCODING CFTR
EP4031662A1 (en) 2019-09-20 2022-07-27 Translate Bio, Inc. Mrna encoding engineered cftr
AU2021230361A1 (en) 2020-03-04 2022-09-08 Poseida Therapeutics, Inc. Compositions and methods for the treatment of Metabolic Liver Disorders
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
US20240269248A1 (en) * 2021-05-19 2024-08-15 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
JP2004527210A (ja) * 2000-08-02 2004-09-09 ザ ジョンズ ホプキンス ユニバーシティー 内皮細胞発現パターンの評価法
US20040142325A1 (en) 2001-09-14 2004-07-22 Liat Mintz Methods and systems for annotating biomolecular sequences
WO2013151665A2 (en) * 2012-04-02 2013-10-10 modeRNA Therapeutics Modified polynucleotides for the production of proteins associated with human disease
CN104411338A (zh) 2012-04-02 2015-03-11 现代治疗公司 用于产生与人类疾病相关的生物制剂和蛋白质的修饰多核苷酸
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
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)

Also Published As

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

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