ES2960692T3 - Composiciones y métodos para el tratamiento de la deficiencia de ornitina transcarbamilasa - Google Patents

Composiciones y métodos para el tratamiento de la deficiencia de ornitina transcarbamilasa Download PDF

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Publication number
ES2960692T3
ES2960692T3 ES19893199T ES19893199T ES2960692T3 ES 2960692 T3 ES2960692 T3 ES 2960692T3 ES 19893199 T ES19893199 T ES 19893199T ES 19893199 T ES19893199 T ES 19893199T ES 2960692 T3 ES2960692 T3 ES 2960692T3
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Spain
Prior art keywords
otc
mrna
seq
protein
polynucleotide
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Active
Application number
ES19893199T
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English (en)
Spanish (es)
Inventor
Carlos Perez-Garcia
Kiyoshi Tachikawa
Daiki Matsuda
Padmanabh Chivukula
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Arcturus Therapeutics Inc
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Arcturus Therapeutics Inc
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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
    • C12N9/10—Transferases (2.)
    • C12N9/1003—Transferases (2.) transferring one-carbon groups (2.1)
    • C12N9/1018—Carboxy- and carbamoyl transferases (2.1.3)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y201/00—Transferases transferring one-carbon groups (2.1)
    • C12Y201/03—Carboxy- and carbamoyltransferases (2.1.3)
    • C12Y201/03003—Ornithine carbamoyltransferase (2.1.3.3)
    • 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
    • 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
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/22—Vectors comprising a coding region that has been codon optimised for expression in a respective host

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  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Engineering & Computer Science (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)
  • Biomedical Technology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Cardiology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Microbiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
ES19893199T 2018-12-06 2019-12-05 Composiciones y métodos para el tratamiento de la deficiencia de ornitina transcarbamilasa Active ES2960692T3 (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201862776302P 2018-12-06 2018-12-06
PCT/US2019/064786 WO2020118115A1 (en) 2018-12-06 2019-12-05 Compositions and methods for treating ornithine transcarbamylase deficiency

Publications (1)

Publication Number Publication Date
ES2960692T3 true ES2960692T3 (es) 2024-03-06

Family

ID=70972469

Family Applications (1)

Application Number Title Priority Date Filing Date
ES19893199T Active ES2960692T3 (es) 2018-12-06 2019-12-05 Composiciones y métodos para el tratamiento de la deficiencia de ornitina transcarbamilasa

Country Status (9)

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US (3) US11685906B2 (https=)
EP (2) EP4299750A3 (https=)
JP (1) JP7445657B2 (https=)
AU (2) AU2019394996B2 (https=)
CA (1) CA3122080A1 (https=)
ES (1) ES2960692T3 (https=)
HU (1) HUE064076T2 (https=)
PL (1) PL3891274T3 (https=)
WO (1) WO2020118115A1 (https=)

Families Citing this family (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP7445657B2 (ja) 2018-12-06 2024-03-07 アークトゥラス・セラピューティクス・インコーポレイテッド オルニチントランスカルバミラーゼ欠損症を処置するための組成物および方法
JP2022520148A (ja) * 2018-12-06 2022-03-29 アークトゥラス・セラピューティクス・インコーポレイテッド 修飾タンパク質および関連の処置方法
JP7832114B2 (ja) * 2020-03-03 2026-03-17 アークトゥラス・セラピューティクス・インコーポレイテッド オルニチントランスカルバミラーゼ欠損症の治療のための組成物及び方法
WO2022204370A1 (en) * 2021-03-24 2022-09-29 Modernatx, Inc. Lipid nanoparticles and polynucleotides encoding ornithine transcarbamylase for the treatment of ornithine transcarbamylase deficiency
KR20240006603A (ko) 2021-05-07 2024-01-15 헬릭스 나노테크놀로지스, 인코포레이티드 변형된 리보핵산 및 이의 용도
US20250127927A1 (en) * 2021-08-23 2025-04-24 The Board Of Trustees Of The Leland Stanford Junior University Targeted integration at beta-globin locus in human hematopoietic stem and progenitor cells

Family Cites Families (65)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP3257675B2 (ja) 1990-10-12 2002-02-18 マックス−プランク−ゲゼルシャフト ツール フェルデルング デル ビッセンシャフテン エー．ファウ． 修飾リボザイム
US5652094A (en) 1992-01-31 1997-07-29 University Of Montreal Nucleozymes
ATE490267T1 (de) 2001-06-05 2010-12-15 Curevac Gmbh Stabilisierte mrna mit erhöhtem g/c-gehalt, kodierend für ein virales antigen
WO2005001062A2 (en) 2003-06-25 2005-01-06 Gencia Corporation Modified vectors for organelle transfection
EP1687017B1 (en) 2003-10-24 2013-03-06 Gencia Corporation Methods and compositions for delivering polynucleotides
EP3611266B1 (en) 2005-08-23 2022-11-09 The Trustees of the University of Pennsylvania Rna containing modified nucleosides and methods of use thereof
EP2049665A2 (en) 2006-07-28 2009-04-22 Applera Corporation Dinucleotide mrna cap analogs
WO2009058911A2 (en) 2007-10-31 2009-05-07 Applied Biosystems Inc. Preparation and isolation of 5' capped mrna
WO2009082817A1 (en) 2007-12-27 2009-07-09 Protiva Biotherapeutics, Inc. Silencing of polo-like kinase expression using interfering rna
WO2009086558A1 (en) 2008-01-02 2009-07-09 Tekmira Pharmaceuticals Corporation Improved compositions and methods for the delivery of nucleic acids
AU2009238175C1 (en) 2008-04-15 2023-11-30 Arbutus Biopharma Corporation Novel lipid formulations for nucleic acid delivery
WO2010037408A1 (en) 2008-09-30 2010-04-08 Curevac Gmbh Composition comprising a complexed (m)rna and a naked mrna for providing or enhancing an immunostimulatory response in a mammal and uses thereof
CN104119242B (zh) 2008-10-09 2017-07-07 泰米拉制药公司 改善的氨基脂质和递送核酸的方法
WO2010048536A2 (en) 2008-10-23 2010-04-29 Alnylam Pharmaceuticals, Inc. Processes for preparing lipids
HUE037082T2 (hu) 2008-11-10 2018-08-28 Arbutus Biopharma Corp Új lipidek és készítmények terápiás hatóanyagok szállítására
EP3243504A1 (en) 2009-01-29 2017-11-15 Arbutus Biopharma Corporation Improved lipid formulation
KR20180094137A (ko) 2009-05-05 2018-08-22 알닐람 파마슈티칼스 인코포레이티드 지질 조성물
MX342785B (es) 2009-06-10 2016-10-12 Alnylam Pharmaceuticals Inc Formulacion mejorada de lipido.
KR101161622B1 (ko) * 2009-08-31 2012-07-04 헬릭스 주식회사 번역 효율 증진용 ｄｎａ 단편 및 이를 포함하는 재조합 벡터
HUE042177T2 (hu) 2009-12-01 2019-06-28 Translate Bio Inc Szteroidszármazék mRNS szállítására humán genetikai betegségekben
SG186085A1 (en) 2010-06-03 2013-01-30 Alnylam Pharmaceuticals Inc Biodegradable lipids for the delivery of active agents
WO2012000104A1 (en) 2010-06-30 2012-01-05 Protiva Biotherapeutics, Inc. Non-liposomal systems for nucleic acid delivery
PL3586861T3 (pl) 2011-06-08 2022-05-23 Translate Bio, Inc. Kompozycje nanocząstek lipidowych i sposoby dostarczania mrna
RU2648950C2 (ru) 2011-10-03 2018-04-02 Модерна Терапьютикс, Инк. Модифицированные нуклеозиды, нуклеотиды и нуклеиновые кислоты и их применение
EP4721752A2 (en) 2011-12-30 2026-04-08 Cellscript, Llc Herstellung und verwendung von in vitro-synthetisierter ssrna zur einführung in säugetierzellen zur induktion einer biologischen oder biochemischen wirkung
US9254311B2 (en) 2012-04-02 2016-02-09 Moderna Therapeutics, Inc. Modified polynucleotides for the production of proteins
US20140090108A1 (en) 2012-09-18 2014-03-27 Freydoun Garabagi Vectors and Methods For Enhancing Recombinant Protein Expression in Plants
WO2014093924A1 (en) 2012-12-13 2014-06-19 Moderna Therapeutics, Inc. Modified nucleic acid molecules and uses thereof
WO2014130722A1 (en) 2013-02-20 2014-08-28 Valerion Therapeutics, Llc Methods and compositions for treatment of forbes-cori disease
US20160022840A1 (en) 2013-03-09 2016-01-28 Moderna Therapeutics, Inc. Heterologous untranslated regions for mrna
US8912147B2 (en) * 2013-04-15 2014-12-16 BioBlast Pharma Ltd. Mitochondrial proteins constructs and uses thereof
EP3027221A1 (en) * 2013-07-30 2016-06-08 PhaseRx, Inc. Block copolymers and their conjugates or complexes with oligonucleotides
WO2015051169A2 (en) 2013-10-02 2015-04-09 Moderna Therapeutics, Inc. Polynucleotide molecules and uses thereof
EA034103B1 (ru) 2013-10-22 2019-12-27 Транслейт Био, Инк. СПОСОБ ЛЕЧЕНИЯ ФЕНИЛКЕТОНУРИИ С ПРИМЕНЕНИЕМ мРНК
US9593077B2 (en) 2013-11-18 2017-03-14 Arcturus Therapeutics, Inc. Ionizable cationic lipid for RNA delivery
RS60902B1 (sr) * 2014-03-09 2020-11-30 Univ Pennsylvania Kompozicije korisne u lečenju nedostatka ornitinske transkarbamilaze (otc)
US10202591B2 (en) 2014-06-13 2019-02-12 Valerion Therapeutics, Llc Methods and compositions for treatment of glycogen storage diseases and glycogen metabolism disorders
AU2015338923B2 (en) 2014-11-02 2021-10-21 Arcturus Therapeutics, Inc. Messenger UNA molecules and uses thereof
WO2016077123A1 (en) 2014-11-10 2016-05-19 Moderna Therapeutics, Inc. Multiparametric nucleic acid optimization
EP3461904A1 (en) 2014-11-10 2019-04-03 ModernaTX, Inc. Alternative nucleic acid molecules containing reduced uracil content and uses thereof
KR102380363B1 (ko) 2014-11-18 2022-03-29 아크투루스 쎄라퓨틱스, 인크. Rna 전달을 위한 이온성 양이온 지질
US9709490B2 (en) 2014-12-08 2017-07-18 Canon Kabushiki Kaisha Refractive index distribution measuring method, refractive index distribution measuring apparatus, and optical element manufacturing method
US11219634B2 (en) * 2015-01-21 2022-01-11 Genevant Sciences Gmbh Methods, compositions, and systems for delivering therapeutic and diagnostic agents into cells
EP3328439B1 (en) 2015-07-31 2020-04-08 Arcturus Therapeutics, Inc. Multiligand agent for drug delivery
ES2964690T3 (es) 2015-09-21 2024-04-09 Trilink Biotechnologies Llc Método para sintetizar ARN con caperuza 5'
US11389546B2 (en) 2015-12-09 2022-07-19 Modernatx, Inc. Heterologous UTR sequences for enhanced mRNA expression
US9834510B2 (en) 2015-12-30 2017-12-05 Arcturus Therapeutics, Inc. Aromatic ionizable cationic lipid
CA3027312A1 (en) * 2016-06-13 2017-12-21 Translate Bio, Inc. Messenger rna therapy for the treatment of ornithine transcarbamylase deficiency
US10576167B2 (en) 2016-08-17 2020-03-03 Factor Bioscience Inc. Nucleic acid products and methods of administration thereof
US10487105B2 (en) 2016-10-19 2019-11-26 Arcturus Therapeutics, Inc. Trinucleotide MRNA cap analogs
BR112019008481A2 (pt) 2016-10-26 2020-03-03 Curevac Ag Vacinas de mrna de nanopartículas lipídicas
MA46761A (fr) 2016-11-10 2019-09-18 Translate Bio Inc Administration sous-cutanée d'arn messager
WO2018119163A1 (en) 2016-12-21 2018-06-28 Payne Joseph E Ionizable cationic lipid for rna delivery
US10383952B2 (en) 2016-12-21 2019-08-20 Arcturus Therapeutics, Inc. Ionizable cationic lipid for RNA delivery
US10526284B2 (en) 2016-12-21 2020-01-07 Arcturus Therapeutics, Inc. Ionizable cationic lipid for RNA delivery
EP3565605A1 (en) * 2017-01-03 2019-11-13 ethris GmbH Ornithine transcarbamylase coding polyribonucleotides and formulations thereof
US10227302B2 (en) 2017-02-09 2019-03-12 Arcturus Therapeutics, Inc. Ionizable cationic lipid for RNA delivery
WO2018160592A1 (en) 2017-02-28 2018-09-07 Arcturus Therapeutics, Inc. Translatable molecules and synthesis thereof
ES3039647T3 (en) 2017-05-31 2025-10-23 Arcturus Therapeutics Inc Synthesis and structure of high potency rna therapeutics
WO2018222926A1 (en) 2017-05-31 2018-12-06 Ultragenyx Pharmaceutical Inc. Therapeutics for glycogen storage disease type iii
EP3714048B1 (en) 2017-11-22 2025-04-09 ModernaTX, Inc. Polynucleotides encoding ornithine transcarbamylase for the treatment of urea cycle disorders
EP3727428A1 (en) 2017-12-20 2020-10-28 Translate Bio, Inc. Improved composition and methods for treatment of ornithine transcarbamylase deficiency
JP7445657B2 (ja) 2018-12-06 2024-03-07 アークトゥラス・セラピューティクス・インコーポレイテッド オルニチントランスカルバミラーゼ欠損症を処置するための組成物および方法
EP3942050A4 (en) 2019-03-19 2023-02-22 Arcturus Therapeutics, Inc. PROCEDURE FOR THE PRODUCTION OF LIPID-COATED RNA NANOPARTICLES
JP7832114B2 (ja) 2020-03-03 2026-03-17 アークトゥラス・セラピューティクス・インコーポレイテッド オルニチントランスカルバミラーゼ欠損症の治療のための組成物及び方法

Also Published As

Publication number Publication date
JP2022513718A (ja) 2022-02-09
JP7445657B2 (ja) 2024-03-07
US20260078356A1 (en) 2026-03-19
CA3122080A1 (en) 2020-06-11
US20200181584A1 (en) 2020-06-11
US12398379B2 (en) 2025-08-26
EP3891274A4 (en) 2022-08-31
AU2019394996B2 (en) 2025-11-06
AU2026200747A1 (en) 2026-02-26
PL3891274T3 (pl) 2024-03-25
US20240002815A1 (en) 2024-01-04
AU2019394996A1 (en) 2021-07-29
EP3891274A1 (en) 2021-10-13
EP3891274B1 (en) 2023-10-18
HUE064076T2 (hu) 2024-02-28
EP3891274C0 (en) 2023-10-18
EP4299750A3 (en) 2024-07-10
EP4299750A2 (en) 2024-01-03
US11685906B2 (en) 2023-06-27
WO2020118115A1 (en) 2020-06-11

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