HUE064076T2 - Készítmények és módszerek az ornitin-transzkarbamiláz hiányának kezelésére - Google Patents
Készítmények és módszerek az ornitin-transzkarbamiláz hiányának kezeléséreInfo
- Publication number
- HUE064076T2 HUE064076T2 HUE19893199A HUE19893199A HUE064076T2 HU E064076 T2 HUE064076 T2 HU E064076T2 HU E19893199 A HUE19893199 A HU E19893199A HU E19893199 A HUE19893199 A HU E19893199A HU E064076 T2 HUE064076 T2 HU E064076T2
- Authority
- HU
- Hungary
- Prior art keywords
- compositions
- methods
- ornithine transcarbamylase
- transcarbamylase deficiency
- treating ornithine
- 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
- C12N9/10—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
Landscapes
- Health & Medical Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Bioinformatics & Cheminformatics (AREA)
- General Health & Medical Sciences (AREA)
- Genetics & Genomics (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- General Engineering & Computer Science (AREA)
- Biochemistry (AREA)
- Medicinal Chemistry (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Molecular Biology (AREA)
- General Chemical & Material Sciences (AREA)
- Cardiology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Heart & Thoracic Surgery (AREA)
- Biomedical Technology (AREA)
- Biotechnology (AREA)
- Microbiology (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)
Applications Claiming Priority (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862776302P | 2018-12-06 | 2018-12-06 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| HUE064076T2 true HUE064076T2 (hu) | 2024-02-28 |
Family
ID=70972469
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| HUE19893199A HUE064076T2 (hu) | 2018-12-06 | 2019-12-05 | Készítmények és módszerek az ornitin-transzkarbamiláz hiányának kezelésére |
Country Status (9)
| Country | Link |
|---|---|
| US (3) | US11685906B2 (enExample) |
| EP (2) | EP3891274B1 (enExample) |
| JP (1) | JP7445657B2 (enExample) |
| AU (2) | AU2019394996B2 (enExample) |
| CA (1) | CA3122080A1 (enExample) |
| ES (1) | ES2960692T3 (enExample) |
| HU (1) | HUE064076T2 (enExample) |
| PL (1) | PL3891274T3 (enExample) |
| WO (1) | WO2020118115A1 (enExample) |
Families Citing this family (6)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CA3122080A1 (en) | 2018-12-06 | 2020-06-11 | Arcturus Therapeutics, Inc. | Compositions and methods for treating ornithine transcarbamylase deficiency |
| 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 |
| EP4114470A4 (en) * | 2020-03-03 | 2024-04-17 | Arcturus Therapeutics, Inc. | COMPOSITIONS AND METHODS FOR TREATING ORNITHINE TRANSCARBAMYLASE DEFICIENCY |
| US20240189449A1 (en) * | 2021-03-24 | 2024-06-13 | Modernatx, Inc. | Lipid nanoparticles and polynucleotides encoding ornithine transcarbamylase for the treatment of ornithine transcarbamylase deficiency |
| EP4334330A1 (en) | 2021-05-07 | 2024-03-13 | Helix Nanotechnologies, Inc. | Modified ribonucleic acids and uses thereof |
| WO2023028469A2 (en) * | 2021-08-23 | 2023-03-02 | 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)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| AU649074B2 (en) | 1990-10-12 | 1994-05-12 | Max-Planck-Gesellschaft Zur Forderung Der Wissenschaften E.V. | Modified ribozymes |
| US5652094A (en) | 1992-01-31 | 1997-07-29 | University Of Montreal | Nucleozymes |
| ATE291925T1 (de) | 2001-06-05 | 2005-04-15 | Curevac Gmbh | Stabilisierte mrna mit erhöhtem g/c-gehalt und optimierter codon usage für die gentherapie |
| JP5248014B2 (ja) | 2003-06-25 | 2013-07-31 | ゲンシア コーポレーション | 細胞小器官トランスフェクションのための改質ベクター |
| AU2004297533B2 (en) | 2003-10-24 | 2010-04-29 | Gencia Corporation | Methods and compositions for delivering polynucleotides |
| PL2578685T3 (pl) | 2005-08-23 | 2020-01-31 | The Trustees Of The University Of Pennsylvania | Rna zawierający zmodyfikowane nukleozydy i sposoby jego zastosowania |
| 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 |
| US9006191B2 (en) | 2007-12-27 | 2015-04-14 | Protiva Biotherapeutics, Inc. | Silencing of polo-like kinase expression using interfering RNA |
| EP3100718B1 (en) | 2008-01-02 | 2019-11-27 | Arbutus Biopharma Corporation | Improved compositions and methods for the delivery of nucleic acids |
| EP2279254B1 (en) | 2008-04-15 | 2017-07-05 | Protiva Biotherapeutics Inc. | 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 |
| AU2009303345B2 (en) | 2008-10-09 | 2015-08-20 | Arbutus Biopharma Corporation | Improved amino lipids and methods for the delivery of nucleic acids |
| WO2010048536A2 (en) | 2008-10-23 | 2010-04-29 | Alnylam Pharmaceuticals, Inc. | Processes for preparing lipids |
| EP3699172A3 (en) | 2008-11-10 | 2020-11-18 | Arbutus Biopharma Corporation | Novel lipids and compositions for the delivery of therapeutics |
| EP2391343B1 (en) | 2009-01-29 | 2017-03-01 | Arbutus Biopharma Corporation | Improved lipid formulation for the delivery of nucleic acids |
| CA3042927C (en) | 2009-05-05 | 2022-05-17 | Arbutus Biopharma Corporation | Lipid compositions for the delivery of therapeutic agents |
| KR20220038506A (ko) | 2009-06-10 | 2022-03-28 | 알닐람 파마슈티칼스 인코포레이티드 | 향상된 지질 조성물 |
| KR101161622B1 (ko) * | 2009-08-31 | 2012-07-04 | 헬릭스 주식회사 | 번역 효율 증진용 ｄｎａ 단편 및 이를 포함하는 재조합 벡터 |
| SI2506857T1 (en) | 2009-12-01 | 2018-08-31 | Translate Bio, Inc. | Delivery of mRNA for the enrichment of proteins and enzymes in human genetic diseases |
| EP4481047A3 (en) | 2010-06-03 | 2025-04-16 | Alnylam Pharmaceuticals, Inc. | Biodegradable lipids for the delivery of active agents |
| US9006417B2 (en) | 2010-06-30 | 2015-04-14 | Protiva Biotherapeutics, Inc. | Non-liposomal systems for nucleic acid delivery |
| KR20220025112A (ko) | 2011-06-08 | 2022-03-03 | 샤이어 휴먼 지네틱 테라피즈 인크. | Mrna 전달을 위한 지질 나노입자 조성물 및 방법 |
| PT3682905T (pt) | 2011-10-03 | 2022-04-07 | Modernatx Inc | Nucleósidos, nucleótidos e ácidos nucleicos modificados e respetivas utilizações |
| DK3421601T3 (da) | 2011-12-30 | 2020-02-24 | Cellscript Llc | Fremstilling og anvendelse af in-vitro-syntetiseret ssRNA til indføring i pattedyrceller med henblik på at inducere en biologisk eller biokemisk effekt |
| US10501512B2 (en) | 2012-04-02 | 2019-12-10 | Modernatx, Inc. | Modified polynucleotides |
| 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 |
| WO2014164253A1 (en) | 2013-03-09 | 2014-10-09 | 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 |
| AU2014296278C1 (en) | 2013-07-30 | 2023-02-02 | Genevant Sciences Gmbh | Block copolymers and their conjugates or complexes with oligonucleotides |
| EP3052511A4 (en) | 2013-10-02 | 2017-05-31 | Moderna Therapeutics, Inc. | Polynucleotide molecules and uses thereof |
| EP3574923A1 (en) | 2013-10-22 | 2019-12-04 | Translate Bio, Inc. | Mrna therapy for phenylketonuria |
| CN110003066B (zh) | 2013-11-18 | 2021-09-03 | 阿克丘勒斯治疗公司 | 用于rna递送的可电离的阳离子脂质 |
| BR112016020688B1 (pt) | 2014-03-09 | 2024-01-30 | The Trustees Of The University Of Pennsylvania | Vetores virais recombinantes, vírus adenoassociado recombinante, composição farmacêutica, bem como uso dos mesmos |
| EP3154570A4 (en) | 2014-06-13 | 2017-12-27 | Valerion Therapeutics, LLC | Methods and compositions for treatment of glycogen storage diseases and glycogen metabolism disorders |
| WO2016070166A2 (en) | 2014-11-02 | 2016-05-06 | Arcturus Therapeutics, Inc. | Messenger una molecules and uses thereof |
| EP3461904A1 (en) | 2014-11-10 | 2019-04-03 | ModernaTX, Inc. | Alternative nucleic acid molecules containing reduced uracil content and uses thereof |
| EP4324473A3 (en) | 2014-11-10 | 2024-05-29 | ModernaTX, Inc. | Multiparametric nucleic acid optimization |
| DK3221293T3 (da) | 2014-11-18 | 2023-04-03 | Arcturus Therapeutics Inc | Ioniserbar kationisk lipid til RNA-afgivelse |
| 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 |
| EP3247328B1 (en) | 2015-01-21 | 2026-01-07 | 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 |
| DK4276189T3 (da) | 2015-09-21 | 2026-05-26 | Trilink Biotechnologies Llc | Initierende oligonukleotidprimere med kappe til syntesering af 5'-ende RNA'er med kappe |
| WO2017100551A1 (en) | 2015-12-09 | 2017-06-15 | Alexion Pharmaceuticals, Inc. | HETEROLOGOUS UTR SEQUENCES FOR ENHANCED mRNA EXPRESSION |
| US9834510B2 (en) | 2015-12-30 | 2017-12-05 | Arcturus Therapeutics, Inc. | Aromatic ionizable cationic lipid |
| AU2017283479B2 (en) | 2016-06-13 | 2023-08-17 | Translate Bio, Inc. | Messenger RNA therapy for the treatment of ornithine transcarbamylase deficiency |
| IL264439B2 (en) | 2016-08-17 | 2024-08-01 | Factor Bioscience Inc | non-viral, cell-free composition comprising a synthetic messenger RNA (MRNA) encoding a gene-editing protein for use in treating cancer, and a synthetic RNA encoding a gene-editing protein for use in treatment |
| US10487105B2 (en) | 2016-10-19 | 2019-11-26 | Arcturus Therapeutics, Inc. | Trinucleotide MRNA cap analogs |
| KR20190093816A (ko) | 2016-10-26 | 2019-08-26 | 큐어백 아게 | 지질 나노입자 mRNA 백신 |
| JP7560946B2 (ja) | 2016-11-10 | 2024-10-03 | トランスレイト バイオ， インコーポレイテッド | メッセンジャーｒｎａの皮下送達 |
| 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 |
| WO2018119163A1 (en) | 2016-12-21 | 2018-06-28 | Payne Joseph E | Ionizable cationic lipid for rna delivery |
| US11104887B2 (en) | 2017-01-03 | 2021-08-31 | 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 |
| EP3589290A4 (en) | 2017-02-28 | 2020-12-30 | Arcturus Therapeutics, Inc. | Translatable molecules and synthesis thereof |
| US11015204B2 (en) | 2017-05-31 | 2021-05-25 | Arcturus Therapeutics, Inc. | Synthesis and structure of high potency RNA therapeutics |
| KR102636537B1 (ko) | 2017-05-31 | 2024-02-15 | 울트라제닉스 파마수티컬 인코포레이티드 | 글리코겐 축적 질환 유형 iii에 대한 치료제 |
| EP3714048B1 (en) * | 2017-11-22 | 2025-04-09 | ModernaTX, Inc. | Polynucleotides encoding ornithine transcarbamylase for the treatment of urea cycle disorders |
| MA51306A (fr) | 2017-12-20 | 2020-10-28 | Translate Bio Inc | Compositions et procédés améliorés pour le traitement du déficit en ornithine transcarbamylase |
| CA3122080A1 (en) | 2018-12-06 | 2020-06-11 | Arcturus Therapeutics, Inc. | Compositions and methods for treating ornithine transcarbamylase deficiency |
| EP3942050A4 (en) | 2019-03-19 | 2023-02-22 | Arcturus Therapeutics, Inc. | PROCEDURE FOR THE PRODUCTION OF LIPID-COATED RNA NANOPARTICLES |
| EP4114470A4 (en) | 2020-03-03 | 2024-04-17 | Arcturus Therapeutics, Inc. | COMPOSITIONS AND METHODS FOR TREATING ORNITHINE TRANSCARBAMYLASE DEFICIENCY |
-
2019
- 2019-12-05 CA CA3122080A patent/CA3122080A1/en active Pending
- 2019-12-05 PL PL19893199.0T patent/PL3891274T3/pl unknown
- 2019-12-05 AU AU2019394996A patent/AU2019394996B2/en active Active
- 2019-12-05 WO PCT/US2019/064786 patent/WO2020118115A1/en not_active Ceased
- 2019-12-05 EP EP19893199.0A patent/EP3891274B1/en active Active
- 2019-12-05 EP EP23195937.0A patent/EP4299750A3/en active Pending
- 2019-12-05 HU HUE19893199A patent/HUE064076T2/hu unknown
- 2019-12-05 JP JP2021531945A patent/JP7445657B2/ja active Active
- 2019-12-05 ES ES19893199T patent/ES2960692T3/es active Active
- 2019-12-05 US US16/705,102 patent/US11685906B2/en active Active
-
2023
- 2023-05-18 US US18/319,977 patent/US12398379B2/en active Active
-
2025
- 2025-07-24 US US19/279,738 patent/US20260078356A1/en active Pending
-
2026
- 2026-02-02 AU AU2026200747A patent/AU2026200747A1/en active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| AU2026200747A1 (en) | 2026-02-26 |
| US20200181584A1 (en) | 2020-06-11 |
| US20240002815A1 (en) | 2024-01-04 |
| PL3891274T3 (pl) | 2024-03-25 |
| AU2019394996A1 (en) | 2021-07-29 |
| EP3891274A4 (en) | 2022-08-31 |
| EP3891274B1 (en) | 2023-10-18 |
| ES2960692T3 (es) | 2024-03-06 |
| CA3122080A1 (en) | 2020-06-11 |
| JP2022513718A (ja) | 2022-02-09 |
| EP4299750A3 (en) | 2024-07-10 |
| US11685906B2 (en) | 2023-06-27 |
| EP3891274C0 (en) | 2023-10-18 |
| EP3891274A1 (en) | 2021-10-13 |
| US12398379B2 (en) | 2025-08-26 |
| US20260078356A1 (en) | 2026-03-19 |
| JP7445657B2 (ja) | 2024-03-07 |
| AU2019394996B2 (en) | 2025-11-06 |
| WO2020118115A1 (en) | 2020-06-11 |
| EP4299750A2 (en) | 2024-01-03 |
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