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 (2) | US11685906B2 (hu) |
| EP (2) | EP4299750A3 (hu) |
| JP (1) | JP7445657B2 (hu) |
| AU (1) | AU2019394996B2 (hu) |
| CA (1) | CA3122080A1 (hu) |
| ES (1) | ES2960692T3 (hu) |
| HU (1) | HUE064076T2 (hu) |
| PL (1) | PL3891274T3 (hu) |
| WO (1) | WO2020118115A1 (hu) |
Families Citing this family (6)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US20220096520A1 (en) * | 2018-12-06 | 2022-03-31 | Arcturus Therapeutics, Inc. | Modified proteins and associated methods of treatment |
| US11685906B2 (en) | 2018-12-06 | 2023-06-27 | Arcturus Therapeutics, Inc. | Compositions and methods for treating ornithine transcarbamylase deficiency |
| CA3169889A1 (en) * | 2020-03-03 | 2021-09-10 | Arcturus Therapeutics, Inc. | Compositions and methods for the treatment of ornithine transcarbamylase deficiency |
| WO2022204370A1 (en) * | 2021-03-24 | 2022-09-29 | Modernatx, Inc. | Lipid nanoparticles and polynucleotides encoding ornithine transcarbamylase for the treatment of ornithine transcarbamylase deficiency |
| CA3214286A1 (en) | 2021-05-07 | 2022-11-10 | Nikhil Dhar | 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 |
|---|---|---|---|---|
| DE69123979T2 (de) | 1990-10-12 | 1997-04-30 | Max Planck Gesellschaft | Abgeänderte ribozyme |
| US5652094A (en) | 1992-01-31 | 1997-07-29 | University Of Montreal | Nucleozymes |
| PT1857122E (pt) | 2001-06-05 | 2011-03-07 | Curevac Gmbh | Arnm estabilizado com teor de g/c aumentado, codificando para um antigénio viral |
| CA2530248A1 (en) | 2003-06-25 | 2005-01-06 | Gencia Corporation | Modified vectors for organelle transfection |
| ES2411962T3 (es) * | 2003-10-24 | 2013-07-09 | Gencia Corporation | Métodos y composiciones para suministrar polinucleótidos |
| ES2937245T3 (es) | 2005-08-23 | 2023-03-27 | Univ Pennsylvania | ARN que contiene nucleósidos modificados y métodos de uso del mismo |
| US8304529B2 (en) | 2006-07-28 | 2012-11-06 | Life Technologies 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 |
| CA2709875C (en) | 2008-01-02 | 2019-07-16 | Tekmira Pharmaceuticals Corporation | Improved compositions and methods for the delivery of nucleic acids |
| CA2721333C (en) | 2008-04-15 | 2020-12-01 | 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 |
| PL2350043T3 (pl) | 2008-10-09 | 2014-09-30 | Tekmira Pharmaceuticals Corp | Ulepszone aminolipidy i sposoby dostarczania kwasów nukleinowych |
| WO2010048536A2 (en) | 2008-10-23 | 2010-04-29 | Alnylam Pharmaceuticals, Inc. | Processes for preparing lipids |
| WO2010054406A1 (en) | 2008-11-10 | 2010-05-14 | Alnylam Pharmaceuticals, Inc. | Novel lipids and compositions for the delivery of therapeutics |
| WO2010088537A2 (en) | 2009-01-29 | 2010-08-05 | Alnylam Pharmaceuticals, Inc. | Improved lipid formulation |
| NZ711583A (en) | 2009-05-05 | 2017-03-31 | Arbutus Biopharma Corp | Lipid compositions |
| KR102066189B1 (ko) | 2009-06-10 | 2020-01-14 | 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 |
| RS58405B1 (sr) | 2009-12-01 | 2019-04-30 | Translate Bio Inc | Stereoidni derivati za isporuku irnk u humanim genetskim oboljenjima |
| SG186085A1 (en) | 2010-06-03 | 2013-01-30 | 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 |
| JP6184945B2 (ja) | 2011-06-08 | 2017-08-23 | シャイアー ヒューマン ジェネティック セラピーズ インコーポレイテッド | ｍＲＮＡ送達のための脂質ナノ粒子組成物および方法 |
| HRP20220250T1 (hr) | 2011-10-03 | 2022-04-29 | Modernatx, Inc. | Modificirani nukleozidi, nukleotidi i nukleinske kiseline, te njihove uporabe |
| AU2012362113B2 (en) | 2011-12-30 | 2017-08-03 | Cellscript, Llc | Making and using in vitro-synthesized ssRNA for introducing into mammalian cells to induce a biological or biochemical effect |
| 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 |
| CA2901969A1 (en) | 2013-02-20 | 2014-08-28 | Valerion Therapeutics, Llc | Methods and compositions for treatment of forbes-cori disease |
| HK1220122A1 (zh) | 2013-03-09 | 2017-04-28 | Modernatx, Inc. | Mrna的异源未转录区域 |
| US8912147B2 (en) * | 2013-04-15 | 2014-12-16 | BioBlast Pharma Ltd. | Mitochondrial proteins constructs and uses thereof |
| CA2919828C (en) * | 2013-07-30 | 2022-07-19 | Phaserx, Inc. | 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 |
| MX2016005239A (es) | 2013-10-22 | 2016-08-12 | Shire Human Genetic Therapies | Tratamiento con acido ribonucleico mensajero para la fenilcetonuria. |
| 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 |
| SI3116900T1 (sl) | 2014-03-09 | 2021-02-26 | The Trustees Of The University Of Pennsylvania | Sestavki uporabni pri zdravljenju pomanjkanja ornitin transkarbamilaze(OTC) |
| WO2015192092A1 (en) | 2014-06-13 | 2015-12-17 | Valerion Therapeutics, Llc | Methods and compositions for treatment of glycogen storage diseases and glycogen metabolism disorders |
| EP3212793B1 (en) | 2014-11-02 | 2020-01-08 | 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 |
| EP3218508A4 (en) | 2014-11-10 | 2018-04-18 | 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 |
| WO2016118697A1 (en) * | 2015-01-21 | 2016-07-28 | Phaserx, Inc. | Methods, compositions, and systems for delivering therapeutic and diagnostic agents into cells |
| JP6784750B2 (ja) | 2015-07-31 | 2020-11-11 | アークトゥルス セラピューティクス， インコーポレイテッド | 薬物送達のためのマルチリガンド剤 |
| HUE065704T2 (hu) | 2015-09-21 | 2024-06-28 | Trilink Biotechnologies Llc | Készítmények és módszerek 5-sapkával ellátott RNS-ek szintetizálására |
| 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 |
| WO2018075827A1 (en) | 2016-10-19 | 2018-04-26 | Arcturus Therapeutics, Inc. | Trinucleotide mrna cap analogs |
| IL317855A (en) | 2016-10-26 | 2025-02-01 | Acuitas Therapeutics Inc | mRNA vaccines with lipid nanoparticles |
| CA3041350A1 (en) | 2016-11-10 | 2018-05-17 | Translate Bio, Inc. | Subcutaneous delivery of messenger rna |
| 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 |
| US10383952B2 (en) | 2016-12-21 | 2019-08-20 | 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 |
| 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 |
| JP7284101B2 (ja) | 2017-05-31 | 2023-05-30 | ウルトラジェニクス ファーマシューティカル インク． | 糖原病ｉｉｉ型のための治療薬 |
| JP7423522B2 (ja) * | 2017-11-22 | 2024-01-29 | モダーナティエックス・インコーポレイテッド | 尿素サイクル異常症の治療のためのオルニチントランスカルバミラーゼをコードするポリヌクレオチド |
| EP3727428A1 (en) | 2017-12-20 | 2020-10-28 | Translate Bio, Inc. | Improved composition and methods for treatment of ornithine transcarbamylase deficiency |
| US11685906B2 (en) | 2018-12-06 | 2023-06-27 | Arcturus Therapeutics, Inc. | Compositions and methods for treating ornithine transcarbamylase deficiency |
| KR20220018960A (ko) | 2019-03-19 | 2022-02-15 | 아크투루스 쎄라퓨틱스, 인크. | 지질-캡슐화된 rna 나노입자 제조 방법 |
| CA3169889A1 (en) | 2020-03-03 | 2021-09-10 | Arcturus Therapeutics, Inc. | Compositions and methods for the treatment of ornithine transcarbamylase deficiency |
-
2019
- 2019-12-05 US US16/705,102 patent/US11685906B2/en active Active
- 2019-12-05 PL PL19893199.0T patent/PL3891274T3/pl unknown
- 2019-12-05 WO PCT/US2019/064786 patent/WO2020118115A1/en not_active Ceased
- 2019-12-05 EP EP23195937.0A patent/EP4299750A3/en active Pending
- 2019-12-05 JP JP2021531945A patent/JP7445657B2/ja active Active
- 2019-12-05 ES ES19893199T patent/ES2960692T3/es active Active
- 2019-12-05 AU AU2019394996A patent/AU2019394996B2/en active Active
- 2019-12-05 HU HUE19893199A patent/HUE064076T2/hu unknown
- 2019-12-05 EP EP19893199.0A patent/EP3891274B1/en active Active
- 2019-12-05 CA CA3122080A patent/CA3122080A1/en active Pending
-
2023
- 2023-05-18 US US18/319,977 patent/US12398379B2/en active Active
Also Published As
| Publication number | Publication date |
|---|---|
| ES2960692T3 (es) | 2024-03-06 |
| EP3891274A4 (en) | 2022-08-31 |
| JP2022513718A (ja) | 2022-02-09 |
| EP4299750A2 (en) | 2024-01-03 |
| PL3891274T3 (pl) | 2024-03-25 |
| EP3891274B1 (en) | 2023-10-18 |
| EP3891274A1 (en) | 2021-10-13 |
| AU2019394996A1 (en) | 2021-07-29 |
| WO2020118115A1 (en) | 2020-06-11 |
| CA3122080A1 (en) | 2020-06-11 |
| AU2019394996B2 (en) | 2025-11-06 |
| US20240002815A1 (en) | 2024-01-04 |
| US20200181584A1 (en) | 2020-06-11 |
| US11685906B2 (en) | 2023-06-27 |
| JP7445657B2 (ja) | 2024-03-07 |
| EP3891274C0 (en) | 2023-10-18 |
| EP4299750A3 (en) | 2024-07-10 |
| US12398379B2 (en) | 2025-08-26 |
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