MA45032A - Polynucléotides codant pour la porphobilinogène désaminase destinés au traitement de la porphyrie intermittente aiguë - Google Patents
Polynucléotides codant pour la porphobilinogène désaminase destinés au traitement de la porphyrie intermittente aiguëInfo
- Publication number
- MA45032A MA45032A MA045032A MA45032A MA45032A MA 45032 A MA45032 A MA 45032A MA 045032 A MA045032 A MA 045032A MA 45032 A MA45032 A MA 45032A MA 45032 A MA45032 A MA 45032A
- Authority
- MA
- Morocco
- Prior art keywords
- desaminase
- intermitten
- porphobilinogen
- porphyria
- acute
- Prior art date
Links
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
- A61K38/45—Transferases (2)
-
- 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
- A61K48/0008—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
- A61K48/0025—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid
- A61K48/0033—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid the non-active part being non-polymeric
-
- 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
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P7/00—Drugs for disorders of the blood or the extracellular fluid
- A61P7/08—Plasma substitutes; Perfusion solutions; Dialytics or haemodialytics; Drugs for electrolytic or acid-base disorders, e.g. hypovolemic shock
-
- 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
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
- C12N15/88—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation using microencapsulation, e.g. using amphiphile liposome vesicle
-
- 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.)
- C12N9/1085—Transferases (2.) transferring alkyl or aryl groups other than methyl groups (2.5)
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y205/00—Transferases transferring alkyl or aryl groups, other than methyl groups (2.5)
- C12Y205/01—Transferases transferring alkyl or aryl groups, other than methyl groups (2.5) transferring alkyl or aryl groups, other than methyl groups (2.5.1)
- C12Y205/01061—Hydroxymethylbilane synthase (2.5.1.61)
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Organic Chemistry (AREA)
- General Health & Medical Sciences (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- Medicinal Chemistry (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Biochemistry (AREA)
- Public Health (AREA)
- Molecular Biology (AREA)
- Veterinary Medicine (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Biomedical Technology (AREA)
- Epidemiology (AREA)
- Microbiology (AREA)
- Hematology (AREA)
- Immunology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Diabetes (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Gastroenterology & Hepatology (AREA)
- General Chemical & Material Sciences (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Enzymes And Modification Thereof (AREA)
- Saccharide Compounds (AREA)
- Medicinal Preparation (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201662338161P | 2016-05-18 | 2016-05-18 | |
| EP17382259 | 2017-05-09 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| MA45032A true MA45032A (fr) | 2019-03-27 |
Family
ID=67223874
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| MA045032A MA45032A (fr) | 2016-05-18 | 2017-05-18 | Polynucléotides codant pour la porphobilinogène désaminase destinés au traitement de la porphyrie intermittente aiguë |
Country Status (3)
| Country | Link |
|---|---|
| US (2) | US20200085916A1 (enExample) |
| JP (2) | JP2019519601A (enExample) |
| MA (1) | MA45032A (enExample) |
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| HRP20220070T1 (hr) | 2014-04-23 | 2022-04-01 | Modernatx, Inc. | Cjepiva nukleinske kiseline |
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| WO2017031232A1 (en) | 2015-08-17 | 2017-02-23 | Modernatx, Inc. | Methods for preparing particles and related compositions |
| EP3364949A4 (en) | 2015-10-22 | 2019-07-31 | ModernaTX, Inc. | CANCER VACCINES |
| EP3364981A4 (en) | 2015-10-22 | 2019-08-07 | ModernaTX, Inc. | VACCINE AGAINST THE HUMAN CYTOMEGALOVIRUS |
| DE20164728T1 (de) | 2015-10-22 | 2021-09-30 | Modernatx, Inc. | Impfstoffe gegen atemwegsvirus |
| SG11201803360UA (en) | 2015-10-22 | 2018-05-30 | Modernatx Inc | Nucleic acid vaccines for varicella zoster virus (vzv) |
| WO2017070624A1 (en) | 2015-10-22 | 2017-04-27 | Modernatx, Inc. | Tropical disease vaccines |
| DK3386484T3 (da) | 2015-12-10 | 2022-07-04 | Modernatx Inc | Sammensætninger og fremgangsmåder til afgivelse af terapeutiske midler |
| WO2017106799A1 (en) | 2015-12-17 | 2017-06-22 | Modernatx, Inc. | POLYNUCLEOTIDES ENCODING METHYLMALONYL-CoA MUTASE |
| US11801227B2 (en) | 2016-05-18 | 2023-10-31 | Modernatx, Inc. | Polynucleotides encoding cystic fibrosis transmembrane conductance regulator for the treatment of cystic fibrosis |
| JP2019519601A (ja) | 2016-05-18 | 2019-07-11 | モダーナティエックス・インコーポレイテッドＭｏｄｅｒｎａＴＸ， Ｉｎｃ． | 急性間欠性ポルフィリン症の治療のためのポルホビリノゲン脱アミノ酵素をコードするポリヌクレオチド |
| US11001861B2 (en) | 2016-05-18 | 2021-05-11 | Modernatx, Inc. | Polynucleotides encoding galactose-1-phosphate uridylyltransferase for the treatment of galactosemia type 1 |
| EP3458106A4 (en) | 2016-05-18 | 2020-03-18 | Modernatx, Inc. | POLYNUCLEOTIDS FOR CODING LIPOPROTEIN LIPASE FOR TREATING HYPERLIPIDEMIA |
| AU2017268396B2 (en) | 2016-05-18 | 2023-05-18 | Modernatx, Inc. | Polynucleotides encoding citrin for the treatment of citrullinemia type 2 |
| US12128113B2 (en) | 2016-05-18 | 2024-10-29 | Modernatx, Inc. | Polynucleotides encoding JAGGED1 for the treatment of Alagille syndrome |
| SG11201901941YA (en) | 2016-09-14 | 2019-04-29 | Modernatx Inc | High purity rna compositions and methods for preparation thereof |
| EP3528821A4 (en) | 2016-10-21 | 2020-07-01 | ModernaTX, Inc. | HUMAN CYTOMEGALOVIRUS VACCINE |
| MA46766A (fr) | 2016-11-11 | 2019-09-18 | Modernatx Inc | Vaccin antigrippal |
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| WO2018170347A1 (en) | 2017-03-17 | 2018-09-20 | Modernatx, Inc. | Zoonotic disease rna vaccines |
| MA48047A (fr) | 2017-04-05 | 2020-02-12 | Modernatx Inc | Réduction ou élimination de réponses immunitaires à des protéines thérapeutiques administrées par voie non intraveineuse, par exemple par voie sous-cutanée |
| WO2018206125A1 (en) * | 2017-05-09 | 2018-11-15 | Fundacion Para La Investigacion Medica Aplicada | Human porphobilinogen deaminase derived proteins and polynucleotides and uses thereof |
| MA49421A (fr) | 2017-06-15 | 2020-04-22 | Modernatx Inc | Formulations d'arn |
| WO2019036685A1 (en) | 2017-08-18 | 2019-02-21 | Modernatx, Inc. | METHODS FOR HPLC ANALYSIS |
| EP3668977A4 (en) | 2017-08-18 | 2021-04-21 | Modernatx, Inc. | HPLC ANALYTICAL PROCESSES |
| WO2019036682A1 (en) | 2017-08-18 | 2019-02-21 | Modernatx, Inc. | RNA VARIANTS POLYMERASE |
| MX2020002348A (es) | 2017-08-31 | 2020-10-08 | Modernatx Inc | Métodos de elaboración de nanopartículas lipídicas. |
| EP3681514A4 (en) | 2017-09-14 | 2021-07-14 | ModernaTX, Inc. | RNA VACZINE AGAINST ZIKA VIRUS |
| CN111712468A (zh) | 2017-12-22 | 2020-09-25 | 北卡罗莱纳州立大学 | 聚合物荧光团、包含其的组合物及制备和使用其的方法 |
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| US12151029B2 (en) | 2018-09-19 | 2024-11-26 | Modernatx, Inc. | PEG lipids and uses thereof |
| US12090235B2 (en) | 2018-09-20 | 2024-09-17 | Modernatx, Inc. | Preparation of lipid nanoparticles and methods of administration thereof |
| CN113939282A (zh) | 2019-01-31 | 2022-01-14 | 摩登纳特斯有限公司 | 制备脂质纳米颗粒的方法 |
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| CN114469996B (zh) * | 2021-12-23 | 2023-10-20 | 中国医学科学院医学生物学研究所 | 一种包含miR-135b-5p的外泌体及在抗轮状病毒感染中的应用 |
| TW202404568A (zh) * | 2022-04-07 | 2024-02-01 | 美商Ｒｎａ免疫公司 | 用於藥物遞送之化合物及組合物 |
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| KR102921785B1 (ko) * | 2022-12-19 | 2026-02-03 | 주식회사 녹십자 | 핵산 전달용 사이클로알칸계 지질 화합물 및 이를 포함하는 지질 나노입자 |
Family Cites Families (21)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| ATE217799T1 (de) | 1998-01-27 | 2002-06-15 | Hemebiotech As | Behandlung von akuter intermittierender porphyria oder anderen porphyrischen krankheiten |
| AU774603B2 (en) | 1999-07-27 | 2004-07-01 | Zymenex A/S | Production of rhPBGD and new therapeutic methods for treating patients with acute intermittent porphyria (AIP) and other porphyric diseases |
| KR20110086553A (ko) * | 2008-09-29 | 2011-07-28 | 암스테르담 몰레큘러 테라퓨틱스 비. 브이. | 포르포빌리노겐 디아미나아제 유전자 치료 |
| 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 |
| JP2013095755A (ja) | 2011-11-02 | 2013-05-20 | Kyowa Hakko Kirin Co Ltd | カチオン性脂質 |
| US20140308304A1 (en) | 2011-12-07 | 2014-10-16 | Alnylam Pharmaceuticals, Inc. | Lipids for the delivery of active agents |
| WO2013151664A1 (en) | 2012-04-02 | 2013-10-10 | modeRNA Therapeutics | Modified polynucleotides for the production of proteins |
| 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 |
| US10501512B2 (en) | 2012-04-02 | 2019-12-10 | Modernatx, Inc. | Modified polynucleotides |
| US20140275229A1 (en) | 2012-04-02 | 2014-09-18 | Moderna Therapeutics, Inc. | Modified polynucleotides encoding udp glucuronosyltransferase 1 family, polypeptide a1 |
| US8980864B2 (en) * | 2013-03-15 | 2015-03-17 | Moderna Therapeutics, Inc. | Compositions and methods of altering cholesterol levels |
| CA2953341C (en) | 2014-06-25 | 2023-01-24 | Acuitas Therapeutics Inc. | Lipids and lipid nanoparticle formulations for delivery of nucleic acids |
| HRP20220156T1 (hr) | 2015-09-17 | 2022-04-15 | Modernatx, Inc. | Spojevi i pripravci za unutarstaničnu isporuku terapeutskih sredstava |
| CN108368028B (zh) * | 2015-10-28 | 2021-09-03 | 爱康泰生治疗公司 | 用于递送核酸的新型脂质和脂质纳米颗粒制剂 |
| WO2017106799A1 (en) | 2015-12-17 | 2017-06-22 | Modernatx, Inc. | POLYNUCLEOTIDES ENCODING METHYLMALONYL-CoA MUTASE |
| EP3458106A4 (en) | 2016-05-18 | 2020-03-18 | Modernatx, Inc. | POLYNUCLEOTIDS FOR CODING LIPOPROTEIN LIPASE FOR TREATING HYPERLIPIDEMIA |
| US11801227B2 (en) | 2016-05-18 | 2023-10-31 | Modernatx, Inc. | Polynucleotides encoding cystic fibrosis transmembrane conductance regulator for the treatment of cystic fibrosis |
| US11001861B2 (en) | 2016-05-18 | 2021-05-11 | Modernatx, Inc. | Polynucleotides encoding galactose-1-phosphate uridylyltransferase for the treatment of galactosemia type 1 |
| AU2017268396B2 (en) | 2016-05-18 | 2023-05-18 | Modernatx, Inc. | Polynucleotides encoding citrin for the treatment of citrullinemia type 2 |
| JP2019519601A (ja) | 2016-05-18 | 2019-07-11 | モダーナティエックス・インコーポレイテッドＭｏｄｅｒｎａＴＸ， Ｉｎｃ． | 急性間欠性ポルフィリン症の治療のためのポルホビリノゲン脱アミノ酵素をコードするポリヌクレオチド |
| WO2017201332A1 (en) | 2016-05-18 | 2017-11-23 | Modernatx, Inc. | Polynucleotides encoding acyl-coa dehydrogenase, very long-chain for the treatment of very long-chain acyl-coa dehydrogenase deficiency |
-
2017
- 2017-05-18 JP JP2019512957A patent/JP2019519601A/ja active Pending
- 2017-05-18 MA MA045032A patent/MA45032A/fr unknown
- 2017-05-18 US US16/302,339 patent/US20200085916A1/en not_active Abandoned
-
2022
- 2022-07-21 US US17/813,984 patent/US12377136B2/en active Active
-
2023
- 2023-02-17 JP JP2023023253A patent/JP2023062097A/ja active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| US20200085916A1 (en) | 2020-03-19 |
| JP2023062097A (ja) | 2023-05-02 |
| US12377136B2 (en) | 2025-08-05 |
| JP2019519601A (ja) | 2019-07-11 |
| US20230112986A1 (en) | 2023-04-13 |
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