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)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- General Health & Medical Sciences (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Medicinal Chemistry (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Biochemistry (AREA)
- Public Health (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Molecular Biology (AREA)
- Veterinary Medicine (AREA)
- Biomedical Technology (AREA)
- Epidemiology (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Immunology (AREA)
- Biophysics (AREA)
- Diabetes (AREA)
- Hematology (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Gastroenterology & Hepatology (AREA)
- Physics & Mathematics (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Enzymes And Modification Thereof (AREA)
- Saccharide Compounds (AREA)
- Medicinal Preparation (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (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 (fr) |
| JP (2) | JP2019519601A (fr) |
| MA (1) | MA45032A (fr) |
Families Citing this family (58)
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| DK3134131T3 (en) | 2014-04-23 | 2022-02-07 | Modernatx Inc | Nucleic acid vaccines |
| US11364292B2 (en) | 2015-07-21 | 2022-06-21 | Modernatx, Inc. | CHIKV RNA vaccines |
| WO2017020026A1 (fr) | 2015-07-30 | 2017-02-02 | Modernatx, Inc. | Arn épitopes peptidiques concatémériques |
| US11564893B2 (en) | 2015-08-17 | 2023-01-31 | Modernatx, Inc. | Methods for preparing particles and related compositions |
| MD3718565T2 (ro) | 2015-10-22 | 2022-09-30 | Modernatx Inc | Vaccinuri împotriva virusului respirator |
| WO2017070601A1 (fr) | 2015-10-22 | 2017-04-27 | Modernatx, Inc. | Vaccins à base d'acide nucléique contre le virus varicelle-zona (vzv) |
| CN108430456B (zh) | 2015-10-22 | 2022-01-18 | 摩登纳特斯有限公司 | 癌症疫苗 |
| EP3364981A4 (fr) | 2015-10-22 | 2019-08-07 | ModernaTX, Inc. | Vaccin contre le cytomégalovirus humain |
| EP3364950A4 (fr) | 2015-10-22 | 2019-10-23 | ModernaTX, Inc. | Vaccins contre des maladies tropicales |
| CA3007955A1 (fr) | 2015-12-10 | 2017-06-15 | Modernatx, Inc. | Nanoparticules de lipide pour l'administration d'agents therapeutiques |
| EP3390630B1 (fr) | 2015-12-17 | 2025-01-22 | ModernaTX, Inc. | Polynucléotides codant pour la méthylmalonyl-coa mutase |
| US11001861B2 (en) | 2016-05-18 | 2021-05-11 | Modernatx, Inc. | Polynucleotides encoding galactose-1-phosphate uridylyltransferase for the treatment of galactosemia type 1 |
| MA45053A (fr) | 2016-05-18 | 2019-03-27 | Modernatx Inc | Polynucléotides codant pour un régulateur de conductance transmembranaire de fibrose kystique pour le traitement de la fibrose kystique |
| US12123030B2 (en) | 2016-05-18 | 2024-10-22 | Modernatx, Inc. | Polynucleotides encoding lipoprotein lipase for the treatment of hyperlipidemia |
| MA45032A (fr) | 2016-05-18 | 2019-03-27 | Fundacion Para La Investig Medica Aplicada | Polynucléotides codant pour la porphobilinogène désaminase destinés au traitement de la porphyrie intermittente aiguë |
| WO2017201349A1 (fr) | 2016-05-18 | 2017-11-23 | Modernatx, Inc. | Polynucléotides codant pour la citrine pour le traitement de la citrullinémie de type 2 |
| US12128113B2 (en) | 2016-05-18 | 2024-10-29 | Modernatx, Inc. | Polynucleotides encoding JAGGED1 for the treatment of Alagille syndrome |
| JP6983455B2 (ja) | 2016-09-14 | 2021-12-17 | モデルナティーエックス, インコーポレイテッド | 高純度rna組成物及びその調製のための方法 |
| AU2017345766A1 (en) | 2016-10-21 | 2019-05-16 | Modernatx, Inc. | Human cytomegalovirus vaccine |
| CN110167587A (zh) | 2016-11-11 | 2019-08-23 | 摩登纳特斯有限公司 | 流感疫苗 |
| EP3551193A4 (fr) | 2016-12-08 | 2020-08-19 | Modernatx, Inc. | Vaccins à acide nucléique contre des virus respiratoires |
| EP3555289A1 (fr) | 2016-12-13 | 2019-10-23 | ModernaTX, Inc. | Purification par affinité d'arn |
| WO2018170270A1 (fr) | 2017-03-15 | 2018-09-20 | Modernatx, Inc. | Vaccin contre le virus varicelle-zona |
| MA47787A (fr) | 2017-03-15 | 2020-01-22 | Modernatx Inc | Vaccin contre le virus respiratoire syncytial |
| US11752206B2 (en) * | 2017-03-15 | 2023-09-12 | Modernatx, Inc. | Herpes simplex virus vaccine |
| WO2018170245A1 (fr) | 2017-03-15 | 2018-09-20 | Modernatx, Inc. | Vaccin à large spectre contre le virus de la grippe |
| KR20190132405A (ko) * | 2017-03-15 | 2019-11-27 | 모더나티엑스, 인크. | 치료제의 세포내 전달을 위한 화합물 및 조성물 |
| WO2018170347A1 (fr) | 2017-03-17 | 2018-09-20 | Modernatx, Inc. | Vaccins à base d'arn contre des maladies zoonotiques |
| WO2018187590A1 (fr) | 2017-04-05 | 2018-10-11 | Modernatx, Inc. | Réduction ou élimination de réponses immunitaires à des protéines thérapeutiques administrées par voie non intraveineuse, par exemple par voie sous-cutanée |
| EP3621637A1 (fr) * | 2017-05-09 | 2020-03-18 | Fundacion para la Investigacion Medica Aplicada | Protéines dérivées de la porphobilinogène désaminase humaine et polynucléotides et leurs utilisations |
| WO2018232357A1 (fr) | 2017-06-15 | 2018-12-20 | Modernatx, Inc. | Formulations d'arn |
| CN111212905A (zh) | 2017-08-18 | 2020-05-29 | 摩登纳特斯有限公司 | Rna聚合酶变体 |
| WO2019036685A1 (fr) | 2017-08-18 | 2019-02-21 | Modernatx, Inc. | Procédés pour analyse par clhp |
| WO2019036683A1 (fr) | 2017-08-18 | 2019-02-21 | Modernatx, Inc. | Procédés analytiques par hplc |
| WO2019046809A1 (fr) | 2017-08-31 | 2019-03-07 | Modernatx, Inc. | Procédés de fabrication de nanoparticules lipidiques |
| US10653767B2 (en) | 2017-09-14 | 2020-05-19 | Modernatx, Inc. | Zika virus MRNA vaccines |
| JP7398115B2 (ja) | 2017-12-22 | 2023-12-14 | ノース カロライナ ステート ユニバーシティ | ポリマーフルオロフォア、それを含む組成物、ならびにその調製および使用方法 |
| WO2019148101A1 (fr) | 2018-01-29 | 2019-08-01 | Modernatx, Inc. | Vaccins à base d'arn contre le vrs |
| US12151029B2 (en) | 2018-09-19 | 2024-11-26 | Modernatx, Inc. | PEG lipids and uses thereof |
| AU2019345067A1 (en) | 2018-09-19 | 2021-04-08 | Modernatx, Inc. | High-purity peg lipids and uses thereof |
| WO2020061457A1 (fr) | 2018-09-20 | 2020-03-26 | Modernatx, Inc. | Préparation de nanoparticules lipidiques et leurs méthodes d'administration |
| JP2022521094A (ja) | 2019-02-20 | 2022-04-05 | モデルナティエックス インコーポレイテッド | 共転写キャッピング用rnaポリメラーゼバリアント |
| US11851694B1 (en) | 2019-02-20 | 2023-12-26 | Modernatx, Inc. | High fidelity in vitro transcription |
| CN113874502A (zh) | 2019-03-11 | 2021-12-31 | 摩登纳特斯有限公司 | 补料分批体外转录方法 |
| WO2020190750A1 (fr) | 2019-03-15 | 2020-09-24 | Modernatx, Inc. | Vaccins à base d'arn contre le vih |
| CN115667207B (zh) * | 2020-04-01 | 2025-09-16 | 翻译生物公司 | 基于酚酸脂质的阳离子脂质 |
| WO2021204179A1 (fr) | 2020-04-09 | 2021-10-14 | Suzhou Abogen Biosciences Co., Ltd. | Vaccins à base d'acide nucléique pour coronavirus |
| KR102924173B1 (ko) | 2020-04-09 | 2026-02-06 | 쑤저우 아보젠 바이오사이언시스 컴퍼니 리미티드 | 지질 나노입자 조성물 |
| CN113521269A (zh) | 2020-04-22 | 2021-10-22 | 生物技术Rna制药有限公司 | 冠状病毒疫苗 |
| US11406703B2 (en) | 2020-08-25 | 2022-08-09 | Modernatx, Inc. | Human cytomegalovirus vaccine |
| KR20230118715A (ko) | 2020-10-14 | 2023-08-11 | 조지 메이슨 리서치 파운데이션 인코포레이티드 | 이온화 가능 지질 및 이의 제조 및 사용 방법 |
| WO2022150717A1 (fr) | 2021-01-11 | 2022-07-14 | Modernatx, Inc. | Vaccins contre le virus de la grippe à arn saisonnière |
| US20220363937A1 (en) | 2021-05-14 | 2022-11-17 | Armstrong World Industries, Inc. | Stabilization of antimicrobial coatings |
| US12186387B2 (en) | 2021-11-29 | 2025-01-07 | BioNTech SE | Coronavirus vaccine |
| US12529047B1 (en) | 2021-12-21 | 2026-01-20 | Modernatx, Inc. | mRNA quantification methods |
| 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 |
| WO2023196615A1 (fr) * | 2022-04-07 | 2023-10-12 | RNAimmune, Inc. | Composés et compositions pour l'administration de médicaments |
| WO2024002985A1 (fr) | 2022-06-26 | 2024-01-04 | BioNTech SE | Vaccin contre le coronavirus |
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 |
| JP2003505057A (ja) | 1999-07-27 | 2003-02-12 | ヘム−バイオテック エイ/エス | rhPBGDの製造及び急性間欠性ポルフィリン症(AIP)及び他のポルフィリン症の患者を処置するための新規な治療方法 |
| AU2009297243B2 (en) * | 2008-09-29 | 2015-10-29 | Proyecto De Biomedicina Cima S.L. | Porphobilinogen deaminase gene therapy |
| PL3338765T3 (pl) | 2009-12-01 | 2019-06-28 | Translate Bio, Inc. | Pochodna steroidowa dla dostarczania mrna w ludzkich chorobach genetycznych |
| JP2013095755A (ja) | 2011-11-02 | 2013-05-20 | Kyowa Hakko Kirin Co Ltd | カチオン性脂質 |
| WO2013086373A1 (fr) | 2011-12-07 | 2013-06-13 | Alnylam Pharmaceuticals, Inc. | Lipides pour l'administration d'agents actifs |
| CN108949772A (zh) | 2012-04-02 | 2018-12-07 | 现代泰克斯公司 | 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 |
| US9254311B2 (en) | 2012-04-02 | 2016-02-09 | Moderna Therapeutics, Inc. | Modified polynucleotides for the production of proteins |
| US20140275229A1 (en) | 2012-04-02 | 2014-09-18 | Moderna Therapeutics, Inc. | Modified polynucleotides encoding udp glucuronosyltransferase 1 family, polypeptide a1 |
| WO2013151663A1 (fr) | 2012-04-02 | 2013-10-10 | modeRNA Therapeutics | Polynucléotides modifiés pour la production de protéines membranaires |
| US8980864B2 (en) * | 2013-03-15 | 2015-03-17 | Moderna Therapeutics, Inc. | Compositions and methods of altering cholesterol levels |
| IL316479A (en) | 2014-06-25 | 2024-12-01 | Acuitas Therapeutics Inc | Novel lipids and lipid nanoparticle compositions for nucleic acid delivery |
| RS63030B1 (sr) | 2015-09-17 | 2022-04-29 | Modernatx Inc | Jedinjenja i kompozicije za intracelularno isporučivanje terapeutskih sredstava |
| RS63986B1 (sr) | 2015-10-28 | 2023-03-31 | Acuitas Therapeutics Inc | Novi lipidi i lipidne formulacije nanočestica za isporuku nukleinskih kiselina |
| EP3390630B1 (fr) | 2015-12-17 | 2025-01-22 | ModernaTX, Inc. | Polynucléotides codant pour la méthylmalonyl-coa mutase |
| MA45032A (fr) | 2016-05-18 | 2019-03-27 | Fundacion Para La Investig Medica Aplicada | Polynucléotides codant pour la porphobilinogène désaminase destinés au traitement de la porphyrie intermittente aiguë |
| US11001861B2 (en) | 2016-05-18 | 2021-05-11 | Modernatx, Inc. | Polynucleotides encoding galactose-1-phosphate uridylyltransferase for the treatment of galactosemia type 1 |
| MA45053A (fr) | 2016-05-18 | 2019-03-27 | Modernatx Inc | Polynucléotides codant pour un régulateur de conductance transmembranaire de fibrose kystique pour le traitement de la fibrose kystique |
| US20190298657A1 (en) | 2016-05-18 | 2019-10-03 | Modernatx, Inc. | Polynucleotides Encoding Acyl-CoA Dehydrogenase, Very Long-Chain for the Treatment of Very Long-Chain Acyl-CoA Dehydrogenase Deficiency |
| US12123030B2 (en) | 2016-05-18 | 2024-10-22 | Modernatx, Inc. | Polynucleotides encoding lipoprotein lipase for the treatment of hyperlipidemia |
| WO2017201349A1 (fr) | 2016-05-18 | 2017-11-23 | Modernatx, Inc. | Polynucléotides codant pour la citrine pour le traitement de la citrullinémie de type 2 |
-
2017
- 2017-05-18 MA MA045032A patent/MA45032A/fr unknown
- 2017-05-18 JP JP2019512957A patent/JP2019519601A/ja active Pending
- 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 |
|---|---|
| US20230112986A1 (en) | 2023-04-13 |
| JP2023062097A (ja) | 2023-05-02 |
| US12377136B2 (en) | 2025-08-05 |
| US20200085916A1 (en) | 2020-03-19 |
| JP2019519601A (ja) | 2019-07-11 |
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