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)
- Pharmacology & Pharmacy (AREA)
- Molecular Biology (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (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)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Saccharide Compounds (AREA)
- Medicinal Preparation (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Enzymes And Modification 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 (38)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
US11364292B2 (en) | 2015-07-21 | 2022-06-21 | Modernatx, Inc. | CHIKV RNA vaccines |
WO2017031232A1 (fr) | 2015-08-17 | 2017-02-23 | Modernatx, Inc. | Procédés de préparation de particules et compositions associées |
WO2017070624A1 (fr) | 2015-10-22 | 2017-04-27 | Modernatx, Inc. | Vaccins contre des maladies tropicales |
JP6921833B2 (ja) | 2015-10-22 | 2021-08-18 | モデルナティーエックス, インコーポレイテッド | ヒトサイトメガロウイルスワクチン |
JP6925688B2 (ja) | 2015-10-22 | 2021-08-25 | モデルナティーエックス, インコーポレイテッド | 水痘帯状疱疹ウイルス(vzv)のための核酸ワクチン |
MD3718565T2 (ro) | 2015-10-22 | 2022-09-30 | Modernatx Inc | Vaccinuri împotriva virusului respirator |
MD3386484T2 (ro) | 2015-12-10 | 2022-11-30 | Modernatx Inc | Compoziții și metode de livrare a unor agenți terapeutici |
CA3007108A1 (fr) | 2015-12-17 | 2017-06-22 | Modernatx, Inc. | Polynucleotides codant pour la methylmalonyl-coa mutase |
MA45036A (fr) | 2016-05-18 | 2019-03-27 | Modernatx Inc | Polynucléotides codant pour la citrine pour le traitement de la citrullinémie de type 2 |
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 |
CA3036831A1 (fr) | 2016-09-14 | 2018-03-22 | Modernatx, Inc. | Compositions d'arn de haute purete et procedes pour leur preparation |
WO2018075980A1 (fr) | 2016-10-21 | 2018-04-26 | Modernatx, Inc. | Vaccin contre le cytomégalovirus humain |
US10925958B2 (en) | 2016-11-11 | 2021-02-23 | Modernatx, Inc. | Influenza vaccine |
WO2018107088A2 (fr) | 2016-12-08 | 2018-06-14 | Modernatx, Inc. | Vaccins à acide nucléique contre des virus respiratoires |
US11384352B2 (en) | 2016-12-13 | 2022-07-12 | Modernatx, Inc. | RNA affinity purification |
WO2018170245A1 (fr) | 2017-03-15 | 2018-09-20 | Modernatx, Inc. | Vaccin à large spectre contre le virus de la grippe |
US11752206B2 (en) * | 2017-03-15 | 2023-09-12 | Modernatx, Inc. | Herpes simplex virus vaccine |
US11045540B2 (en) | 2017-03-15 | 2021-06-29 | Modernatx, Inc. | Varicella zoster virus (VZV) vaccine |
US11464848B2 (en) | 2017-03-15 | 2022-10-11 | Modernatx, Inc. | Respiratory syncytial virus vaccine |
EP3595676A4 (fr) | 2017-03-17 | 2021-05-05 | Modernatx, Inc. | Vaccins à base d'arn contre des maladies zoonotiques |
EP3607074A4 (fr) | 2017-04-05 | 2021-07-07 | Modernatx, Inc. | Réduction ou élimination de réponses immunitaires à des protéines thérapeutiques administrées par voie non intraveineuse, par exemple par voie sous-cutanée |
WO2018206125A1 (fr) * | 2017-05-09 | 2018-11-15 | 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 |
WO2019036685A1 (fr) | 2017-08-18 | 2019-02-21 | Modernatx, Inc. | Procédés pour analyse par clhp |
CN111212905A (zh) | 2017-08-18 | 2020-05-29 | 摩登纳特斯有限公司 | Rna聚合酶变体 |
US11866696B2 (en) | 2017-08-18 | 2024-01-09 | Modernatx, Inc. | Analytical HPLC methods |
WO2019046809A1 (fr) | 2017-08-31 | 2019-03-07 | Modernatx, Inc. | Procédés de fabrication de nanoparticules lipidiques |
EP3681514A4 (fr) | 2017-09-14 | 2021-07-14 | ModernaTX, Inc. | Vaccins à arn contre le virus zika |
MA54676A (fr) | 2018-01-29 | 2021-11-17 | Modernatx Inc | Vaccins à base d'arn contre le vrs |
US11851694B1 (en) | 2019-02-20 | 2023-12-26 | Modernatx, Inc. | High fidelity in vitro transcription |
WO2021204179A1 (fr) | 2020-04-09 | 2021-10-14 | Suzhou Abogen Biosciences Co., Ltd. | Vaccins à base d'acide nucléique pour coronavirus |
TW202204309A (zh) | 2020-04-09 | 2022-02-01 | 大陸商蘇州艾博生物科技有限公司 | 脂質奈米粒子組成物 |
WO2021213924A1 (fr) | 2020-04-22 | 2021-10-28 | BioNTech SE | Vaccin contre un coronavirus |
US11406703B2 (en) | 2020-08-25 | 2022-08-09 | Modernatx, Inc. | Human cytomegalovirus vaccine |
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 (3)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
ES2507540T3 (es) * | 2008-09-29 | 2014-10-15 | Uniqure Ip B.V. | Terapia génica de porfobilinógeno deaminasa |
JP2013095755A (ja) * | 2011-11-02 | 2013-05-20 | Kyowa Hakko Kirin Co Ltd | カチオン性脂質 |
WO2013151667A1 (fr) * | 2012-04-02 | 2013-10-10 | modeRNA Therapeutics | Polynucléotides modifiés |
-
2017
- 2017-05-18 JP JP2019512957A patent/JP2019519601A/ja active Pending
- 2017-05-18 US US16/302,339 patent/US20200085916A1/en not_active Abandoned
- 2017-05-18 MA MA045032A patent/MA45032A/fr unknown
-
2022
- 2022-07-21 US US17/813,984 patent/US20230112986A1/en active Pending
-
2023
- 2023-02-17 JP JP2023023253A patent/JP2023062097A/ja active Pending
Also Published As
Publication number | Publication date |
---|---|
JP2023062097A (ja) | 2023-05-02 |
US20200085916A1 (en) | 2020-03-19 |
US20230112986A1 (en) | 2023-04-13 |
JP2019519601A (ja) | 2019-07-11 |
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