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)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- Biotechnology (AREA)
- Medicinal Chemistry (AREA)
- General Engineering & Computer Science (AREA)
- Biochemistry (AREA)
- Molecular Biology (AREA)
- Veterinary Medicine (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Pharmacology & Pharmacy (AREA)
- Biomedical Technology (AREA)
- Epidemiology (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Immunology (AREA)
- Physics & Mathematics (AREA)
- Diabetes (AREA)
- Hematology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Gastroenterology & Hepatology (AREA)
- Biophysics (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)
- Peptides Or Proteins (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 (cg-RX-API-DMAC7.html) |
| JP (2) | JP2019519601A (cg-RX-API-DMAC7.html) |
| MA (1) | MA45032A (cg-RX-API-DMAC7.html) |
Families Citing this family (56)
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| RS66380B1 (sr) | 2014-04-23 | 2025-02-28 | Modernatx Inc | Vakcine nukleinske kiseline |
| US11364292B2 (en) | 2015-07-21 | 2022-06-21 | Modernatx, Inc. | CHIKV RNA vaccines |
| WO2017020026A1 (en) | 2015-07-30 | 2017-02-02 | Modernatx, Inc. | Concatemeric peptide epitopes rnas |
| WO2017031232A1 (en) | 2015-08-17 | 2017-02-23 | Modernatx, Inc. | Methods for preparing particles and related compositions |
| EP4349405A3 (en) | 2015-10-22 | 2024-06-19 | ModernaTX, Inc. | Respiratory virus vaccines |
| 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 |
| WO2017070624A1 (en) | 2015-10-22 | 2017-04-27 | Modernatx, Inc. | Tropical disease vaccines |
| TN2018000152A1 (en) | 2015-10-22 | 2019-10-04 | Modernatx Inc | Nucleic acid vaccines for varicella zoster virus (vzv) |
| WO2017099823A1 (en) | 2015-12-10 | 2017-06-15 | Modernatx, Inc. | Compositions and methods for delivery of therapeutic agents |
| CA3007108A1 (en) | 2015-12-17 | 2017-06-22 | Modernatx, Inc. | Polynucleotides encoding methylmalonyl-coa mutase |
| WO2017201342A1 (en) | 2016-05-18 | 2017-11-23 | Modernatx, Inc. | Polynucleotides encoding jagged1 for the treatment of alagille syndrome |
| MA45041A (fr) | 2016-05-18 | 2019-03-27 | Modernatx Inc | Polynucléotides codant pour la galactose-1-phosphate uridylyltransférase destinés au traitement de la galactosémie de type 1 |
| SG11201810162PA (en) | 2016-05-18 | 2018-12-28 | Modernatx Inc | Polynucleotides encoding citrin for the treatment of citrullinemia type 2 |
| WO2017201333A1 (en) | 2016-05-18 | 2017-11-23 | Modernatx, Inc. | Polynucleotides encoding lipoprotein lipase for the treatment of hyperlipidemia |
| 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 |
| CA3036831A1 (en) | 2016-09-14 | 2018-03-22 | Modernatx, Inc. | High purity rna compositions and methods for preparation thereof |
| EP3528821A4 (en) | 2016-10-21 | 2020-07-01 | ModernaTX, Inc. | HUMAN CYTOMEGALOVIRUS VACCINE |
| EP3538146A4 (en) | 2016-11-11 | 2020-07-15 | ModernaTX, Inc. | INFLUENZA VACCINE |
| US11103578B2 (en) | 2016-12-08 | 2021-08-31 | Modernatx, Inc. | Respiratory virus nucleic acid vaccines |
| US11384352B2 (en) | 2016-12-13 | 2022-07-12 | Modernatx, Inc. | RNA affinity purification |
| US11464848B2 (en) | 2017-03-15 | 2022-10-11 | Modernatx, Inc. | Respiratory syncytial virus vaccine |
| US11576961B2 (en) | 2017-03-15 | 2023-02-14 | Modernatx, Inc. | Broad spectrum influenza virus vaccine |
| RS63953B1 (sr) * | 2017-03-15 | 2023-02-28 | Modernatx Inc | Jedinjenje i kompozicije za intracelularnu isporuku terapeutskih sredstava |
| US11752206B2 (en) * | 2017-03-15 | 2023-09-12 | Modernatx, Inc. | Herpes simplex virus vaccine |
| WO2018170270A1 (en) | 2017-03-15 | 2018-09-20 | Modernatx, Inc. | Varicella zoster virus (vzv) vaccine |
| MA47790A (fr) | 2017-03-17 | 2021-05-05 | Modernatx Inc | Vaccins à base d'arn contre des maladies zoonotiques |
| EP3607074A4 (en) | 2017-04-05 | 2021-07-07 | Modernatx, Inc. | REDUCTION OR ELIMINATION OF IMMUNE RESPONSES TO NON-INTRAVENOUS THERAPEUTIC PROTEINS, FOR EXAMPLE SUBCUTANEOUSLY |
| WO2018206125A1 (en) * | 2017-05-09 | 2018-11-15 | Fundacion Para La Investigacion Medica Aplicada | Human porphobilinogen deaminase derived proteins and polynucleotides and uses thereof |
| EP3638215A4 (en) | 2017-06-15 | 2021-03-24 | Modernatx, Inc. | RNA FORMULATIONS |
| EP3668977A4 (en) | 2017-08-18 | 2021-04-21 | Modernatx, Inc. | HPLC ANALYTICAL PROCESSES |
| EP3668971B8 (en) | 2017-08-18 | 2024-05-29 | ModernaTX, Inc. | Rna polymerase variants |
| EP3668979A4 (en) | 2017-08-18 | 2021-06-02 | Modernatx, Inc. | METHOD OF HPLC ANALYSIS |
| CA3073211A1 (en) | 2017-08-31 | 2019-03-07 | Modernatx, Inc. | Methods of making lipid nanoparticles |
| WO2019055807A1 (en) | 2017-09-14 | 2019-03-21 | Modernatx, Inc. | RNA VACCINES AGAINST ZIKA VIRUS |
| WO2019126144A1 (en) | 2017-12-22 | 2019-06-27 | North Carolina State University | Polymeric fluorophores, compositions comprising the same, and methods of preparing and using the same |
| US11911453B2 (en) | 2018-01-29 | 2024-02-27 | Modernatx, Inc. | RSV RNA vaccines |
| EP3852732A1 (en) | 2018-09-19 | 2021-07-28 | ModernaTX, Inc. | Peg lipids and uses thereof |
| CN112996854B (zh) | 2018-09-19 | 2024-08-30 | 摩登纳特斯有限公司 | 高纯度peg脂质和其用途 |
| WO2020061457A1 (en) | 2018-09-20 | 2020-03-26 | Modernatx, Inc. | Preparation of lipid nanoparticles and methods of administration thereof |
| MA55037A (fr) | 2019-02-20 | 2021-12-29 | Modernatx Inc | Variants d'arn polymérase pour le coiffage co-transcriptionnel |
| US11851694B1 (en) | 2019-02-20 | 2023-12-26 | Modernatx, Inc. | High fidelity in vitro transcription |
| KR20210149727A (ko) | 2019-03-11 | 2021-12-09 | 모더나티엑스, 인크. | 유가식(fed-batch) 시험관내 전사 공정 |
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| KR102813425B1 (ko) | 2020-04-22 | 2025-05-28 | 바이오엔테크 에스이 | 코로나바이러스 백신 |
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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 |
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Family Cites Families (21)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO1999037325A2 (en) | 1998-01-27 | 1999-07-29 | Hemebiotech A/S | Method for treating acute intermittent porphyria (aip) and other porphyric diseases |
| WO2001007065A2 (en) | 1999-07-27 | 2001-02-01 | Hemebiotech A/S | PRODUCTION OF rhPBGD AND NEW THERAPEUTIC METHODS FOR TREATING PATIENTS WITH ACUTE INTERMITTENT PORPHYRIA (AIP) AND OTHER PORPHYRIC DISEASES |
| EA201170506A1 (ru) | 2008-09-29 | 2011-12-30 | Амстердам Молекьюлар Терапьютикс (Амт) Б.В. | Генная терапия порфобилиногендезаминазой |
| US20110244026A1 (en) | 2009-12-01 | 2011-10-06 | Braydon Charles Guild | Delivery of mrna for the augmentation 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 |
| WO2013086373A1 (en) | 2011-12-07 | 2013-06-13 | Alnylam Pharmaceuticals, Inc. | Lipids for the delivery of active agents |
| US20140275229A1 (en) | 2012-04-02 | 2014-09-18 | Moderna Therapeutics, Inc. | Modified polynucleotides encoding udp glucuronosyltransferase 1 family, polypeptide a1 |
| US10501512B2 (en) | 2012-04-02 | 2019-12-10 | Modernatx, Inc. | Modified polynucleotides |
| 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 |
| EP2834260A4 (en) | 2012-04-02 | 2016-08-10 | Moderna Therapeutics Inc | MODIFIED POLYNUCLEOTIDES FOR THE PREPARATION OF MEMBRANE PROTEINS |
| US8980864B2 (en) * | 2013-03-15 | 2015-03-17 | Moderna Therapeutics, Inc. | Compositions and methods of altering cholesterol levels |
| IL298516B2 (en) | 2014-06-25 | 2025-03-01 | Acuitas Therapeutics Inc | Novel lipids and lipid nanoparticle compositions for nucleic acid delivery |
| ES2910425T3 (es) | 2015-09-17 | 2022-05-12 | Modernatx Inc | Compuestos y composiciones para la administración intracelular de agentes terapéuticos |
| SMT202300052T1 (it) * | 2015-10-28 | 2023-05-12 | Acuitas Therapeutics Inc | Nuovi lipidi e formulazioni di nanoparticelle lipidiche per il rilascio di acidi nucleici |
| CA3007108A1 (en) | 2015-12-17 | 2017-06-22 | Modernatx, Inc. | Polynucleotides encoding methylmalonyl-coa mutase |
| 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 |
| 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 |
| WO2017201333A1 (en) | 2016-05-18 | 2017-11-23 | Modernatx, Inc. | Polynucleotides encoding lipoprotein lipase for the treatment of hyperlipidemia |
| MA45041A (fr) | 2016-05-18 | 2019-03-27 | Modernatx Inc | Polynucléotides codant pour la galactose-1-phosphate uridylyltransférase destinés au traitement de la galactosémie de type 1 |
| SG11201810162PA (en) | 2016-05-18 | 2018-12-28 | Modernatx Inc | Polynucleotides encoding citrin for the treatment of citrullinemia type 2 |
-
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 |
|---|---|
| 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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