MA47436A - Traitement de la mucopolysaccharidose i avec une alpha-l-iduronidase glycosylée entièrement humaine (idua) - Google Patents
Traitement de la mucopolysaccharidose i avec une alpha-l-iduronidase glycosylée entièrement humaine (idua)Info
- Publication number
- MA47436A MA47436A MA047436A MA47436A MA47436A MA 47436 A MA47436 A MA 47436A MA 047436 A MA047436 A MA 047436A MA 47436 A MA47436 A MA 47436A MA 47436 A MA47436 A MA 47436A
- Authority
- MA
- Morocco
- Prior art keywords
- idua
- iduronidase
- glycosylate
- mucopolysaccharidosis
- treatment
- Prior art date
Links
- 101001019502 Homo sapiens Alpha-L-iduronidase Proteins 0.000 title 2
- 102100035028 Alpha-L-iduronidase Human genes 0.000 title 1
- 206010056886 Mucopolysaccharidosis I Diseases 0.000 title 1
- 102000056929 human IDUA Human genes 0.000 title 1
Classifications
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/33—Heterocyclic compounds
- A61K31/335—Heterocyclic compounds having oxygen as the only ring hetero atom, e.g. fungichromin
- A61K31/365—Lactones
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/33—Heterocyclic compounds
- A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
- A61K31/435—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom
- A61K31/4353—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom ortho- or peri-condensed with heterocyclic ring systems
- A61K31/436—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom ortho- or peri-condensed with heterocyclic ring systems the heterocyclic ring system containing a six-membered ring having oxygen as a ring hetero atom, e.g. rapamycin
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/56—Compounds containing cyclopenta[a]hydrophenanthrene ring systems; Derivatives thereof, e.g. steroids
- A61K31/57—Compounds containing cyclopenta[a]hydrophenanthrene ring systems; Derivatives thereof, e.g. steroids substituted in position 17 beta by a chain of two carbon atoms, e.g. pregnane or progesterone
- A61K31/573—Compounds containing cyclopenta[a]hydrophenanthrene ring systems; Derivatives thereof, e.g. steroids substituted in position 17 beta by a chain of two carbon atoms, e.g. pregnane or progesterone substituted in position 21, e.g. cortisone, dexamethasone, prednisone or aldosterone
-
- 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
- A61K38/46—Hydrolases (3)
- A61K38/47—Hydrolases (3) acting on glycosyl compounds (3.2), e.g. cellulases, lactases
-
- 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
- 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
- A61K48/0083—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 administration regime
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
- A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K2300/00—Mixtures or combinations of active ingredients, wherein at least one active ingredient is fully defined in groups A61K31/00 - A61K41/00
-
- 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
- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14141—Use of virus, viral particle or viral elements as a vector
- C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y302/00—Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
- C12Y302/01—Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
- C12Y302/01076—L-Iduronidase (3.2.1.76)
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- General Health & Medical Sciences (AREA)
- Veterinary Medicine (AREA)
- Medicinal Chemistry (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Engineering & Computer Science (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Epidemiology (AREA)
- Organic Chemistry (AREA)
- Biomedical Technology (AREA)
- Neurology (AREA)
- Neurosurgery (AREA)
- Genetics & Genomics (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Biotechnology (AREA)
- Molecular Biology (AREA)
- Gastroenterology & Hepatology (AREA)
- Immunology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Psychiatry (AREA)
- Hospice & Palliative Care (AREA)
- Biochemistry (AREA)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201762452769P | 2017-01-31 | 2017-01-31 | |
| US201762485655P | 2017-04-14 | 2017-04-14 | |
| US201762529366P | 2017-07-06 | 2017-07-06 | |
| US201762579690P | 2017-10-31 | 2017-10-31 | |
| US201862616234P | 2018-01-11 | 2018-01-11 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| MA47436A true MA47436A (fr) | 2019-12-11 |
Family
ID=63041038
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| MA047436A MA47436A (fr) | 2017-01-31 | 2018-01-30 | Traitement de la mucopolysaccharidose i avec une alpha-l-iduronidase glycosylée entièrement humaine (idua) |
Country Status (12)
| Country | Link |
|---|---|
| US (2) | US20190358303A1 (fr) |
| EP (1) | EP3576768A4 (fr) |
| JP (2) | JP2020508289A (fr) |
| KR (1) | KR20190109506A (fr) |
| AU (1) | AU2018216807A1 (fr) |
| BR (1) | BR112019015482A2 (fr) |
| CA (1) | CA3049915A1 (fr) |
| IL (1) | IL268076B2 (fr) |
| MA (1) | MA47436A (fr) |
| SG (1) | SG11201906452PA (fr) |
| UY (1) | UY37587A (fr) |
| WO (1) | WO2018144441A1 (fr) |
Families Citing this family (6)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| BR112020000063A2 (pt) | 2017-07-06 | 2020-07-14 | The Trustees Of The University Of Pennsylvania | terapia gênica mediada por aav9 para tratamento de mucopolissacaridose tipo i |
| AU2018335752A1 (en) | 2017-09-22 | 2020-03-12 | Christian HINDERER | Gene therapy for treating Mucopolysaccharidosis type ii |
| JP2021531276A (ja) * | 2018-07-18 | 2021-11-18 | レジェンクスバイオ インコーポレーテッド | 完全ヒトグリコシル化ヒトα−Ｌ−イズロニダーゼ（ＩＤＵＡ）を用いたムコ多糖症Ｉ型の治療 |
| AU2021211416A1 (en) * | 2020-01-22 | 2022-08-11 | Regenxbio Inc. | Treatment of mucopolysaccharidosis I with fully-human glycosylated human alpha-L-iduronidase (IDUA) |
| EP4284335A1 (fr) | 2021-02-01 | 2023-12-06 | RegenxBio Inc. | Thérapie génique de céroïdes-lipofuscinoses neuronales |
| CA3236188A1 (fr) * | 2021-10-27 | 2023-05-04 | The University Of North Carolina At Chapel Hill | Vecteur aav-idua pour le traitement de mps i |
Family Cites Families (13)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5196315A (en) * | 1990-05-01 | 1993-03-23 | The Johns Hopkins University | Human neuronal cell line |
| JPH06504449A (ja) * | 1991-11-14 | 1994-05-26 | ウイミンズ・アンド・チルドレンズ・ホスピタル | 合成アルファ−ｌ−イズロニダーゼおよびそれをコード化する遺伝子配列 |
| TWI235158B (en) * | 1997-09-01 | 2005-07-01 | Kirin Brewery | Human thrombopoietin produced by human established cell lines, processes foe preparing the same, and pharmaceutical composition comprising the same |
| CA2328518A1 (fr) * | 1998-05-13 | 1999-11-18 | Harbor-Ucla | (alpha)-l-iduronidase recombinee, procedes de production et de purification de celle-ci et methodes de traitement de maladies dues a des deficits en celle-ci |
| US6585971B1 (en) * | 1999-11-12 | 2003-07-01 | Harbor-Ucla Research And Education Institute | Recombinant α-L-iduronidase, methods for producing and purifying the same and methods for treating disease caused by deficiencies thereof |
| US6426208B1 (en) * | 1999-11-12 | 2002-07-30 | Harbor-Ucla Research And Education Institute | Recombinant α-L-iduronidase, methods for producing and purifying the same and methods for treating diseases caused by deficiencies thereof |
| CA2412882A1 (fr) * | 2000-06-30 | 2002-01-10 | Maxygen Aps | Polypeptides glycosyles a extension peptidique |
| US9375440B2 (en) * | 2006-11-03 | 2016-06-28 | Medtronic, Inc. | Compositions and methods for making therapies delivered by viral vectors reversible for safety and allele-specificity |
| BR112015028605A8 (pt) * | 2013-05-15 | 2019-12-24 | Univ Minnesota | uso de uma composição e uso de um imunossupressor e uma composição |
| CN105555317A (zh) * | 2013-06-11 | 2016-05-04 | 药物运输有限公司 | 与特异性生物活性货物肽偶联的源自人类的细胞渗透肽的结构、制造和用途 |
| WO2015009961A1 (fr) * | 2013-07-19 | 2015-01-22 | Armagen Technologies, Inc. | Compositions et procédés associés à des structures qui traversent la barrière hémato-encéphalique |
| KR20190060016A (ko) * | 2014-10-20 | 2019-05-31 | 뉴럴스템, 인크. | 성장 인자를 코딩하는 외인성 폴리뉴클레오티드를 포함하는 안정한 신경 줄기세포 및 그의 사용 방법 |
| EP3218000A2 (fr) * | 2014-11-10 | 2017-09-20 | Alexion Pharmaceuticals, Inc. | Compositions thérapeutiques à base d'alpha-l-iduronidase, d'iduronate-2-sulfatase et d'alpha-galactosidase a et leurs procédés d'utilisation |
-
2018
- 2018-01-30 BR BR112019015482-5A patent/BR112019015482A2/pt unknown
- 2018-01-30 JP JP2019541264A patent/JP2020508289A/ja active Pending
- 2018-01-30 CA CA3049915A patent/CA3049915A1/fr active Pending
- 2018-01-30 MA MA047436A patent/MA47436A/fr unknown
- 2018-01-30 SG SG11201906452PA patent/SG11201906452PA/en unknown
- 2018-01-30 UY UY0001037587A patent/UY37587A/es not_active Application Discontinuation
- 2018-01-30 US US16/482,057 patent/US20190358303A1/en not_active Abandoned
- 2018-01-30 IL IL268076A patent/IL268076B2/en unknown
- 2018-01-30 WO PCT/US2018/015910 patent/WO2018144441A1/fr unknown
- 2018-01-30 AU AU2018216807A patent/AU2018216807A1/en not_active Abandoned
- 2018-01-30 KR KR1020197025133A patent/KR20190109506A/ko not_active Application Discontinuation
- 2018-01-30 EP EP18748381.3A patent/EP3576768A4/fr active Pending
-
2023
- 2023-10-03 US US18/480,172 patent/US20240050537A1/en active Pending
-
2024
- 2024-05-15 JP JP2024079088A patent/JP2024112876A/ja active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| EP3576768A1 (fr) | 2019-12-11 |
| UY37587A (es) | 2018-08-31 |
| US20190358303A1 (en) | 2019-11-28 |
| CA3049915A1 (fr) | 2018-08-09 |
| SG11201906452PA (en) | 2019-08-27 |
| KR20190109506A (ko) | 2019-09-25 |
| IL268076B1 (en) | 2024-06-01 |
| US20240050537A1 (en) | 2024-02-15 |
| EP3576768A4 (fr) | 2020-11-04 |
| JP2024112876A (ja) | 2024-08-21 |
| AU2018216807A1 (en) | 2019-08-08 |
| BR112019015482A2 (pt) | 2020-03-31 |
| WO2018144441A1 (fr) | 2018-08-09 |
| IL268076B2 (en) | 2024-10-01 |
| JP2020508289A (ja) | 2020-03-19 |
| IL268076A (en) | 2019-09-26 |
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| MA47436A (fr) | Traitement de la mucopolysaccharidose i avec une alpha-l-iduronidase glycosylée entièrement humaine (idua) | |
| CY1122184T1 (el) | Συνθεσεις περιλαμβανουσες βακτηριακα στελεχη | |
| MA52789A (fr) | Méthodes de traitement du cancer avec des anticorps anti-pd-1 | |
| MD3209309T2 (ro) | Compoziții care conțin tulpini bacteriene | |
| EP3452166A4 (fr) | Stimulation intranasale pour le traitement de la maladie de la glande de meibomius et de la blépharite | |
| MA52962A (fr) | Méthodes de traitement du cancer avec des anticorps bispécifiques anti-cd3xmuc16 et des anticorps anti-pd-1 | |
| MA43190A (fr) | Traitement de la dyslipidémie mixte | |
| MA51815A (fr) | Méthodes de traitement ou de prévention de migraines avec anticorps contra le receptor de cgrp | |
| MA56212A (fr) | Compositions et méthodes de traitement du cancer | |
| MA46990A (fr) | Compositions de glp-1 et leurs utilisations | |
| BR112016022213A2 (pt) | combinações de ingredientes ativos inseticidas e nematicidas | |
| MA48730A (fr) | Compositions et méthodes de traitement de synucléinopathies | |
| MA45675A (fr) | Méthodes et compositions permettant le traitement du cancer | |
| MA49059A (fr) | Traitement des cancers positifs à her2 | |
| MA46742A (fr) | Composition pharmaceutique, méthodes de traitement et leurs utilisations | |
| MA46361A (fr) | Traitement du cancer de la prostate | |
| MA47408A (fr) | Traitement du cancer | |
| EA201691887A1 (ru) | Лечение внутрипеченочных холестатических заболеваний | |
| ZA201807438B (en) | Methods of treating ocular conditions | |
| CL2018000433A1 (es) | Procedimientos para mejorar la salud de peces de cultivo. | |
| EA201892475A1 (ru) | Оксабороловые сложные эфиры, их применение | |
| MA42930A (fr) | Traitement de maladies neurodégénératives | |
| EA201892297A1 (ru) | Способы лечения холестатических и фибротических заболеваний | |
| ZA201703467B (en) | Methods of treating ocular conditions | |
| FR3022139B1 (fr) | Nouvelles compositions pour le traitement des rougeurs cutanees et de la rosacee |