UY37587A - Tratamiento de mucopolisacaridosis i con alfa-l-iduronidasa (idua) humana glicosilada completamente humana - Google Patents
Tratamiento de mucopolisacaridosis i con alfa-l-iduronidasa (idua) humana glicosilada completamente humanaInfo
- Publication number
- UY37587A UY37587A UY0001037587A UY37587A UY37587A UY 37587 A UY37587 A UY 37587A UY 0001037587 A UY0001037587 A UY 0001037587A UY 37587 A UY37587 A UY 37587A UY 37587 A UY37587 A UY 37587A
- Authority
- UY
- Uruguay
- Prior art keywords
- human
- idua
- mucopolisacaridosis
- iduronidasa
- glicosilated
- Prior art date
Links
- 102100035028 Alpha-L-iduronidase Human genes 0.000 title abstract 3
- 101001019502 Homo sapiens Alpha-L-iduronidase Proteins 0.000 title abstract 2
- 210000003169 central nervous system Anatomy 0.000 abstract 2
- 108010003381 Iduronidase Proteins 0.000 abstract 1
- 206010056886 Mucopolysaccharidosis I Diseases 0.000 abstract 1
- 210000001175 cerebrospinal fluid Anatomy 0.000 abstract 1
- 238000003745 diagnosis Methods 0.000 abstract 1
- 238000000034 method Methods 0.000 abstract 1
- 239000000203 mixture Substances 0.000 abstract 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
- 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
- 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
- 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)
- Medicinal Chemistry (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Engineering & Computer Science (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Epidemiology (AREA)
- Organic Chemistry (AREA)
- Neurosurgery (AREA)
- Neurology (AREA)
- Biomedical Technology (AREA)
- Genetics & Genomics (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Biotechnology (AREA)
- Molecular Biology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Immunology (AREA)
- Gastroenterology & Hepatology (AREA)
- Psychiatry (AREA)
- Hospice & Palliative Care (AREA)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Biochemistry (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)
Abstract
Se describen composiciones y métodos para la administración de una a-L-iduronidasa (IDUA) glicosilada completamente humana (HuGly) al líquido cefalorraquídeo del sistema nervioso central (SNC) de un sujeto humano con diagnóstico de mucopolisacaridosis I (MPS I).
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 |
---|---|
UY37587A true UY37587A (es) | 2018-08-31 |
Family
ID=63041038
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
UY0001037587A UY37587A (es) | 2017-01-31 | 2018-01-30 | Tratamiento de mucopolisacaridosis i con alfa-l-iduronidasa (idua) humana glicosilada completamente humana |
Country Status (12)
Country | Link |
---|---|
US (2) | US20190358303A1 (es) |
EP (1) | EP3576768A4 (es) |
JP (1) | JP2020508289A (es) |
KR (1) | KR20190109506A (es) |
AU (1) | AU2018216807A1 (es) |
BR (1) | BR112019015482A2 (es) |
CA (1) | CA3049915A1 (es) |
IL (1) | IL268076B1 (es) |
MA (1) | MA47436A (es) |
SG (1) | SG11201906452PA (es) |
UY (1) | UY37587A (es) |
WO (1) | WO2018144441A1 (es) |
Families Citing this family (6)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
EP3676385A1 (en) | 2017-07-06 | 2020-07-08 | The Trustees of The University of Pennsylvania | Aav9-mediated gene therapy for treating mucopolysaccharidosis type i |
EP3684938A1 (en) | 2017-09-22 | 2020-07-29 | The Trustees of the University of Pennsylvania | Gene therapy for treating mucopolysaccharidosis type ii |
AU2019306236A1 (en) * | 2018-07-18 | 2021-03-11 | Regenxbio Inc. | Treatment of mucopolysaccharidosis I with fully-human glycosylated human alpha-L-iduronidase (IDUS) |
BR112022014563A2 (pt) * | 2020-01-22 | 2022-09-13 | Regenxbio Inc | Método para tratamento de um sujeito humano diagnosticado com mucopolissacaridose i (mps i) |
EP4284335A1 (en) | 2021-02-01 | 2023-12-06 | RegenxBio Inc. | Gene therapy for neuronal ceroid lipofuscinoses |
CA3236188A1 (en) * | 2021-10-27 | 2023-05-04 | The University Of North Carolina At Chapel Hill | Aav-idua vector for treatment of 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 |
KR930703447A (ko) * | 1991-11-14 | 1993-11-30 | 지오프리 태터살 | 합성 알파-엘-이두로니다제 및 이를 암호화하는 유전자 서열 |
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 |
JP2002514429A (ja) * | 1998-05-13 | 2002-05-21 | ハーバー−ユーシーエルエー | 組換えα−L−イズロニダーゼ、その生成及び精製方法及びその欠損により引き起こされる疾病の治療方法 |
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 |
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 |
JP2004504016A (ja) * | 2000-06-30 | 2004-02-12 | マキシゲン・エイピーエス | ペプチド拡張されたグリコシル化ポリペプチド |
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 |
AU2014310360B2 (en) * | 2013-06-11 | 2019-10-24 | PPL (BVI) Limited | Structure, manufacturing and uses of human-derived cell-permeable peptides conjugated with specific biologically active cargo peptides |
US10906981B2 (en) * | 2013-07-19 | 2021-02-02 | The Regents Of The University Of California | Compositions and methods related to structures that cross the blood brain barrier |
WO2016064737A1 (en) * | 2014-10-20 | 2016-04-28 | Neuralstem, Inc. | Stable neural stem cells comprising an exogenous polynucleotide coding for a growth factor and methods of use thereof |
US20180185495A1 (en) * | 2014-11-10 | 2018-07-05 | Alexion Pharmaceuticals, Inc. | Therapeutic compositions of alpha-l-iduronidase, iduronate-2-sulfatase, and alpha-galactosidase a and methods of use thereof |
-
2018
- 2018-01-30 EP EP18748381.3A patent/EP3576768A4/en active Pending
- 2018-01-30 SG SG11201906452PA patent/SG11201906452PA/en unknown
- 2018-01-30 UY UY0001037587A patent/UY37587A/es unknown
- 2018-01-30 AU AU2018216807A patent/AU2018216807A1/en active Pending
- 2018-01-30 US US16/482,057 patent/US20190358303A1/en not_active Abandoned
- 2018-01-30 IL IL268076A patent/IL268076B1/en unknown
- 2018-01-30 JP JP2019541264A patent/JP2020508289A/ja active Pending
- 2018-01-30 KR KR1020197025133A patent/KR20190109506A/ko not_active Application Discontinuation
- 2018-01-30 MA MA047436A patent/MA47436A/fr unknown
- 2018-01-30 BR BR112019015482-5A patent/BR112019015482A2/pt unknown
- 2018-01-30 WO PCT/US2018/015910 patent/WO2018144441A1/en unknown
- 2018-01-30 CA CA3049915A patent/CA3049915A1/en active Pending
-
2023
- 2023-10-03 US US18/480,172 patent/US20240050537A1/en active Pending
Also Published As
Publication number | Publication date |
---|---|
SG11201906452PA (en) | 2019-08-27 |
CA3049915A1 (en) | 2018-08-09 |
JP2020508289A (ja) | 2020-03-19 |
WO2018144441A1 (en) | 2018-08-09 |
US20240050537A1 (en) | 2024-02-15 |
IL268076B1 (en) | 2024-06-01 |
IL268076A (en) | 2019-09-26 |
KR20190109506A (ko) | 2019-09-25 |
EP3576768A1 (en) | 2019-12-11 |
MA47436A (fr) | 2019-12-11 |
EP3576768A4 (en) | 2020-11-04 |
AU2018216807A1 (en) | 2019-08-08 |
BR112019015482A2 (pt) | 2020-03-31 |
US20190358303A1 (en) | 2019-11-28 |
Similar Documents
Publication | Publication Date | Title |
---|---|---|
UY37587A (es) | Tratamiento de mucopolisacaridosis i con alfa-l-iduronidasa (idua) humana glicosilada completamente humana | |
CL2018001372A1 (es) | Moléculas de anticuerpo contra april y usos de las mismas | |
EA201792613A1 (ru) | Модуляторы cot и способы их применения | |
CL2017002066A1 (es) | Proteasas de cisteína | |
EA201792547A1 (ru) | Популяции бактерий для стимуляции состояния здоровья | |
EA201692529A1 (ru) | Способы и композиции для лечения расстройств с использованием фоллистатиновых полипептидов | |
EA201791866A1 (ru) | Бициклические гетероциклы в качестве ингибиторов fgfr | |
MA51815A (fr) | Méthodes de traitement ou de prévention de migraines avec anticorps contra le receptor de cgrp | |
BR112016016158A2 (pt) | Derivados de heterociclila bicíclicos como inibidores de irak4, sua composição farmacêutica e seus usos | |
EA201691582A1 (ru) | Новые фармацевтические препараты | |
BR112017003863A2 (pt) | “modulador óptico e sistema óptico modulador de múltiplos segmentos” | |
BR112017008659A2 (pt) | ?métodos e compostos de agonista de gip? | |
BR112017018181A2 (pt) | plugue médico e conector de engate para estabelecer uma comunicação fluida entre dois sistemas. | |
EA201692031A1 (ru) | Фармацевтическая композиция с активным действием в отношении хронических бактериальных инфекций | |
CL2019002251A1 (es) | Anticuerpos contra triptasa, composiciones de estos y usos de estos. | |
UY37679A (es) | Tratamiento de mucopolisacaridosis ii con iduronato-2-sulfatasa (ids) humana recombinante producida por células neurales o gliales humanas | |
EA201792612A1 (ru) | 6-аминохинолин-3-карбонитрилы в качестве модуляторов cot | |
MX2019006281A (es) | Cartuchos de filtro tubulares y ovoides y sistemas de filtro que los utilizan. | |
MX2019002924A (es) | Composiciones purificadas de oligosacaridos de leche humana. | |
ITUB20152054A1 (it) | Sistema di monitoraggio dello stato di salute e/o di attività fisica | |
MX2020009709A (es) | Composiciones farmaceuticas que comprenden acido dihomo-gamma-linolenico (dgla) y uso de las mismas. | |
UY36173A (es) | Metodo de tratamiento de enfermedades relacionadas con el factor inducible por hipoxia | |
FR3022139B1 (fr) | Nouvelles compositions pour le traitement des rougeurs cutanees et de la rosacee | |
BR112022014563A2 (pt) | Método para tratamento de um sujeito humano diagnosticado com mucopolissacaridose i (mps i) | |
EA202090270A1 (ru) | Новые замещенные производные ксантина |