SG11201906452PA - Treatment of mucopolysaccharidosis i with fully-human glycosylated human alpha-l-iduronidase (idua) - Google Patents
Treatment of mucopolysaccharidosis i with fully-human glycosylated human alpha-l-iduronidase (idua)Info
- Publication number
- SG11201906452PA SG11201906452PA SG11201906452PA SG11201906452PA SG11201906452PA SG 11201906452P A SG11201906452P A SG 11201906452PA SG 11201906452P A SG11201906452P A SG 11201906452PA SG 11201906452P A SG11201906452P A SG 11201906452PA SG 11201906452P A SG11201906452P A SG 11201906452PA
- Authority
- SG
- Singapore
- Prior art keywords
- international
- human
- iduronidase
- idua
- mucopolysaccharidosis
- Prior art date
Links
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)
- Pharmacology & Pharmacy (AREA)
- Medicinal Chemistry (AREA)
- Veterinary Medicine (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Engineering & Computer Science (AREA)
- Epidemiology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Organic Chemistry (AREA)
- Neurosurgery (AREA)
- Neurology (AREA)
- Biomedical Technology (AREA)
- Genetics & Genomics (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Chemical Kinetics & Catalysis (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 (6)
| 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 | |
| PCT/US2018/015910 WO2018144441A1 (en) | 2017-01-31 | 2018-01-30 | Treatment of mucopolysaccharidosis i with fully-human glycosylated human alpha-l-iduronidase (idua) |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| SG11201906452PA true SG11201906452PA (en) | 2019-08-27 |
Family
ID=63041038
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| SG11201906452PA SG11201906452PA (en) | 2017-01-31 | 2018-01-30 | Treatment of mucopolysaccharidosis i with fully-human glycosylated human alpha-l-iduronidase (idua) |
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) | IL268076A (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 |
| 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 |
| KR20220131522A (ko) * | 2020-01-22 | 2022-09-28 | 리젠엑스바이오 인크. | 완전-인간 글리코실화된 인간 알파-l-이두로니다아제 (idua)를 사용한 점액다당류증 i의 치료 |
| WO2022165313A1 (en) | 2021-02-01 | 2022-08-04 | 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 (10)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5196315A (en) * | 1990-05-01 | 1993-03-23 | The Johns Hopkins University | Human neuronal cell line |
| 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 |
| EP1299535A2 (en) * | 2000-06-30 | 2003-04-09 | Maxygen Aps | Peptide extended glycosylated polypeptides |
| KR20220119187A (ko) * | 2013-05-15 | 2022-08-26 | 리젠츠 오브 더 유니버시티 오브 미네소타 | 중추 신경계로의 아데노-연관 바이러스 매개 유전자 전달 |
| DK3007729T3 (da) * | 2013-06-11 | 2019-09-30 | Portage Pharmaceuticals Ltd | Struktur, fremstilling og anvendelser af humanderiverede cellegennemtrængelige peptider, der er konjugeret med specifikke biologisk aktive cargo-peptider |
| 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 |
| EP3218000A2 (en) * | 2014-11-10 | 2017-09-20 | 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 KR KR1020197025133A patent/KR20190109506A/ko not_active Application Discontinuation
- 2018-01-30 SG SG11201906452PA patent/SG11201906452PA/en unknown
- 2018-01-30 WO PCT/US2018/015910 patent/WO2018144441A1/en unknown
- 2018-01-30 AU AU2018216807A patent/AU2018216807A1/en active Pending
- 2018-01-30 CA CA3049915A patent/CA3049915A1/en active Pending
- 2018-01-30 US US16/482,057 patent/US20190358303A1/en not_active Abandoned
- 2018-01-30 UY UY0001037587A patent/UY37587A/es unknown
- 2018-01-30 JP JP2019541264A patent/JP2020508289A/ja active Pending
- 2018-01-30 EP EP18748381.3A patent/EP3576768A4/en active Pending
- 2018-01-30 BR BR112019015482-5A patent/BR112019015482A2/pt unknown
- 2018-01-30 MA MA047436A patent/MA47436A/fr unknown
-
2019
- 2019-07-12 IL IL268076A patent/IL268076A/en unknown
-
2023
- 2023-10-03 US US18/480,172 patent/US20240050537A1/en active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| EP3576768A1 (en) | 2019-12-11 |
| MA47436A (fr) | 2019-12-11 |
| WO2018144441A1 (en) | 2018-08-09 |
| KR20190109506A (ko) | 2019-09-25 |
| UY37587A (es) | 2018-08-31 |
| US20240050537A1 (en) | 2024-02-15 |
| CA3049915A1 (en) | 2018-08-09 |
| US20190358303A1 (en) | 2019-11-28 |
| JP2020508289A (ja) | 2020-03-19 |
| AU2018216807A1 (en) | 2019-08-08 |
| BR112019015482A2 (pt) | 2020-03-31 |
| IL268076A (en) | 2019-09-26 |
| EP3576768A4 (en) | 2020-11-04 |
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