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 humana

Info

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
Application number
UY0001037587A
Other languages
English (en)
Inventor
Stephen Yoo
Matthew Simpson Curran
Zhuchun Wu
Robert Reinhardt Rickey
Original Assignee
Regenxbio Inc
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Regenxbio Inc filed Critical Regenxbio Inc
Publication of UY37587A publication Critical patent/UY37587A/es

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)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Engineering & Computer Science (AREA)
  • Epidemiology (AREA)
  • Bioinformatics & Cheminformatics (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)

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).
UY0001037587A 2017-01-31 2018-01-30 Tratamiento de mucopolisacaridosis i con alfa-l-iduronidasa (idua) humana glicosilada completamente humana UY37587A (es)

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) IL268076A (es)
MA (1) MA47436A (es)
SG (1) SG11201906452PA (es)
UY (1) UY37587A (es)
WO (1) WO2018144441A1 (es)

Families Citing this family (6)

* Cited by examiner, † Cited by third party
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
JP7449223B2 (ja) 2017-09-22 2024-03-13 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア ムコ多糖症ｉｉ型を治療するための遺伝子療法
US20210275647A1 (en) * 2018-07-18 2021-09-09 Regenxbio Inc. Treatment of mucopolysaccharidosis i with fully-human glycosylated human alpha-l-iduronidase (idua)
AU2021211416A1 (en) * 2020-01-22 2022-08-11 Regenxbio Inc. Treatment of mucopolysaccharidosis I with fully-human glycosylated human alpha-L-iduronidase (IDUA)
CA3209779A1 (en) 2021-02-01 2022-08-04 Regenxbio Inc. Gene therapy for neuronal ceroid lipofuscinoses
WO2023076972A1 (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)

* Cited by examiner, † Cited by third party
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
EP1078075A2 (en) * 1998-05-13 2001-02-28 Harbor-Ucla Research and Education Institute Recombinant (alpha)-l-iduronidase, methods for producing and purifying the same and methods for treating diseases 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
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
WO2002002597A2 (en) * 2000-06-30 2002-01-10 Maxygen Aps Peptide extended glycosylated polypeptides
KR20160010526A (ko) * 2013-05-15 2016-01-27 리젠츠 오브 더 유니버시티 오브 미네소타 중추 신경계로의 아데노-연관 바이러스 매개 유전자 전달
DK3581207T3 (da) * 2013-06-11 2023-02-06 Ppl Bvi Ltd Fusionsproteiner omfattende hoxd12 og nemo-bindingsdomæne og anvendelse deraf
KR20190060016A (ko) * 2014-10-20 2019-05-31 뉴럴스템, 인크. 성장 인자를 코딩하는 외인성 폴리뉴클레오티드를 포함하는 안정한 신경 줄기세포 및 그의 사용 방법
JP2017534640A (ja) * 2014-11-10 2017-11-24 アレクシオン ファーマシューティカルズ， インコーポレイテッド アルファ−ｌ−イズロニダーゼ、イズロン酸−２−スルファターゼおよびアルファガラクトシダーゼａの治療用組成物ならびにそれらの使用方法

Also Published As

Publication number Publication date
JP2020508289A (ja) 2020-03-19
AU2018216807A1 (en) 2019-08-08
CA3049915A1 (en) 2018-08-09
KR20190109506A (ko) 2019-09-25
EP3576768A4 (en) 2020-11-04
BR112019015482A2 (pt) 2020-03-31
WO2018144441A1 (en) 2018-08-09
US20240050537A1 (en) 2024-02-15
MA47436A (fr) 2019-12-11
US20190358303A1 (en) 2019-11-28
IL268076A (en) 2019-09-26
EP3576768A1 (en) 2019-12-11
SG11201906452PA (en) 2019-08-27

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