KR20220148162A - 인간 신경 또는 신경아교 세포에 의해 생성된 재조합 인간 이두로네이트-2-설파타아제 (ids)를 사용한 점액다당류증 ii의 치료 - Google Patents

인간 신경 또는 신경아교 세포에 의해 생성된 재조합 인간 이두로네이트-2-설파타아제 (ids)를 사용한 점액다당류증 ii의 치료 Download PDF

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KR20220148162A
KR20220148162A KR1020227027253A KR20227027253A KR20220148162A KR 20220148162 A KR20220148162 A KR 20220148162A KR 1020227027253 A KR1020227027253 A KR 1020227027253A KR 20227027253 A KR20227027253 A KR 20227027253A KR 20220148162 A KR20220148162 A KR 20220148162A
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human
certain embodiments
recombinant
ids
dose
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Korean (ko)
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스티븐 조셉 파콜라
파울로 팔라벨라
마리-로르 뉴보렛
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리젠엑스바이오 인크.
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0075—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 delivery route, e.g. oral, subcutaneous
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    • A61K31/00—Medicinal preparations containing organic active ingredients
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    • 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
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    • 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
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    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
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    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y301/00—Hydrolases acting on ester bonds (3.1)
    • C12Y301/06—Sulfuric ester hydrolases (3.1.6)
    • C12Y301/06013—Iduronate-2-sulfatase (3.1.6.13)
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    • G01N2333/914—Hydrolases (3)
    • G01N2333/916—Hydrolases (3) acting on ester bonds (3.1), e.g. phosphatases (3.1.3), phospholipases C or phospholipases D (3.1.4)
    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N2400/00—Assays, e.g. immunoassays or enzyme assays, involving carbohydrates
    • G01N2400/10—Polysaccharides, i.e. having more than five saccharide radicals attached to each other by glycosidic linkages; Derivatives thereof, e.g. ethers, esters
    • G01N2400/38—Heteroglycans, i.e. polysaccharides having more than one sugar residue in the main chain in either alternating or less regular sequence, e.g. gluco- or galactomannans, Konjac gum, Locust bean gum or Guar gum
    • G01N2400/40—Glycosaminoglycans, i.e. GAG or mucopolysaccharides, e.g. chondroitin sulfate, dermatan sulfate, hyaluronic acid, heparin, heparan sulfate, and related sulfated polysaccharides

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  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Peptides Or Proteins (AREA)
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KR1020227027253A 2020-01-29 2021-01-28 인간 신경 또는 신경아교 세포에 의해 생성된 재조합 인간 이두로네이트-2-설파타아제 (ids)를 사용한 점액다당류증 ii의 치료 Pending KR20220148162A (ko)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US202062967494P 2020-01-29 2020-01-29
US62/967,494 2020-01-29
US202063066625P 2020-08-17 2020-08-17
US63/066,625 2020-08-17
US202063088305P 2020-10-06 2020-10-06
US63/088,305 2020-10-06
PCT/US2021/015446 WO2021154963A1 (en) 2020-01-29 2021-01-28 Treatment of mucopolysaccharidosis ii with recombinant human iduronate-2-sulfatase (ids) produced by human neural or glial cells

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KR20220148162A true KR20220148162A (ko) 2022-11-04

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KR1020227027253A Pending KR20220148162A (ko) 2020-01-29 2021-01-28 인간 신경 또는 신경아교 세포에 의해 생성된 재조합 인간 이두로네이트-2-설파타아제 (ids)를 사용한 점액다당류증 ii의 치료

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Country Link
US (1) US20230088992A1 (https=)
EP (1) EP4096631A1 (https=)
JP (1) JP2023512242A (https=)
KR (1) KR20220148162A (https=)
AU (1) AU2021214174A1 (https=)
BR (1) BR112022015183A2 (https=)
CA (1) CA3167741A1 (https=)
IL (1) IL294636A (https=)
TW (1) TW202142695A (https=)
WO (1) WO2021154963A1 (https=)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR20190139951A (ko) 2017-04-14 2019-12-18 리젠엑스바이오 인크. 인간 뉴런 또는 신경교 세포에 의해 생성된 재조합 인간 이두로네이트-2 설파타제(ids)를 이용한 뮤코다당증 ii형의 치료
US11822232B2 (en) * 2020-07-28 2023-11-21 Synopsys, Inc. Dose information generation and communication for lithography manufacturing systems
EP4291249A2 (en) 2021-02-10 2023-12-20 RegenxBio Inc. Treatment of mucopolysaccharidosis ii with recombinant human iduronate-2-sulfatase (ids)
CN120550109B (zh) * 2025-05-29 2026-04-17 普大生物科技(泰州)有限公司 一种包含多价肺炎球菌多糖-蛋白结合物的组合物及其制备方法和应用

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PT1453547T (pt) 2001-12-17 2016-12-28 Univ Pennsylvania Sequências do vírus adeno-associado (aav) do serotipo 8, vetores contendo as mesmas, e utilizações destas
EP2345731B1 (en) 2003-09-30 2015-10-21 The Trustees of the University of Pennsylvania Adeno-associated virus (AAV) clades, sequences, vectors containing same, and uses thereof
ES2525067T3 (es) 2005-04-07 2014-12-17 The Trustees Of The University Of Pennsylvania Método de incremento de la función de un vector de AAV
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KR20230159646A (ko) * 2010-06-25 2023-11-21 샤이어 휴먼 지네틱 테라피즈 인크. 이듀로네이트-2-설파타제의 cns 전달을 위한 방법들 및 조성물들
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KR20190139951A (ko) * 2017-04-14 2019-12-18 리젠엑스바이오 인크. 인간 뉴런 또는 신경교 세포에 의해 생성된 재조합 인간 이두로네이트-2 설파타제(ids)를 이용한 뮤코다당증 ii형의 치료
KR20220131522A (ko) * 2020-01-22 2022-09-28 리젠엑스바이오 인크. 완전-인간 글리코실화된 인간 알파-l-이두로니다아제 (idua)를 사용한 점액다당류증 i의 치료

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EP4096631A1 (en) 2022-12-07
US20230088992A1 (en) 2023-03-23
JP2023512242A (ja) 2023-03-24
IL294636A (en) 2022-09-01
BR112022015183A2 (pt) 2022-12-20
WO2021154963A1 (en) 2021-08-05
AU2021214174A1 (en) 2022-08-18
CA3167741A1 (en) 2021-08-05
TW202142695A (zh) 2021-11-16

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