KR20200053468A - I형 점액다당류증을 치료하기 위한 유전자 요법 - Google Patents

I형 점액다당류증을 치료하기 위한 유전자 요법 Download PDF

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KR20200053468A
KR20200053468A KR1020207003304A KR20207003304A KR20200053468A KR 20200053468 A KR20200053468 A KR 20200053468A KR 1020207003304 A KR1020207003304 A KR 1020207003304A KR 20207003304 A KR20207003304 A KR 20207003304A KR 20200053468 A KR20200053468 A KR 20200053468A
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vector
hidua
months
human
dose
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Korean (ko)
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크리스티안 힌더러
제임스 엠. 윌슨
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더 트러스티스 오브 더 유니버시티 오브 펜실바니아
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Priority to KR1020247013510A priority Critical patent/KR20240063170A/ko
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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
    • A61K48/005—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
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • A—HUMAN NECESSITIES
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    • 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
    • A61K31/34—Heterocyclic compounds having oxygen as the only ring hetero atom, e.g. fungichromin having five-membered rings with one oxygen as the only ring hetero atom, e.g. isosorbide
    • A61K31/343—Heterocyclic compounds having oxygen as the only ring hetero atom, e.g. fungichromin having five-membered rings with one oxygen as the only ring hetero atom, e.g. isosorbide condensed with a carbocyclic ring, e.g. coumaran, bufuralol, befunolol, clobenfurol, amiodarone
    • A—HUMAN NECESSITIES
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    • 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
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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
    • A61K31/439—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 the ring forming part of a bridged ring system, e.g. quinuclidine
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    • A61K31/33—Heterocyclic compounds
    • A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/535—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with at least one nitrogen and one oxygen as the ring hetero atoms, e.g. 1,2-oxazines
    • A61K31/5375—1,4-Oxazines, e.g. morpholine
    • A61K31/5377—1,4-Oxazines, e.g. morpholine not condensed and containing further heterocyclic rings, e.g. timolol
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    • 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
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    • A61K38/47—Hydrolases (3) acting on glycosyl compounds (3.2), e.g. cellulases, lactases
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    • A61K9/0012—Galenical forms characterised by the site of application
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    • C12N15/09—Recombinant DNA-technology
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    • C12N9/14—Hydrolases (3)
    • C12N9/24—Hydrolases (3) acting on glycosyl compounds (3.2)
    • C12N9/2402—Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1)
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    • C12Y302/01—Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
    • C12Y302/01076—L-Iduronidase (3.2.1.76)
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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
    • A—HUMAN NECESSITIES
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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
    • A61K48/0083—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
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
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    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
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  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
KR1020207003304A 2017-07-06 2018-07-05 I형 점액다당류증을 치료하기 위한 유전자 요법 Ceased KR20200053468A (ko)

Priority Applications (1)

Application Number Priority Date Filing Date Title
KR1020247013510A KR20240063170A (ko) 2017-07-06 2018-07-05 I형 점액다당류증을 치료하기 위한 유전자 요법

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US201762529385P 2017-07-06 2017-07-06
US62/529,385 2017-07-06
US201762530614P 2017-07-10 2017-07-10
US62/530,614 2017-07-10
US201862616106P 2018-01-11 2018-01-11
US62/616,106 2018-01-11
PCT/US2018/040957 WO2019010335A1 (en) 2017-07-06 2018-07-05 AAV9 MEDIATED GENE THERAPY FOR TREATING MUCOPOLYSACCHARIDOSIS TYPE I

Related Child Applications (1)

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KR1020247013510A Division KR20240063170A (ko) 2017-07-06 2018-07-05 I형 점액다당류증을 치료하기 위한 유전자 요법

Publications (1)

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KR20200053468A true KR20200053468A (ko) 2020-05-18

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KR1020207003304A Ceased KR20200053468A (ko) 2017-07-06 2018-07-05 I형 점액다당류증을 치료하기 위한 유전자 요법
KR1020247013510A Pending KR20240063170A (ko) 2017-07-06 2018-07-05 I형 점액다당류증을 치료하기 위한 유전자 요법

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KR1020247013510A Pending KR20240063170A (ko) 2017-07-06 2018-07-05 I형 점액다당류증을 치료하기 위한 유전자 요법

Country Status (11)

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US (2) US11890329B2 (https=)
EP (1) EP3676385A1 (https=)
JP (2) JP2020526514A (https=)
KR (2) KR20200053468A (https=)
AU (1) AU2018298133A1 (https=)
BR (1) BR112020000063A2 (https=)
CA (1) CA3068328A1 (https=)
IL (1) IL271780A (https=)
MA (1) MA50016A (https=)
SG (1) SG11201912631PA (https=)
WO (1) WO2019010335A1 (https=)

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EP3411488A1 (en) 2016-02-03 2018-12-12 The Trustees Of The University Of Pennsylvania Gene therapy for treating mucopolysaccharidosis type i
MA50016A (fr) 2017-07-06 2020-07-08 Univ Pennsylvania Thérapie génique médiée par aav9 pour traiter la mucopolysaccharidose de type i
TWI835747B (zh) 2017-09-22 2024-03-21 賓州大學委員會 用於治療黏多醣病 ii 型之基因治療
WO2020113034A1 (en) * 2018-11-30 2020-06-04 Avexis, Inc. Aav viral vectors and uses thereof
WO2021081217A1 (en) * 2019-10-23 2021-04-29 The Trustees Of The University Of Pennsylvania Compositions for drg-specific reduction of transgene expression
WO2021150570A1 (en) * 2020-01-22 2021-07-29 Regenxbio Inc. Treatment of mucopolysaccharidosis i with fully-human glycosylated human alpha-l-iduronidase (idua)
IL300407A (en) * 2020-08-10 2023-04-01 Prevail Therapeutics Inc Gene therapies for lysosomal disorders
US20230346979A1 (en) * 2020-08-10 2023-11-02 Prevail Therapeutics, Inc. Gene therapies for neurodegenerative disorders
WO2022165313A1 (en) 2021-02-01 2022-08-04 Regenxbio Inc. Gene therapy for neuronal ceroid lipofuscinoses
WO2022170082A1 (en) * 2021-02-05 2022-08-11 Regents Of The University Of Minnesota Methods for preventing cardiac or skeletal defects in diseases including mucopolysaccharidoses
EP4294461A4 (en) * 2021-02-22 2025-06-25 Decibel Therapeutics, Inc. METHODS AND COMPOSITIONS FOR REDUCING NUCLEIC ACID VECTOR-INDUCED TOXICITY IN THE INNER EAR
WO2024205528A1 (en) * 2023-03-31 2024-10-03 Erci̇yes Üni̇versi̇tesi̇ Strateji̇ Geli̇şti̇rme Dai̇re Başkanliği A multi-head for use in electrohydrodynamic

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