KR20200104864A - 뮤코다당류증 iiib형에 대한 유전자 요법 - Google Patents

뮤코다당류증 iiib형에 대한 유전자 요법 Download PDF

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KR20200104864A
KR20200104864A KR1020207018109A KR20207018109A KR20200104864A KR 20200104864 A KR20200104864 A KR 20200104864A KR 1020207018109 A KR1020207018109 A KR 1020207018109A KR 20207018109 A KR20207018109 A KR 20207018109A KR 20200104864 A KR20200104864 A KR 20200104864A
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제임스 엠. 윌슨
크리스티안 힌더러
줄리에트 호르도
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더 트러스티스 오브 더 유니버시티 오브 펜실바니아
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  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
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KR1020207018109A 2017-11-30 2018-11-29 뮤코다당류증 iiib형에 대한 유전자 요법 Withdrawn KR20200104864A (ko)

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Application Number Priority Date Filing Date Title
US201762593090P 2017-11-30 2017-11-30
US62/593,090 2017-11-30
PCT/US2018/063166 WO2019108856A1 (en) 2017-11-30 2018-11-29 Gene therapy for mucopolysaccharidosis iiib

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KR20200104864A true KR20200104864A (ko) 2020-09-04

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US (2) US11723989B2 (https=)
EP (1) EP3717652A4 (https=)
JP (1) JP7384797B2 (https=)
KR (1) KR20200104864A (https=)
CN (1) CN111989396A (https=)
AU (1) AU2018375163A1 (https=)
BR (1) BR112020010977A2 (https=)
CA (1) CA3083761A1 (https=)
CL (1) CL2020001428A1 (https=)
EA (1) EA202091352A1 (https=)
IL (1) IL274972A (https=)
MX (1) MX2020005673A (https=)
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US12605465B2 (en) 2020-03-23 2026-04-21 The University Of North Carolina At Chapel Hill AAV-NAGLU vectors for treatment of mucopolysaccharidosis IIIB
US20230183741A1 (en) * 2020-05-11 2023-06-15 University Of Florida Research Foundation, Incorporated Disease correction by delivery of aav8 vectors expressing codon optimized naglu
MX2022014258A (es) 2020-05-12 2023-02-22 Univ Pennsylvania Composiciones para la reducción específica de un fármaco de la expresión de transgén.
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WO2022165313A1 (en) 2021-02-01 2022-08-04 Regenxbio Inc. Gene therapy for neuronal ceroid lipofuscinoses
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WO2023077133A2 (en) * 2021-10-29 2023-05-04 The Johns Hopkins University Controlling homeostatic regulatory circuitry in hypothalamus
US20250295807A1 (en) 2021-11-15 2025-09-25 The Trustees Of The University Of Pennsylvania Compositions for drg-specific reduction of transgene expression
WO2024258961A1 (en) 2023-06-12 2024-12-19 The Trustees Of The University Of Pennsylvania Aav gene therapy for mucopolysaccharidosis iiib
WO2025157848A1 (en) * 2024-01-23 2025-07-31 Institut National de la Santé et de la Recherche Médicale Methods for the intravenous treatment of sanfilippo syndrome type iiib

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CN111989396A (zh) 2020-11-24
SG11202004991SA (en) 2020-06-29
US20200289675A1 (en) 2020-09-17
AU2018375163A1 (en) 2020-06-11
EP3717652A1 (en) 2020-10-07
JP7384797B2 (ja) 2023-11-21
JP2021507687A (ja) 2021-02-25
CL2020001428A1 (es) 2020-10-23
CA3083761A1 (en) 2019-06-06
US11723989B2 (en) 2023-08-15
US20240009327A1 (en) 2024-01-11
IL274972A (en) 2020-07-30
WO2019108856A1 (en) 2019-06-06
BR112020010977A2 (pt) 2020-11-17
EA202091352A1 (ru) 2020-08-27
MX2020005673A (es) 2020-12-03
EP3717652A4 (en) 2021-11-24

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