KR102752457B1 - 청력 상실의 치료를 위한 유전자 요법 구축물 및 방법 - Google Patents

청력 상실의 치료를 위한 유전자 요법 구축물 및 방법 Download PDF

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KR102752457B1
KR102752457B1 KR1020197030012A KR20197030012A KR102752457B1 KR 102752457 B1 KR102752457 B1 KR 102752457B1 KR 1020197030012 A KR1020197030012 A KR 1020197030012A KR 20197030012 A KR20197030012 A KR 20197030012A KR 102752457 B1 KR102752457 B1 KR 102752457B1
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hearing loss
leu
tmprss3
gly
asp
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KR20190131052A (ko
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힌리치 스타에커
쉐 중 류
정-이 천
시저 제임스 아얄라
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레스큐 히어링 인크
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    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K67/00—Rearing or breeding animals, not otherwise provided for; New or modified breeds of animals
    • A01K67/027—New or modified breeds of vertebrates
    • A01K67/0275—Genetically modified vertebrates, e.g. transgenic
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K67/00—Rearing or breeding animals, not otherwise provided for; New or modified breeds of animals
    • A01K67/027—New or modified breeds of vertebrates
    • A01K67/0275—Genetically modified vertebrates, e.g. transgenic
    • A01K67/0278—Knock-in vertebrates, e.g. humanised vertebrates
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
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    • A61K35/76—Viruses; Subviral particles; Bacteriophages
    • A—HUMAN NECESSITIES
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    • 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
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    • 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
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    • 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
    • A—HUMAN NECESSITIES
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
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    • A01K2217/07—Animals genetically altered by homologous recombination
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    • A—HUMAN NECESSITIES
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KR1020197030012A 2017-03-17 2018-03-16 청력 상실의 치료를 위한 유전자 요법 구축물 및 방법 Active KR102752457B1 (ko)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201762472790P 2017-03-17 2017-03-17
US62/472,790 2017-03-17
US201762531522P 2017-07-12 2017-07-12
US62/531,522 2017-07-12
PCT/US2018/022873 WO2018170402A1 (en) 2017-03-17 2018-03-16 Gene therapy constructs and methods for treatment of hearing loss

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KR20190131052A KR20190131052A (ko) 2019-11-25
KR102752457B1 true KR102752457B1 (ko) 2025-01-09

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US (2) US20200248203A1 (enExample)
EP (1) EP3595634B1 (enExample)
JP (1) JP2020509786A (enExample)
KR (1) KR102752457B1 (enExample)
CN (1) CN110709060A (enExample)
BR (1) BR112019019367A2 (enExample)
CA (1) CA3054941A1 (enExample)
ES (1) ES2982270T3 (enExample)
IL (1) IL268912B2 (enExample)
WO (1) WO2018170402A1 (enExample)

Families Citing this family (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US12391936B2 (en) 2017-03-17 2025-08-19 Rescue Hearing, Inc. Gene therapy systems and related methods for treatment of hearing loss
CN112442528B (zh) * 2019-08-30 2022-08-12 深圳华大基因股份有限公司 Loxhd1基因突变体及其应用
KR20220137652A (ko) * 2020-02-05 2022-10-12 레스큐 히어링 인크 청력 손실 치료를 위한 유전자 요법 시스템 및 관련 방법
EP4008788A1 (en) * 2020-12-03 2022-06-08 Medizinische Hochschule Hannover Lentiviral vector technology for inner ear gene therapy
CN113584082B (zh) * 2021-06-22 2023-07-25 复旦大学附属眼耳鼻喉科医院 CRISPR/Cas9基因编辑系统及其在制备治疗遗传性感音神经性聋的药物中的应用
CN113215248B (zh) * 2021-06-25 2022-04-22 中国人民解放军空军军医大学 一种感音神经性耳聋相关的myo15a基因突变检测试剂盒
WO2024259309A1 (en) * 2023-06-15 2024-12-19 Regeneron Pharmaceuticals, Inc. Gene therapy for hearing disorders

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WO2004099779A1 (en) * 2003-05-05 2004-11-18 Bayer Healthcare Ag Diagnostics and therapeutics for diseases associated with human transmembrane serine protease 3 (tmprss3)
WO2011075838A1 (en) * 2009-12-21 2011-06-30 Audigen Inc. Method of treating or preventing hearing loss
US20150050354A1 (en) * 2012-04-02 2015-02-19 Moderna Therapeutics, Inc. Modified polynucleotides for the treatment of otic diseases and conditions
US20150329875A1 (en) * 2014-05-13 2015-11-19 Sangamo Biosciences, Inc. Methods and compositions for prevention or treatment of a disease

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WO2001083781A2 (en) * 2000-04-28 2001-11-08 Millennium Pharmaceuticals, Inc. 14094, a novel human trypsin family member and uses thereof
JP2009504183A (ja) * 2005-08-15 2009-02-05 ジェネンテック・インコーポレーテッド 遺伝子破壊、それに関連する組成物および方法
CN105188767A (zh) 2012-07-25 2015-12-23 布罗德研究所有限公司 可诱导的dna结合蛋白和基因组干扰工具及其应用
US20160237455A1 (en) 2013-09-27 2016-08-18 Editas Medicine, Inc. Crispr-related methods and compositions
NZ758025A (en) 2013-10-11 2022-07-01 Massachusetts Eye & Ear Infirmary Methods of predicting ancestral virus sequences and uses thereof
EP3212221B1 (en) 2014-10-29 2023-12-06 Massachusetts Eye & Ear Infirmary Efficient delivery of therapeutic molecules in vitro and in vivo
WO2017100791A1 (en) * 2015-12-11 2017-06-15 Massachusetts Eye And Ear Infirmary Materials and methods for delivering nucleic acids to cochlear and vestibular cells

Patent Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2004099779A1 (en) * 2003-05-05 2004-11-18 Bayer Healthcare Ag Diagnostics and therapeutics for diseases associated with human transmembrane serine protease 3 (tmprss3)
WO2011075838A1 (en) * 2009-12-21 2011-06-30 Audigen Inc. Method of treating or preventing hearing loss
US20150050354A1 (en) * 2012-04-02 2015-02-19 Moderna Therapeutics, Inc. Modified polynucleotides for the treatment of otic diseases and conditions
US20150329875A1 (en) * 2014-05-13 2015-11-19 Sangamo Biosciences, Inc. Methods and compositions for prevention or treatment of a disease

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EP3595634A1 (en) 2020-01-22
CN110709060A (zh) 2020-01-17
KR20190131052A (ko) 2019-11-25
JP2020509786A (ja) 2020-04-02
CA3054941A1 (en) 2018-09-20
EP3595634A4 (en) 2020-11-04
US20200248203A1 (en) 2020-08-06
EP3595634B1 (en) 2024-03-27
ES2982270T3 (es) 2024-10-15
US20200114023A1 (en) 2020-04-16
BR112019019367A2 (pt) 2020-04-14
WO2018170402A1 (en) 2018-09-20
IL268912B1 (en) 2024-07-01
IL268912A (en) 2019-10-31
IL268912B2 (en) 2024-11-01

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