JP2020509786A - 聴覚損失の治療のための遺伝子治療構成物および方法 - Google Patents
聴覚損失の治療のための遺伝子治療構成物および方法 Download PDFInfo
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- JP2020509786A JP2020509786A JP2020500024A JP2020500024A JP2020509786A JP 2020509786 A JP2020509786 A JP 2020509786A JP 2020500024 A JP2020500024 A JP 2020500024A JP 2020500024 A JP2020500024 A JP 2020500024A JP 2020509786 A JP2020509786 A JP 2020509786A
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- C12N9/14—Hydrolases (3)
- C12N9/48—Hydrolases (3) acting on peptide bonds (3.4)
- C12N9/50—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25)
- C12N9/64—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue
- C12N9/6421—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue from mammals
- C12N9/6424—Serine endopeptidases (3.4.21)
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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
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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
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- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P27/00—Drugs for disorders of the senses
- A61P27/16—Otologicals
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- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
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- C12N5/06—Animal cells or tissues; Human cells or tissues
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- 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
- A01K2217/00—Genetically modified animals
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- A01K2217/072—Animals genetically altered by homologous recombination maintaining or altering function, i.e. knock in
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- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
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- 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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- A01K2227/10—Mammal
- A01K2227/105—Murine
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- 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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- C12N2750/14011—Parvoviridae
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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 |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2020509786A true JP2020509786A (ja) | 2020-04-02 |
| JP2020509786A5 JP2020509786A5 (enExample) | 2021-03-11 |
Family
ID=63522674
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020500024A Pending JP2020509786A (ja) | 2017-03-17 | 2018-03-16 | 聴覚損失の治療のための遺伝子治療構成物および方法 |
Country Status (10)
| Country | Link |
|---|---|
| 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) |
Cited By (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP2024500314A (ja) * | 2020-12-03 | 2024-01-09 | メディツィーニシェ・ホーホシューレ・ハノーファー | 内耳遺伝子治療のためのレンチウイルスベクター技術 |
Families Citing this family (6)
| 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 |
| CN115820654A (zh) * | 2019-08-30 | 2023-03-21 | 深圳华大基因股份有限公司 | Loxhd1基因突变体及其应用 |
| IL295265A (en) * | 2020-02-05 | 2022-10-01 | Rescue Hearing Inc | Gene therapy systems and related methods for treatment of hearing loss |
| 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 |
Citations (5)
| 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) |
| 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 |
| US20130095071A1 (en) * | 2009-12-21 | 2013-04-18 | 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 |
Family Cites Families (6)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP1280923A2 (en) * | 2000-04-28 | 2003-02-05 | Millennium Pharmaceuticals, Inc. | 14094, a human trypsin family member and uses thereof |
| EP2877213B1 (en) | 2012-07-25 | 2020-12-02 | The Broad Institute, Inc. | Inducible dna binding proteins and genome perturbation tools and applications thereof |
| US20160237455A1 (en) | 2013-09-27 | 2016-08-18 | Editas Medicine, Inc. | Crispr-related methods and compositions |
| JP6552511B2 (ja) | 2013-10-11 | 2019-07-31 | マサチューセッツ アイ アンド イヤー インファーマリー | 祖先ウイルス配列を予測する方法およびその使用 |
| WO2016069910A1 (en) | 2014-10-29 | 2016-05-06 | Massachusetts Eye And Ear Infirmary | Methods for efficient delivery of therapeutic molecules in vitro and in vivo |
| JP6990182B2 (ja) * | 2015-12-11 | 2022-02-15 | マサチューセッツ アイ アンド イヤー インファーマリー | 蝸牛および前庭細胞に核酸を送達するための材料および方法 |
-
2018
- 2018-03-16 KR KR1020197030012A patent/KR102752457B1/ko active Active
- 2018-03-16 BR BR112019019367A patent/BR112019019367A2/pt unknown
- 2018-03-16 WO PCT/US2018/022873 patent/WO2018170402A1/en not_active Ceased
- 2018-03-16 ES ES18768248T patent/ES2982270T3/es active Active
- 2018-03-16 US US16/488,103 patent/US20200248203A1/en active Pending
- 2018-03-16 CN CN201880028001.3A patent/CN110709060A/zh active Pending
- 2018-03-16 EP EP18768248.9A patent/EP3595634B1/en active Active
- 2018-03-16 IL IL268912A patent/IL268912B2/en unknown
- 2018-03-16 JP JP2020500024A patent/JP2020509786A/ja active Pending
- 2018-03-16 CA CA3054941A patent/CA3054941A1/en active Pending
-
2019
- 2019-12-24 US US16/726,495 patent/US20200114023A1/en active Pending
Patent Citations (5)
| 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) |
| 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 |
| US20130095071A1 (en) * | 2009-12-21 | 2013-04-18 | 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 |
Non-Patent Citations (4)
| Title |
|---|
| "Definition:Homo sapiens cDNA FLJ32670 fis, clone TESTI1000127, weakly similar to POLYCYSTIN PRECURSO", DATABASE GENBANK[ONLINE], JPN6022046834, 9 January 2008 (2008-01-09), ISSN: 0004915293 * |
| "Definition:Homo sapiens transmembrane protease, serine 3. mRNA(cDNA clone MGC:104100 IMAGE:30915553)", DATABASE GENBANK[ONLINE], JPN6022046836, 15 July 2006 (2006-07-15), ISSN: 0004915292 * |
| CHUNG J, ET AL., J MOL MED, vol. 92(6), JPN6022046835, 2014, pages 651 - 663, ISSN: 0004915294 * |
| GRILLET N, ET AL.: "Mutations in LOXHD1, an evolutionarily conserved stereociliary protein, disrupt hair cell function i", AMJ HUM GENET, vol. 2009;85(3), JPN6021052615, 2009, pages 328 - 337, ISSN: 0004915291 * |
Cited By (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP2024500314A (ja) * | 2020-12-03 | 2024-01-09 | メディツィーニシェ・ホーホシューレ・ハノーファー | 内耳遺伝子治療のためのレンチウイルスベクター技術 |
Also Published As
| Publication number | Publication date |
|---|---|
| BR112019019367A2 (pt) | 2020-04-14 |
| US20200114023A1 (en) | 2020-04-16 |
| US20200248203A1 (en) | 2020-08-06 |
| KR20190131052A (ko) | 2019-11-25 |
| CN110709060A (zh) | 2020-01-17 |
| CA3054941A1 (en) | 2018-09-20 |
| KR102752457B1 (ko) | 2025-01-09 |
| IL268912B1 (en) | 2024-07-01 |
| EP3595634A4 (en) | 2020-11-04 |
| WO2018170402A1 (en) | 2018-09-20 |
| IL268912A (en) | 2019-10-31 |
| EP3595634A1 (en) | 2020-01-22 |
| ES2982270T3 (es) | 2024-10-15 |
| IL268912B2 (en) | 2024-11-01 |
| EP3595634B1 (en) | 2024-03-27 |
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