KR20210057720A - Clrn1 관련된 청력 손실 및/또는 시력 손실의 치료 방법 - Google Patents
Clrn1 관련된 청력 손실 및/또는 시력 손실의 치료 방법 Download PDFInfo
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- KR20210057720A KR20210057720A KR1020217002300A KR20217002300A KR20210057720A KR 20210057720 A KR20210057720 A KR 20210057720A KR 1020217002300 A KR1020217002300 A KR 1020217002300A KR 20217002300 A KR20217002300 A KR 20217002300A KR 20210057720 A KR20210057720 A KR 20210057720A
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- C—CHEMISTRY; METALLURGY
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- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
- C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
- C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
- C12N15/86—Viral vectors
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- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
- C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
- C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- 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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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7088—Compounds having three or more nucleosides or nucleotides
- A61K31/713—Double-stranded nucleic acids or oligonucleotides
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- 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
- A61K35/12—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
- A61K35/30—Nerves; Brain; Eyes; Corneal cells; Cerebrospinal fluid; Neuronal stem cells; Neuronal precursor cells; Glial cells; Oligodendrocytes; Schwann cells; Astroglia; Astrocytes; Choroid plexus; Spinal cord tissue
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- 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
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- 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
- A61K48/0091—Purification or manufacturing processes for gene therapy compositions
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/0012—Galenical forms characterised by the site of application
- A61K9/0046—Ear
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/0012—Galenical forms characterised by the site of application
- A61K9/0048—Eye, e.g. artificial tears
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/0012—Galenical forms characterised by the site of application
- A61K9/0048—Eye, e.g. artificial tears
- A61K9/0051—Ocular inserts or implants
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- 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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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- 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
- C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
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- C07—ORGANIC CHEMISTRY
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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
- C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
- C12N15/67—General methods for enhancing the expression
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- C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
- C12N5/06—Animal cells or tissues; Human cells or tissues
- C12N5/0602—Vertebrate cells
- C12N5/0618—Cells of the nervous system
- C12N5/062—Sensory transducers, e.g. photoreceptors; Sensory neurons, e.g. for hearing, taste, smell, pH, touch, temperature, pain
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- C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
- C12N5/06—Animal cells or tissues; Human cells or tissues
- C12N5/0602—Vertebrate cells
- C12N5/0618—Cells of the nervous system
- C12N5/0621—Eye cells, e.g. cornea, iris pigmented cells
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2510/00—Genetically modified cells
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14141—Use of virus, viral particle or viral elements as a vector
- C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
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- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/42—Vector systems having a special element relevant for transcription being an intron or intervening sequence for splicing and/or stability of RNA
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- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/50—Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal
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- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- General Health & Medical Sciences (AREA)
- Genetics & Genomics (AREA)
- Medicinal Chemistry (AREA)
- Organic Chemistry (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Veterinary Medicine (AREA)
- Biomedical Technology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biotechnology (AREA)
- Zoology (AREA)
- Molecular Biology (AREA)
- Epidemiology (AREA)
- Biochemistry (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- Biophysics (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Cell Biology (AREA)
- Microbiology (AREA)
- Ophthalmology & Optometry (AREA)
- Immunology (AREA)
- Toxicology (AREA)
- Gastroenterology & Hepatology (AREA)
- Neurology (AREA)
- Physics & Mathematics (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Neurosurgery (AREA)
- Plant Pathology (AREA)
- Virology (AREA)
- Developmental Biology & Embryology (AREA)
- Manufacturing & Machinery (AREA)
- Analytical Chemistry (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862689660P | 2018-06-25 | 2018-06-25 | |
| US62/689,660 | 2018-06-25 | ||
| PCT/US2019/039030 WO2020005974A1 (en) | 2018-06-25 | 2019-06-25 | Methods of treating clrn1-associated hearing loss and/or vision loss |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| KR20210057720A true KR20210057720A (ko) | 2021-05-21 |
Family
ID=68985009
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| KR1020217002300A Ceased KR20210057720A (ko) | 2018-06-25 | 2019-06-25 | Clrn1 관련된 청력 손실 및/또는 시력 손실의 치료 방법 |
Country Status (14)
| Country | Link |
|---|---|
| US (1) | US20210277417A1 (https=) |
| EP (1) | EP3824090A4 (https=) |
| JP (3) | JP2021529519A (https=) |
| KR (1) | KR20210057720A (https=) |
| CN (1) | CN112639107B (https=) |
| AU (2) | AU2019294603B2 (https=) |
| BR (1) | BR112020026606A2 (https=) |
| CA (1) | CA3104330A1 (https=) |
| CL (1) | CL2020003346A1 (https=) |
| EA (1) | EA202190114A1 (https=) |
| IL (1) | IL279551A (https=) |
| MX (1) | MX2020013628A (https=) |
| SG (1) | SG11202012710WA (https=) |
| WO (1) | WO2020005974A1 (https=) |
Families Citing this family (3)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US20230340038A1 (en) * | 2020-09-14 | 2023-10-26 | President And Fellows Of Harvard College | Recombinant adeno associated virus (raav) encoding gjb2 and uses thereof |
| MX2023007800A (es) * | 2020-12-29 | 2023-07-11 | Akouos Inc | Composiciones y metodos para tratar la perdida de audicion y/o la perdida de vision asociada a clrn1. |
| CA3233522A1 (en) * | 2021-09-30 | 2023-04-06 | Katherine Diane GRIBBLE | Gene therapy delivery compositions and methods for treating hearing loss |
Family Cites Families (13)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US8980952B2 (en) * | 2002-03-20 | 2015-03-17 | University Of Maryland, Baltimore | Methods for treating brain swelling with a compound that blocks a non-selective cation channel |
| KR20080016786A (ko) * | 2005-02-03 | 2008-02-22 | 주식회사 바이오텍 아이 | 당뇨병 치료용 조성물 및 당뇨병 치료 방법 |
| JP2009511014A (ja) * | 2005-10-05 | 2009-03-19 | ベイヒル セラピューティクス インコーポレーティッド | 自己免疫疾患の処置のための組成物および方法 |
| US20090215178A1 (en) * | 2008-02-22 | 2009-08-27 | Zequn Tang | Methods to enhance the stability and homogeneity of transgene expression in clonal cell lines |
| WO2011061937A1 (en) * | 2009-11-19 | 2011-05-26 | Immuno Tec Laboratory Co., Ltd. | Methods for producing antibody-producing cells that produce desired polypeptides |
| RU2658490C2 (ru) * | 2012-03-27 | 2018-06-21 | Кьюрвак Аг | Искусственные молекулы нуклеиновых кислот для улучшенной экспрессии белков или пептидов |
| CN102978284A (zh) * | 2012-11-19 | 2013-03-20 | 金子兵 | 一种筛查各类眼科遗传性疾病的基因芯片及其制备和使用方法 |
| ES2697249T3 (es) * | 2013-03-11 | 2019-01-22 | Fond Telethon | miR-204 y miR-211 y usos de los mismos |
| CA2909733C (en) * | 2013-04-18 | 2023-10-17 | Fondazione Telethon | Effective delivery of large genes by dual aav vectors |
| WO2016073900A1 (en) * | 2014-11-06 | 2016-05-12 | Case Western Reserve University | Compositions and methods of treating usher syndrome iii |
| CN107921118B (zh) * | 2015-06-12 | 2022-11-08 | 葛兰素史密丝克莱恩生物有限公司 | 腺病毒多核苷酸和多肽 |
| SG11201804814YA (en) * | 2015-12-11 | 2018-07-30 | Massachusetts Eye & Ear Infirmary | Materials and methods for delivering nucleic acids to cochlear and vestibular cells |
| JP2019530737A (ja) * | 2016-08-23 | 2019-10-24 | アコーオス インコーポレイテッド | ヒト対象において非加齢性聴力障害を治療するための組成物および方法 |
-
2019
- 2019-06-25 SG SG11202012710WA patent/SG11202012710WA/en unknown
- 2019-06-25 WO PCT/US2019/039030 patent/WO2020005974A1/en not_active Ceased
- 2019-06-25 CN CN201980055197.XA patent/CN112639107B/zh active Active
- 2019-06-25 JP JP2020572414A patent/JP2021529519A/ja active Pending
- 2019-06-25 CA CA3104330A patent/CA3104330A1/en active Pending
- 2019-06-25 AU AU2019294603A patent/AU2019294603B2/en active Active
- 2019-06-25 EP EP19827048.0A patent/EP3824090A4/en active Pending
- 2019-06-25 KR KR1020217002300A patent/KR20210057720A/ko not_active Ceased
- 2019-06-25 EA EA202190114A patent/EA202190114A1/ru unknown
- 2019-06-25 BR BR112020026606-0A patent/BR112020026606A2/pt unknown
- 2019-06-25 US US17/253,658 patent/US20210277417A1/en active Pending
- 2019-06-25 MX MX2020013628A patent/MX2020013628A/es unknown
-
2020
- 2020-12-17 IL IL279551A patent/IL279551A/en unknown
- 2020-12-22 CL CL2020003346A patent/CL2020003346A1/es unknown
-
2023
- 2023-08-23 JP JP2023135405A patent/JP2023153320A/ja active Pending
-
2025
- 2025-07-03 JP JP2025112689A patent/JP2025156349A/ja active Pending
-
2026
- 2026-02-24 AU AU2026201371A patent/AU2026201371A1/en active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| EP3824090A1 (en) | 2021-05-26 |
| JP2025156349A (ja) | 2025-10-14 |
| CL2020003346A1 (es) | 2021-05-14 |
| JP2021529519A (ja) | 2021-11-04 |
| AU2026201371A1 (en) | 2026-03-19 |
| AU2019294603B2 (en) | 2025-11-27 |
| SG11202012710WA (en) | 2021-01-28 |
| CN112639107A (zh) | 2021-04-09 |
| EA202190114A1 (ru) | 2021-05-12 |
| CN112639107B (zh) | 2025-03-04 |
| BR112020026606A2 (pt) | 2021-04-06 |
| JP2023153320A (ja) | 2023-10-17 |
| US20210277417A1 (en) | 2021-09-09 |
| WO2020005974A1 (en) | 2020-01-02 |
| CA3104330A1 (en) | 2020-01-02 |
| IL279551A (en) | 2021-01-31 |
| AU2019294603A1 (en) | 2021-01-14 |
| EP3824090A4 (en) | 2022-06-01 |
| MX2020013628A (es) | 2021-05-27 |
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