CL2020003346A1 - Métodos para tratar la pérdida de la audición y/o pérdida de la visión asociadas con clrn1 - Google Patents

Métodos para tratar la pérdida de la audición y/o pérdida de la visión asociadas con clrn1

Info

Publication number
CL2020003346A1
CL2020003346A1 CL2020003346A CL2020003346A CL2020003346A1 CL 2020003346 A1 CL2020003346 A1 CL 2020003346A1 CL 2020003346 A CL2020003346 A CL 2020003346A CL 2020003346 A CL2020003346 A CL 2020003346A CL 2020003346 A1 CL2020003346 A1 CL 2020003346A1
Authority
CL
Chile
Prior art keywords
clrn1
methods
loss
treating hearing
vision
Prior art date
Application number
CL2020003346A
Other languages
English (en)
Inventor
Emmanuel J Simons
Robert Ng
Original Assignee
Akouos Inc
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Akouos Inc filed Critical Akouos Inc
Publication of CL2020003346A1 publication Critical patent/CL2020003346A1/es

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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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    • 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
    • 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
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • 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
    • 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
    • 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
    • A—HUMAN NECESSITIES
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    • 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
    • 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
    • A—HUMAN NECESSITIES
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • 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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    • 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
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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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    • 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
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    • C12N2830/00—Vector systems having a special element relevant for transcription
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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)
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  • Animal Behavior & Ethology (AREA)
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  • General Engineering & Computer Science (AREA)
  • Biophysics (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Cell Biology (AREA)
  • Microbiology (AREA)
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  • Ophthalmology & Optometry (AREA)
  • Toxicology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Physics & Mathematics (AREA)
  • Neurology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Plant Pathology (AREA)
  • Neurosurgery (AREA)
  • Developmental Biology & Embryology (AREA)
  • Virology (AREA)
  • Manufacturing & Machinery (AREA)
  • Acoustics & Sound (AREA)

Abstract

Se divulgan composiciones que incluyen un solo vector de ácido nucleico y dos vectores de ácido nucleico diferentes, y el uso de estas composiciones para tratar la pérdida de la audición y/o la pérdida de la visión en un sujeto.
CL2020003346A 2018-06-25 2020-12-22 Métodos para tratar la pérdida de la audición y/o pérdida de la visión asociadas con clrn1 CL2020003346A1 (es)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US201862689660P 2018-06-25 2018-06-25

Publications (1)

Publication Number Publication Date
CL2020003346A1 true CL2020003346A1 (es) 2021-05-14

Family

ID=68985009

Family Applications (1)

Application Number Title Priority Date Filing Date
CL2020003346A CL2020003346A1 (es) 2018-06-25 2020-12-22 Métodos para tratar la pérdida de la audición y/o pérdida de la visión asociadas con clrn1

Country Status (14)

Country Link
US (1) US20210277417A1 (es)
EP (1) EP3824090A4 (es)
JP (3) JP2021529519A (es)
KR (1) KR20210057720A (es)
CN (1) CN112639107B (es)
AU (2) AU2019294603B2 (es)
BR (1) BR112020026606A2 (es)
CA (1) CA3104330A1 (es)
CL (1) CL2020003346A1 (es)
EA (1) EA202190114A1 (es)
IL (1) IL279551A (es)
MX (1) MX2020013628A (es)
SG (1) SG11202012710WA (es)
WO (1) WO2020005974A1 (es)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
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
JP2024500786A (ja) * 2020-12-29 2024-01-10 アコーオス インコーポレイテッド Ｃｌｒｎ１関連難聴及び／または視力低下を治療するための組成物及び方法
JP2024537796A (ja) * 2021-09-30 2024-10-16 アコーオス インコーポレイテッド 難聴を治療するための遺伝子療法送達組成物及び方法

Family Cites Families (13)

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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
EP1855726A2 (en) * 2005-02-03 2007-11-21 Biotech Institute For International Innovation Inc. Compositions and methods for the therapeutic treatment of diabetes
WO2007044394A2 (en) * 2005-10-05 2007-04-19 Bayhill Therapeutics, Inc. Compositions and methods for treatment of autoimmune disease
US20090215178A1 (en) * 2008-02-22 2009-08-27 Zequn Tang Methods to enhance the stability and homogeneity of transgene expression in clonal cell lines
CN102762728A (zh) * 2009-11-19 2012-10-31 株式会社免疫工学研究所 用于制备产生期望的多肽的抗体生产细胞的方法
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
CN105209619B (zh) * 2013-03-11 2018-10-12 泰莱托恩基金会 miR-204和miR-211及其用途
ES2704677T3 (es) * 2013-04-18 2019-03-19 Fond Telethon Suministro eficaz de genes grandes por vectores de AAV duales
US11827680B2 (en) * 2014-11-06 2023-11-28 Case Western Reserve University Compounds and methods of treating usher syndrome III
JP6983665B2 (ja) * 2015-06-12 2021-12-17 グラクソスミスクライン バイオロジカルズ ソシエテ アノニム アデノウイルスポリヌクレオチド及びポリペプチド
ES2903000T3 (es) * 2015-12-11 2022-03-30 Massachusetts Eye & Ear Infirmary Materiales y métodos para administrar ácidos nucleicos a las células cocleares y vestibulares
CN110225975A (zh) * 2016-08-23 2019-09-10 阿库斯股份有限公司 用于治疗人受试者中非年龄相关的听力损害的组合物和方法

Also Published As

Publication number Publication date
KR20210057720A (ko) 2021-05-21
CA3104330A1 (en) 2020-01-02
JP2023153320A (ja) 2023-10-17
EA202190114A1 (ru) 2021-05-12
US20210277417A1 (en) 2021-09-09
CN112639107B (zh) 2025-03-04
EP3824090A4 (en) 2022-06-01
AU2019294603A1 (en) 2021-01-14
MX2020013628A (es) 2021-05-27
AU2019294603B2 (en) 2025-11-27
JP2021529519A (ja) 2021-11-04
IL279551A (en) 2021-01-31
WO2020005974A1 (en) 2020-01-02
EP3824090A1 (en) 2021-05-26
BR112020026606A2 (pt) 2021-04-06
AU2026201371A1 (en) 2026-03-19
JP2025156349A (ja) 2025-10-14
CN112639107A (zh) 2021-04-09
SG11202012710WA (en) 2021-01-28

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