MX2020013628A - Métodos para tratar la pérdida de audición y/o la pérdida de visión asociada a clarina 1 (clrn1). - Google Patents

Métodos para tratar la pérdida de audición y/o la pérdida de visión asociada a clarina 1 (clrn1).

Info

Publication number
MX2020013628A
MX2020013628A MX2020013628A MX2020013628A MX2020013628A MX 2020013628 A MX2020013628 A MX 2020013628A MX 2020013628 A MX2020013628 A MX 2020013628A MX 2020013628 A MX2020013628 A MX 2020013628A MX 2020013628 A MX2020013628 A MX 2020013628A
Authority
MX
Mexico
Prior art keywords
loss
clrn1
treating
methods
vision
Prior art date
Application number
MX2020013628A
Other languages
English (en)
Spanish (es)
Inventor
Robert Ng
Emmanuel John Simons
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 MX2020013628A publication Critical patent/MX2020013628A/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
    • 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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    • 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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    • 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
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    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • 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
    • 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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    • 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
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    • C12N5/0618—Cells of the nervous system
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    • 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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    • 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
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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)
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  • Genetics & Genomics (AREA)
  • Medicinal Chemistry (AREA)
  • Organic Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
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  • Veterinary Medicine (AREA)
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  • 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)
  • Gastroenterology & Hepatology (AREA)
  • Toxicology (AREA)
  • Neurology (AREA)
  • Physics & Mathematics (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
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  • Neurosurgery (AREA)
  • Plant Pathology (AREA)
  • Developmental Biology & Embryology (AREA)
  • Virology (AREA)
  • Manufacturing & Machinery (AREA)
  • Analytical Chemistry (AREA)
MX2020013628A 2018-06-25 2019-06-25 Métodos para tratar la pérdida de audición y/o la pérdida de visión asociada a clarina 1 (clrn1). MX2020013628A (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201862689660P 2018-06-25 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
MX2020013628A true MX2020013628A (es) 2021-05-27

Family

ID=68985009

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2020013628A MX2020013628A (es) 2018-06-25 2019-06-25 Métodos para tratar la pérdida de audición y/o la pérdida de visión asociada a clarina 1 (clrn1).

Country Status (14)

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

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
CN117157108A (zh) * 2020-12-29 2023-12-01 阿库斯股份有限公司 用于治疗clrn1相关听力损失和/或视力损失的组合物和方法
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)

* Cited by examiner, † Cited by third party
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
JP2008529498A (ja) * 2005-02-03 2008-08-07 バイオテック インスティテュート フォー インターナショナル イノベーション，インク． 糖尿病治療用組成物類および方法
US20100160415A1 (en) * 2005-10-05 2010-06-24 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
US20130071881A1 (en) * 2009-11-19 2013-03-21 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
CN105209619B (zh) * 2013-03-11 2018-10-12 泰莱托恩基金会 miR-204和miR-211及其用途
DK2986635T3 (en) * 2013-04-18 2019-01-28 Fond Telethon EFFECTIVE DELIVERY OF BIG GENES THROUGH DUAL-AAV VECTORS
WO2016073900A1 (en) * 2014-11-06 2016-05-12 Case Western Reserve University Compositions and methods of treating usher syndrome iii
WO2016198621A1 (en) * 2015-06-12 2016-12-15 Glaxosmithkline Biologicals Sa Adenovirus polynucleotides and polypeptides
CA3007476A1 (en) * 2015-12-11 2017-06-15 Massachusetts Eye And Ear Infirmary Materials and methods for delivering nucleic acids to cochlear and vestibular cells
WO2018039375A1 (en) * 2016-08-23 2018-03-01 Akouos, Inc. Compositions and methods for treating non-age-associated hearing impairment in a human subject

Also Published As

Publication number Publication date
JP2023153320A (ja) 2023-10-17
KR20260073466A (ko) 2026-05-21
CN112639107B (zh) 2025-03-04
CL2020003346A1 (es) 2021-05-14
AU2019294603A1 (en) 2021-01-14
CA3104330A1 (en) 2020-01-02
BR112020026606A2 (pt) 2021-04-06
CN112639107A (zh) 2021-04-09
WO2020005974A1 (en) 2020-01-02
KR20210057720A (ko) 2021-05-21
US20210277417A1 (en) 2021-09-09
AU2019294603B2 (en) 2025-11-27
EP3824090A1 (en) 2021-05-26
EP3824090A4 (en) 2022-06-01
JP2025156349A (ja) 2025-10-14
JP2021529519A (ja) 2021-11-04
AU2026201371A1 (en) 2026-03-19
IL279551A (en) 2021-01-31
SG11202012710WA (en) 2021-01-28
EA202190114A1 (ru) 2021-05-12

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