EA202190114A1 - Способы лечения clrn1-ассоциированной потери слуха и/или потери зрения - Google Patents

Способы лечения clrn1-ассоциированной потери слуха и/или потери зрения

Info

Publication number
EA202190114A1
EA202190114A1 EA202190114A EA202190114A EA202190114A1 EA 202190114 A1 EA202190114 A1 EA 202190114A1 EA 202190114 A EA202190114 A EA 202190114A EA 202190114 A EA202190114 A EA 202190114A EA 202190114 A1 EA202190114 A1 EA 202190114A1
Authority
EA
Eurasian Patent Office
Prior art keywords
loss
treatment
clrn1
methods
associated hearing
Prior art date
Application number
EA202190114A
Other languages
English (en)
Inventor
Эммануэль Дж. Саймонс
Роберт НГ
Original Assignee
Акуос, Инк.
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 Акуос, Инк. filed Critical Акуос, Инк.
Publication of EA202190114A1 publication Critical patent/EA202190114A1/ru

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    • C12N15/09—Recombinant DNA-technology
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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
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • 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
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    • 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
    • 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, ocular implants
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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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
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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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    • C12N15/09—Recombinant DNA-technology
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    • 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
    • C12N2830/42—Vector systems having a special element relevant for transcription being an intron or intervening sequence for splicing and/or stability of RNA
    • C—CHEMISTRY; METALLURGY
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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

Abstract

В изобретении предложены композиции, которые содержат один вектор нуклеиновой кислоты или два разных вектора нуклеиновых кислот, и применение этих композиций для лечения потери слуха и/или потери зрения у субъекта.
EA202190114A 2018-06-25 2019-06-25 Способы лечения clrn1-ассоциированной потери слуха и/или потери зрения EA202190114A1 (ru)

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
EA202190114A1 true EA202190114A1 (ru) 2021-05-12

Family

ID=68985009

Family Applications (1)

Application Number Title Priority Date Filing Date
EA202190114A EA202190114A1 (ru) 2018-06-25 2019-06-25 Способы лечения clrn1-ассоциированной потери слуха и/или потери зрения

Country Status (14)

Country Link
US (1) US20210277417A1 (ru)
EP (1) EP3824090A4 (ru)
JP (2) JP2021529519A (ru)
KR (1) KR20210057720A (ru)
CN (1) CN112639107A (ru)
AU (1) AU2019294603A1 (ru)
BR (1) BR112020026606A2 (ru)
CA (1) CA3104330A1 (ru)
CL (1) CL2020003346A1 (ru)
EA (1) EA202190114A1 (ru)
IL (1) IL279551A (ru)
MX (1) MX2020013628A (ru)
SG (1) SG11202012710WA (ru)
WO (1) WO2020005974A1 (ru)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA3233522A1 (en) * 2021-09-30 2023-04-06 Katherine Diane GRIBBLE Gene therapy delivery compositions and methods for treating hearing loss

Family Cites Families (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR20080016786A (ko) * 2005-02-03 2008-02-22 주식회사 바이오텍 아이 당뇨병 치료용 조성물 및 당뇨병 치료 방법
ES2654205T3 (es) * 2012-03-27 2018-02-12 Curevac Ag Moléculas artificiales de ácido nucleico para la expresión mejorada de proteínas o péptidos
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
US10369231B2 (en) * 2013-03-11 2019-08-06 Fondazione Telethon MiR-204 and miR-211 and uses thereof
US11827680B2 (en) * 2014-11-06 2023-11-28 Case Western Reserve University Compounds and methods of treating usher syndrome III
US11167042B2 (en) * 2015-12-11 2021-11-09 Massachusetts Eye And 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

Also Published As

Publication number Publication date
EP3824090A1 (en) 2021-05-26
SG11202012710WA (en) 2021-01-28
CA3104330A1 (en) 2020-01-02
WO2020005974A1 (en) 2020-01-02
MX2020013628A (es) 2021-05-27
EP3824090A4 (en) 2022-06-01
IL279551A (en) 2021-01-31
JP2023153320A (ja) 2023-10-17
JP2021529519A (ja) 2021-11-04
KR20210057720A (ko) 2021-05-21
AU2019294603A1 (en) 2021-01-14
US20210277417A1 (en) 2021-09-09
BR112020026606A2 (pt) 2021-04-06
CL2020003346A1 (es) 2021-05-14
CN112639107A (zh) 2021-04-09

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