AR122078A1 - Composiciones y métodos para tratar la pérdida auditiva asociada a slc26a4 - Google Patents

Composiciones y métodos para tratar la pérdida auditiva asociada a slc26a4

Info

Publication number
AR122078A1
AR122078A1 ARP210101313A ARP210101313A AR122078A1 AR 122078 A1 AR122078 A1 AR 122078A1 AR P210101313 A ARP210101313 A AR P210101313A AR P210101313 A ARP210101313 A AR P210101313A AR 122078 A1 AR122078 A1 AR 122078A1
Authority
AR
Argentina
Prior art keywords
methods
hearing loss
slc26a4
compositions
treating
Prior art date
Application number
ARP210101313A
Other languages
English (en)
Inventor
Emmanuel John Simons
Robert Ng
Yen-Fu Cheng
Original Assignee
Akouos Inc
Taipei Veterans General Hospital
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, Taipei Veterans General Hospital filed Critical Akouos Inc
Publication of AR122078A1 publication Critical patent/AR122078A1/es

Links

Classifications

    • 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
    • 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
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/1703—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • A61K38/1709—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • 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
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • 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
    • 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
    • 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
    • C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; CARE OF BIRDS, FISHES, INSECTS; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2267/00—Animals characterised by purpose
    • A01K2267/03—Animal model, e.g. for test or diseases
    • A01K2267/0306—Animal model for genetic diseases
    • 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
    • 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

Abstract

La presente invención se refiere a constructos que comprenden una secuencia codificante enlazada operativamente a un promotor, en donde la secuencia codificante codifica una proteína pendrina. Los constructos ejemplares incluyen constructos de AAV. También se proporcionan métodos de uso de constructos descritos para el tratamiento de la pérdida auditiva y/o sordera.
ARP210101313A 2020-05-13 2021-05-12 Composiciones y métodos para tratar la pérdida auditiva asociada a slc26a4 AR122078A1 (es)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US202063024466P 2020-05-13 2020-05-13

Publications (1)

Publication Number Publication Date
AR122078A1 true AR122078A1 (es) 2022-08-10

Family

ID=76584538

Family Applications (1)

Application Number Title Priority Date Filing Date
ARP210101313A AR122078A1 (es) 2020-05-13 2021-05-12 Composiciones y métodos para tratar la pérdida auditiva asociada a slc26a4

Country Status (14)

Country Link
US (1) US20230201372A1 (es)
EP (1) EP4165195A2 (es)
JP (1) JP2023526047A (es)
KR (1) KR20230041965A (es)
CN (1) CN116801911A (es)
AR (1) AR122078A1 (es)
AU (1) AU2021272971A1 (es)
BR (1) BR112022022906A2 (es)
CA (1) CA3183171A1 (es)
CL (1) CL2022003153A1 (es)
IL (1) IL298128A (es)
MX (1) MX2022014204A (es)
TW (1) TW202208405A (es)
WO (1) WO2021231567A2 (es)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2023122720A1 (en) * 2021-12-23 2023-06-29 University Of Rochester Compositions and methods for delivery of agents to inner ear

Family Cites Families (34)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP0173552B1 (en) 1984-08-24 1991-10-09 The Upjohn Company Recombinant dna compounds and the expression of polypeptides such as tpa
US5168062A (en) 1985-01-30 1992-12-01 University Of Iowa Research Foundation Transfer vectors and microorganisms containing human cytomegalovirus immediate-early promoter-regulatory DNA sequence
US4797368A (en) 1985-03-15 1989-01-10 The United States Of America As Represented By The Department Of Health And Human Services Adeno-associated virus as eukaryotic expression vector
US5139941A (en) 1985-10-31 1992-08-18 University Of Florida Research Foundation, Inc. AAV transduction vectors
US5436146A (en) 1989-09-07 1995-07-25 The Trustees Of Princeton University Helper-free stocks of recombinant adeno-associated virus vectors
US6268213B1 (en) 1992-06-03 2001-07-31 Richard Jude Samulski Adeno-associated virus vector and cis-acting regulatory and promoter elements capable of expressing at least one gene and method of using same for gene therapy
US5478745A (en) 1992-12-04 1995-12-26 University Of Pittsburgh Recombinant viral vector system
US5869305A (en) 1992-12-04 1999-02-09 The University Of Pittsburgh Recombinant viral vector system
US6204059B1 (en) 1994-06-30 2001-03-20 University Of Pittsburgh AAV capsid vehicles for molecular transfer
US6093570A (en) 1995-06-07 2000-07-25 The University Of North Carolina At Chapel Hill Helper virus-free AAV production
US5741683A (en) 1995-06-07 1998-04-21 The Research Foundation Of State University Of New York In vitro packaging of adeno-associated virus DNA
US6001650A (en) 1995-08-03 1999-12-14 Avigen, Inc. High-efficiency wild-type-free AAV helper functions
WO1998010088A1 (en) 1996-09-06 1998-03-12 Trustees Of The University Of Pennsylvania An inducible method for production of recombinant adeno-associated viruses utilizing t7 polymerase
AU728220B2 (en) 1997-04-14 2001-01-04 Cell Genesys, Inc. Methods for increasing the efficiency of recombinant AAV product
US6156303A (en) 1997-06-11 2000-12-05 University Of Washington Adeno-associated virus (AAV) isolates and AAV vectors derived therefrom
US6146874A (en) 1998-05-27 2000-11-14 University Of Florida Method of preparing recombinant adeno-associated virus compositions
CA2348382C (en) 1998-11-10 2013-09-17 The University Of North Carolina At Chapel Hill Chimeric parvovirus vectors and methods of making and administering the same
DE60139471D1 (de) 2000-06-01 2009-09-17 Univ North Carolina Verfahren und zusammensetzungen zur kontrollierter abgabe von rekombinant parvovirus vektoren
EP2305299B1 (en) 2001-05-31 2017-03-01 GlaxoSmithKline Biologicals SA Chimeric alphavirus replicon particles
DE60233061D1 (de) 2001-09-06 2009-09-03 Alphavax Inc Alphavirus replikon-vektorsysteme
NZ532635A (en) 2001-11-13 2007-05-31 Univ Pennsylvania A method of identifying unknown adeno-associated virus (AAV) sequences and a kit for the method
PT1453547T (pt) 2001-12-17 2016-12-28 Univ Pennsylvania Sequências do vírus adeno-associado (aav) do serotipo 8, vetores contendo as mesmas, e utilizações destas
AU2003274397A1 (en) 2002-06-05 2003-12-22 University Of Florida Production of pseudotyped recombinant aav virions
ES2648241T3 (es) 2003-09-30 2017-12-29 The Trustees Of The University Of Pennsylvania Clados de virus adenoasociados (AAV), secuencias, vectores que contienen el mismo, y usos de los mismos
US7557197B2 (en) 2004-01-28 2009-07-07 Oregon Health & Science University Human soluble neuropilin-1 primary polyadenylation signal and uses thereof
WO2006012414A2 (en) 2004-07-20 2006-02-02 Critical Therapeutics, Inc. Novel polyadenylation signal for use in expression vectors
ES2525067T3 (es) 2005-04-07 2014-12-17 The Trustees Of The University Of Pennsylvania Método de incremento de la función de un vector de AAV
WO2007120542A2 (en) 2006-03-30 2007-10-25 The Board Of Trustees Of The Leland Stanford Junior University Aav capsid library and aav capsid proteins
EP2831225A1 (en) 2012-03-26 2015-02-04 The United States of America, As Represented by the Secretary, Dept. of Health & Human Services Office of Technology Transfer Delivery of packaged rna to mammalian cells
GB201518979D0 (en) 2015-10-27 2015-12-09 Univ Leuven Kath Treatment of hepatic steatosis related oligo-ovulation
US20190038778A1 (en) * 2016-02-05 2019-02-07 The General Hospital Corporation Hybrid System for Efficient Gene Delivery to Cells of the Inner Ear
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
AU2019224121A1 (en) 2018-02-22 2020-08-27 Akouos, Inc. Compositions and methods for treating non-age-associated hearing impairment in a human subject
WO2020077295A1 (en) * 2018-10-11 2020-04-16 Decibel Therapeutics, Inc. Aav1 vectors and uses thereof for treatment of otic indications

Also Published As

Publication number Publication date
WO2021231567A3 (en) 2021-12-23
CL2022003153A1 (es) 2023-07-28
US20230201372A1 (en) 2023-06-29
KR20230041965A (ko) 2023-03-27
MX2022014204A (es) 2023-04-14
JP2023526047A (ja) 2023-06-20
EP4165195A2 (en) 2023-04-19
TW202208405A (zh) 2022-03-01
BR112022022906A2 (pt) 2023-01-17
IL298128A (en) 2023-01-01
AU2021272971A1 (en) 2023-02-02
CN116801911A (zh) 2023-09-22
CA3183171A1 (en) 2021-11-18
WO2021231567A2 (en) 2021-11-18

Similar Documents

Publication Publication Date Title
CL2022003147A1 (es) Composiciones y métodos para tratar pérdida auditiva asociada con gjb2
CO2021001893A2 (es) Anticuerpos anti-gdf15, composiciones y métodos de uso
CL2022003153A1 (es) Composiciones y métodos para tratar la pérdida auditiva asociada a slc26a4
PE20180241A1 (es) Adenovirus oncolitico que codifica una proteina b7
CL2019002368A1 (es) Composiciones y métodos para el tratamiento de cáncer.
EA202091995A1 (ru) Композиции и способы лечения не связанного с возрастом нарушения слуха у субъекта-человека
CO2020009402A2 (es) Proteínas de fusión fc il-22 y métodos de uso
CO2021014768A2 (es) Anticuerpos anti-sema3a y sus usos para tratar enfermedades oculares
PE20230252A1 (es) Anticuerpos anti-interleucina-33 y sus usos de estos
BR112022017192A2 (pt) Variantes de transglutaminase
CL2022002276A1 (es) Composiciones y métodos para tratar discapacidad auditiva no asociada con la edad en un sujeto
CL2021001615A1 (es) Anticuerpos anti il-36 y procedimientos de uso de estos
CO2022002573A2 (es) Anticuerpos contra ilt2 y uso de los mismos
CO2023006684A2 (es) Anticuerpos contra sars-cov-2 y usos de los mismos
CO2020015052A2 (es) Anticuerpos anti-cd63, conjugados y usos de estos
CL2019003167A1 (es) Proteínas de unión al antígeno anti-jagged1.
CO2023017812A2 (es) Ligandos crbn de (r)-glutarimida y métodos de uso
DOP2023000132A (es) Composiciones y métodos para tratar la pérdida de audición y/o la pérdida de visión asociada a clrn1
ECSP23093588A (es) Composiciones de suministro de terapia génica y métodos para tratar perdida auditiva
ECSP21087921A (es) Materiales y métodos para modular la inmunidad mediada por células t
AR122213A1 (es) Anticuerpos anti-cd200r1 y métodos de uso de estos
BR112022006820A2 (pt) Composições e métodos para inibir a perda de colágeno.
BR112022002761A2 (pt) Proteínas de ligação a 4-1bb e ox40 e composições e métodos relacionados, anticorpos contra 4-1bb, anticorpos contra ox40
BR112022015952A2 (pt) Composições de peptídeos e métodos de uso das mesmas
ECSP22020678A (es) Anticuerpos anti-nrp1a y sus usos para tratar enfermedades oculares