EP 4211151 A1 20230719 - RECOMBINANT ADENO ASSOCIATED VIRUS (RAAV) ENCODING GJB2 AND USES THEREOF
Title (en)
RECOMBINANT ADENO ASSOCIATED VIRUS (RAAV) ENCODING GJB2 AND USES THEREOF
Title (de)
FÜR GJB2 CODIERENDES REKOMBINANTES ADENO-ASSOZIIERTES VIRUS (RAAV) UND VERWENDUNGEN DAVON
Title (fr)
VIRUS ADÉNO-ASSOCIÉ RECOMBINANT (RVAA) CODANT GJB2 ET SES UTILISATIONS
Publication
Application
Priority
- US 202063078233 P 20200914
- US 202163161619 P 20210316
- US 2021050205 W 20210914
Abstract (en)
[origin: WO2022056444A1] The present disclosure, at least in part, relates to compositions (e.g., isolated nucleic acid and rAAVs) and methods for treating Non-syndromic hearing loss and deafness (DFNB1) by delivering gap junction beta 2 (GJB2) protein to inner ear cells that normally express GJB2 (e.g., fibrocytes and supporting cells of the organ of Corti and nearby regions). The isolated nucleic acid of the present disclosure comprises an expression cassette, wherein the expression cassette comprises a gap junction beta 2 (GJB2) gene regulatory element (GRE) (e.g., GJB2 enhancers, GJB2 promoters, GJB2 5' UTR, and/or GJB2 3' UTR), and a nucleotide sequence encoding a GJB2 protein.
IPC 8 full level
C07K 14/705 (2006.01); C12N 15/864 (2006.01)
CPC (source: EP IL KR)
A61K 48/005 (2013.01 - KR); A61P 27/16 (2018.01 - KR); C07K 14/47 (2013.01 - EP IL KR); C12N 15/86 (2013.01 - EP IL KR); C12N 2750/14143 (2013.01 - EP IL KR); C12N 2830/008 (2013.01 - EP IL KR); C12N 2830/48 (2013.01 - EP IL KR); C12N 2830/50 (2013.01 - EP IL KR)
Designated contracting state (EPC)
AL AT BE BG CH CY CZ DE DK EE ES FI FR GB GR HR HU IE IS IT LI LT LU LV MC MK MT NL NO PL PT RO RS SE SI SK SM TR
Designated extension state (EPC)
BA ME
Designated validation state (EPC)
KH MA MD TN
DOCDB simple family (publication)
WO 2022056444 A1 20220317; AU 2021339843 A1 20230406; BR 112023004605 A2 20230411; CA 3191533 A1 20220317; EP 4211151 A1 20230719; IL 301057 A 20230501; JP 2023541443 A 20231002; KR 20230069157 A 20230518; MX 2023002978 A 20230601; TW 202227476 A 20220716
DOCDB simple family (application)
US 2021050205 W 20210914; AU 2021339843 A 20210914; BR 112023004605 A 20210914; CA 3191533 A 20210914; EP 21867807 A 20210914; IL 30105723 A 20230301; JP 2023516689 A 20210914; KR 20237012321 A 20210914; MX 2023002978 A 20210914; TW 110134291 A 20210914