EP 4069841 A1 20221012 - TARGETED TRANSFER RNAS FOR TREATMENT OF DISEASES
Title (en)
TARGETED TRANSFER RNAS FOR TREATMENT OF DISEASES
Title (de)
GEZIELTE TRANSFER-RNAS ZUR BEHANDLUNG VON KRANKHEITEN
Title (fr)
ARN DE TRANSFERT CIBLÉS POUR LE TRAITEMENT DE MALADIES
Publication
Application
Priority
- US 201962942690 P 20191202
- US 201962942667 P 20191202
- US 202063010856 P 20200416
- US 202063111856 P 20201110
- US 2020062671 W 20201201
Abstract (en)
[origin: WO2021113218A1] Provided are engineered tRNA molecules and vectors encoding engineered suppressor tRNA molecules that recognize and readthrough of disease-causing premature stop codons. Pharmaceutical formulations of the engineered tRNA or vectors encoding the engineered tRNA are provided for use in treating diseases associated with truncation of a protein caused by premature stop codons present in the nucleic acid sequence encoding the protein.
IPC 8 full level
A61K 31/7088 (2006.01); A61K 48/00 (2006.01); C12N 15/11 (2006.01)
CPC (source: EP)
A61K 31/7088 (2013.01); A61K 31/712 (2013.01); C12N 15/11 (2013.01); C12N 15/85 (2013.01); C12N 15/86 (2013.01); C12N 2310/51 (2013.01); C12N 2320/51 (2013.01); C12N 2750/14143 (2013.01)
Citation (search report)
See references of WO 2021113218A1
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
DOCDB simple family (publication)
WO 2021113218 A1 20210610; AU 2020396868 A1 20220714; CA 3163514 A1 20210610; CN 115397983 A 20221125; EP 4069841 A1 20221012; JP 2023504661 A 20230206
DOCDB simple family (application)
US 2020062671 W 20201201; AU 2020396868 A 20201201; CA 3163514 A 20201201; CN 202080093906 A 20201201; EP 20829427 A 20201201; JP 2022533139 A 20201201