EP 4121120 A2 20230125 - METHODS OF TREATING MITOCHONDRIAL DISORDERS
Title (en)
METHODS OF TREATING MITOCHONDRIAL DISORDERS
Title (de)
VERFAHREN ZUR BEHANDLUNG VON MITOCHONDRIALEN ERKRANKUNGEN
Title (fr)
MÉTHODES DE TRAITEMENT DE TROUBLES MITOCHONDRIAUX
Publication
Application
Priority
- US 202016820368 A 20200316
- US 2021021850 W 20210311
Abstract (en)
[origin: WO2021188349A2] Provided herein are methods for treating a disease or disorder associated with mitochondrial dysfunction through ex vivo introduction of a nucleic acid molecule into hematopoietic stem and progenitor cells (HSPCs) followed by transplantation of the HSPCs into a subject in need of treatment. The nucleic acid molecule may include a functional human frataxin (hFXN) or may include a gene editing system that when transfected into the cells removes a trinucleotide extension mutation of endogenous hFXN.
IPC 8 full level
A61K 48/00 (2006.01); A61K 35/28 (2006.01); C12N 15/11 (2006.01); C12N 15/63 (2006.01); C12N 15/86 (2006.01)
CPC (source: EP)
A61K 39/461 (2023.05); A61K 39/4614 (2023.05); A61K 39/4622 (2023.05); A61K 39/46432 (2023.05); A61K 48/005 (2013.01); C07K 14/47 (2013.01); C12N 9/22 (2013.01); C12N 15/113 (2013.01); C12N 15/907 (2013.01); A61K 2239/31 (2023.05); A61K 2239/38 (2023.05); C12N 2310/20 (2017.05); C12N 2740/16043 (2013.01)
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 2021188349 A2 20210923; WO 2021188349 A3 20211021; AU 2021239908 A1 20221006; CA 3171432 A1 20210923; EP 4121120 A2 20230125
DOCDB simple family (application)
US 2021021850 W 20210311; AU 2021239908 A 20210311; CA 3171432 A 20210311; EP 21771515 A 20210311