EP 4359006 A1 20240501 - ANTISENSE COMPOUNDS AND METHODS FOR TARGETING CUG REPEATS
Title (en)
ANTISENSE COMPOUNDS AND METHODS FOR TARGETING CUG REPEATS
Title (de)
ANTISENSE-VERBINDUNGEN UND VERFAHREN ZUM TARGETING VON CUG-WIEDERHOLUNGEN
Title (fr)
COMPOSÉS ANTISENS ET MÉTHODES DE CIBLAGE DE RÉPÉTITIONS DE CUG
Publication
Application
Priority
- US 202163213900 P 20210623
- US 202163239671 P 20210901
- US 202163239847 P 20210901
- US 202163290892 P 20211217
- US 202163290960 P 20211217
- US 202263298565 P 20220111
- US 202263305071 P 20220131
- US 202263268577 P 20220225
- US 202263314369 P 20220226
- US 202263316634 P 20220304
- US 202263317856 P 20220308
- US 202263326201 P 20220331
- US 202263362295 P 20220331
- US 202263327179 P 20220404
- US 202263339250 P 20220506
- US 2022034517 W 20220622
Abstract (en)
[origin: WO2022271818A1] Compounds comprising a cyclic peptide, such as a cyclic cell penetrating peptide, and an antisense compound are provided. The antisense compound binds to a gene having an expanded CTG repeat or a gene transcript having an expanded CUG repeat. The compounds can be delivered to subjects to treat diseases associated with expanded CTG·CUG repeats, such as myotonic dystrophy type 1 (DM1), spinocerebellar ataxia-8 (SCA8), and Huntington disease like-2 (HDL2).
IPC 8 full level
A61K 47/64 (2017.01); C07K 7/64 (2006.01); C12N 15/113 (2010.01)
CPC (source: EP IL KR)
A61K 31/712 (2013.01 - EP IL KR); A61K 38/00 (2013.01 - IL); A61K 47/6455 (2017.08 - EP IL KR); A61P 21/00 (2018.01 - EP IL KR); A61P 25/00 (2018.01 - EP IL); C07K 7/64 (2013.01 - EP IL KR); C07K 19/00 (2013.01 - EP IL KR); C12N 15/113 (2013.01 - EP IL KR); A01K 2217/072 (2013.01 - EP IL); A01K 2227/105 (2013.01 - EP IL); A01K 2267/0306 (2013.01 - EP IL); A61K 38/00 (2013.01 - EP); A61K 2300/00 (2013.01 - IL); C12N 2310/11 (2013.01 - EP IL KR); C12N 2310/3233 (2013.01 - EP IL KR); C12N 2310/3513 (2013.01 - EP IL KR)
C-Set (source: EP)
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 2022271818 A1 20221229; AU 2022298774 A1 20231214; CA 3222824 A1 20221229; CO 2023018002 A2 20240205; DO P2023000279 A 20240430; EP 4359006 A1 20240501; IL 309001 A 20240201; KR 20240038967 A 20240326
DOCDB simple family (application)
US 2022034517 W 20220622; AU 2022298774 A 20220622; CA 3222824 A 20220622; CO 2023018002 A 20240109; DO 2023000279 A 20231218; EP 22744043 A 20220622; IL 30900123 A 20231130; KR 20247002577 A 20220622