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CO2023018002A2 - Antisense compounds and methods for targeting cug repeats - Google Patents

Antisense compounds and methods for targeting cug repeats

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Publication number
CO2023018002A2
CO2023018002A2CONC2023/0018002ACO2023018002ACO2023018002A2CO 2023018002 A2CO2023018002 A2CO 2023018002A2CO 2023018002 ACO2023018002 ACO 2023018002ACO 2023018002 A2CO2023018002 A2CO 2023018002A2
Authority
CO
Colombia
Prior art keywords
methods
antisense compounds
cug repeats
expanded
compounds
Prior art date
Application number
CONC2023/0018002A
Other languages
Spanish (es)
Inventor
Xiulong Shen
Ziqing Qian
Patrick Dougherty
Mahboubeh Kheirabadi
Xiang Li
Original Assignee
Entrada Therapeutics Inc
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 Entrada Therapeutics IncfiledCriticalEntrada Therapeutics Inc
Publication of CO2023018002A2publicationCriticalpatent/CO2023018002A2/en

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Abstract

Translated fromSpanish

Se proporcionan compuestos que comprenden un péptido cíclico, tal como un péptido penetrante de células cíclico, y un compuesto antisentido. El compuesto antisentido se une a un gen que tiene una repetición CTG expandida o un transcrito génico que tiene una repetición CUG expandida. Los compuestos pueden suministrarse a los sujetos para tratar enfermedades asociadas con repeticiones CTG·CUG expandidas, tales como la distrofia miotónica tipo 1 (DM1), la ataxia espinocerebelosa-8 (SCA8) y la enfermedad similar a Huntington-2 (HDL2).Provided are compounds comprising a cyclic peptide, such as a cyclic cell penetrating peptide, and an antisense compound. The antisense compound binds to a gene that has an expanded CTG repeat or a gene transcript that has an expanded CUG repeat. The compounds can be administered to subjects to treat diseases associated with expanded CTG·CUG repeats, such as myotonic dystrophy type 1 (DM1), spinocerebellar ataxia-8 (SCA8) and Huntington-like disease-2 (HDL2).

CONC2023/0018002A2021-06-232024-01-09 Antisense compounds and methods for targeting cug repeatsCO2023018002A2 (en)

Applications Claiming Priority (16)

Application NumberPriority DateFiling DateTitle
US202163213900P2021-06-232021-06-23
US202163239671P2021-09-012021-09-01
US202163239847P2021-09-012021-09-01
US202163290892P2021-12-172021-12-17
US202163290960P2021-12-172021-12-17
US202263298565P2022-01-112022-01-11
US202263305071P2022-01-312022-01-31
US202263268577P2022-02-252022-02-25
US202263314369P2022-02-262022-02-26
US202263316634P2022-03-042022-03-04
US202263317856P2022-03-082022-03-08
US202263326201P2022-03-312022-03-31
US202263362295P2022-03-312022-03-31
US202263327179P2022-04-042022-04-04
US202263339250P2022-05-062022-05-06
PCT/US2022/034517WO2022271818A1 (en)2021-06-232022-06-22Antisense compounds and methods for targeting cug repeats

Publications (1)

Publication NumberPublication Date
CO2023018002A2true CO2023018002A2 (en)2024-02-05

Family

ID=82608300

Family Applications (1)

Application NumberTitlePriority DateFiling Date
CONC2023/0018002ACO2023018002A2 (en)2021-06-232024-01-09 Antisense compounds and methods for targeting cug repeats

Country Status (13)

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EP (1)EP4359006A1 (en)
JP (1)JP2024524291A (en)
KR (1)KR20240038967A (en)
AU (1)AU2022298774A1 (en)
CA (1)CA3222824A1 (en)
CL (1)CL2023003840A1 (en)
CO (1)CO2023018002A2 (en)
DO (1)DOP2023000279A (en)
EC (1)ECSP23096162A (en)
IL (1)IL309001A (en)
MX (1)MX2023015509A (en)
PE (1)PE20242357A1 (en)
WO (1)WO2022271818A1 (en)

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Also Published As

Publication numberPublication date
WO2022271818A1 (en)2022-12-29
IL309001A (en)2024-02-01
MX2023015509A (en)2024-01-22
CA3222824A1 (en)2022-12-29
EP4359006A1 (en)2024-05-01
CL2023003840A1 (en)2024-07-12
PE20242357A1 (en)2024-12-16
KR20240038967A (en)2024-03-26
JP2024524291A (en)2024-07-05
DOP2023000279A (en)2024-04-30
AU2022298774A1 (en)2023-12-14
ECSP23096162A (en)2024-03-01

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