US2022133704A1PendingUtilityA1
P38 kinase inhibitors reduce dux4 and downstream gene expression for the treatment of fshd
Est. expiryOct 5, 2037(~11.2 yrs left)· nominal 20-yr term from priority
Inventors:Angela CacaceLuis Gustavo Alejandro Rojas SotoLorin A. Thompson, IiiOwen Brendan WallaceLucienne V. RoncoNing ShenAlan Scott RobertsonAaron Nakwon Chang
A61K 31/4418A61P 21/00A61K 45/00C12Y 207/11024A61K 31/496A61K 31/5377A61K 45/06A61K 31/519A61K 31/416A61K 31/53C12Q 1/686C12Q 2600/106A61K 31/513C12Q 1/6883A61K 31/4439C12Q 2600/156A61K 9/0053A61K 9/0073A61K 31/437C12N 15/113G01N 33/5061G01N 33/68G01N 2500/10C07K 14/4707A61K 31/454A61K 31/00C12N 9/12A61K 31/506C12Q 1/6827A61K 9/0019
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Claims
Abstract
The disclosure relates to methods and compositions including p38 kinase inhibitors and agents that regulate expression of DUX4 and downstream genes including but not restricted to ZSCAN4, LEUTX, PRAMEF2, TRIM43, MBD3L2, KHDC1L, RFPL2, CCNA1, SLC34A2, TPRX1, PRAMEF20, TRIM49, PRAMEF4, PRAME6, PRAMEF15, or ZNF280A. Methods useful for treating a disease associated with abnormal DUX4 and downstream gene expression (e.g., Fascioscapulohumeral muscular dystrophy) are disclosed.
Claims
exact text as granted — not AI-modified1 - 30 . (canceled)
31 . A pharmaceutically acceptable tablet for orally delivering 15 mg of a compound represented by Formula (V′):
comprising:
15 mg of the compound; and
one or more pharmaceutically acceptable excipients.
32 . A pharmaceutically acceptable tablet for orally delivering 15 mg of a compound represented by Formula (V′):
comprising:
15 mg of the compound provided as a pharmaceutically acceptable salt; and
one or more pharmaceutically acceptable excipients.Cited by (0)
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