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US 12,171,755 B2Granted
PAPD5 inhibitors and methods of use thereof
AssigneesChildren's Medical Center Corporation, The Brigham and Women's Hospital, Inc.
InventorsSuneet Agarwal, Neha Nagpal, Yick Fong
Life of this patent
Estimating the term…
Abstract
The disclosure relates to compounds that are, e.g., PAP Associated Domain Containing 5 (PAPD5) inhibitors and methods of use thereof.
Claims16
A method of treating a disease or condition selected from:
- a disorder associated with telomere or telomerase dysfunction, a degenerative disorder associated with aging, a pre-leukemic or pre-cancerous condition, a neurodevelopmental disorder, and an acquired or genetic disease or condition associated with alterations in RNA, the method comprising administering to a subject in need thereof a therapeutically effective amount of a compound of Formula (II-IIb):
- or a pharmaceutically acceptable salt thereof, wherein:
- R2, R4, R6, and R7 are each independently selected from H, C1-6 alkyl, ORa1, C(O)ORa1, C(O)Rb1, NRc1Rd1, and NRc1C(O)Rb1;
- R1 is selected from H, C1-6 alkyl, C(O)ORa1, C(O)Rb1, NRc1Rd1, and NRc1C(O)Rb1;
- R3 is selected from H, C1-6 alkyl, ORa1, C(O)Rb1, NRc1Rd1, and NRc1C(O)Rb1;
- each RCy1 is independently selected from C1-6 alkyl, C(O)ORa4, ORa4, C(O)Rb4, and halo;
- Ra1, Rb1, Rb4, Rc1, and Rd1 are each independently selected from C1-6 alkyl, and C1-4 haloalkyl;
- and
- Ra4 is selected from H, C1-6 alkyl, and C1-4 haloalkyl.
6 more elements
A method of treating dyskeratosis congenita, the method comprising administering to a subject in need thereof a therapeutically effective amount of a compound of formula:
- or a pharmaceutically acceptable salt thereof.
A method of treating a disease or condition selected from:
- a disorder associated with telomere or telomerase dysfunction, a disorder associated with aging, a pre-leukemic or pre-cancerous condition, a neurodevelopmental disorder, and an acquired or genetic disease or condition associated with alterations in RNA, the method comprising administering to a subject in need thereof a therapeutically effective amount of a compound of formula:
- or a pharmaceutically acceptable salt thereof, wherein:
- R2, R3, R4, R6, and R7 are each independently selected from H, halo Cy1, C1-6 alkyl, ORa1, SRa1, C(O)ORa1, C(O)NRc1Rd1, NRc1C(O)Rb1, NRc1Rd1, S(O)2Rb1 and C(O)Rb1;
- R1 is selected from ORa1 and SRa1;
- each Cy1 is independently selected from C6-10 aryl, C3-10 cycloalkyl, 5-10 membered heteroaryl, and 4-12 membered heterocycloalkyl, each of which is optionally substituted with 1, 2, 3, 4, or 5 substituents independently selected from RCy1;
- each RCy1 is independently selected from C(O)ORa4, ORa4, C(O)Rb4, and halo;
- Ra1, Rb1, Rb4, Rc1, and Rd1 are each independently selected from C1-6 alkyl, and C1-4 haloalkyl;
- and
- Ra4 is selected from H, C1-6 alkyl, and C1-4 haloalkyl.
6 more elements
Dependencies are inferred from the claim text.