3-(5-oxy)-1-oxoisoindolin-2-yl)piperidine-2,6-dione derivatives and uses thereof

US12103919B2 · kind B2 · utility

Assignee

• Novartis AG · CH

Inventors

• Simone BONAZZI · Cambridge, US
• Artiom CERNIJENKO · Cambridge, US
• Jennifer Stroka Cobb · Stow, US
• Janetta Dewhurst · Framingham, US
• John Ryan Kerrigan · Wakefield, US
• Gary O'Brien · Maynard, US
• MooJe Sung · Belmont, US
• Noel Marie-France Thomsen · Cambridge, US
• Pamela YF TING · Somerville, US

Key dates

• Filing date: Jun 1, 2022
• Grant date: Oct 1, 2024
• Expiry date: Jun 1, 2042

Classification

• Technology area (CPC C): Chemistry; Metallurgy
• CPC primary: C07D471/08
• WIPO field: Pharmaceuticals
• WIPO sector: Chemistry

Abstract

The present disclosure relates to compounds of formula (I) and pharmaceutical compositions and their use in reducing Widely Interspaced Zinc Finger Motifs (WIZ) expression levels, or inducing fetal hemoglobin (HbF) expression, and in the treatment of inherited blood disorders (e.g., hemoglobinopathies, e.g., beta-hemoglobinopathies), such as sickle cell disease and beta-thalassemia.