피라졸로피리딘 유도체 및 이의 용도

• Main Authors: BONAZZI SIMONE, MANNING JAMES R, DEWHURST JANETTA, DALES NATALIE ALYSIA, MALIK HASNAIN AHMED, O'BRIEN GARY, HESSE MATTHEW JAMES, PATTERSON ANDREW W, CERNIJENKO ARTIOM, COBB JENNIFER STROKA, JAIN RAMA, KERRIGAN JOHN RYAN, TING PAMELA YF, THOMSEN NOEL MARIE FRANCE
• Format: Patent
• Language: Korean
• Published: 14.11.2023
• Subjects: CHEMISTRY; HETEROCYCLIC COMPOUNDS; HUMAN NECESSITIES; HYGIENE; MEDICAL OR VETERINARY SCIENCE; METALLURGY; ORGANIC CHEMISTRY; PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES; SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS ORMEDICINAL PREPARATIONS

본 발명은 화학식 I의 화합물 및 제약 조성물, 및 넓은 간격의 징크 핑거 모티프(Widely Interspaced Zinc Finger Motif; WIZ) 발현 수준의 감소, 또는 태아 헤모글로빈(HbF) 발현의 유도, 및 겸상적혈구병 및 베타 지중해빈혈과 같은 유전성 혈액 장애(예를 들어, 혈색소병증, 예를 들어 베타 혈색소병증)의 치료에서의 이의 용도에 관한 것이다. 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.