피루베이트 키나제 (PKR) 활성화제로서의 피롤로피롤 조성물

• Main Authors: GUSTAFSON GARY, HAN BINGSONG, WANG ZHONGGUO, MITCHELL LORNA, SHELEKHIN TATIANA, ZHENG XIAOZHANG, LANCIA JR. DAVID R, RICHARD DAVID, GREEN NEAL, SMITH CHASE C, ERICSSON ANNA
• Format: Patent
• Language: Korean
• Published: 20.12.2018
• 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 피루베이트 키나제의 조절에 관한 것이며, PKR의 활성화제로서 유용한 화학식 (I)의 신규한 화합물뿐만 아니라 이들 화합물의 다양한 용도를 제공한다. PKR 활성화 화합물은 PKR 및/또는 PKM2와 관련된 질병 및 장애, 예컨대 피루베이트 키나제 결핍증 (PKD), 겸상 적혈구 질병 (SCD), 및 지중해빈혈의 치료에 유용하다: [화학식 (I)]. The disclosure relates to modulating pyruvate kinase and provides novel chemical compounds useful as activators of PKR, as well as various uses of these compounds. PKR activating compounds are useful in the treatment of diseases and disorders associated with PKR and/or PKM2, such as pyruvate kinase deficiency (PKD), sickle cell disease (SCD), and thalassemia.