COMPOUNDS FOR TREATING SPINAL MUSCULAR ATROPHY

• Main Authors: EMMANUEL PINARD, MARLA L WEETALL, JANA NARASIMHAN, LUKE GREEN, HASANE RATNI, ANTHONY A TURPOFF, AMAL DAKKA, ZHANG XIAOYAN, ZHANG NANJING, GARY MITCHELL KARP, MATTHEW G WOLL, CHOI SOONGYU, YANG TIANLE, ZHOU XING, ELLEN WELCH, QI HONGYAN, NIKOLAI NARYSHKIN
• Format: Patent
• Language: English; Japanese
• Published: 13.07.2017
• 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

PROBLEM TO BE SOLVED: To provide compounds for treating spinal muscular atrophy by contact thereof with a human cell to enhance insertion of exon 7 of SMN2 into mRNA that is transcribed from the SMN2 gene; and pharmaceutical compositions containing the compounds.SOLUTION: The invention provides compounds represented by the formula in the figure and pharmaceutical compositions containing the compounds. [Ris piperazinyl, morpholinyl, 1,4-diazepanyl, or the like; Ris phenyl; Ra is H, halogen, or Calkyl; Rb is H, halogen, Calkyl, Calkoxy, or the like; Rc is H, halogen, or Calkyl.]SELECTED DRAWING: Figure 1 【課題】ヒト細胞と接触させ、ＳＭＮ２遺伝子から転写されたｍＲＮＡにＳＭＮ２のエクソン７が挿入されることを促進することにより、脊髄性筋萎縮症を治療するための化合物、及び該化合物を含有する薬学的組成物の提供。【解決手段】下式で示される化合物、及び該化合物を含有する薬学的組成物。［Ｒ１は、ピペラジニル、モルホリニル、１，４−ジアゼパニル等；Ｒ２は、フェニル；Ｒａは、Ｈ、ハロゲン、又はＣ１−８アルキル；Ｒｂは、Ｈ、ハロゲン、Ｃ１−８アルキル、又はＣ１−８アルコキシ等；Ｒｃは、Ｈ、ハロゲン、又はＣ１−８アルキル］【選択図】図１