Ptc Therapeutics, Inc.

F. Hoffmann-La Roche Ag

Pharmacyclics, Inc.

Pharmacylics, Inc.

Chang-Sun Lee

Young-Choon Moon

Steven David Paget

Hongyu Ren

Nadiya Sydorenko

Ramil Y Baiazitov

Wu Du

Soongyu Choi

Richard Gerald Wilde

Hasane Ratni

Erik J Verner

Gary Mitchell Karp

Joseph J Buggy

Sriram Balasubramanian

Ronggang Liu

Vincent W F Tai

Emmanuel Pinard

Guangming Chen

Ellen Welch

Nikolai A Naryshkin

Xiaoyan Zhang

Hongyan Qi

Liangxian Cao

Marla L Weetall

Nanjing Zhang

Jana Narasimhan

Anthony A Turpoff

Xin Zhao

Thomas W Davis

Matthew G Woll

Amal Dakka

Tianle Yang

Chunshi Li

Sergey Paushkin

Song Xiao Huang

Hiroo Koyama

Seongwoo Hwang

Song Huang

Amine substituted reverse pyrimidine compounds and forms thereof that inhibit the function and reduce the level of B-cell specific Moloney murine leukemia virus integration site 1 (Bmi-1) protein and methods for their use to inhibit Bmi-1 function and reduce the level of Bmi-1 to treat a cancer mediated by Bmi-1 are described herein.

Substituted reverse pyrimidine Bmi-1 inhibitors

Amine substituted pyridine and pyrazine compounds and forms thereof that inhibit the function and reduce the level of B-cell specific Moloney murine leukemia virus integration site 1 (Bmi-1) protein and methods for their use to inhibit Bmi-1 function and reduce the level of Bmi-1 to treat a cancer mediated by Bmi-1 are described herein.

Substituted pyridine and pyrazine BMI-1 inhibitors

Provided herein are compounds, compositions thereof and uses therewith for treating spinal muscular atrophy. In a specific embodiment, provided herein are compounds of a form that may be used to modulate the inclusion of exon 7 of SMN2 into mRNA that is transcribed from the SMN2 gene. In another specific embodiment, provided herein are compounds of a form that may be used to modulate the inclusion of exon 7 of SMN1 into mRNA that is transcribed from the SMN1 gene. In yet another embodiment, provided herein are compounds of a form that may be used to modulate the inclusion of exon 7 of SMN1 and SMN2 into mRNA that is transcribed from the SMN1 and SMN2 genes, respectively.

Compounds for treating spinal muscular atrophy

Amine substituted pyrimidine compounds and forms thereof that inhibit the function and reduce the level of B-cell specific Moloney murine leukemia virus integration site 1 (Bmi-1) protein and methods for their use to inhibit Bmi-1 function and reduce the level of Bmi-1 to treat a cancer mediated by Bmi-1 are described herein.

Substituted pyrimidine Bmi-1 inhibitors

Amine substituted triazine compounds and forms thereof that inhibit the function and reduce the level of B-cell specific Moloney murine leukemia virus integration site 1 (Bmi-1) protein and methods for their use to inhibit Bmi-1 function and reduce the level of Bmi-1 to treat a cancer mediated by Bmi-1 are described herein.

Substituted triazine BMI-1 inhibitors

Provided herein are compounds, compositions thereof and uses therewith for treating spinal muscular atrophy.

Described herein are compounds and pharmaceutical compositions containing such compounds, which inhibit the activity of histone deacetylase 8 (HDAC8). Also described herein are methods of using such HDAC8 inhibitors, alone and in combination with other compounds, for treating diseases or conditions that would benefit from inhibition of HDAC8 activity.

Indole derivatives as inhibitors of histone deacetylase

Growing community of inventors

Belle Mead, NJ, United States of America

Chang-Sun Lee