Compounds for treating spinal muscular atrophy

USRE47689E1 · kind E1 · reissue

Assignees

• PTC THERAPEUTICS, INC. · US
• F. Hoffmann-La Roche AG · CH

Inventors

• Matthew G. Woll · Madison, US
• Guangming Chen · Bound Brook, US
• Soongyu Choi · Skillman, US
• Amal Dakka · Whitehouse Station, US
• Song Huang
• Gary Mitchell Karp · Princeton, US
• Chang-Sun Lee · Montgomery, US
• Chunshi Li · East Brunswick Township, US
• Jana Narasimhan · Scotch Plains, US
• Nikolai Naryshkin · East Brunswick Township, US
• Sergey V. Paushkin · Montgomery, US
• Hongyan Qi · Plainsboro, US
• Anthony Turpoff
• Marla L. Weetall · Morristown, US
• Ellen Welch · Califon, US
• Tianle Yang · Mountainside, US
• Nanjing Zhang · Princeton, US
• Xiaoyan Zhang · Lexington, US
• Xin Zhao · Austin, US
• Emmanuel Pinard · Rixheim, FR
• Hasane Ratni · Habsheim, FR

Key dates

• Filing date: Sep 5, 2017
• Grant date: Nov 5, 2019
• Expiry date: Sep 5, 2037

Classification

• Technology area (CPC —): General

Abstract

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.