No single group can research and develop a treatment for SMA alone—it requires collaboration between academics, industry, government, and the SMA community. We bring all those groups together and lead the SMA Industry Collaboration to advance goals important to the SMA community.

One of the most distinctive aspects of Cure SMA’s research program is the SMA Industry Collaboration. Established in 2016, the SMA Industry Collaboration is a multi-faceted partnership that brings together pharmaceutical companies, Cure SMA, and other nonprofit organizations, to share information, ideas, and data. The SMA Industry Collaboration works together to address scientific, clinical, and regulatory topics that are critical to advancing drug development in spinal muscular atrophy (SMA) and will benefit the broader SMA community. It is currently comprised of our partners at Novartis Gene Therapies, Biogen, Genentech/Roche Pharmaceuticals, Scholar Rock, and SMA Europe.

Through the SMA Industry Collaboration, we fund research to ensure that effective, safe treatments can progress through clinical trials quickly and gain approval from the U.S. Food and Drug Administration (FDA) and international regulators. Our research also ensures these treatments address the unmet needs of the SMA community, and that the community’s priorities and goals are incorporated into the development, review, and approval of therapies.

The industry collaboration currently consists of four topic groups: 

  • Regulatory interaction and outcome measure development.

  • Education and awareness.

  • Clinical trials.

  • Patient reported data project.

Current and Past SMA Industry Collaboration Projects

 SMA Industry Collaboration Publications

Publishing our work allows us to share ideas and work collaboratively to advance SMA drug development. To access manuscripts, toolkits, and other resources generated by the collaboration, please visit Cure SMA’s Published SMA Research page.

SMA Industry Collaboration Latest News