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

  • Conducting clinical trial readiness and education initiatives to increase the number of sites that conduct SMA clinical trials (e.g., toolkits and workshops for clinical trial coordinators and physical therapists).

  • Developing and refining outcome measures to ensure that SMA clinical trials are measuring the results most important to those with SMA.

  • Creating, executing, and publishing results of the benefit-risk survey to better understand what benefits the SMA community is looking for in approved therapies and what risks they are willing to accept.

  • Holding a Patient-Focused Drug Development Meeting with the FDA and publishing a Voice of the Patient report based on those findings.

  • Launching an annual Community Update Survey to better understand the impact of SMA and how things change as therapies are made available.

  • Offering SMArt Moves, an education and awareness campaign to reduce the time to diagnosis so babies with SMA can get the most benefit from approved treatments.

  • Conducting a Critical Path Innovation Meeting (CPIM) with the FDA to unmet discuss the community’s ongoing unmet medical needs.

  • Developing a competency-based formal education program for SMA physical therapists.

  • Evaluating opportunities to “bring trials to patients” via adoption of telemedicine and remote monitoring.

 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 Clinical Care and Research Publications page.

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