The Cure SMA Drug Pipeline is the primary ways we evaluate the success of our research program. It identifies the major drug programs in development and tracks their progress from basic research through FDA approval and beyond.
Key Measurements
The spinal muscular atrophy (SMA) pipeline measures the progress of our research in two key ways. First, by tracking each individual program all the way to U.S. Food and Drug Administration (FDA) approval. Second, by tracking how these programs are spread among the different therapeutic approaches.
Progress of Individual Drug Programs
Cure SMA’s research model funds drug programs at all stages of development. Our basic research program studies the biology and causes of SMA, often revealing new and more effective ways of making drugs. These basic research ideas are then converted into practical drug candidates through drug discovery. Finally, those drug candidates move through the clinical trial process. Our drug pipeline monitors each individual program as it moves through these stages.
Approved Therapies for SMA
Several therapies have been approved for SMA: Evrysdi, marketed by Genentech/Roche; Spinraza, marketed by Biogen; and Zolgensma, marketed by Novartis Gene Therapies. The drug pipeline also tracks the implementation of these and other therapies as they are approved.
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Balance of Therapeutic Approaches
In order to find a cure for SMA, we know it’s crucial to attack SMA from all sides. As with all scientific research, it’s difficult to predict which SMA drug programs might be successful. By investing in a diversity of approaches, we maximize our chances for success. If one drug candidate or one approach fails, we have others to take its place. The therapeutic approaches section of our website describes each of these approaches in more detail.
The Cure SMA Drug Pipeline identifies several possible treatment targets:
Replacement or correction of the faulty SMN1 gene.
Modulation of the low functioning SMN2 “back-up gene.”
Muscle protection to prevent or restore the loss of muscle function in SMA.
Neuroprotection of the motor neurons affected by loss of SMN protein.
Newer approaches that identify additional systems and pathways affected by SMA.