SMA’s Recent Breakthrough: How Salanersen is Bringing Hope
SMA is most commonly linked to changes in the SMN1 gene, which reduces the survival motor neuron protein the body needs for healthy motor neurons and muscle function. Salanersen, also known as BIIB115 or ION306, is an investigational antisense oligonucleotide being studied for the treatment of Spinal Muscular Atrophy. Its role is to target the backup SMN2 gene and help the body produce more SMN protein, which is why it has become an important topic in SMA treatment awareness. Salanersen works by helping the SMN2 gene make more SMN protein.
Salanersen is bringing hope because it may build on the same general treatment pathway as Spinraza while potentially offering greater potency and less frequent dosing. Biogen has described salanersen as a novel antisense oligonucleotide designed with the potential for high efficacy and once-yearly dosing in SMA. In 2026, the FDA granted salanersen Breakthrough Therapy Designation for Spinal Muscular Atrophy, a designation meant to help speed development and review for treatments that show early clinical promise in serious conditions. Biogen reported that the designation was supported by exploratory Phase 1b data in children with SMA who previously received gene therapy and had suboptimal clinical status, with observed clinically meaningful motor improvements and slowing of neurodegeneration after salanersen treatment.
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