SMN2 gene — Drug Target

All drugs that target SMN2 gene — marketed and clinical-stage. Includes 1 drug classes acting on this target.

Drug classes

antisense oligonucleotide

Phase 3 pipeline (1)

• RO7234292 (RG6042) · Hoffmann-La Roche · antisense oligonucleotide · Neurology
  RO7234292 is a small molecule antisense oligonucleotide that targets the SMN2 gene to increase production of the survival motor neuron protein.