SOD1 gene — Drug Target

All drugs that target SOD1 gene — marketed and clinical-stage. Includes 2 drug classes acting on this target.

Drug classes

antisense oligonucleotide · Gene therapy

Phase 3 pipeline (1)

• Tivanisiran sodium ophthalmic solution · Sylentis, S.A. · antisense oligonucleotide · Ophthalmology
  Tivanisiran sodium ophthalmic solution is an antisense oligonucleotide that targets the SOD1 gene to reduce the production of superoxide dismutase 1 protein.

Phase 2 pipeline (1)

• CTI-1601 · Larimar Therapeutics, Inc. · Gene therapy · Neurology
  CTI-1601 is a gene therapy that aims to restore motor neuron function in patients with amyotrophic lateral sclerosis (ALS).