SMN1 gene — Drug Target

All drugs that target SMN1 gene — marketed and clinical-stage. Includes 3 drug classes acting on this target.

Drug classes

Gene Therapy · Gene therapy · Gene editing therapy

Marketed (2)

• Onasemnogene Abeparvovec-brve · Novartis Pharmaceuticals · Gene Therapy · Neuroscience
  ITVISMA delivers a functional SMN1 gene using an AAV9 vector to promote SMN protein expression in motor neurons.
• Onasemnogene Abeparvovec-xioi · Novartis Gene Therapies · Gene therapy · Neurology
  Onasemnogene abeparvovec-xioi is a gene therapy that delivers a functional copy of the SMN1 gene via an adeno-associated virus vector to restore survival motor neuron protein production in patients with spinal muscular atrophy.

Phase 2 pipeline (1)

• CT001 · Cessatech A/S · Gene editing therapy · Neurology
  CT001 is a gene editing therapy that uses CRISPR-Cas9 to edit the SMN1 gene.

Phase 1 pipeline (1)

• AVXS-101 · Novartis Gene Therapies · Gene therapy · Rare Disease
  Gene therapy for spinal muscular atrophy