Gene editing therapy

This page covers all Gene editing therapy drugs tracked by Drug Landscape: marketed products and active clinical-stage compounds, targeting SMN1 gene.

Targets

SMN1 gene

Phase 2 pipeline (1)

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