DT-216P2
Friedreich's Ataxia (FA)
Phase 1/2Active
Key Facts
About Design Therapeutics
Design Therapeutics is a clinical-stage biotech focused on developing disease-modifying therapies for severe genetic disorders driven by nucleotide repeat expansions. Its core innovation is the GeneTAC™ platform, which creates bifunctional small molecules that can precisely upregulate or downregulate disease-causing genes without altering DNA. The company's strategy targets monogenic diseases with high unmet need, advancing a pipeline led by DT-216P2 for Friedreich's ataxia, now in Phase 1/2 trials, alongside preclinical programs in Fuchs endothelial corneal dystrophy and myotonic dystrophy type 1.
View full company profileTherapeutic Areas
Other Friedreich's Ataxia (FA) Drugs
| Drug | Company | Phase |
|---|---|---|
| CAP-004 | Capsida Biotherapeutics | IND-enabling |
| Vatiquinone (EPI-743) | PTC Therapeutics | Phase 3 |