Company intelligence / Profile preview
Design Therapeutics
Carlsbad, California, United Statesdesigntx.com ↗
- Ownership
- Public · DSGN
- Employees
- ~100
- Development stage
- Other
01
Overview
Design Therapeutics has advanced its lead program DT‑216P2 for Friedreich ataxia into a Phase 1 single ascending dose trial in healthy volunteers; patient dosing expected mid‑2025. Its second program DT‑168 for FECD completed Phase 1 healthy volunteer dosing with data expected first half of 2025. A development candidate selection for DM1 is anticipated in 2025. The company reports preclinical progress on additional programs and discovery efforts across multiple genetic diseases caused by nucleotide repeat expansions. Early clinical results have demonstrated target engagement and modulation after a single dose of their lead compound.
- Therapeutic areas
- OphthalmologyNeurology
- Modalities
- Small molecules (specifically GeneTAC® gene targeted chimera small molecules)
- Industry
- Biotech
02
Drug pipeline
8 assets7 more assets in the full pipeline.
03
Partnerships
No major pharma partnerships disclosed as of May 2025.
04
Financials
- Market cap
- $605.2M
- Total revenue
- $0
- Enterprise value
- $399.1M
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