Design Therapeutics, Inc.
DSGN evidence brief
What we can confirm
- Market Cap:
- $786.7M
What it means
What to watch
Prepared research question
What matters most for DSGN—catalysts, financial risk and supporting evidence?
Traceable citations · Unknowns marked
BioSniper AI · DSGN
Design Therapeutics, Inc. (DSGN) is a clinical-stage biotechnology company developing gene-expression modulation therapies for inherited neurological and ophthalmic diseases, with its lead candidate DT-216P2 in a registrational trial for Friedreich's ataxia.
Key risk: No revenue, high cash burn
Market Cap
$786.7M
Cash Runway
—
Lead Asset
—
Next Catalyst
—
Financial data as of Jun 30, 2026.
Tracking 5 clinical trials for Design Therapeutics, Inc..
Design Therapeutics, Inc. (DSGN) is a clinical-stage biotechnology company developing gene-expression modulation therapies for inherited neurological and ophthalmic diseases, with its lead candidate DT-216P2 in a registrational trial for Friedreich's ataxia.
- Lead candidate DT-216P2 is in a registrational trial for Friedreich's ataxia (FA), with an update planned for Q4 2026 and 12-week results expected Q1 2027 [news].
- Cash, cash equivalents and investment securities were $207.4 million as of June 30, 2026, with an accumulated deficit of $334.8 million [sec_mda].
- Net income for the six months ended June 30, 2026 was -$20.2 million; shareholders' equity was $201.9 million [sec].
- DT-216P2 was generally well-tolerated in early studies with no serious adverse events or treatment discontinuations; biomarker data showed meaningful increases in FXN mRNA and protein [sec_mda].
- A Phase 2 biomarker trial for DT-168 (Fuchs endothelial corneal dystrophy) is ongoing, with data now expected in 2027 due to a delay [sec_mda].
What works
- DT-216P2 has shown biomarker activity (increased FXN mRNA and protein) and clinical improvements in FA patients with no serious adverse events [sec_mda].
- The company has a cash runway of more than 12 months from August 2026 based on its operating plan [sec_mda].
- The HD GeneTAC program demonstrated >50% reduction in mutant huntingtin mRNA and protein in an animal model while preserving wild-type huntingtin [sec_mda].
What to weigh
- The company has no approved products and has not generated any revenue from product sales; accumulated deficit was $334.8 million as of June 30, 2026 [sec_mda].
- Clinical development is costly and uncertain; the company may expend capital resources sooner than expected [sec_mda].
- Pursuing a biomarker-driven strategy carries increased risks as there are currently a limited number of approved biomarker-specific therapies [sec].
- The Phase 2 biomarker trial for DT-168 has experienced a delay, with data now expected in 2027 [sec_mda].
From the filings
Quoted directly from source documents.
DT-216P2 registrational trial update planned for Q4 2026 and 12-week results Q1 2027
“Design Therapeutics plans registrational FA trial for DT-216P2 with results in 2026-2027.”
News · October 4, 2026
Cash, cash equivalents and investment securities were $207.4 million as of June 30, 2026
“Our cash, cash equivalents and investment securities balance as of June 30, 2026 was $207.4 million.”
SEC MD&A · August 3, 2026
Accumulated deficit of $334.8 million as of June 30, 2026
“since our inception and as of June 30, 2026 had an accumulated deficit of $334.8 million.”
SEC MD&A · August 3, 2026
DT-216P2 was generally well-tolerated with no serious adverse events
“DT-216P2 was generally well-tolerated, with no serious adverse events or treatment discontinuations reported.”
SEC MD&A · August 3, 2026
Catalyst timeline
- 2025-06-01 · Phase 2 start for DT-216P2 in FA — A Phase 1/2 open-label multiple ascending dose study of DT-216P2 in FA patients began.
- 2025-07-30 · Phase 2 start for DT-168 in FECD — A Phase 2 multicenter open-label study of DT-168 ophthalmic solution in FECD patients undergoing keratoplasty began.
Risks & what to watch (4)›
Key risks
- No revenue, high cash burn — No approved products; accumulated deficit $334.8M as of June 30, 2026; net loss $20.2M in H1 2026 [sec_mda].
- Clinical trial delays — Phase 2 DT-168 trial data delayed to 2027; timing of progress and expenses uncertain [sec_mda].
- Biomarker strategy risk — Limited number of approved biomarker-specific therapies increases development risk [sec].
- Capital consumption risk — Company could expend capital resources sooner than expected due to uncertain trial costs [sec_mda].
What to watch
- Registrational trial update for DT-216P2 expected in Q4 2026 [news].
- 12-week results from the DT-216P2 registrational trial expected in Q1 2027 [news].
- Phase 2 biomarker trial data for DT-168 (FECD) expected in 2027 [sec_mda].
- Cash runway and burn rate updates in future SEC filings [sec_mda].
Sources: Catalysts · News · SEC filings · SEC MD&A · Generated October 13, 2026 · How we verify sources →
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Evidence & data
Regulatory
Financials
Institutional Holdings
Source: SEC Form 13F · as of Mar 2026Institutional holders
125
Total value held
$395.2M
| Holder | Shares | Value |
|---|---|---|
| sr one capital management, lp | 6,526,476 | $69.4M |
| ra capital management, l.p. | 2,972,973 | $31.6M |
| point72 asset management, l.p. | 2,722,754 | $29.0M |
| baker bros. advisors lp | 2,585,978 | $27.5M |
| blackrock, inc. | 2,133,764 | $22.7M |
| logos global management lp | 1,950,000 | $20.7M |
| driehaus capital management llc | 1,744,987 | $18.6M |
| vivo capital, llc | 1,612,903 | $17.2M |
| almitas capital llc | 1,530,335 | $16.3M |
| vanguard capital management llc | 1,349,850 | $14.4M |
Showing top 10 of 125 institutional holders of Design Therapeutics, Inc.. Open the full 13F history after sign-in.
Short Interest
as of Apr 28, 2026- % of float
- 4.8%
- Days to cover
- 6.7
- Shares short
- 2,238,096
- Daily short volume
- -
Short percent of float and days-to-cover reflect reported short positioning. Daily short volume is the exchange-reported short share of a single day's trading volume and is noisier. See the most-shorted biotech ranking →
DSGN short interestGovernance
Frequently asked questions
What is the AI investment thesis for Design Therapeutics, Inc.?
Design Therapeutics, Inc. (DSGN) is a clinical-stage biotechnology company developing gene-expression modulation therapies for inherited neurological and ophthalmic diseases, with its lead candidate DT-216P2 in a registrational trial for Friedreich's ataxia.
Who is the CEO of Design Therapeutics, Inc.?
Mr. Pratik Shah Ph.D. is the chief executive officer of Design Therapeutics, Inc. (DSGN).
Where is Design Therapeutics, Inc. headquartered?
Design Therapeutics, Inc. (DSGN) is headquartered in Carlsbad, CA, United States.
What therapeutic areas does Design Therapeutics, Inc. focus on?
Design Therapeutics, Inc. develops therapies across Oncology, Rare Disease, Neurology, Ophthalmology, Respiratory, Dermatology.
The AI overview and BioSniper Score are neutral, data-derived signals built from public SEC filings, ClinicalTrials.gov and FDA records — not investment advice.