Stoke Therapeutics (STOK)
Develops antisense medicines that restore protein expression in severe genetic diseases.
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Develops TANGO antisense medicines that increase protein expression from working gene copies, led by pivotal-stage zorevunersen for Dravet syndrome and early-stage STK-002 for inherited optic atrophy. Has no approved products; revenue comes from Biogen and Acadia collaborations.
Develops antisense oligonucleotide medicines that use the TANGO platform to redirect RNA splicing and increase protein expression from a working gene copy. Zorevunersen, for SCN1A-related Dravet syndrome, has a fully enrolled primary analysis cohort in the global pivotal EMPEROR study. STK-002 is dose escalating in a first-in-human study for OPA1-linked autosomal dominant optic atrophy, while a partnered SYNGAP1 program and earlier central-nervous-system research extend the pipeline.
Leads zorevunersen's global development and retains commercial rights in the United States, Canada, and Mexico; Biogen holds rights elsewhere and shares development costs. Collaboration and licensing agreements with Biogen and Acadia generate current revenue. The same splice-switching design underpins its clinical programs across haploinsufficiency disorders. No products are approved, and operations depend on trial outcomes, regulatory clearance, collaborators, and third-party manufacturers.
Company facts
Target rangeAs of Sep 22, 2026
Latest analyst notes
| Date | Firm | Action | Prior | Current |
|---|---|---|---|---|
| Aug 4, 2026 | Canaccord Genuity | Reiterate | Rating: —Target: $36 | Target: $58 |
| Jul 13, 2026 | Goldman Sachs | Initiate | Rating: —Target: — | Target: $44 |
| Feb 23, 2026 | Wolfe Research | Target | Target: — | Target: $40 |
| Feb 5, 2026 | Guggenheim | Target | Target: — | Target: $60 |
| Jan 12, 2026 | Wedbush | Reiterate | Rating: —Target: — | Target: $36 |
EPSReportedEstimate
RevenueReportedEstimate
Balance sheet, Jun 30, 2026Cash & short-term investments $292.8M · Total debt $2.7M · Total assets $394.0M · Shareholders' equity $345.5M
- Revenue
- $9.3M
- Operating income
- −$65.4M
- Net income
- −$61.6M
No dividends on record.
| Institutions | ≈ 118.68% | 76.58M shares | |
|---|---|---|---|
| 203 holders · as of Jun 30, 2026 · largest filers plus last quarter's tail | |||
| Insiders | 0.78% | 505.2K shares | |
| 8 holders · as of Sep 3, 2026 | |||
| Adds to | 119.46% | ||
64.53M company shares as of Sep 23, 2026 · institutions as of Jun 30, 2026 · holders as of their latest filing
This quarter's institutional total failed a plausibility check; shown: the largest filers plus last quarter's remainder.
These groups come from separate filings that can overlap, so they add to 119.46%; nothing is left for Other.
Top shareholders13F · 13D/G · Form 4
| Holder | Stake | Shares | Date |
|---|---|---|---|
BlackRock, Inc.Institution | 9.11% | 5.88M | as of Jun 30, 2026 |
RTW Investments, LPInstitution | 8.72% | 5.62M | as of Jun 30, 2026 |
FMR LLCInstitution | 8.23% | 5.31M | as of Jun 30, 2026 |
LYNX1 Capital Management LPInstitution | 6.26% | 4.04M | as of Jun 30, 2026 |
Redmile Group, LLCInstitution | 6.12% | 3.95M | as of Jun 30, 2026 |
Insider tradesForm 4
| Date | Insider | Role | Shares | Avg price |
|---|---|---|---|---|
| Sep 1, 2026 | Barry Ticho | officer: CHIEF MEDICAL OFFICER | −5,545 | $30.67 |
| Aug 18, 2026 | Edward M. MD Kaye | director | −12,567 | $32.03 |
| Aug 18, 2026 | Barry Ticho | officer: CHIEF MEDICAL OFFICER | −4,767 | $32.03 |
| Aug 18, 2026 | Jonathan Allan | officer: GENERAL COUNSEL & CORP SEC | −2,861 | $32.03 |
| Aug 3, 2026 | Barry Ticho | officer: CHIEF MEDICAL OFFICER | −7,176 | $29.04 |
- Jazz Pharmaceuticals
Owns Epidiolex, the leading approved Dravet anti-seizure blockbuster
- Harmony Biosciences
Phase 3 oral 5-HT2 agonist EPX-100 targets the same Dravet population
- Ionis Pharmaceuticals
ASO pioneer with direct SCN1A Dravet program (ION337) mirroring zorevunersen
- Bright Minds Biosciences
Phase 2 oral 5-HT2C agonist BMB-101 spans DEE incl. Dravet; Phase 3 planned
- Xenon Pharmaceuticals
Pre-clinical oral NaV1.1 potentiator XPC-837 targets the same SCN1A biology
Compare
| Company | Price | Change | |||||
|---|---|---|---|---|---|---|---|
| Stoke Therapeutics | $27.50 | −5.84% | $1.88B | — | — | 68.3 | −32.5% |
| Jazz Pharmaceuticals | $235.58 | −2.07% | $15.11B | 16.4 | 9.4 | 3.3 | +15.6% |
| Harmony Biosciences | $40.73 | −1.94% | $2.40B | 13.5 | 10.2 | 2.5 | +30.3% |
| Ionis Pharmaceuticals | $43.32 | −5.31% | $7.60B | — | — | 8.7 | −40.7% |
| Bright Minds Biosciences | $57.81 | −4.71% | $593.8M | — | — | — | — |
| Xenon Pharmaceuticals | $36.71 | −6.09% | $3.78B | — | — | — | — |
| Median | 15.0 | 9.8 | 3.3 | +15.6% |







