Rocket Pharmaceuticals (RCKT)
Develops gene therapies for rare immune and cardiovascular disorders.
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Develops gene therapies for rare inherited disorders, with KRESLADI approved for severe LAD-I and a prioritized AAV cardiovascular pipeline. Operations are concentrated in R&D and commercial readiness, with execution tied to treatment-center logistics, post-approval follow-up, and resumed Danon dosing after a clinical hold.
Operates as a one-segment gene-therapy developer focused on rare inherited disease. KRESLADI is the approved lentiviral therapy for severe LAD-I, while the active AAV cardiovascular pipeline centers on RP-A501 for Danon disease, RP-A601 for PKP2-associated arrhythmogenic cardiomyopathy, and RP-A701 for BAG3-associated dilated cardiomyopathy.
Work is anchored in Cranbury, New Jersey, with most employees in the United States and small teams in Europe. The business has not yet shifted into material product revenue, so commercial execution depends on specialized treatment-center onboarding, manufacturing, and vein-to-vein logistics.
Current strategy is more concentrated after the 2025 reorganization. KRESLADI approval depends on longer-term follow-up and post-marketing data, and the Danon program depends on successful lower-dose dosing after its clinical hold was lifted.
Company facts
Target rangeAs of Sep 22, 2026
Latest analyst notes
| Date | Firm | Action | Prior | Current |
|---|---|---|---|---|
| Sep 16, 2026 | Needham | Upgrade | Target: — | Target: $9 |
| Jan 8, 2026 | Morgan Stanley | Target | Target: $45 | Target: $5 |
| Nov 18, 2025 | JP Morgan | Downgrade | ||
| Oct 3, 2025 | Leerink Partners | Reiterate | Rating: —Target: — | Target: $7 |
| Aug 20, 2025 | B of A Securities | Upgrade |
EPSReportedEstimate
RevenueReportedEstimate
Balance sheet, Jun 30, 2026Cash & short-term investments $283.7M · Total debt $24.8M · Total assets $423.1M · Shareholders' equity $368.8M
- Revenue
- $0
- Operating income
- −$46.9M
- Net income
- $123.2M
No dividends on record.
| Institutions | 91.52% | 100.46M shares | |
|---|---|---|---|
| 185 holders · as of Jun 30, 2026 | |||
| Insiders | 3.61% | 3.96M shares | |
| 20 holders · as of Sep 15, 2026 | |||
| Other | 4.87% | 5.35M shares | |
| remainder | |||
109.77M company shares as of Sep 22, 2026 · institutions as of Jun 30, 2026 · holders as of their latest filing
Top shareholders13F · 13D/G · Form 4
| Holder | Stake | Shares | Date |
|---|---|---|---|
RTW Investments, LPInstitution | 16.11% | 17.69M | as of Jun 30, 2026 |
BlackRock, Inc.Institution | 9.04% | 9.92M | as of Jun 30, 2026 |
Vanguard Portfolio Management LLCInstitution | 5.29% | 5.81M | as of Jun 30, 2026 |
Citigroup IncInstitution | 4.43% | 4.87M | as of Jun 30, 2026 |
Marshall Wace, LLPInstitution | 3.67% | 4.03M | as of Jun 30, 2026 |
Insider tradesForm 4
| Date | Insider | Role | Shares | Avg price |
|---|---|---|---|---|
| Sep 11, 2026 | Martin Wilson | officer: General Counsel | −75,364 | $3.40 |
| Aug 18, 2026 | Martin Wilson | officer: General Counsel | −631 | $3.38 |
| Aug 18, 2026 | Gaurav Shah | officer: CEO | −2,293 | $3.38 |
| Aug 13, 2026 | Gaurav Shah | officer: CEO | −2,837 | $3.38 |
| Aug 13, 2026 | Martin Wilson | officer: General Counsel | −3,493 | $3.38 |
- Lexeo Therapeutics
Direct PKP2-ACM and FA cardiomyopathy AAV peer
- Tenaya Therapeutics
Direct genetic cardiomyopathy AAV rival
- Solid Biosciences
Cardiac genetic medicine and AAV capsid platform peer
- Sarepta Therapeutics
Commercial rare-disease AAV gene therapy leader
- REGENXBIO
AAV platform and rare-disease gene therapy peer
- Krystal Biotech
Commercial rare-disease genetic medicine peer
CompareAfter hours
| Company | Price | Change | |||||
|---|---|---|---|---|---|---|---|
| Rocket Pharmaceuticals | $3.38 | +0.75% | $357.1M | — | — | — | — |
| Lexeo Therapeutics | $4.07 | 0.00% | $320.4M | — | — | — | — |
| Tenaya Therapeutics | $0.6811 | −1.28% | $146.2M | — | — | 109.5 | — |
| Solid Biosciences | $8.61 | −3.31% | $840.8M | — | — | — | — |
| Sarepta Therapeutics | $21.07 | +1.20% | $2.13B | — | 36.2 | 1.1 | −34.3% |
| REGENXBIO | $8.15 | −0.12% | $407.4M | — | — | 2.3 | +405.7% |
| Krystal Biotech | $353.02 | +0.61% | $9.84B | 41.8 | 41.3 | 22.3 | +24.1% |
| Median | 41.8 | 38.8 | 12.3 | +24.1% |