Rocket Pharmaceuticals
Develops gene therapies for rare immune and cardiovascular disorders.
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Profile
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
Key statistics
Year to date +11.4% · Average volume (30d) 1.5M
Analysts
Ratings
Target range
Latest analyst notes
| Date | Firm | Action | Prior rating | Rating | Prior target | Target |
|---|---|---|---|---|---|---|
| Jan 8, 2026 | Morgan Stanley | Target | $45 | $5 | ||
| Nov 18, 2025 | JP Morgan | Downgrade | Overweight | Underweight | ||
| Oct 3, 2025 | Leerink Partners | Reiterate | Market Perform | $7 | ||
| Aug 20, 2025 | B of A Securities | Upgrade | Neutral | Buy | ||
| Jul 25, 2025 | B of A Securities | Downgrade | Buy | Neutral |
Earnings
EPS
Revenue
History
| Report date | EPS est | EPS | Surprise | Revenue est | Revenue | Surprise | Next session |
|---|---|---|---|---|---|---|---|
| Aug 10, 2026Q2 FY26 | $0.86 | $1.08 | +25.6% | $18.2M | — | — | −3.85% |
| May 7, 2026Q1 FY26 | −$0.41 | −$0.42 | −2.4% | $4.5M | — | — | −1.09% |
| Feb 26, 2026Q4 FY25 | −$0.42 | −$0.38 | +9.5% | $277.8K | — | — | +10.60% |
| Nov 6, 2025Q3 FY25 | −$0.48 | −$0.45 | +6.2% | $312.5K | — | — | −1.86% |
Financial highlights
| 12 quarters | Q1 FY25 | Q2 FY25 | Q3 FY25 | Q4 FY25 | Q1 FY26 | Q2 FY26 | |
|---|---|---|---|---|---|---|---|
| Revenue | $0 | $0 | $0 | $0 | $0 | $0 | |
| Gross profit | $0 | $0 | $0 | $0 | −$2.1M | $0 | |
| Operating income | −$64.4M | −$71.1M | −$52.2M | −$44.0M | −$48.5M | −$46.9M | |
| Net income | −$61.3M | −$68.9M | −$50.3M | −$42.5M | −$47.6M | $123.2M | |
| Diluted EPS | −$0.56 | −$0.62 | −$0.45 | −$0.38 | −$0.42 | $1.08 | |
| Gross margin | — | — | — | — | — | — | |
| Operating margin | — | — | — | — | — | — |
Fiscal years (4-quarter sums)FY21 $0 · FY22 $0 · FY23 $0 · FY24 $0 · FY25 $0
Balance sheet, Jun 30, 2026Cash & short-term investments $283.7M · Total debt $24.8M · Total assets $423.1M · Shareholders' equity $368.8M
Dividends
No dividends on record.
Ownership
Institutions and insiders are filed separately and overlap, so together they can pass the whole company. No split is drawn from them.
Shares outstanding 109.19M · Float 86.96M · 79.6% free float
Top shareholders13F · 13D/G · Form 4
| Holder | Shares | % outstanding | Form | Filed |
|---|---|---|---|---|
| RTW Investments, LP | 17.69M | 16.20% | 13F | Aug 14, 2026 |
| BlackRock, Inc. | 9.92M | 9.09% | 13F | Aug 7, 2026 |
| Vanguard Portfolio Management | 5.85M | 5.35% | 13G | Jul 31, 2026 |
| Citigroup Inc | 4.87M | 4.46% | 13F | Aug 3, 2026 |
| Marshall Wace, LLP | 4.03M | 3.69% | 13F | Aug 13, 2026 |
Insider tradesForm 4
| Date | Insider | Role | Shares | Avg price |
|---|---|---|---|---|
| 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 |
| Aug 11, 2026 | Sarbani Chaudhuri | officer: See Remarks | −4,701 | $3.50 |
Peers
- 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 close
| Company | Price | Change | |||||
|---|---|---|---|---|---|---|---|
| Rocket Pharmaceuticals | $3.82 | −0.91% | $421.5M | — | — | — | — |
| Lexeo Therapeutics | $4.77 | −1.95% | $381.6M | — | — | — | — |
| Tenaya Therapeutics | $0.6956 | +0.40% | $149.8M | — | — | 112.2 | — |
| Solid Biosciences | $9.77 | −0.10% | $961.9M | — | — | — | — |
| Sarepta Therapeutics | $21.05 | +0.98% | $2.20B | — | 26.3 | 1.1 | −34.3% |
| REGENXBIO | $9.65 | −0.21% | $499.4M | — | — | 2.9 | +405.7% |
| Krystal Biotech | $351.46 | +0.05% | $10.36B | 44.0 | 41.3 | 23.5 | +24.1% |
| Median | 44.0 | 33.8 | 13.2 | +24.1% |