RCKT (Rocket Pharmaceuticals Inc.) stock: $3.33, $365.8M market cap, $283.7M cash, $82.1M EV, $377.3M fully diluted market cap. Next expected readout: Apr-28. Lead program: RP-A501 (Danon Disease), Phase 2. Data from Fully Diluted's biotech stocks database.
Balance sheet
| Cash | $283.7M |
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| Debt | $0.0M |
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| Net cash | $283.7M |
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| Enterprise value | $82.1M |
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| Basic shares | 109.5M shares |
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| Fully diluted shares | 113.3M shares |
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| Fully diluted market cap | $377.3M |
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| Cash per share | $2.59 |
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Cash figures as of 2026-06-30. Database snapshot: 2026-09-22.
Price history
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Upcoming catalysts
| Expected | Drug | Phase | Indication | Mgmt guide |
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| May 13 - Jun 24 | RP-A501 | Phase 2 | Danon Disease | — |
Estimated readout windows: registry primary-completion date + 6-12 weeks. Windows already open are shown from "Now". Drug names link to ClinicalTrials.gov.
Full readout calendar →
Pipeline assets
KRESLADI
Phase 3 · Active Modality: ex vivo gene therapy (lentiviral-modified autologous hematopoietic stem cells)
Aliases RP-L201, marnetegragene autotemcel
Target ITGB2 (CD18)
Partnerships In-licensed from CIEMAT, CIBERER and Instituto de Investigación Sanitaria Fundación Jiménez Díaz; LV vector developed in collaboration with University College London and CIEMAT
Indications- Leukocyte adhesion deficiency-I (LAD-I) (Phase 3)
Status note Phase 1/2 registration-enabling trial treated 9 severe LAD-I patients (published NEJM May 2025). BLA accepted with priority review (Sept 2023); review extended to June 2024; FDA issued CRL June 2024 (limited additional CMC). BLA resubmission accepted October 2025 with PDUFA March 28, 2026. RMAT, Rare Pediatric, Fast Track, PRIME, ATMP, Orphan designations.
RP-A501
Phase 2 · Active Modality: gene therapy (AAV9 capsid, human LAMP2B transgene, single IV infusion)
Target LAMP2
Partnerships CIRM: up to $5.8M CLIN2 grant (Aug 2024) supporting clinical development
Indications- Danon disease (DD) (Phase 2)
Status note Global single-arm Phase 2 pivotal trial (12 patients). Enrollment completed Sep 2024. Dosing paused after two SAEs (capillary leak syndrome, one death) in May 2025; FDA clinical hold May 23, 2025 lifted August 2025. Resuming at recalibrated dose 3.8e13 GC/kg with sequential dosing. RMAT, Fast Track, Rare Pediatric, Orphan, ATMP, PRIME designations.
RP-A601
Phase 1 · Active Modality: gene therapy (AAVrh74 capsid, human PKP2 transgene, single IV infusion)
Target PKP2
Indications- Plakophilin-2 arrhythmogenic cardiomyopathy (PKP2-ACM) (Phase 1)
Status note Single-arm open-label Phase 1 ongoing in adults with ICDs (3 treated; 8e13 GC/kg selected as development dose; generally well tolerated; PKP2 protein expression increased in all 3). RMAT, Fast Track, Orphan designations.
RP-A701
Preclinical · Active Modality: gene therapy (AAVrh74 capsid, human BAG3 transgene)
Target BAG3
Partnerships Acquired via Renovacor acquisition (December 2022)
Indications- BAG3 dilated cardiomyopathy (BAG3-DCM) (Preclinical)
Status note IND cleared June 2025; Fast Track granted July 2025. First-in-human multi-center dose-escalation Phase 1 in adults with BAG3-DCM planned.
RP-L102
Phase 2 · Active Modality: ex vivo gene therapy (lentiviral-modified autologous hematopoietic stem cells)
Target FANCA
Indications- Fanconi anemia (FA) (Phase 2)
Status note Phase 2 study treated 14 FA type A patients without myeloablative conditioning. As of July 2025, no internal resources allocated to regulatory filings/commercialization; company exploring external partnership options (decision based on business strategy, not safety/efficacy).
RP-L301
Phase 1 · Active Modality: ex vivo gene therapy (lentiviral-modified autologous hematopoietic stem cells)
Target PKLR
Partnerships In-licensed from CIEMAT, CIBERER and Instituto de Investigación Sanitaria Fundación Jiménez Díaz
Indications- Pyruvate kinase deficiency (PKD) (Phase 1)
Status note Phase 1 study treated 2 adult and 2 pediatric patients. FDA alignment on 10-patient pivotal Phase 2 for accelerated approval, but no internal resources allocated and no enrollment planned; exploring external partnerships.
Per-asset detail extracted from the 10-Q filed 2026-08-10. Fields the filing does not state are marked "not disclosed" rather than filled from other sources.