Biotech · Public company · New York, NY, US
Rocket Pharmaceuticals, Inc.
Ticker RCKT · SEC CIK 0001281895
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Clinical trials158 recruiting or active
Pipeline programs9From trials and the company's site
Disclosed deals0None found yet
Cash runwayNot availableNo recent cash burn filed
Pipeline9
- RP-A501Phase 2 · Danon DiseaseClinicalTrials.gov · fetched Nov 12, 2026
- RP-L102Phase 2 · Fanconi Anemia Complementation Group AClinicalTrials.gov · fetched Nov 12, 2026
- RP-L201Phase 2 · Leukocyte Adhesion Defect - Type IClinicalTrials.gov · fetched Nov 12, 2026
- RP-L301Phase 2 · Pyruvate Kinase DeficiencyClinicalTrials.gov · fetched Nov 12, 2026
- RP-A601Phase 1 · PKP2 Arrhythmogenic Cardiomyopathy (PKP2-ACM)ClinicalTrials.gov · fetched Nov 12, 2026
- RP-A701 is a recombinant viral vector composed of an AAV serotype rh.74 (AAVrh.74) capsid encapsulating the transgene, BCL2-associated Athanogene 3 (BAG3)Phase 1 · Dilated Cardiomyopathy (DCM)ClinicalTrials.gov · fetched Nov 12, 2026
- RP-L401Phase 1 · Infantile Malignant OsteopetrosisClinicalTrials.gov · fetched Nov 12, 2026
- No interventionDanon DiseaseClinicalTrials.gov · fetched Nov 12, 2026
- RP-L102Fanconi AnemiaClinicalTrials.gov · fetched Nov 12, 2026
Clinical trials15
- PKP2-ACM Natural History StudyRECRUITING · started May 15, 2026ClinicalTrials.gov NCT06644742 · fetched Nov 12, 2026
- A Phase 1 AAV Gene Therapy Trial Evaluating Safety and Preliminary Efficacy of RP-A701 in Subjects With BAG3 Dilated CardiomyopathyPHASE1 · RECRUITING · started Apr 17, 2026ClinicalTrials.gov NCT07137338 · fetched Nov 12, 2026
- Clinical Trial to Evaluate the Efficacy of Gene Therapy for Pyruvate Kinase DeficiencyPHASE2 · SUSPENDED · started Apr 1, 2026ClinicalTrials.gov NCT06422351 · fetched Nov 12, 2026
- Danon Disease Natural History StudyRECRUITING · started Dec 20, 2023ClinicalTrials.gov NCT06214507 · fetched Nov 12, 2026
- A Gene Therapy Study of RP-A501 in Male Patients With Danon DiseasePHASE2 · RECRUITING · started Sep 5, 2023ClinicalTrials.gov NCT06092034 · fetched Nov 12, 2026
- A Phase 1, Dose Escalation Trial of RP-A601 in Subjects With PKP2 Variant-Mediated Arrhythmogenic Cardiomyopathy (PKP2-ACM)PHASE1 · RECRUITING · started Aug 29, 2023ClinicalTrials.gov NCT05885412 · fetched Nov 12, 2026
- Long-Term Follow-Up (LTFU) for Gene Therapy of Leukocyte Adhesion Deficiency-I (LAD-I)ACTIVE_NOT_RECRUITING · started Mar 9, 2022ClinicalTrials.gov NCT06282432 · fetched Nov 12, 2026
- Long-Term Follow-up: Phase I/II Clinical Study to Evaluate the Safety and Efficacy of the Infusion of RP-L102ENROLLING_BY_INVITATION · started Feb 1, 2022ClinicalTrials.gov NCT07527975 · fetched Nov 12, 2026
- A Trial to Evaluate Safety and Efficacy of RP-L401-0120 in Subjects With Infantile Malignant OsteopetrosisPHASE1 · TERMINATED · started Nov 19, 2020ClinicalTrials.gov NCT04525352 · fetched Nov 12, 2026
- Gene Therapy for Fanconi Anemia, Complementation Group APHASE2 · ACTIVE_NOT_RECRUITING · started Jul 15, 2020ClinicalTrials.gov NCT04248439 · fetched Nov 12, 2026
- Gene Therapy for Pyruvate Kinase Deficiency (PKD)PHASE1 · COMPLETED · started Jul 6, 2020ClinicalTrials.gov NCT04105166 · fetched Nov 12, 2026
- A Clinical Trial to Evaluate the Safety and Efficacy of RP-L201 in Subjects With Leukocyte Adhesion Deficiency-IPHASE1_PHASE2 · COMPLETED · started Aug 30, 2019ClinicalTrials.gov NCT03812263 · fetched Nov 12, 2026
- Gene Therapy for Male Patients With Danon Disease (DD) Using RP-A501; AAV9.LAMP2BPHASE1 · COMPLETED · started Apr 17, 2019ClinicalTrials.gov NCT03882437 · fetched Nov 12, 2026
- A Clinical Trial to Evaluate the Safety of RP-L102 in Pediatric Subjects With Fanconi Anemia Subtype APHASE1 · UNKNOWN · started Jan 11, 2019ClinicalTrials.gov NCT03814408 · fetched Nov 12, 2026
- The Natural History of Danon DiseaseUNKNOWN · started Nov 30, 2018ClinicalTrials.gov NCT03766386 · fetched Nov 12, 2026
SEC filings12
- 8-K · 8-KSEC EDGAR · filed Oct 6, 2026 · fetched Nov 12, 2026
- 8-K · 8-KSEC EDGAR · filed Sep 15, 2026 · fetched Nov 12, 2026
- 8-K · 8-KSEC EDGAR · filed Aug 27, 2026 · fetched Nov 12, 2026
- 8-K · 8-KSEC EDGAR · filed Aug 10, 2026 · fetched Nov 12, 2026
- 10-Q · 10-QSEC EDGAR · filed Aug 10, 2026 · fetched Nov 12, 2026
- 8-K · 8-KSEC EDGAR · filed Aug 3, 2026 · fetched Nov 12, 2026
- 8-K · 8-KSEC EDGAR · filed Jun 30, 2026 · fetched Nov 12, 2026
- 8-K · 8-KSEC EDGAR · filed Jun 12, 2026 · fetched Nov 12, 2026
- 8-K · 8-KSEC EDGAR · filed May 21, 2026 · fetched Nov 12, 2026
- 8-K · 8-KSEC EDGAR · filed May 7, 2026 · fetched Nov 12, 2026
- 10-Q · 10-QSEC EDGAR · filed May 7, 2026 · fetched Nov 12, 2026
- 8-K · 8-KSEC EDGAR · filed Apr 28, 2026 · fetched Nov 12, 2026