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ROCKET PHARMACEUTICALS, INC. (RCKT)

Rocket Pharmaceuticals, Inc., operating alongside its affiliates, is an innovative biotechnology enterprise dedicated to pioneering gene therapies for rare and severely debilitating diseases. The company's active clinical pipeline features multiple promising programs. It is currently progressing three ex vivo lentiviral vector therapies aimed at specific genetic conditions: Fanconi anemia, characterized by a bone marrow defect that hinders blood cell production; leukocyte adhesion deficiency-I, an inherited disorder resulting in a compromised immune system; and pyruvate kinase deficiency, a rare autosomal recessive red blood cell ailment leading to chronic non-spherocytic hemolytic anemia. Complementing these, Rocket Pharmaceuticals also has an in vivo adeno-associated virus program underway for Danon disease, a serious multi-organ lysosomal storage disorder that tragically often results in early mortality due to heart failure. To facilitate its extensive research and development efforts, the company has established key licensing partnerships with esteemed institutions such as the Fred Hutchinson Cancer Research Center, European research bodies including CIEMAT and its collaborators (Centro de Investigacion Biomedica En Red and Fundacion Instituto de investigacion Sanitaria Fundacion Jimenez Diaz), UCL Business PLC (in conjunction with CIEMAT), The Regents of the University of California, and REGENXBIO, Inc. Rocket Pharmaceuticals, Inc. maintains its primary corporate location in Cranbury, New Jersey.

Sector: Pharmaceutical Preparations

Industry: Biotechnology

Rocket Pharmaceuticals, Inc. logo

Rocket Pharmaceuticals, Inc.

NASDAQ:RCKTHealthcare
-3.94%$2.44
AH$2.40-1.64%vs close
Mkt Cap$267.85M
Float84.59M
O/S109.77M
Vol
$ Vol
RVOL
Inst Own74.7%
SI19.3%
Earnings
Dilution
Rocket Pharmaceuticals, Inc. logoNASDAQ:RCKT$2.44-3.94%
Phase Distribution
26 lead-sponsored trials·9 active·10 completed
Phase 18
Phase 1/22
Phase 28
Phase 31
Not applicable7
(7 more as collaborator)
Status Overview
Not yet recruiting1
Recruiting6
Active, not recruiting3
Completed10
Stopped4
Unknown2

Trials need funding. See this company's dilution risk and cash position.

Clinical Trials
Sponsor role
Status
Phase
  • Phase 3
    Completed

    Study of Trabodenoson in Adults With Ocular Hypertension or Primary Open-angle Glaucoma

    trabodenoson 4.5% BID, trabodenoson 6.0% QD

    Primary Open-Angle Glaucoma (POAG), Ocular Hypertension (OHT)

    Enrollment
    303
    Start
    Sep 2015
    Primary Completion
    Nov 2016
    Completion
    Nov 2016
    NCT02565173
  • Phase 2
    Terminated

    A Trial of INO-1001 in Patients Undergoing Heart Surgery That Involves Heart-lung Bypass

    INO-1001

    Heart Diseases, Postoperative Complications

    Enrollment
    162
    Start
    Oct 2005
    Primary Completion
    -
    Completion
    Jun 2006
    NCT00271167
  • Phase 2
    Completed

    A Study of INO-1001, an Intravenous PARP (Poly [ADP Ribose] Polymerase) Inhibitor in Acute Heart Attack Patients Undergoing Primary Percutaneous Coronary Intervention

    INO-1001

    Acute Myocardial Infarction

    Enrollment
    40
    Start
    Jan 2004
    Primary Completion
    -
    Completion
    Jun 2006
    NCT00271765
  • Phase 2
    Completed

    A Phase II Study to Evaluate the Additivity of Trabodenoson to Latanoprost in Adults With Ocular Hypertension or Primary Open-Angle Glaucoma

    Trabodenoson, Latanoprost

    Primary Open-Angle Glaucoma (POAG), Ocular Hypertension (OHT)

    Enrollment
    101
    Start
    Aug 2013
    Primary Completion
    Aug 2014
    Completion
    Sep 2014
    NCT01917383
  • Phase 2
    Completed

    Study of Fixed-Dose Combinations of Trabodenoson and Latanoprost in Adults With OHT or POAG

    trabodenoson 6.0% / latanoprost 0.005% QD, trabodenoson 3.0% / latanoprost 0.005% QD

    Primary Open-Angle Glaucoma (POAG), Ocular Hypertension (OHT)

    Enrollment
    201
    Start
    Aug 2016
    Primary Completion
    May 2017
    Completion
    May 2017
    NCT02829996
  • Phase 2
    Unknown status

    Lentiviral-mediated Gene Therapy for Pediatric Patients With Fanconi Anemia Subtype A

    RP-L102

    Fanconi Anemia Complementation Group A

    Enrollment
    7
    Start
    Nov 2019
    Primary Completion
    Feb 2025est.
    Completion
    Feb 2025est.
    NCT04069533
  • Phase 2
    Active, not recruiting

    Gene Therapy for Fanconi Anemia, Complementation Group A

    RP-L102

    Fanconi Anemia Complementation Group A

    Enrollment
    5
    Start
    Jul 2020
    Primary Completion
    May 2026est.
    Completion
    May 2026est.
    NCT04248439
  • Phase 2
    Recruiting

    A Gene Therapy Study of RP-A501 in Male Patients With Danon Disease

    RP-A501

    Danon Disease

    Enrollment
    18
    Start
    Sep 2023
    Primary Completion
    Jun 2028est.
    Completion
    Jun 2032est.
    NCT06092034
  • Phase 2
    Suspended

    Clinical Trial to Evaluate the Efficacy of Gene Therapy for Pyruvate Kinase Deficiency

    RP-L301

    Pyruvate Kinase Deficiency

    Enrollment
    10
    Start
    Apr 2026est.
    Primary Completion
    Jan 2029est.
    Completion
    Jan 2029est.
    NCT06422351
  • Phase 1/2
    Completed

    A Dose-Escalation Study Designed to Evaluate the Tolerability, Safety, Pharmacokinetics (PK), and Efficacy of Chronic Topical Ocular Application of INO-8875 in Adults With Ocular Hypertension or Primary Open-Angle Glaucoma

    INO-8875, Placebo

    Glaucoma, Ocular Hypertension

    Enrollment
    144
    Start
    May 2010
    Primary Completion
    Dec 2011
    Completion
    Jan 2012
    NCT01123785
  • Phase 1/2
    Completed

    A Clinical Trial to Evaluate the Safety and Efficacy of RP-L201 in Subjects With Leukocyte Adhesion Deficiency-I

    RP-L201

    Leukocyte Adhesion Defect - Type I

    Enrollment
    9
    Start
    Aug 2019
    Primary Completion
    Sep 2023
    Completion
    Sep 2023
    NCT03812263
  • Phase 1
    Completed

    Phase I Dose-Escalation Study to Evaluate Tolerability, Safety & PK of INO-8875 in Healthy Older-Adult Volunteers

    INO-8875

    Health

    Enrollment
    70
    Start
    May 2010
    Primary Completion
    Feb 2011
    Completion
    Feb 2011
    NCT01123772
  • Phase 1
    Unknown status

    A Clinical Trial to Evaluate the Safety of RP-L102 in Pediatric Subjects With Fanconi Anemia Subtype A

    RP-L102

    Fanconi Anemia Complementation Group A

    Enrollment
    2
    Start
    Jan 2019
    Primary Completion
    Dec 2020est.
    Completion
    Mar 2022est.
    NCT03814408
  • Phase 1
    Withdrawn

    A Gene Therapy Trial to Evaluate the Safety and Efficacy of RP-L201 in Subjects With Leukocyte Adhesion Deficiency-I

    RP-L201

    Leukocyte Adhesion Defect - Type I

    Enrollment
    0
    Start
    Apr 2019
    Primary Completion
    Oct 2021est.
    Completion
    Oct 2021est.
    NCT03825783
  • Phase 1
    Completed

    Gene Therapy for Male Patients With Danon Disease (DD) Using RP-A501; AAV9.LAMP2B

    RP-A501

    Danon Disease

    Enrollment
    7
    Start
    Apr 2019
    Primary Completion
    Jul 2025
    Completion
    Jul 2025
    NCT03882437
  • Phase 1
    Completed

    Gene Therapy for Pyruvate Kinase Deficiency (PKD)

    RP-L301

    Pyruvate Kinase Deficiency

    Enrollment
    4
    Start
    Jul 2020
    Primary Completion
    Jun 2025
    Completion
    Jun 2025
    NCT04105166
  • Phase 1
    Terminated

    A Trial to Evaluate Safety and Efficacy of RP-L401-0120 in Subjects With Infantile Malignant Osteopetrosis

    RP-L401

    Infantile Malignant Osteopetrosis

    Enrollment
    1
    Start
    Nov 2020
    Primary Completion
    May 2021
    Completion
    May 2021
    NCT04525352
  • Phase 1
    Recruiting

    A Phase 1, Dose Escalation Trial of RP-A601 in Subjects With PKP2 Variant-Mediated Arrhythmogenic Cardiomyopathy (PKP2-ACM)

    RP-A601

    PKP2 Arrhythmogenic Cardiomyopathy (PKP2-ACM)

    Enrollment
    9
    Start
    Aug 2023
    Primary Completion
    Sep 2028est.
    Completion
    Sep 2029est.
    NCT05885412
  • Phase 1
    Recruiting

    A Phase 1 AAV Gene Therapy Trial Evaluating Safety and Preliminary Efficacy of RP-A701 in Subjects With BAG3 Dilated Cardiomyopathy

    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)

    Dilated Cardiomyopathy (DCM)

    Enrollment
    8
    Start
    Apr 2026
    Primary Completion
    Jun 2029est.
    Completion
    Jun 2029est.
    NCT07137338
  • Observational
    Active, not recruiting

    Long-Term Follow-up of Subjects With Fanconi Anaemia Subtype A Treated With ex Vivo Gene Therapy

    Safety and efficacy assessments

    Fanconi Anemia Complementation Group A, Fanconi Anemia

    Enrollment
    9
    Start
    Jun 2020
    Primary Completion
    Jan 2034est.
    Completion
    Jan 2034est.
    NCT04437771
  • Observational
    Completed

    Natural History of Danon Disease

    Heart Transplant, Cardiac Assistive Device

    Danon Disease

    Enrollment
    59
    Start
    Sep 2022
    Primary Completion
    Oct 2023
    Completion
    Oct 2023
    NCT05548855
  • Observational
    Recruiting

    Danon Disease Natural History Study

    No intervention

    Danon Disease

    Enrollment
    60
    Start
    Dec 2023
    Primary Completion
    Dec 2029est.
    Completion
    Mar 2030est.
    NCT06214507
  • Observational
    Active, not recruiting

    Long-Term Follow-Up (LTFU) for Gene Therapy of Leukocyte Adhesion Deficiency-I (LAD-I)

    -

    Leukocyte Adhesion Deficiency

    Enrollment
    9
    Start
    Mar 2022
    Primary Completion
    Oct 2036est.
    Completion
    Oct 2036est.
    NCT06282432
  • Observational
    Recruiting

    PKP2-ACM Natural History Study

    -

    Cardiomyopathies, Heart Diseases +2

    Enrollment
    36
    Start
    May 2026
    Primary Completion
    Dec 2030est.
    Completion
    Apr 2031est.
    NCT06644742
  • Observational
    Not yet recruiting

    BAG3-DCM Natural History Study

    -

    Dilated Cardiomyopathy (DCM), Cardiovascular Diseases +2

    Enrollment
    30
    Start
    Dec 2026est.
    Primary Completion
    Dec 2032est.
    Completion
    Mar 2033est.
    NCT07486752

1-25 of 26

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