Rocket Pharmaceuticals

🇺🇸United States

Country: 🇺🇸United States

Ownership: Public

Established: 2015-01-01

Employees: 268

Market Cap: $1.7B

Website: http://www.rocketpharma.com

Introduction: Rocket Pharmaceuticals, Inc. is a clinical-stage biotechnology company, which engages in the development of gene therapy treatment options for rare and devastating pediatric diseases. Its multi-platform development approach applies the lentiviral vector and adeno-associated viral vector gene therapy platforms. Its clinical program is an LVV-based gene therapy for the treatment of Fanconi Anemia (FA), a difficult to treat genetic disease that leads to bone marrow failure and potentially cancer. The company was founded on July 7, 1999 and is headquartered in Cranbury, NJ.

Clinical Trials

PKP2-ACM Natural History Study

Not yet recruiting

Conditions: Cardiomyopathies
Heart Diseases
Cardiovascular Diseases
Genetic Diseases

First Posted Date: 2024-10-16

Last Posted Date: 2024-10-16

Lead Sponsor: Rocket Pharmaceuticals Inc.

Target Recruit Count: 36

Registration Number: NCT06644742

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

Phase 2

Not yet recruiting

Conditions: Pyruvate Kinase Deficiency

Interventions: Biological: RP-L301

First Posted Date: 2024-05-21

Last Posted Date: 2024-05-22

Lead Sponsor: Rocket Pharmaceuticals Inc.

Target Recruit Count: 10

Registration Number: NCT06422351

Locations: 🇺🇸
Stanford University, Palo Alto, California, United States
🇪🇸
Hospital Infantil Universitario Niño Jesús, Madrid, Spain
🇪🇸
Hospital Universitario Fundación Jiménez Díaz, Madrid, Spain

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

Conditions: Leukocyte Adhesion Deficiency

First Posted Date: 2024-02-28

Last Posted Date: 2024-02-28

Lead Sponsor: Rocket Pharmaceuticals Inc.

Target Recruit Count: 9

Registration Number: NCT06282432

Locations: 🇬🇧
University College London Great Ormond Street Institute of Child Health (GOSH), London, United Kingdom
🇺🇸
University of California, Los Angeles (UCLA), Los Angeles, California, United States
🇪🇸
Hospital Infantil Universitario Niño Jesús, Madrid, Spain

Danon Disease Natural History Study

Recruiting

Conditions: Danon Disease

Interventions: Other: No intervention

First Posted Date: 2024-01-19

Last Posted Date: 2024-11-28

Lead Sponsor: Rocket Pharmaceuticals Inc.

Target Recruit Count: 46

Registration Number: NCT06214507

Locations: 🇺🇸
University of Florida College of Medicine, Gainesville, Florida, United States
🇺🇸
University of California, San Diego, La Jolla, California, United States
🇺🇸
Texas Children's Hospital, Houston, Texas, United States

and more 8 locations

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

Phase 2

Recruiting

Conditions: Danon Disease

Interventions: Genetic: RP-A501

First Posted Date: 2023-10-23

Last Posted Date: 2024-07-10

Lead Sponsor: Rocket Pharmaceuticals Inc.

Target Recruit Count: 12

Registration Number: NCT06092034

Locations: 🇺🇸
Boston Children's Hospital, Boston, Massachusetts, United States
🇮🇹
Meyer Childrens Hospital, Florence, Italy
🇺🇸
University of California, San Diego, La Jolla, California, United States

and more 3 locations

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

Phase 1

Recruiting

Conditions: PKP2 Arrhythmogenic Cardiomyopathy (PKP2-ACM)

Interventions: Genetic: RP-A601

First Posted Date: 2023-06-02

Last Posted Date: 2024-10-04

Lead Sponsor: Rocket Pharmaceuticals Inc.

Target Recruit Count: 9

Registration Number: NCT05885412

Locations: 🇺🇸
Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, United States
🇺🇸
Duke University, Durham, North Carolina, United States
🇺🇸
University of California, San Diego, La Jolla, California, United States

Natural History of Danon Disease

Completed

Conditions: Danon Disease

Interventions: Procedure: Heart Transplant
Device: Cardiac Assistive Device

First Posted Date: 2022-09-22

Last Posted Date: 2023-11-21

Lead Sponsor: Rocket Pharmaceuticals Inc.

Target Recruit Count: 59

Registration Number: NCT05548855

Locations: 🇮🇹
Ospedale Papa Giovanni XXIII di Bergamo ASST Pap Giovanni XXIII Hospital, Bergamo, Italy
🇪🇸
Hospital Álvaro Cunqueiro, Pontevedra, Spain
🇫🇷
CHU de Nantes, Hôpital Laennec, Nantes, France

and more 12 locations

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

Phase 1

Terminated

Conditions: Infantile Malignant Osteopetrosis

Interventions: Biological: RP-L401

First Posted Date: 2020-08-25

Last Posted Date: 2022-07-13

Lead Sponsor: Rocket Pharmaceuticals Inc.

Target Recruit Count: 1

Registration Number: NCT04525352

Locations: 🇺🇸
University of California, Los Angeles, Los Angeles, California, United States

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

Conditions: Fanconi Anemia Complementation Group A
Fanconi Anemia

Interventions: Other: Safety and efficacy assessments

First Posted Date: 2020-06-18

Last Posted Date: 2020-06-22

Lead Sponsor: Rocket Pharmaceuticals Inc.

Target Recruit Count: 9

Registration Number: NCT04437771

Locations: 🇪🇸
Hospital Infantil Universitario Niño Jesús (HIUNJ), Madrid, Spain

Gene Therapy for Fanconi Anemia, Complementation Group A

Phase 2

Active, not recruiting

Conditions: Fanconi Anemia Complementation Group A

Interventions: Biological: RP-L102

First Posted Date: 2020-01-30

Last Posted Date: 2024-04-10

Lead Sponsor: Rocket Pharmaceuticals Inc.

Target Recruit Count: 5

Registration Number: NCT04248439

Locations: 🇺🇸
Stanford University, Stanford, California, United States
🇺🇸
University of Minnesota, Minneapolis, Minnesota, United States

© Copyright 2025. All Rights Reserved by MedPath

Home Terms & Conditions Privacy Policy