
-
Rocket Pharmaceuticals NasdaqGM:RCKT Rocket Pharmaceuticals, Inc., together with its subsidiaries, operates as a late-stage biotechnology company that focuses on developing gene therapies for rare and devastating diseases in the United States. It develops vivo adeno-associated viral (AAV) programs, which include programs for Danon disease (DD), a multi-organ lysosomal-associated disorder leading to early death due to heart failure (RP-A501), which is in Phase 2 trial; Plakophilin-2 Arrhythmogenic Cardiomyopathy (PKP2-ACM), an inheritable cardiac disorder that is characterized by a progressive loss of cardiac muscle mass, severe right ventricular dilation, dysplasia, fibrofatty replacement of the myocardium and a high propensity to arrhythmias and sudden death (RP-A601), which is in phase 1; and BAG3 Dilated Cardiomyopathy (BAG3-DCM), which is a form of cardiomyopathy and is characterized by progressive thinning of the walls of the heart, which is in preclinical stage. It also develops vivo lentiviral (LV) programs, which include programs for Leukocyte Adhesion Deficiency-I (LAD-I), a genetic disorder that causes the immune system to malfunction (RP-L201); Fanconi Anemia (FA), a genetic defect in the bone marrow that reduces production of blood cells or promotes the production of faulty blood cells (RP-L102); and Pyruvate Kinase Deficiency (PKD), a red blood cell autosomal recessive disorder that results in chronic non-spherocytic hemolytic anemia (RP-L301). It has license agreements with Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas, Centro de Investigacion Biomedica En Red, and Fundacion Instituto de investigacion Sanitaria Fundacion Jimenez Diaz; UCL Business PLC; The Regents of the University of California; REGENXBIO, Inc.; and Temple University. Rocket Pharmaceuticals, Inc. was founded in 1999 and is headquartered in Cranbury, New Jersey.
Location: 9 Cedarbrook Drive, Cranbury, NJ, 08512, United States | Website: https://www.rocketpharma.com | Industry: Biotechnology | Sector: Healthcare
Market Cap
NaN
52 Wk Range
$ - $
Previous Close
$N/A
Open
$N/A
Day Range
N/A - N/A
Volume
N/A
Avg Volume
N/A
Enterprise Value
407.5M
Cash
318.2M
Avg Qtr Burn
-52.16M
Short % of Float
14.99%
Insider Ownership
2.74%
Institutional Own.
99.31%
Qtr Updated
03/31/25
Drug Pipeline
Powered by

Only showing assets and stage. See catalyst data with a free or premium account. Unlock more data with a free account.
Drug & Indication | Stage & Event | Catalyst Date |
---|---|---|
KRESLADI (Marnetegragene autotemcel) /RP-L201 Details Immunodeficiency, Leukocyte Adhesion Deficiency | BLA Resubmission | |
RP-L102 Details Rare diseases, Blood disorder, Fanconi Anemia | BLA Submission | |
RP-A501 Details Immunodeficiency, Danon Disease | Phase 2 Update | |
RP-L301 Details Pyruvate Kinase Deficiency | Phase 2 Initiation | |
RP-A601 Details Arrhythmogenic cardiomyopathy | Phase 1 Data readout | |
RP-L401 Details Infantile Malignant Osteopetrosis | Failed Discontinued |