Rocket Pharmaceuticals is focused on developing innovative gene therapies aimed at treating rare genetic disorders, particularly those that have significant unmet medical needs. Their approach includes utilizing ex vivo lentiviral (LV) and in vivo adeno-associated viral (AAV) platforms, which allow for tailored therapies that address the root causes of diseases. One of their key products, RP-A501, is specifically designed for Danon Disease, a severe genetic condition affecting heart muscle function. The company is pioneering clinical trials that assess both the safety and efficacy of this gene therapy, with a pivotal Phase 2 trial currently underway, evaluating critical endpoints such as LAMP2 protein expression and left ventricular mass reduction.

In addition to their therapeutic development, Rocket Pharmaceuticals is also conducting natural history studies to gather comprehensive data on the progression of Danon Disease. These observational studies aim to collect longitudinal clinical information from patients, which is essential for understanding the disease's impact and informing future clinical trials. By engaging in these studies, Rocket seeks to enhance patient care and contribute to the broader understanding of rare diseases, ultimately empowering patient communities and improving healthcare outcomes.

United States

Rocket Pharmaceuticals (NASDAQ: RCKT), Ltd is a biotechnology company focused on seeking cures through molecular and gene therapy.

We are developing first-in-class treatments for patients with rare and often devastating orphan diseases using a lentiviral-based and adeno-associated virus (AAV) therapy approach. We have developed rewarding partnerships with a number of leading international research institutions as well as with centers of manufacturing and cell processing expertise, while building an internal team to drive the research, development, clinical and regulatory programs. Ultimately we aim to launch and globally commercialize innovative gene therapies in disease areas with high unmet need.

Cardiovascular Diseases

Genetic Diseases

Hematologic Diseases

Metabolic Diseases

Rare diseases

Rocket Pharmaceuticals

Blood disorder

Fanconi Anemia

Rocket’s pipeline is comprised of first-in-class gene therapies that incorporate both adeno-associated viral vector (AAV) and lentiviral vector (LVV) approaches to gene therapy.
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 <li>LVV gene therapy that elicits phenotypic correction of blood cells and stabilization of previously declining blood counts</li>
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Gene therapy

RP-L102

Immunodeficiency

Leukocyte Adhesion Deficiency

KRESLADI (Marnetegragene autotemcel) /RP-L201 

Pyruvate Kinase Deficiency

RP-L301 

Danon Disease

RP-A501

Arrhythmogenic cardiomyopathy

Heart disease

RP-A601, an AAV.rh74-based gene therapy candidate for the treatment of arrhythmogenic cardiomyopathy due to plakophilin 2 pathogenic variants (PKP2-ACM) a devastating inherited heart disease that can lead to life-threatening arrhythmias, cardiac structural abnormalities, and sudden cardiac death. The current standard of care for patients with PKP2-ACM consists of medical therapy, implantable cardioverter defibrillators (ICDs), and ablations, which are not curative.
Gene therapy

Rocket Pharmaceuticals

Company Details