Rocket Pharmaceuticals

Rocket Pharmaceuticals is a biopharmaceutical company focused on seeking gene therapy cures for patients with rare and devastating genetic diseases. The company describes its mission as pursuing these cures through innovative science and platforms, and its vision as freeing patients and the world from rare genetic diseases. Its stated values are Trust, Curiosity, Generosity and Elevate. The company’s work includes research and development of gene therapy programs and supporting research such as natural history studies. These observational studies gather information about how a disease progresses and affects patients; participants do not receive investigational treatments as part of the studies. Rocket says that information can support the research and development of its gene therapy programs. One named program is RP-A501, which Rocket is developing for Danon disease, a rare genetic disorder that can cause thickening and weakening of the heart muscle. The company reports that an open-label, global pivotal Phase 2 trial has commenced. Its disclosed trial plans describe assessment of LAMP2 protein expression and reduction in left ventricular mass at 12 months. Rocket also sponsors a multi-center natural history study for PKP2-related arrhythmogenic cardiomyopathy. The supplied company materials identify trial and study sites in the United States and Europe. The available evidence does not establish the company’s founding date, headquarters, ownership, listing status, or commercial product sales.

The supplied materials describe Rocket Pharmaceuticals as a biopharmaceutical company whose mission is to seek gene therapy cures for people with devastating rare diseases. They emphasize the company’s stated focus on innovative science and platforms, and its vision of freeing patients and the wider world from rare genetic diseases. The materials do not provide a founding date, a founder, a headquarters, an ownership history, or other dated corporate-history details. Rocket’s described research approach includes both gene therapy development and natural history studies. The company explains that natural history studies are observational: they collect information about disease progression and effects on patients, without providing investigational treatments or interventions to participants. Rocket presents these studies as a way to build understanding of the conditions it researches and to support development of its gene therapy programs. The company materials identify RP-A501 as a gene therapy program being developed for Danon disease, a rare genetic disorder associated with thickening and weakening of the heart muscle. Rocket reports that an open-label, global pivotal Phase 2 clinical trial has commenced. The disclosed plan describes enrollment of male patients from the United States and Europe and evaluation of LAMP2 protein expression and change in left ventricular mass at 12 months. The available material does not establish trial outcomes or regulatory approval. Rocket also describes sponsoring a multi-center natural history study for plakophilin-2-related arrhythmogenic cardiomyopathy (PKP2-ACM). The stated purpose is to characterize the condition using de-identified longitudinal clinical information. The supplied pages name research or trial sites in the United States and Europe. No additional dated milestones, commercial launches, leadership changes, acquisitions, or ownership events are established by the provided evidence.

A rare-disease biopharmaceutical company focused on developing gene therapies intended to address disease at its root cause.

• RP-A501

  An investigational gene therapy program being developed for Danon disease; the supplied company page reports that a pivotal Phase 2 trial has commenced.

1. 1Home | Rocket Pharmaceuticalsrocketpharma.com
2. 2About Us | Rocket Pharmaceuticalsrocketpharma.com
3. 3Natural History Studies | Rocket Pharmaceuticalsrocketpharma.com
4. 4PKP2-ACM | Rocket Pharmaceuticalsrocketpharma.com
5. 5Rocket Pharmaceuticals Inc.wikidata.org