RP-A501 is being developed for Danon Disease, a rare genetic disorder characterized by thickening and weakening of the heart muscle, often resulting in heart failure, and for male patients, frequent death during adolescence or early adulthood. Rocket is the first company demonstrating safety and efficacy data in clinical studies for gene therapy targeting the heart.

An open-label, global Phase 2 pivotal clinical trial of RP-A501 for the treatment of Danon Disease has commenced. The trial is expected to enroll 12 male patients from the U.S. and EU. The trial will assess a co-primary endpoint consisting of LAMP2 protein expression and left ventricular (LV) mass reduction from baseline at 12 months.

Phase 2 Trial Sites

For more details about the RP-A501 for Danon Disease clinical trial, visit clinicaltrials.gov.

Frequently Asked Questions

The Natural History of a Disease is the typical progression of the disease over a person’s lifetime starting from its onset

Natural History studies are observational studies that gather comprehensive data on the course of the disease to gain a better understanding of its progression and impact on patients. Data captured may include information on the physical signs and symptoms patients experience, test results (for example blood tests or imaging), genetic information, and health-related quality of life, over a period of time. This information helps describe the disease course and identify important factors (e.g. demographic, genetic, and/or lab test results), that may contribute to better or worse disease outcomes.

Travel, accommodation, and food stipend would be provided. Study assessments are provided free of charge.

Natural History Studies are particularly important for rare diseases. Given the diversity of symptoms and the small size of the patient population, these studies are often the only opportunity for researchers and physicians to address significant knowledge gaps and obtain a robust understanding of the disease and patient experience.

Engaging in a Natural History Study offers numerous benefits to the community, such as:

  • Raising awareness in medical, academic and pharmaceutical sectors
  • Empowering rare disease patient communities with knowledge and support for advocating better healthcare policies and support services
  • Enhancing patient care with improved insights into disease progression, allowing for more personalized treatment plans and potentially earlier detection of complications
  • Contributing to advancements in healthcare

These studies provide various avenues to help inform and design clinical trials, such as:

  • Highlighting areas of unmet medical need
  • Understanding characteristics of the patient population to define a treatment benefit from a patient’s perspective.
  • Describing disease progression, enabling the identification of clinical outcome measures and biomarkers
  • Provide patient-centric data for health authorities such as the FDA, and other public health institutions to use in their review processes of investigational treatments.
Learn more about Rocket’s clinical trial for FA

Interested in learning more?
Visit our Patients & Caregivers page or email us at clinicaltrials@nullrocketpharma.com

View more details about the Phase 2 pivotal trial of RP-A501 and natural history study on clinicaltrials.gov.

To read our Expanded Access statement, click here.

DNA Is No Longer Destiny

Seeking gene therapy cures that correct rare and devastating diseases at their root cause. Cures that represent freedom – freedom for patients and their loved ones to fully live in a world without the burden of genetic disease. A world where DNA is not destiny and genetic disease is history.

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Mission, Vision & Values

Our mission is to seek gene therapy cures for patients with devastating rare diseases through the most innovative science and platforms to fulfill the promise of gene therapy for the betterment of our industry, science and humanity.

Trust

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Trust is given and trust is earned – it’s a balance. The word “trust” comes from the “Proto-Indo-European word “deru” which means “to be firm, solid, steadfast.” Trust is the ground and foundation for everything we do.

Generosity

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Being generous means following up, sharing our best ideas, forgiving ourselves and others, asking who needs us, treating our word as gold, taking time to truly see others, and so many other things. The word “generous” has the same root as the word “gene” – which means “to beget.” Genes thrive on the generosity of others. What more is there to say?

Curiosity

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The wonder of a child staring up at the night sky. Humility, selflessness. No single one of us can do this job alone and it is ok to ask for help. Curiosity is derived from the Latin word, “cura” which gave birth to the word “care” as well as “cure.” Generosity is to curiosity what gene is to cure.

Elevate

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Derived from the Latin word “levis,” which means “light” as opposed to heavy. How can we bring trust, generosity and curiosity to elevate ourselves, each other, the pipeline and ultimately the life experience of patients and their families?
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Patient Community

Patients and their caregivers are our North Star—the inspiration and guiding force behind everything we do. We are also acutely aware that rare diseases have a ripple effect, impacting the lives of patients as well as the families, friends, and caregivers who love them. We aim to create a community where rare disease patients and their families can come together and feel supported through every step of their journey.

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Our Science

Our thoughtful multi-platform approach gives our team the unique advantage to develop therapies specifically for conditions with high unmet needs. This allows us to seek diseases where cures seem impossible or those that are often overlooked. Having the flexibility to select the best platform for the disease—ex vivo lentiviral (LV) or in vivo adeno-associated viral (AAV) — keeps patients at the core of everything we do.

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Our People

The Rocket team possesses decades of experience and leadership in the biotech industry. This expertise, combined with our passion, creativity, a commitment to patients and curiosity, has enabled us to build and advance a pipeline of programs targeting genetic bone marrow-derived and cardiovascular diseases.

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