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.

Genetic Testing

Rocket’s unwavering commitment to seeking gene therapies for the rare disease patient community means that, in addition to our leading clinical research, we actively seek ways to address their urgent and often unmet needs.

Benefits of Genetic Testing

Uncover the Mystery

1.

Uncovering a mystery that could reveal the true cause of a patient’s symptoms.
Care Plan

2.

Identifying the optimal care plan with a patient’s health care providers.
Clinical Research

3.

Considering the pursuit of clinical research studies.
Relief

4.

Providing relief from the uncertainty they may have been experiencing.

Approximately 80 percent of rare diseases are estimated to have a genetic origin. For rare disease patients, the diagnostic delay can take months or decades, averaging four to five years. To compound that journey, on average, patients with rare diseases receive three misdiagnoses and consult with five doctors before receiving an accurate diagnosis.

Thanks to scientific advancements, patients may pursue genetic testing with the goal of receiving an accurate and early diagnosis.

Rocket’s Commitment to Genetic Testing

Through ongoing partnerships with some of today’s leading minds in genetic testing, we’re advocating for earlier diagnosis, advancing access, and promoting the best possible care for people living with rare genetic conditions.

Rocket is committed to raising awareness of programs that can shorten the diagnostic journey and reduce the mental and emotional toll. Lack of widespread early and accurate testing creates inequity and blocks proper care, ultimately hindering access to the curative treatments that we so passionately pursue on behalf of the rare disease community. 

Mission: Genome™ NO-CHARGE GENETIC TESTING AND COUNSELING PROGRAM

Mission: Genome is a sponsored, no-charge, third-party genetic counseling and testing program for people with a family history or suspected diagnosis of certain genetic conditions.

The Mission: Genome program was developed to provide genetic testing and counseling services to people and their families so that they and their healthcare providers may make more informed decisions about their health. Through the Mission: Genome program, genetic testing and counseling is offered at no charge to individuals who meet the eligibility criteria. Both genetic testing and genetic counseling are available in the U.S. and Puerto Rico. No-charge family variant testing may also be available for blood relatives of diagnosed patients.

What Is Genetic Testing?

Genetic testing can identify changes to DNA that may lead to a genetic condition. The results of a genetic test can confirm or rule out a suspected genetic disease. Additionally, genetic testing can help determine an individual’s chance of developing or passing on a genetic condition.

To learn more, please click here.

What Is Genetic Counseling?

Genetic counseling is a service that provides information and support for people who have, or may be at risk for, genetic conditions. Genetic counselors are trained healthcare professionals who can discuss genetics, the likelihood of inheritance, and the risks of developing or passing along certain genetic diseases. Genetic counselors are also able to discuss the benefits, limitations, and potential implications of genetic testing.

To learn more, please click here.

Talk to your Doctor or Healthcare Provider

Start a conversation with your doctor or healthcare provider to determine if you could benefit from genetic testing and counseling.

Clinical Trials

Patients and families interested in learning more about available clinical trials and research opportunities in these disease areas should visit our Clinical Trials Page.

Learn More

Patient Advocacy

Patients and families interested in accessing educational and advocacy group resources about any of these disease areas should visit our Patients & Caregivers Page

Learn More

Newborn Screening

We’re also a founding member of the BeginNGS™ consortium, partnering with Rady Children’s Institute for Genomic Medicine to drive early diagnosis of rare genetic diseases by facilitating newborn screenings using rapid Whole Genome Sequencing (rWGS®). The BeginNGS™ consortium serves as a think tank across public and private institutions. Rocket plays a leading role in advocating for early diagnosis of rare genetic diseases.

For more information, please visit BeginNGS.

Learn More Here about Mission: Genome

Learn More
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