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.

Join Us

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Who We Are

The Rocket team is a creative, compassionate and driven group of individuals pushing the boundaries of gene therapy to create potentially curative medicines for rare, genetic diseases. Backed by industry visionaries with rich experience and a growing team with a profound sense of purpose, we are laser focused on the needs of patients. Our passionate team thrives on blue-sky thinking while possessing the expertise and drive to turn those dreams into reality. Our unyielding devotion to our patients is key to what sets us apart from other organizations.

We are always looking for passionate and inspiring team members to join us on our quest for gene therapy cures.

Interested in joining our team?

Check out our open positions

What We Do

We’re harnessing cutting-edge science and innovation to correct diseases at the genetic level to enable people to live long and full lives. Our focused pipeline and efficient execution have positioned us to bring transformative, potentially curative therapies to patients and the rare disease community.

Rocket selects the brightest minds—from new scientists to seasoned gene therapy experts—and combines their multi-disciplinary experiences along with their personal passion to make gene therapy a reality. Insatiably curious, our people are committed to making a difference and sharing their knowledge and experience for the benefit of patients, the scientific community and humanity as a whole.

what we do
life at rocket

Life at Rocket

We believe in having an environment where all ideas are welcome, and every team member’s opinion is valued. One mindset that remains constant among all Rocket employees is our steadfast commitment to the work we do. Our goal is simple – to treat each patient with the highest quality product as if they were our loved ones.

By choosing Rocket, you will have the opportunity to work with some of the most brilliant experts in their related fields. We offer internal development programs, informal mentoring programs, as well as a plan to help you get to where you want to be in your career. We believe that you are the pilot of your career, and we will help you ascend! Rocket is comprised of and celebrates diversity in all its forms. We celebrate the unique differences of all our employees and create a safe, inclusive work environment where all employees have a voice and opportunity. We have built our company like a puzzle – each of our differences become part of our whole, a team that can move mountains.

View our wall of stars video
For me, leadership means ongoing self-improvement and striving towards goals that better support our team and patients. I’m grateful to Rocket for giving me opportunities to advance in my career development so that I can effectively assist others in advancing theirs too. Leadership represents a journey of service, guiding others towards a greater purpose of serving patients, echoing the motto: 'In the joy of others lies our own.'
Mihir Patel
Mihir PatelProcess Development
Rocket is a company that is truly unique where the “sky is the limit”, and we continue to nurture an employee community where everyone feels welcome. At Rocket, we’re deeply dedicated to fostering an inclusive culture not defined by who you are, gender, age, ethnicity, or degree, but by the ideas and passion we bring to our work every day.
Sanchali Kasbekar
Sanchali KasbekarRegulatory Affairs
As a manager at Rocket, I am dedicated to ensuring my team’s success and fostering a collaborative environment where everyone can effectively achieve their goals. I frequently emphasize that the hardest part of a job well done is the journey. Each step is a lesson for the future. Enjoy the process.
Donald Francios
Donald FrancoisQuality
Working at Rocket has provided the fantastic opportunity to expand my industry experience by learning and utilizing cutting edge assays, which elevates my portfolio as a researcher. It’s inspiring to be a part of a team that provides so many opportunities to promote my career growth.
Madeline Doyle
Madeline DoyleResearch
The rigorous scientific methodology taught in academia is quintessential to developing cutting-edge, safe, and efficacious therapies for patients in need. Academia's appreciation of the value of each team member is the fabric of day-to-day gene therapy development at Rocket.
Maria Chitty-Lopez
Maria Chitty-LopezMedical Director
Working on rare diseases is a privilege, and being a part of the Rocket team who are dedicated to advancing gene therapies for patients living with rare and ultra-rare diseases, makes it even more special. The opportunity to build the marketing function and a team to deliver on the strategic vision for each disease area and brand and bring our gene therapies to patients is a once-in-a-lifetime experience.
Charles Shiner
Charlie ShinerCommercial
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What We Offer

Rocket is dedicated to its employees and firmly believes that a robust rewards philosophy will keep employees engaged and healthy, mentally, physically and financially. In addition to a competitive salary and bonus structure, we offer medical, dental, vision and life insurance, 401k match and generous vesting schedule, vacation time, holidays and so much more.

To support their onboarding and integration, new employees become part of a cohort—named for planets, stars, and constellations—and meet periodically to share their unique experiences and perspectives. We shine a light on these new teammates through our Rocket First Connections program that includes employee spotlights and other programming amplified across the company. The goal is to set up new employees for success and build a company that is even more powerfully united in our mission for patients.

Whether we work on site, hybrid or remote, the Rocket employee community enjoys a variety of opportunities to come together throughout the year. These include quarterly Townhalls in which we celebrate our people, various team building events, a holiday gathering and our annual Rare Disease Day celebration.

The inclusive gathering of various viewpoints and voices at Rocket, conducted through a thorough set of focus groups, led to the development of the company’s groundbreaking Employee Value Proposition (EVP). The three pillars of the EVP, Opportunity, Well-Being and Connection, are derived from what employees said they wanted from their work and workplace and serve as a framework for ensuring Rocket is meeting the needs of our team.

Opportunity: an opportunity to develop and grow our careers

Well-being: a place where well-being is a priority

Connection: a collaborative, inclusive community that brings out our best

The EVP serves as a roadmap for meeting the needs of our team and for attracting, developing and retaining the best people in the industry.

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