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.

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.

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.

About Us

team photos - about us

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.

Our vision is to free every patient and the world from rare genetic diseases.

Rocket’s unique set of core values – “Trust,” “Curiosity,” “Generosity” and “Elevate” – is a true beacon of hope within and outside the company. Trust is the bedrock, the ground upon which everything is built. Generosity and curiosity, derived from the same root words as “gene” and “cure,” describe a spirit of giving, aspiring for something greater than oneself, and humility in the search for excellence. Ultimately, the company’s associates and partners seek to elevate themselves, one another and the lives of patients and families around the world through gene therapy.

Trust

+
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

+
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

+
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 okay 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

+
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?

“For the first time in the history of our species, we are discussing not just effective treatments, but potentially total cures at the genetic level, which is the deepest essence of who we are as physical living beings.”

— Gaurav Shah, MD

Diversity, Equity and Inclusion at Rocket

Rocket celebrates diversity and our team is a testament to that. We foster an open environment where all ideas are welcome at the table, and every team member’s voice is valued.

Rocket is firmly committed to fostering a diverse workforce and advancing gender equality.

  • About half (47%) of the Rocket team identifies as female*
  • Over half of Rocket’s senior leadership team (64%) and employee base (48%) are BIPOC (Black, Indigenous and People of Color)*

Ensuring a diverse workforce and gender equality is something we take seriously here at Rocket. We’re committed to creating a collaborative space where differences are celebrated and unique perspectives are appreciated, united behind our mission of seeking gene therapy cures.

All team members are encouraged to embrace opportunities for learning and professional growth, regardless of experience or titles. We have a genuine commitment to lifelong learning and believe everyone, even the most accomplished among us, has room to grow.

Advocacy and Partnerships

Rocket cultivates trusted partnerships with patients and families, advocacy organizations, scientists, healthcare providers and health authorities to help guide us on our mission—and we execute on every commitment we make. 

Patients with rare diseases are our North Star—our constant reminder keeping us centered on our path seeking cures through gene therapy. Only by understanding their journey can we move forward in ours.

History

May 2024
may 2024 orphan medical product designation

Received Orphan Medicinal Product Designation from the European Commission for RP-A601 for PKP2-ACM

May 2024
May 2024 -ASGCT Annual Meeting

Rocket presents data from PKD, FA, and LAD-I programs at the American Society of Gene and Cell Therapy (ASGCT) Annual Meeting

April 2024

Rocket announces European Medicines Agency acceptance of RP-L102 Marketing Authorization Application for Fanconi Anemia

March 2024

Rocket Pharmaceuticals Announces Appointment of Aaron Ondrey as Chief Financial Officer and Additional Updates to Corporate Leadership Team

Leadership

Gaurav Shah, MD

Chief Executive Officer

Kinnari Patel, PharmD, MBA

Kinnari Patel, PharmD, MBA

President, Head of R&D and Chief Operating Officer

Jonathan Schwartz, MD

Chief Medical & Gene Therapy Officer

Aaron Ondrey

Aaron Ondrey

Chief Financial Officer

Isabel Carmona, JD

Isabel Carmona, JD

Chief People Officer

Carlos Martin

Chief Commercial Operations and Revenue Officer

Raj Prabhakar, MBA

Chief Business Officer

Mayo Pujols

Mayo Pujols

Chief Technical Officer

Gayatri R. Rao, MD, JD

Chief Regulatory Officer & SVP, Clinical Safety

Mark White, MB.ChB

General Manager, Commercial Affairs

Martin Wilson, JD

General Counsel & Chief Corporate Officer

Board of Directors

Roderick Wong, MD, MBA

Chairman of the Board

Elisabeth Björk, MD, PhD

Independent Director

Carsten Boess, MBA

Independent Director

Pedro Granadillo, BS

Independent Director

Gotham Makker, MD

Independent Director

Gaurav Shah, MD

Chief Executive Officer

David Southwell, MBA

Director

Naveen Yalamanchi, MD

Independent Director

Fady Malik, MD, PhD

Independent Director

R. Keith Woods

Independent Director

Mikael Dolsten, M.D, PhD

Independent Director

Scientific Advisors

Juan Bueren, PhD
Head of the Hematopoietic Innovative Therapies Division at CIEMAT and CIBERER

Hans-Peter Kiem, MD, PhD
Deputy Director and Professor, Translational Science and Therapeutics Division, Fred Hutch Cancer Center

Donald Kohn, MD, UUC, BS, MS
Director of the UCLA Human Gene and Cell Therapy Program

Axel Schambach, PhD
Professor and Director of the Institute of Experimental Hematology

Jose Carlos Segovia, PhD
Divisional Head, Cell Technology Division, CIEMAT

Matthew Taylor, MD, PhD
Clinical Genetics and Genomics, UCHealth University of Colorado Hospital

John Wagner, MD
Director of the Institute of Cell, Gene and Immunotherapeutics, University of Minnesota

Barry Byrne, MD, PhD
Associate Chair of Pediatrics, Director of the Powell Gene Therapy Center, University of Florida

Partners

Children’s Hospital of Philadelphia (CHOP)

1 of 13

Temple University

2 of 13

REGENXBIO

3 of 13

Memorial Sloan Kettering Cancer Center

4 of 13

NYU

5 of 13

CIRM

6 of 13

University of Minnesota

7 of 13

Stanford Medical School

8 of 13

Forty Seven, a Gilead company

9 of 13

University of Colorado

10 of 13

University of California, Los Angeles

11 of 13

University of California, San Diego

12 of 13

Fred Hutchinson Cancer Research Center

13 of 13

CIBER

CIBERER

CIEMAT

1 of 5

University College London Great Ormond Street Hospital for Children

2 of 5

Lund University

3 of 5

MolMed

4 of 5

University of Hannover

5 of 5
Scroll to Top