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.

RARE DISEASE DAY 2020

I AM RARE, HEAR ME ROAR

Rocket Pharma was proud to commemorate Rare Disease Day in New York City at “I am Rare, Hear me Roar,” an inspiring patient-centered event at the Weill Music Room in iconic Carnegie Hall. Ushering in 2020, we hope to channel the Roaring ‘20s, a decade that saw the discovery of ground-breaking medicines. Over 200 members of the rare disease and broader New York City biotech communities came together to learn firsthand the impact of rare diseases on patients and family members and how to partner in the fight to find new treatments. The event was capped with the lighting of the Empire State Building in Rare Disease Day colors to honor those affected by rare diseases and stand in solidarity with the rare disease community.

Rocket Pharma Rare Disease Day 2020
Rocket Pharma Rare Disease Day 2020
Rocket Pharma Rare Disease Day 2020
Rocket Pharma Rare Disease Day 2020
Rocket Pharma Rare Disease Day 2020
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Rare Disease Day 2020 Video Presentation

Welcome
Stephanie Sirota – Chief Business Officer, RTW Investments

Introduction to Rare Disease Day Part 1
Kinnari Patel, PharmD – COO & EVP, Development, Rocket Pharma

Introduction to Rare Disease Day Part 2
Kinnari Patel, PharmD – COO & EVP, Development, Rocket Pharma

Introduction to Congresswoman Carolyn Maloney
Dr. Gayatri Rao – VP, Rocket Pharma

Remarks by Congresswoman Carolyn Maloney
U.S. Representative for New York’s 12th Congressional District

Diagnostic Journey & Impact of a Rare Disease Diagnosis Part 1
Marlene Botta
Ashlin Veselka

Diagnostic Journey & Impact of a Rare Disease Diagnosis Part 2
Marlene Botta
Ashlin Veselka

Diagnostic Journey & Impact of a Rare Disease Diagnosis Part 3
Marlene Botta
Ashlin Veselka

Diagnostic Journey & Impact of a Rare Disease Diagnosis Part 4
Marlene Botta
Ashlin Veselka

Diagnostic Journey & Impact of a Rare Disease Diagnosis Part 5
Marlene Botta
Ashlin Veselka

Book Reading from “Structured Love” Part 1
Allie M. Jones

Book Reading from “Structured Love” Part 2
Allie M. Jones

The Promise of Gene Therapy Part 1
Marley Gaskins & Tamara Hogue
Dr. Don Kohn – Professor of MIMG, Pediatrics and MMP at UCLA and Principal Investigator (LAD-I)

The Promise of Gene Therapy Part 2
Marley Gaskins & Tamara Hogue
Dr. Don Kohn – Professor of MIMG, Pediatrics and MMP at UCLA and Principal Investigator (LAD-I)

The Promise of Gene Therapy Part 3
Marley Gaskins & Tamara Hogue
Dr. Don Kohn – Professor of MIMG, Pediatrics and MMP at UCLA and Principal Investigator (LAD-I)

The Promise of Gene Therapy Part 4
Marley Gaskins & Tamara Hogue
Dr. Don Kohn – Professor of MIMG, Pediatrics and MMP at UCLA and Principal Investigator (LAD-I)

Embracing the Rare
Part 1
Jack Timperley

Embracing the Rare
Part 2
Jack Timperley

Call to Action
Part 1
Dr. John Wagner – Director, Institute for Cell, Gene and Immunotherapeutics, University of Minnesota and Scientific Advisor (Fanconi Anemia)

Call to Action
Part 2
Dr. John Wagner – Director, Institute for Cell, Gene and Immunotherapeutics, University of Minnesota and Scientific Advisor (Fanconi Anemia)

Call to Action
Part 3
Dr. John Wagner – Director, Institute for Cell, Gene and Immunotherapeutics, University of Minnesota and Scientific Advisor (Fanconi Anemia)

Closing Remarks
Dr. Roderick Wong – Chairman, Rocket Pharma Managing Partner, RTW Investments

Introduction of BioMusica and Opening of the Reception
Part 1
Dr. Gaurav Shah – CEO and President, Rocket Pharma

Introduction of BioMusica and Opening of the Reception
Part 2
Kinnari Patel, PharmD – COO & EVP, Development, Rocket Pharma

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