FDA Clears Rocket Pharmaceuticals' IND for RP-A701 in BAG3-associated Dilated Cardiomyopathy Treatment

Rocket Pharmaceuticals, a prominent biotechnology firm, has reached a significant milestone in the development of RP-A701, a pioneering gene therapy targeting BAG3-associated dilated cardiomyopathy (BAG3-DCM). The U.S. Food and Drug Administration (FDA) has cleared Rocket's Investigational New Drug (IND) application for RP-A701. This groundbreaking therapy employs an AAVrh.74 vector to address a severe heart failure condition marked by progressive ventricular enlargement and compromised systolic function. The approval represents a crucial step for Rocket in its mission to deliver potentially curative gene therapies for rare cardiovascular disorders.

RP-A701 is designed to treat BAG3-DCM, a genetic heart disease that stems from mutations in the BAG3 gene. This condition leads to early onset and rapid progression of heart failure, resulting in significant morbidity and mortality. The BAG3 protein plays a vital role in several cellular functions, including maintaining cardiac contractility, protein quality control, and supporting cardiomyocyte structures. Mutations in the BAG3 gene disrupt these functions, causing the heart muscle to accumulate misfolded and damaged proteins, ultimately impairing cardiac function and leading to heart failure and premature death.

Rocket Pharmaceuticals is set to begin a Phase 1 clinical trial to test RP-A701. This initial trial will be a multi-center, dose-escalation study focusing on evaluating the safety, biological activity, and initial efficacy of RP-A701 in adult patients diagnosed with BAG3-DCM. The study will involve patients who already have implantable cardioverter defibrillators (ICDs) and are at an advanced stage of the disease, posing a high risk for further heart failure and potential cardiac death. Participants will receive a single dose of RP-A701, and researchers will closely monitor BAG3 protein expression levels, changes in cardiac biomarkers, and clinical predictors of disease progression.

The trial aims to provide hope for the estimated 30,000 individuals in the United States affected by BAG3-associated DCM. Currently, available medical treatments, including ICDs, cardiac resynchronization devices, and heart transplants, fail to consistently stop disease progression and often introduce their own complications, such as inappropriate ICD shocks. These interventions do not address the genetic root of the condition, highlighting a significant unmet medical need for patients with BAG3-DCM.

Rocket Pharmaceuticals is renowned for its innovative approach to addressing rare genetic disorders. In addition to RP-A701, its adeno-associated viral (AAV) vector-based cardiovascular portfolio includes advanced clinical programs for Danon Disease, a heart failure condition characterized by heart thickening, and early-stage efforts targeting PKP2-arrhythmogenic cardiomyopathy (ACM), which can lead to life-threatening arrhythmias and sudden cardiac death.

Rocket’s lentiviral (LV) vector-based hematology portfolio comprises late-stage clinical trials for several challenging genetic conditions. These include Fanconi Anemia (FA), a genetic disorder that causes bone marrow failure and potential cancer, Leukocyte Adhesion Deficiency-I (LAD-I), a pediatric disorder causing recurrent, life-threatening infections, and Pyruvate Kinase Deficiency (PKD), a blood disorder resulting in increased red cell destruction and varying degrees of anemia.

Rocket Pharmaceuticals remains committed to advancing its pipeline and developing transformative therapies that address the underlying causes of complex and rare medical conditions, offering patients the possibility of a more hopeful future.