RP-A701 is a first-in-class gene therapy for the treatment of BAG3-associated dilated cardiomyopathy
BAG3-associated dilated cardiomyopathy is a rare, inherited heart condition caused by mutations in the BAG3 gene, leading to early-onset, progressive heart failure due to impaired cardiac function, high morbidity, and premature mortality
Rocket plans to conduct a Phase 1 trial in the U.S. and is working towards the first patient treatment
CRANBURY, NJ, USA I June 30, 2025 I Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a fully integrated, late-stage biotechnology company advancing a sustainable pipeline of genetic therapies for rare disorders with high unmet need, today announced that it has received clearance from the U.S. Food and Drug Administration (FDA) for the Company’s Investigational New Drug (IND) application for RP-A701, an AAVrh.74-based gene therapy candidate for the treatment of BAG3-associated Dilated Cardiomyopathy (BAG3-DCM), a severe form of heart failure characterized by progressive ventricular enlargement and impaired systolic function.
“The FDA clearance of RP-A701, our third clinical-stage gene therapy candidate from our AAV cardiovascular portfolio, is an important milestone for Rocket,” said Kinnari Patel, PharmD, MBA, President, Head of R&D and Chief Operating Officer of Rocket Pharma. “With programs in the clinic for each of the major types of genetic cardiomyopathies – hypertrophic, dilated, and arrhythmogenic – we are advancing our mission to bring potentially curative gene therapies to patients with rare and life-threatening cardiovascular diseases. Phase 1 trial start-up activities are currently underway for RP-A701, and we are working towards treating the first patient.”
The first-in-human Phase 1 clinical trial will be a multi-center, dose-escalation study designed to evaluate the safety, biological activity, and preliminary efficacy of RP-A701 in adults with BAG3-DCM. Initial study participants will include adults with implantable cardioverter defibrillators (ICDs) and advanced disease at high risk for heart failure progression and cardiac death. Participants will receive a single dose of RP-A701, and the trial will assess BAG3 protein expression, changes in cardiac biomarkers, and clinical predictors of disease progression.
About BAG3-associated Dilated Cardiomyopathy (DCM)
BAG3-DCM is an inherited heart disease caused by mutations in the BAG3 gene (Bcl2-associated athanogene 3) resulting in early onset, rapidly progressing heart failure, and significant morbidity and mortality. The BAG3 protein contributes to multiple cellular functions, including cardiac contractility, protein quality control (as a co-chaperone), cardiomyocyte structural support, and regulation of autophagy and apoptosis. Loss of BAG3 leads to an accumulation of misfolded and damaged proteins, which can impair the heart’s ability to contract, leading to impaired cardiac function, heart failure, and even premature death. We estimate that the prevalence of BAG3-associated DCM in the U.S. is as many as 30,000 individuals. Patients living with BAG3-DCM have an urgent unmet medical need, as current medical and interventional therapies (including implantable cardioverter defibrillator [ICD], cardiac resynchronization devices, and heart transplant) do not consistently prevent disease progression, are associated with significant morbidity including inappropriate ICD shocks and device, procedure, and transplant-related complications, and do not address the underlying pathophysiology or genetic mutation.
About Rocket Pharmaceuticals, Inc.
Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) is a fully integrated, late-stage biotechnology company advancing a sustainable pipeline of investigational genetic therapies designed to correct the root cause of complex and rare disorders. Rocket’s innovative multi-platform approach allows us to design the optimal gene therapy for each indication, creating potentially transformative options that enable people living with devastating rare diseases to experience long and full lives.
Rocket’s adeno-associated viral (AAV) vector-based cardiovascular portfolio includes a late-stage clinical program for Danon Disease, a devastating heart failure condition resulting in thickening of the heart, and an early-stage clinical program for PKP2-arrhythmogenic cardiomyopathy (ACM), a life-threatening heart failure disease causing ventricular arrhythmias and sudden cardiac death. Rocket has also received IND clearance for its AAV-based gene therapy for BAG3-associated dilated cardiomyopathy (DCM), a heart failure condition that causes enlarged ventricles.
Rocket’s lentiviral (LV) vector-based hematology portfolio consists of late-stage clinical programs for Fanconi Anemia (FA), a difficult-to-treat genetic disease that leads to bone marrow failure (BMF) and potentially cancer, Leukocyte Adhesion Deficiency-I (LAD-I), a severe pediatric genetic disorder that causes recurrent and life-threatening infections which are frequently fatal, and Pyruvate Kinase Deficiency (PKD), a monogenic red blood cell disorder resulting in increased red cell destruction and mild to life-threatening anemia.
For more information about Rocket, please visit www.rocketpharma.com and follow us on LinkedIn, YouTube, and X.
SOURCE: Rocket Pharmaceuticals
Post Views: 1,395
RP-A701 is a first-in-class gene therapy for the treatment of BAG3-associated dilated cardiomyopathy
BAG3-associated dilated cardiomyopathy is a rare, inherited heart condition caused by mutations in the BAG3 gene, leading to early-onset, progressive heart failure due to impaired cardiac function, high morbidity, and premature mortality
Rocket plans to conduct a Phase 1 trial in the U.S. and is working towards the first patient treatment
CRANBURY, NJ, USA I June 30, 2025 I Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a fully integrated, late-stage biotechnology company advancing a sustainable pipeline of genetic therapies for rare disorders with high unmet need, today announced that it has received clearance from the U.S. Food and Drug Administration (FDA) for the Company’s Investigational New Drug (IND) application for RP-A701, an AAVrh.74-based gene therapy candidate for the treatment of BAG3-associated Dilated Cardiomyopathy (BAG3-DCM), a severe form of heart failure characterized by progressive ventricular enlargement and impaired systolic function.
“The FDA clearance of RP-A701, our third clinical-stage gene therapy candidate from our AAV cardiovascular portfolio, is an important milestone for Rocket,” said Kinnari Patel, PharmD, MBA, President, Head of R&D and Chief Operating Officer of Rocket Pharma. “With programs in the clinic for each of the major types of genetic cardiomyopathies – hypertrophic, dilated, and arrhythmogenic – we are advancing our mission to bring potentially curative gene therapies to patients with rare and life-threatening cardiovascular diseases. Phase 1 trial start-up activities are currently underway for RP-A701, and we are working towards treating the first patient.”
The first-in-human Phase 1 clinical trial will be a multi-center, dose-escalation study designed to evaluate the safety, biological activity, and preliminary efficacy of RP-A701 in adults with BAG3-DCM. Initial study participants will include adults with implantable cardioverter defibrillators (ICDs) and advanced disease at high risk for heart failure progression and cardiac death. Participants will receive a single dose of RP-A701, and the trial will assess BAG3 protein expression, changes in cardiac biomarkers, and clinical predictors of disease progression.
About BAG3-associated Dilated Cardiomyopathy (DCM)
BAG3-DCM is an inherited heart disease caused by mutations in the BAG3 gene (Bcl2-associated athanogene 3) resulting in early onset, rapidly progressing heart failure, and significant morbidity and mortality. The BAG3 protein contributes to multiple cellular functions, including cardiac contractility, protein quality control (as a co-chaperone), cardiomyocyte structural support, and regulation of autophagy and apoptosis. Loss of BAG3 leads to an accumulation of misfolded and damaged proteins, which can impair the heart’s ability to contract, leading to impaired cardiac function, heart failure, and even premature death. We estimate that the prevalence of BAG3-associated DCM in the U.S. is as many as 30,000 individuals. Patients living with BAG3-DCM have an urgent unmet medical need, as current medical and interventional therapies (including implantable cardioverter defibrillator [ICD], cardiac resynchronization devices, and heart transplant) do not consistently prevent disease progression, are associated with significant morbidity including inappropriate ICD shocks and device, procedure, and transplant-related complications, and do not address the underlying pathophysiology or genetic mutation.
About Rocket Pharmaceuticals, Inc.
Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) is a fully integrated, late-stage biotechnology company advancing a sustainable pipeline of investigational genetic therapies designed to correct the root cause of complex and rare disorders. Rocket’s innovative multi-platform approach allows us to design the optimal gene therapy for each indication, creating potentially transformative options that enable people living with devastating rare diseases to experience long and full lives.
Rocket’s adeno-associated viral (AAV) vector-based cardiovascular portfolio includes a late-stage clinical program for Danon Disease, a devastating heart failure condition resulting in thickening of the heart, and an early-stage clinical program for PKP2-arrhythmogenic cardiomyopathy (ACM), a life-threatening heart failure disease causing ventricular arrhythmias and sudden cardiac death. Rocket has also received IND clearance for its AAV-based gene therapy for BAG3-associated dilated cardiomyopathy (DCM), a heart failure condition that causes enlarged ventricles.
Rocket’s lentiviral (LV) vector-based hematology portfolio consists of late-stage clinical programs for Fanconi Anemia (FA), a difficult-to-treat genetic disease that leads to bone marrow failure (BMF) and potentially cancer, Leukocyte Adhesion Deficiency-I (LAD-I), a severe pediatric genetic disorder that causes recurrent and life-threatening infections which are frequently fatal, and Pyruvate Kinase Deficiency (PKD), a monogenic red blood cell disorder resulting in increased red cell destruction and mild to life-threatening anemia.
For more information about Rocket, please visit www.rocketpharma.com and follow us on LinkedIn, YouTube, and X.
SOURCE: Rocket Pharmaceuticals
Post Views: 1,395