What is Rocket Pharma?
Rocket Pharma is a gene therapy company listed on NASDAQ (RCKT), focusing on developing treatments for bone marrow-derived genetic diseases and cardiac conditions. Our mission is to harness the power of gene therapy to address these serious health challenges, aiming to provide transformative treatments for patients in need.
How disruptive are the diseases you target to those affected, particularly in relation to the launch of KRESLADI?
Leukocyte adhesion deficiency type 1 (LAD-1) is a devastating genetic disorder affecting neutrophils, leading to severe infections, frequent hospitalization, and early childhood mortality. Our gene therapy approach aims to correct the genetic mutation at its root, aiming to transform the lives of patients by restoring normal neutrophil function. This not only significantly reduces the need for hospital visits but also extends the potential lifespan of affected children , offering hope where previously there was little.
How do you choose therapeutic areas for your gene therapy treatments?
Our therapeutic focus areas are derived from strategic selection based on unmet medical needs, scientific feasibility, and potential for clinical and commercial success. For bone marrow-derived diseases, we utilize ex vivo lentiviral vectors , while for cardiac and other conditions, we use in vivo AAV vectors. Our R&D process involves a thorough evaluation of diseases where gene therapy could offer a significant benefit, focusing on those with a clear path to making a meaningful impact on patients’ lives. This approach has resulted in six disclosed programs across our world-class pipeline.
What potential do you see for AAV-based gene therapy in treating pediatric heart failures, such as Danon Disease?
AAV-based gene therapy has shown promising results in treating Danon Disease, one of the most aggressive cardiomyopathies.
Our therapy has enabled patients, who otherwise faced poor prognosis, to return to what we believe are normal activities, highlighting the potential of gene therapy to address not just Danon Disease but also other genetic cardiac conditions.
This success opens the door for further innovations in treating cardiovascular diseases at the genetic level. Rocket is also researching a gene therapy for PKP2 arrhythmogenic cardiomyopathy (ACM) in a Phase 1 clinical study and is working towards bringing an asset for BAG3-associated dilated cardiomyopathy (DCM) into clinical trials. Taken together, Rocket’s AAV cardiovascular gene therapy portfolio has the potential to impact more than 100,000 patients in the U.S. and EU.
How will the launch of KRESLADI be managed, especially given its targeted patient population?
The anticipated launch of KRESLADITM (marnetegragene autotemcel) for severe LAD-I will be focused on providing access to treatment for a relatively small, but severely affected patient population. KRESLADI is under Priority Review with the FDA, with a PDUFA date of June 30, 2024. Regulatory discussions are underway to finalize approval details, with a commercial strategy centered around medical education and facilitating access rather than extensive marketing. Treatment centers in the U.S. will be identified to serve patients, emphasizing a streamlined patient journey from diagnosis to treatment.
What are your thoughts on the evolution of gene therapy and the role of AI in advancing treatments?
Gene therapy is poised to address a wide range of genetic diseases, initially focusing on monogenic disorders before tackling more complex conditions. The integration of AI and data analysis will be crucial in understanding and developing treatments for multigenic diseases. While AI will enhance our ability to decipher complex genetic interactions and optimize therapeutic vectors, the inherent unpredictability of human biology underscores the importance of clinical trials and a holistic approach to drug development.
How do you balance the urgency of developing treatments with the inherent challenges of drug development?
Balancing urgency with the meticulous nature of drug development requires a multifaceted approach, including cost-consciousness, platform innovation, and regulatory collaboration. The FDA's support through initiatives like the Priority Review Voucher and a focus on platform approaches helps streamline development for rare diseases. Moreover, building relationships with investors who understand the long-term vision and challenges of gene therapy is crucial for sustaining progress.
What qualities are essential for a CEO in the biotech industry during this transformative period for gene therapy?
Leading a biotech company through the evolution of gene therapy requires curiosity, humility, a sense of urgency, and the ability to foster deep, meaningful collaborations with the medical community and patient advocates. Balancing the excitement of pioneering treatments with the practicalities of biotech development demands both a visionary outlook and a pragmatic approach to overcoming the unique challenges of this field.
