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Rocket Pharmaceuticals
Rocket Pharmaceuticals (RCKT) investor relations material
Rocket Pharmaceuticals 44th Annual J.P. Morgan Healthcare Conference summary
Complete event summary combining all related documents: earnings call transcript, report, and slide presentation.44th Annual J.P. Morgan Healthcare Conference summary14 Jan, 2026
Strategic focus and pipeline prioritization
Refocused on cardiovascular gene therapies with clear biological rationale and regulatory paths, deprioritizing Fanconi anemia and PKD to concentrate resources on high-impact programs.
Three main cardiac programs: Danon disease, PKP2 arrhythmogenic cardiomyopathy (ACM), and BAG3 dilated cardiomyopathy (DCM), each targeting major genetic cardiomyopathy phenotypes.
In-house AAV manufacturing supports all disclosed programs, enabling cost control, process improvement, and scalability for future launches.
Wave two pipeline remains active but slowed to focus on highest value drivers; additional programs are ready to advance as resources allow.
Seeking partnerships for deprioritized programs to ensure patient access.
Financial position and capital strategy
Reported $222.8 million in cash as of September 30, 2025, sufficient to fund operations into Q2 2027, excluding potential PRV sale.
Potential Kresladi approval in March 2026 could yield a PRV, providing strategic non-dilutive capital and extending cash runway.
Commercial launch of Kresladi expected to build infrastructure for future AAV program launches.
Danon disease program updates
Danon disease gene therapy (RP-A501) shows durable LAMP2 expression and robust efficacy in Phase I, with patients thriving beyond typical survival ages.
Clinical hold in 2025 due to a safety event was resolved in under three months after protocol modifications and dose recalibration.
Phase II pivotal trial to resume with a staggered safety run-in at a lower dose, aiming to replicate Phase I efficacy and safety.
Natural history study validates rapid disease progression, supporting a small pivotal trial design.
Strong community interest persists despite prior safety events, with most males expected to be eligible for therapy.
What data supports Danon's recalibrated dose safety/efficacy?
PKP2-ACM registrational pathway and endpoint strategy?
Update on Fanconi/PKD deprioritization partner plans
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Next Rocket Pharmaceuticals earnings date
Q4 202527 Feb, 2026
Rocket Pharmaceuticals
Next Rocket Pharmaceuticals earnings date
Q4 202527 Feb, 2026
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Frequently asked questions
Rocket Pharmaceuticals Inc is a clinical-stage biotechnology company focused on developing gene therapies for rare and life-threatening genetic diseases. The company’s pipeline includes gene therapy programs targeting both hematologic and cardiovascular disorders. Rocket Pharmaceuticals uses lentiviral and adeno-associated virus (AAV) vectors to deliver corrective genes to patients' cells, aiming to provide long-term, potentially curative treatments. Its therapies are designed to address the underlying genetic causes of diseases, with a focus on disorders with limited treatment options. The company is headquartered in Cranbury, New Jersey, and its shares are listed on the NASDAQ.
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