Welcome to our dedicated page for Rocket Pharmaceu news (Ticker: RCKT), a resource for investors and traders seeking the latest updates and insights on Rocket Pharmaceu stock.
Rocket Pharmaceu (RCKT) is a pioneering biotechnology company developing gene therapies for rare pediatric diseases through innovative lentiviral-based approaches. This page provides investors and industry observers with a comprehensive repository of official company announcements, clinical development updates, and strategic partnership news.
Access timely updates on RCKT's research milestones, regulatory progress, and financial performance. Our curated collection includes press releases related to clinical trial outcomes, manufacturing advancements, and collaborations with leading medical institutions. Users will find verified information on therapeutic developments targeting high-need genetic disorders.
Key areas covered include novel treatment pipelines, FDA designations, intellectual property updates, and scientific presentations. Bookmark this page to stay informed about Rocket Pharmaceu's contributions to gene therapy innovation and its evolving position in the biopharmaceutical sector.
Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) presented positive long-term data updates from its LV vector hematology portfolio at the ASGCT meeting. The data demonstrated sustained safety and efficacy in pivotal studies of KRESLADI™ for severe LAD-I, RP-L102 for Fanconi Anemia, and RP-L301 for PKD. KRESLADI™ showed 100% survival without HSCT, RP-L102 had genetic and phenotypic correction, and RP-L301 had clinically meaningful hemoglobin improvement. The safety profiles were well-tolerated with no drug-related serious adverse events.
Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) reported strong financial results for the first quarter of 2024. The company advanced its RP-L102 for Fanconi Anemia towards regulatory reviews, with an anticipated BLA submission in 2024. The EMA accepted the MAA for review. KRESLADI for severe LAD-I is on track for a PDUFA date of June 30, 2024. Aaron Ondrey was appointed as CFO. Rocket's cash, cash equivalents, and investments stood at $330.3M, providing operational runway into 2026.
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