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 (NASDAQ: RCKT) has received a Complete Response Letter (CRL) from the FDA regarding its Biologics License Application for KRESLADI™ (marnetegragene autotemcel; marne-cel), a gene therapy for severe leukocyte adhesion deficiency-I (LAD-I). The FDA has requested additional Chemistry Manufacturing and Controls (CMC) information. Rocket met with FDA leaders to expedite the approval process. The global Phase 1/2 study showed 100% overall survival at 12 months for all nine patients, with significant reductions in infections and improvements in LAD-I symptoms. KRESLADI™ was well tolerated with no serious adverse events reported.
Rocket Pharmaceuticals (NASDAQ: RCKT) has received orphan medicinal product designation from the European Commission for its gene therapy candidate, RP-A601, aimed at treating plakophilin-2 related arrhythmogenic cardiomyopathy (PKP2-ACM). This inherited heart disease affects around 50,000 individuals in the U.S. and Europe, leading to life-threatening arrhythmias and sudden cardiac death. Currently, there are no curative treatments available. The orphan designation provides various financial and regulatory benefits, including a 10-year market exclusivity post-approval. Rocket is conducting a Phase 1 trial to evaluate the safety and preliminary efficacy of RP-A601 in adult PKP2-ACM patients.
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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