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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) announced key advancements in its gene therapy pipeline including RP-A601 for treating arrhythmogenic cardiomyopathy (PKP2-ACM), with an IND submission anticipated in Q2 2023. The company reported promising Phase 1 results for Danon Disease, showing significant clinical improvements and biomarker stability. The expected BLA filings for Fanconi Anemia and Leukocyte Adhesion Deficiency are scheduled for Q2 and Q4 2023, respectively. Rocket is well-capitalized with $401 million in cash, ensuring operational funding through 2024.
Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) is set to present at the 41st Annual J.P. Morgan Healthcare Conference on January 9, 2023, at 2:15 p.m. PT. CEO Gaurav Shah will represent the company, known for its innovative approach to developing genetic therapies aimed at rare childhood disorders. The live audio webcast will be accessible under the Investors section of the company's website, with a replay available post-conference. Rocket's pipeline includes therapies for Fanconi Anemia, Leukocyte Adhesion Deficiency-I, Pyruvate Kinase Deficiency, and Danon Disease.
Rocket Pharmaceuticals (NASDAQ: RCKT) announced updates regarding its gene therapy, RP-A501, for Danon Disease following an end-of-Phase 1 meeting with the FDA. The company plans to advance with a pivotal trial utilizing a biomarker-based composite endpoint, aiming to initiate the study in 1H 2023. The Phase 1 trial showed favorable results with consistent improvements in biomarkers and quality of life across both pediatric and adult patients. The therapy is designed to address a significant unmet medical need in a condition with no current targeted treatments.
Rocket Pharmaceuticals (NASDAQ: RCKT) announced promising updates on its lentiviral gene therapy programs at the American Society of Hematology (ASH) Annual Meeting, with both RP-L102 for Fanconi Anemia and RP-L301 for Pyruvate Kinase Deficiency showing significant success. Six out of ten evaluated FA patients demonstrated sustained genetic correction and hematologic stabilization after 12-36 months. Furthermore, both patients treated with RP-L301 exhibited normalized hemoglobin levels at 24 months, ensuring an improved quality of life. Regulatory filings are anticipated in 2023.
Rocket Pharmaceuticals, Inc. has announced the acquisition of Renovacor, Inc., aimed at enhancing its leadership in AAV-based cardiac gene therapy. Under the merger terms established on September 19, 2022, Renovacor shareholders received 0.1763 shares of Rocket for each Renovacor share. The acquisition adds Renovacor's leading program, REN-001, targeting BAG3-associated dilated cardiomyopathy, which has no current treatments addressing its underlying causes, to Rocket's pipeline. Renovacor shares ceased trading on the NYSE as of December 1, 2022.
Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) reported strong progress in Q3 2022, particularly in its Phase 1 trial for Danon Disease, where RP-A501 showed durable therapeutic effects. The company anticipates FDA feedback for its Phase 2 pivotal study design by Q4 2022. Additionally, Rocket plans to acquire Renovacor to enhance its AAV-based cardiac gene therapy capabilities. With $306.5M in cash and recent equity financing boosting its runway into H2 2024, the company is well-positioned for ongoing clinical trials and regulatory filings in 2023.
Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) announced data presentations at the upcoming 75th American Heart Association Annual Meeting (Nov 5-7, 2022) and the 64th American Society of Hematology Annual Meeting (Dec 10-13, 2022). Data will focus on its gene therapies for rare diseases, including RP-A501 for Danon Disease and RP-L102 for Fanconi Anemia. Presenters include experts from renowned institutions. The company aims to address high unmet medical needs in pediatric populations with genetic disorders.
Rocket Pharmaceuticals (NASDAQ: RCKT) announced that Chief Business Officer Raj Prabhakar will present at the 2022 Cell & Gene Meeting on October 11 at 2:30 p.m. PT in Carlsbad, CA. The presentation aims to highlight the company's pipeline of genetic therapies targeting rare childhood disorders. A live audio webcast will be available for registered attendees, with on-demand viewing post-event. Rocket focuses on innovative treatments for conditions like Fanconi Anemia and Danon Disease, showcasing its commitment to addressing unmet medical needs.
Rocket Pharmaceuticals (NASDAQ: RCKT) has announced the pricing of a public offering of 6,800,000 shares at $14.75 each, aiming to raise approximately