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Rocket Pharmaceuticals, Inc. (symbol: RCKT) is a pioneering biotechnology company dedicated to advancing gene therapy treatments for rare and orphan diseases. The company's mission is to seek cures through molecular and gene therapy, leveraging a lentiviral-based approach to deliver groundbreaking medical advancements.
Core Business: The company focuses on developing first-in-class treatments aimed at addressing significant unmet medical needs. Rocket Pharmaceuticals has a robust pipeline of gene therapy products, including:
- RP-A501 for Danon Disease
- RP-L102 for Fanconi Anemia
- RP-L201 for Leukocyte Adhesion
- RP-L301 for Pyruvate Kinase Deficiency
- RP-L401 for Infantile Malignant Osteopetrosis
These pipeline products are in various stages of clinical development, showcasing the company's commitment to innovative solutions for devastating pediatric diseases.
Partnerships and Collaborations: Rocket Pharmaceuticals has built rewarding partnerships with leading international research institutions and centers of manufacturing and cell processing expertise. These collaborations enhance the company's capacity to conduct rigorous research and development, clinical trials, and regulatory programs.
Financial Condition and Achievements: As a late-stage biopharmaceutical company, Rocket Pharmaceuticals is well-positioned in the market, with strong backing from investors and a promising future outlook. The company’s recent achievements include significant progress in clinical trials and obtaining key regulatory milestones.
Current Projects: The company is actively working on several projects aimed at bringing innovative gene therapies to market. These projects include ongoing clinical trials for their pipeline products and efforts to expand their research and development capabilities.
Looking Forward: Rocket Pharmaceuticals aims to launch and globally commercialize its gene therapies to bring hope and relief to patients suffering from rare diseases. The company's approach combines high scientific standards with a patient-centric focus, driving its mission to transform the landscape of gene therapy.
For more information, please visit www.rocketpharma.com.
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
Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) has initiated an underwritten public offering of $100 million in common stock, with a potential additional $15 million option for underwriters. The offering will be managed by Morgan Stanley, J.P. Morgan, and SVB Securities. This offering is subject to market conditions, and all shares will be sold by Rocket. The process follows a shelf registration statement previously filed with the SEC. The final terms will be disclosed in a subsequent prospectus supplement.
Rocket Pharmaceuticals (RCKT) announced positive updates from its Phase 1 Danon Disease Trial for RP-A501, showing initial improvements in pediatric patients and sustained clinical benefits in adults. The trial includes both safety and efficacy data, indicating that RP-A501 is generally well tolerated across cohorts. Notably, patients exhibited significant reductions in heart failure markers like BNP and improvements in NYHA class. The company anticipates support for advancing to a Phase 2 pivotal study with FDA feedback expected later this year.
Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a clinical biotech firm focused on genetic therapies for rare childhood disorders, will have CEO Gaurav Shah participate in a fireside chat at the Chardan 6th Annual Genetic Medicines Conference on October 4 at 2:00 p.m. ET, in New York City. The event will be accessible via a live audio webcast on the company's website, with a replay available later. Rocket is advancing therapies for conditions like Fanconi Anemia, Leukocyte Adhesion Deficiency-I, Pyruvate Kinase Deficiency, and Danon Disease.
Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) has announced the acquisition of Renovacor, Inc. (NYSE: RCOR) in an all-stock transaction valued at approximately $2.60 per share. This acquisition aims to enhance Rocket's leadership in AAV-based cardiac gene therapy, targeting BAG3-associated dilated cardiomyopathy, which presents a significant unmet medical need. The deal is expected to close by Q1 2023, pending shareholder and regulatory approvals. Rocket anticipates the acquisition will create strong synergies, adding approximately $38M in projected cash at closing and extending its cash runway into Q2 2024.
Rocket Pharmaceuticals is set to present updated data from its Phase 1 Danon Disease trial on September 30, 2022, at the Heart Failure Society of America Annual Scientific Meeting. The presentation will reveal efficacy and safety updates from both pediatric and adult cohorts. An investor event discussing this data will stream at 8:00 a.m. ET. The trial aims to address the serious pediatric heart condition, Danon Disease, utilizing gene therapy.
Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) announced that CEO Gaurav Shah will participate in a fireside chat at the Morgan Stanley 20th Annual Global Healthcare Conference on Monday, Sept. 13, at 3:30 p.m. ET. A live audio webcast will be available on the company’s Investors section of their website. Rocket focuses on developing genetic therapies for rare childhood disorders, including Fanconi Anemia, Leukocyte Adhesion Deficiency-I, and Pyruvate Kinase Deficiency. For more information, visit www.rocketpharma.com.
Rocket Pharmaceuticals (NASDAQ: RCKT) reported positive safety data from its Phase 1 Danon Disease study, indicating RP-A501 was well-tolerated in pediatric patients. They announced 100% overall survival in a pivotal Phase 2 trial for severe LAD-I, with regulatory filings expected in early 2023. The primary endpoint for the Fanconi Anemia trial was met, initiating FDA dialogue for BLA planning. The company reported a cash position of $321.4 million, ensuring operational runway into H1 2024. The appointment of Mayo Pujols as Chief Technical Officer bolsters its leadership in gene therapy.
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