Welcome to our dedicated page for Rocket Pharmaceuticals news (Ticker: RCKT), a resource for investors and traders seeking the latest updates and insights on Rocket Pharmaceuticals stock.
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) has released preliminary data from its Phase 1 clinical trial of RP-A501, a gene therapy targeting Danon Disease. The therapy was well tolerated in the low dose cohort, showing early signs of clinical benefit, including increased gene expression and improved biomarkers for cardiac function. The trial reported manageable safety profiles, with no severe adverse events noted. Additionally, the preliminary results indicate potential for RP-A501 to offer significant therapeutic benefits for patients suffering from this rare genetic disorder.
Rocket Pharmaceuticals (NASDAQ: RCKT) presented updated interim data on its gene therapies for Fanconi Anemia (FA) and Leukocyte Adhesion Deficiency-I (LAD-I) at the 62nd ASH Annual Meeting. The results indicate promising efficacy and durability, particularly with RP-L102 for FA, showing engraftment in 5 out of 7 patients, and stabilization of blood counts in those with 12-month follow-ups. For LAD-I, RP-L201 demonstrated sustained CD18 expression between 23%-40%, exceeding survival thresholds. The company aims to progress these therapies towards potential registration.
Rocket Pharmaceuticals (NASDAQ: RCKT) presented preliminary data from its Phase 1 trial of RP-L301 for Pyruvate Kinase Deficiency (PKD) at the ASH Annual Meeting. The results indicated a significant increase in hemoglobin levels from 7.4 g/dL to 14.3 g/dL after three months of treatment. The therapy achieved transfusion independence for the first patient, demonstrating a promising safety profile with no serious complications. The company aims to provide longer-term data in the second half of 2021, focusing on transforming PKD management.
Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) has announced a webcast scheduled for December 7, 2020, at 6:00 PM EST. The event will discuss key presentations from the 62nd American Society of Hematology (ASH) Annual Meeting, focusing on data related to the Fanconi Anemia, Leukocyte Adhesion Deficiency-I, and Pyruvate Kinase Deficiency programs. Following the presentation, management and key opinion leaders will hold a Q&A session. Investors can access the webcast through Rocket's website.
Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) announces its participation in two upcoming healthcare conferences. The Piper Sandler 32nd Annual Virtual Healthcare Conference will take place on December 1, 2020. Additionally, Gaurav Shah, M.D., President and CEO, will participate in a fireside chat at the Evercore ISI 3rd Annual HealthCONx Conference on December 2, 2020, at 3:30 p.m. ET. Both events will feature live audio webcasts available on the company’s website, www.rocketpharma.com, with replays accessible post-conference.
Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) received a $3.7 million CLIN2 grant from the California Institute for Regenerative Medicine to advance its gene therapy, RP-L401, aimed at treating Infantile Malignant Osteopetrosis (IMO), a severe disorder with an incidence of 1 in 200,000. The funding will support clinical trial costs for a Phase 1 trial at UCLA, which evaluates safety and efficacy in pediatric patients. RP-L401 has Fast Track designation from the FDA and represents a potential curative option beyond current treatments like bone marrow transplants.
Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) announced two oral presentations and one poster presentation at the 62nd American Society of Hematology Annual Meeting, scheduled for December 5-8, 2020. The oral presentations will focus on clinical data for Fanconi Anemia and Leukocyte Adhesion Deficiency-I, while the poster will present preliminary data on Pyruvate Kinase Deficiency. These programs leverage Rocket's "Process B" manufacturing platform, and aim to address critical therapies for rare childhood disorders.
Rocket Pharmaceuticals (NASDAQ: RCKT) reported its Q3 2020 financial results, highlighting advancements across its gene therapy pipeline for rare childhood disorders. The company noted positive data from the RP-L201 trial for LAD-I and initiated a higher dose trial of RP-A501 for Danon Disease. Despite challenges posed by the pandemic, patient enrollment continued. Rocket's cash position stood at $228.7 million, expecting to fund operations through Q2 2022, yet reported a net loss of $29 million or $0.53 per share.
Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) presented promising clinical data at the ESID 2020 Meeting for its gene therapy candidates RP-L201 and RP-L401. The Phase 1/2 trial of RP-L201 for Leukocyte Adhesion Deficiency-I (LAD-I) showed significant therapeutic benefits, with patients achieving CD18 expression levels well above survival thresholds. RP-L401 for Infantile Malignant Osteopetrosis (IMO) also demonstrated preclinical efficacy, supporting its progression to a Phase 1 trial. These advancements indicate Rocket's potential to address critical unmet medical needs in severe pediatric disorders.
Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) announced two presentations at the European Society for Immunodeficiencies (ESID) 2020 meeting, scheduled from October 14-17, 2020. The oral presentation will update data from its Phase 1/2 trial of RP-L201 for Leukocyte Adhesion Deficiency-I (LAD-I), while an e-poster will present preclinical results for RP-L401 targeting Infantile Malignant Osteopetrosis (IMO). Both presentations highlight Rocket’s commitment to addressing significant medical needs in rare pediatric diseases.
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