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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, Inc. (NASDAQ: RCKT) announced that CEO Gaurav Shah will participate in a fireside chat at the Stifel 2021 Virtual Healthcare Conference on November 16, 2021, at 2:40 p.m. ET. A live audio webcast will be available on the company's investor website, with a replay following the conference. Rocket is focused on developing genetic therapies for rare childhood disorders, targeting conditions like Fanconi Anemia and Danon Disease. The company emphasizes its integrated pipeline and platform-agnostic approach to addressing complex medical needs in pediatric patients.
Rocket Pharmaceuticals (NASDAQ: RCKT) announced updated clinical data from its ongoing Phase 1 trial of RP-A501 for Danon Disease, set to be presented at the AHA Scientific Sessions from November 13-15, 2021. The trial aims to evaluate the gene therapy's safety and efficacy, with preliminary results indicating it is generally well tolerated and shows improved cardiac function. Danon Disease, a rare genetic disorder, currently lacks specific treatment options, further emphasizing the potential significance of RP-A501 for patients.
Rocket Pharmaceuticals (NASDAQ: RCKT) announces upcoming presentations at the 63rd American Society of Hematology Annual Meeting from December 11-14, 2021. The presentations will feature updated clinical data from three gene therapy programs targeting Pyruvate Kinase Deficiency (PKD), Leukocyte Adhesion Deficiency-I (LAD-I), and Fanconi Anemia (FA). Highlights include an oral presentation on the Phase 1 study of RP-L301 for PKD and two poster presentations on RP-L201 for LAD-I and RP-L102 for FA, showcasing interim results and addressing severe unmet medical needs.
Rocket Pharmaceuticals (NASDAQ: RCKT) reported Q3 2021 financial results with a net loss of $50.1 million or $0.79 per share, up from $29.1 million or $0.53 per share in Q3 2020. The company holds $421.5 million in cash, expected to fund operations into 2H 2023. Treatment in the Danon Disease trial commenced for pediatric patients, while the LAD-I trial reported positive interim results with favorable safety and efficacy in all seven patients. Comprehensive clinical updates are anticipated at upcoming AHA and ASH meetings.
Frazier Healthcare Partners announced the closing of Frazier Life Sciences Public Fund, raising nearly
Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) announces CEO Gaurav Shah will present at the 2021 Cell & Gene Meeting on the Mesa in Carlsbad, CA, on Tuesday, October 12, at 2:15 p.m. PST. The presentation will be available via a live audio webcast for registered attendees, with a recorded version accessible within 24 hours. Rocket focuses on genetic therapies for rare childhood disorders, with programs targeting conditions such as Fanconi Anemia, Leukocyte Adhesion Deficiency-I, and Infantile Malignant Osteopetrosis, utilizing both lentiviral vector and adeno-associated virus technologies.
Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) announced participation in the Chardan Virtual 5th Annual Genetic Medicines Conference. CEO Gaurav Shah will join a fireside chat on October 5, 2021, at 10:30 am ET, while CMO Jonathan Schwartz will discuss genetic medicines on a panel on October 4, 2021, at 3:00 pm ET. The event's webcast will be available on Rocket's website, with an archive accessible for 30 days.
Rocket focuses on genetic therapies for rare childhood disorders, including Fanconi Anemia and Danon disease.
Rocket Pharmaceuticals (NASDAQ: RCKT) presented positive data from its ongoing Phase 1 trial of RP-A501 for Danon Disease at the HFSA Annual Scientific Meeting 2021. The low-dose cohort (6.7e13 vg/kg) demonstrated that RP-A501 was well tolerated, with all patients showing clinical improvement, sustained cardiac expression, and a manageable safety profile. Rocket plans to initiate treatment in a pediatric cohort in Q3 and expects comprehensive updates in Q4.
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