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Rocket Pharmaceu Stock Price, News & Analysis

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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.

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Rocket Pharmaceuticals (NASDAQ: RCKT) announced a significant setback in its Phase 2 pivotal trial of RP-A501, its gene therapy for Danon disease. A patient experienced a serious adverse event (SAE) involving capillary leak syndrome and subsequently passed away due to an acute systemic infection. The FDA has placed a clinical hold on the trial as of May 23, 2025, following Rocket's voluntary pause in dosing. The company is conducting a root cause analysis, focusing on a recently introduced novel immune suppression agent in the pre-treatment regimen. The trial was designed to evaluate 12 patients, including a pediatric safety run-in, with a dose level of 6.7 x 1013 GC/kg. As of March 31, 2025, Rocket had $318.2 million in cash and investments, expecting to fund operations into 2027.

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Rocket Pharmaceuticals (NASDAQ: RCKT) presented promising preliminary data from its Phase 1 clinical trial of RP-A601 for PKP2 Arrhythmogenic Cardiomyopathy. The trial showed positive results in all three patients with up to 12 months follow-up at a dose of 8.0E13 GC/kg. Key findings include:

- Well-tolerated safety profile with no dose-limiting toxicities - Increased PKP2 protein expression (110% and 398% improvements in two patients) - Enhanced protein expression and desmosomal localization of key proteins - Improved/stabilized heart function and reduced arrhythmia burden - Quality of life improvements with KCCQ-12 score increases of 34-41 points - PVC reductions of 9% to 63% from baseline - Improved NYHA class from Class II to Class I in patients followed beyond six months

This marks Rocket's second successful gene therapy from their AAV cardiovascular portfolio, advancing their mission to develop curative treatments for rare heart conditions.

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Rocket Pharmaceuticals (NASDAQ: RCKT) has announced that data from its Phase 1 clinical trial of RP-A601, a gene therapy treatment for plakophilin-2 related arrhythmogenic cardiomyopathy (PKP2-ACM), will be presented at the American Society of Gene and Cell Therapy (ASGCT) Annual Meeting in New Orleans. The presentation, titled "Preliminary Data from a Phase I Gene Therapy Trial of RP-A601 (AAVrh.74-PKP2a) for Adult Patients with PKP2-ACM," will be delivered by Dr. Barry H. Greenberg on May 15, 2025. Following the presentation, Rocket will host an investor webinar at 4:30 p.m. ET to discuss the findings.
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Rocket Pharmaceuticals (NASDAQ: RCKT) reported its Q1 2025 financial results and pipeline updates. The company ended Q1 with $318.2 million in cash and investments, providing runway into Q4 2026. Key pipeline developments include ongoing Phase 2 pivotal study of RP-A501 for Danon disease with updates expected mid-2025, anticipated initial Phase 1 data for RP-A601 in PKP2-ACM in May 2025, and planned IND submission for BAG3-DCM program mid-2025.

Financial highlights show R&D expenses decreased to $35.9 million from $45.2 million year-over-year, while G&A expenses increased to $28.4 million from $22.1 million. Net loss was $61.3 million ($0.56 per share). The company appointed Sarbani Chaudhuri as Chief Commercial & Medical Affairs Officer and continues regulatory progression for RP-L102 and KRESLADITM programs.

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Rocket Pharmaceuticals (NASDAQ: RCKT), a late-stage biotechnology company focused on genetic therapies for rare disorders, has announced its participation in the 24th Annual Needham Virtual Healthcare Conference.

The company's CEO, Gaurav Shah, M.D., will engage in a fireside chat scheduled for 8:45 a.m. ET on Tuesday, April 8, 2025. The presentation will be accessible via webcast on the company's Investors section website, with replay availability for approximately 30 days after the event.

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Rocket Pharmaceuticals (NASDAQ: RCKT) reported its Q4 and full-year 2024 financial results, highlighting significant progress in its gene therapy pipeline. The company's cash position stands at $372.3 million, providing runway into Q3 2026.

Key developments include ongoing dosing in the Phase 2 pivotal study of RP-A501 for Danon disease, with program updates expected in H1 2025. Long-term data from the Phase 1 study showed sustained efficacy up to five years. The company completed enrollment in the low-dose cohort of RP-A601 for PKP2-ACM, with initial data expected in H1 2025.

Financial results show R&D expenses decreased to $171.2 million from $186.3 million in 2023, while G&A expenses increased to $102.0 million from $73.3 million. Net loss was $258.7 million ($2.73 per share) compared to $245.6 million ($2.92 per share) in 2023. The company raised $182.5 million through a public offering in December 2024.

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Rocket Pharmaceuticals (NASDAQ: RCKT), a late-stage biotechnology company focused on genetic therapies for rare disorders, has announced its participation in the 43rd Annual J.P. Morgan Healthcare Conference in San Francisco. Gaurav Shah, M.D., the company's CEO, will deliver a presentation on Monday, January 13, at 3:45 p.m. PT and will be available for investor meetings throughout the conference.

The presentation will be accessible via webcast and will be made available on the company's website in the Investors section. Rocket Pharmaceuticals specializes in developing sustainable pipeline solutions for rare disorders with high unmet medical needs.

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Rocket Pharmaceuticals (NASDAQ: RCKT) has announced the pricing of an upsized public offering of 13,200,000 shares of common stock at $12.50 per share. The offering is expected to generate gross proceeds of $165 million before deducting underwriting costs and expenses. The company has granted underwriters a 30-day option to purchase up to an additional 1,980,000 shares. The offering is anticipated to close around December 12, 2024. Morgan Stanley, Leerink Partners, and Cantor are serving as joint book-running managers, with LifeSci Capital acting as lead manager.

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Rocket Pharmaceuticals (NASDAQ: RCKT), a late-stage biotechnology company focused on genetic therapies for rare disorders, has announced plans for a $150 million public offering of common stock. The company will grant underwriters a 30-day option to purchase up to an additional 15% of shares. Morgan Stanley, Leerink Partners, and Cantor are serving as joint book-running managers, with LifeSci Capital as lead manager.

The offering will be conducted through a previously filed shelf registration statement with the SEC. The final terms and completion of the offering are subject to market conditions.

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Rocket Pharmaceuticals (NASDAQ: RCKT), a late-stage biotechnology company focused on genetic therapies for rare disorders, announced its participation in the 7th Annual Evercore HealthCONx Conference in Miami, Florida. CEO Gaurav Shah will conduct a fireside chat on Tuesday, December 3, at 1:45 p.m. ET and hold investor meetings throughout the day. The fireside chat will be accessible via webcast and will be available on the company's Investors section of their website.

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FAQ

What is the current stock price of Rocket Pharmaceu (RCKT)?

The current stock price of Rocket Pharmaceu (RCKT) is $2.48 as of May 28, 2025.

What is the market cap of Rocket Pharmaceu (RCKT)?

The market cap of Rocket Pharmaceu (RCKT) is approximately 738.0M.
Rocket Pharmaceu

Nasdaq:RCKT

RCKT Rankings

RCKT Stock Data

738.00M
103.56M
3.02%
106.9%
10.35%
Biotechnology
Pharmaceutical Preparations
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United States
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