Rocket Pharmaceuticals
🇺🇸United States
- Country
- 🇺🇸United States
- Ownership
- Public
- Established
- 2015-01-01
- Employees
- 268
- Market Cap
- $1.7B
- Website
- http://www.rocketpharma.com
- Introduction
Rocket Pharmaceuticals, Inc. is a clinical-stage biotechnology company, which engages in the development of gene therapy treatment options for rare and devastating pediatric diseases. Its multi-platform development approach applies the lentiviral vector and adeno-associated viral vector gene therapy platforms. Its clinical program is an LVV-based gene therapy for the treatment of Fanconi Anemia (FA), a difficult to treat genetic disease that leads to bone marrow failure and potentially cancer. The company was founded on July 7, 1999 and is headquartered in Cranbury, NJ.
K&L Gates Advises Rocket Pharmaceuticals on Public Offering of Common Stock
K&L Gates LLP advised Rocket Pharmaceuticals, Inc. in an underwritten public offering of 15,180,000 shares of common stock, raising US$189.8 million, and a concurrent private placement of pre-funded warrants, raising US$5.0 million. The firm's Corporate practice, with hundreds of lawyers globally, provided legal solutions for the transactions.
Pompe Disease Clinical Trials 2024: EMA, PDMA, FDA Approval, Medication, Pipeline
DelveInsight's 'Pompe Disease Pipeline Insights, 2024' covers 15+ companies and 20+ pipeline drugs, including clinical and nonclinical stage products, with insights on product type, stage, route of administration, and molecule type. Key companies include Spark Therapeutics, Amicus Therapeutics, and Novartis AG. Report highlights recent clinical trial results and emerging therapies.
Weekly Rewind – December 6, 2024
Rocket Pharmaceuticals' AAV9 treatment shows good safety and efficacy in a 3-cohort trial. Updated efficacy data from phase 1/2 announced. Phase 2 to assess Hansa's imlifidase as pretreatment for Genethon's GNT-0003. Sana's SC291 CAR T-cell therapy targets lupus. Cartesian plans AURORA phase 3 trial in H1 next year.
Gene Therapy RP-A501 Shows Promising Phase 1 Results in Danon Disease
Rocket Pharmaceuticals' RP-A501 gene therapy for Danon disease showed sustained LAMP2 protein expression and reduced LV mass index in patients up to 60 months post-treatment, with generally well-tolerated safety profile.
Related Clinical Trials:
2024 BioTech Breakthrough Awards Program Celebrates Groundbreaking Biotechnology
BioTech Breakthrough announces 4th annual awards recognizing top life sciences and biotechnology innovations, with winners including Leyden Labs, Rocket Pharmaceuticals, Phathom Pharmaceuticals, and more.
2024 BioTech Breakthrough Awards Program Celebrates
BioTech Breakthrough announced winners of its 4th annual awards, recognizing top biotech companies and innovations in areas like BioPharma, Genomics, Therapeutics, and AI. The program attracted thousands of nominations from 14 countries, highlighting advancements in biological sciences and AI, such as CRISPR and precision medicine.
Rocket Pharmaceuticals Reports Third Quarter 2024 Financial Results and Highlights
Rocket Pharmaceuticals reports Q3 2024 financial and operational results, including completion of enrollment in RP-A501 Phase 2 pivotal study for Danon disease, initiation of rolling BLA submission for RP-L102 for Fanconi Anemia, and appointment of Mikael Dolsten to the Board of Directors. The company anticipates FDA approval for KRESLADI for severe LAD-I in 2025 and expects its cash, cash equivalents, and investments to fund operations into 2026.
SG Americas Securities LLC Takes $113,000 Position in Rocket Pharmaceuticals, Inc
SG Americas Securities LLC bought 6,128 Rocket Pharmaceuticals shares in Q3, valued at $113,000. Other investors also increased holdings, with 98.39% of stock owned by institutions. RCKT stock opened at $17.04 on Friday, with a market cap of $1.55 billion. Rocket Pharmaceuticals reported a Q2 loss of $0.74 per share, missing estimates by $0.02. Analysts maintain 'buy' ratings with a consensus target price of $51.75.
Scotiabank says its 3 biotech 'top pick' stocks have more than 100% upside potential
Scotiabank identifies Rocket Pharmaceuticals, Liquidia Corp, and Ocular Therapeutix as top biotech picks, predicting over 100% stock growth in 12 months due to anticipated macroeconomic changes and strong sector execution.
Gene Therapy Clinical Trial Pipeline Gains Momentum: 180+
The gene therapy market is rapidly expanding, with over 180 companies developing more than 200 gene therapies. Key players include Ultragenyx Pharmaceutical Inc, Rocket Pharmaceuticals, and Adverum Biotechnologies. Prominent therapies in the pipeline include DTX401, RP-L102, and ADVM-022. Gene therapy aims to treat diseases by modifying genes, offering potential long-lasting effects with fewer side effects. Challenges include immune reactions, long-term effects, and high costs.
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