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.
Gene Therapy Clinical Trial Pipeline Gains Momentum: 180+ Companies Lead the Charge
Gene therapy clinical trials gain momentum with over 180 companies advancing treatments, leveraging technologies like CRISPR/Cas9 for precision. Key therapies include ZOLGENSMA for spinal muscular atrophy and LUXTURNA for inherited retinal diseases. Challenges include immune reactions, long-term effects, and high costs, necessitating ethical oversight and equitable access.
Pompe Disease Clinical Trials 2024 (Updates): EMA, PDMA, FDA
DelveInsight's 'Pompe Disease Pipeline Insights, 2024' covers 15+ companies and 20+ pipeline drugs, including clinical and nonclinical stage products. Key companies include Spark Therapeutics, Amicus Therapeutics, and Asklepios Biopharmaceutical. Recent updates include study results from Astellas Gene Therapies, Amicus Therapeutics, and Genzyme. Pompe disease is a rare genetic disorder with treatments like enzyme replacement therapy.
World Heart Day 2024: Looking Back at Cardiology's Year of Progress in Cell and Gene Therapy
Cardiology gene therapy and cell therapy progress includes Rocket Pharmaceuticals completing enrollment for Danon disease gene therapy RP-A501, LX2006 improving Friedreich Ataxia cardiomyopathy biomarkers, Nexcella dosing first patient in light chain amyloidosis CAR-T trial, Verve Therapeutics starting phase 1b trial for VERVE-102, and Sardocor receiving fast track designation for heart failure gene therapy SRD-001.
Related Clinical Trials:
Rocket Pharmaceuticals Maxes Out Recruitment in Pivotal Trial for Danon Disease Gene Therapy
Rocket Pharmaceuticals completed enrollment in a phase 2 trial (NCT06092034) evaluating RP-A501, a gene therapy for Danon disease, with 12 male patients receiving a dose of 6.7x10^13 GC/kg. The trial's primary end points include LAMP2 expression and left ventricular mass reduction, with secondary end points including troponin levels and natriuretic peptide levels. Rocket plans to seek regulatory filings for RP-A501 both inside and outside the US.
Rocket Pharmaceuticals Announces Completion of Enrollment in Phase 2 Pivotal Trial of RP-A501 for Danon Disease
Rocket Pharmaceuticals, Inc. announced full enrollment in its global Phase 2 trial evaluating RP-A501 for Danon disease, a rare genetic disorder. The trial aims to assess the efficacy and safety of RP-A501, a gene therapy designed to restore cardiac function by delivering functional LAMP2B genes to heart tissue.
Related Clinical Trials:
Rocket Pharmaceuticals: Pioneering Gene Therapy for Rare Diseases
Gene therapy aims to cure rare diseases, freeing patients from genetic burdens. Trust, generosity, curiosity, and patient community support are core values. Innovative science targets diseases with high unmet needs, focusing on patient-centered solutions.
© Copyright 2025. All Rights Reserved by MedPath