RCKT
- Rocket Pharmaceuticals, Inc.
()
Overview
Company Summary
Rocket Pharmaceuticals, Inc. (RCKT) is a biotechnology company focused on the development of transformative gene therapies for patients suffering from rare genetic disorders.
The company's primary goal is to address the underlying cause of these genetic diseases by utilizing the groundbreaking technology of gene therapy. Gene therapy involves introducing genetic material into a patient's cells to correct or replace faulty genes that lead to these disorders. This approach has the potential to provide long-term or even permanent treatments for individuals affected by such conditions.
Rocket Pharmaceuticals is dedicated to developing a portfolio of gene therapy candidates targeting various rare genetic diseases, including rare hematologic (blood-related) disorders and rare childhood-onset neurogenetic diseases. These conditions typically have significant unmet medical needs, with limited treatment options available.
The company's gene therapy candidates are designed to deliver corrected or functional copies of genes to the patients' cells, with the ultimate aim of restoring or improving normal cellular and physiological functions. This approach has the potential to offer a transformative solution by addressing the root cause of these genetic disorders rather than simply managing their symptoms.
Rocket Pharmaceuticals is actively involved in preclinical and clinical studies to evaluate the safety and effectiveness of their gene therapy candidates. These studies involve extensive research and development activities, collaboration with medical professionals and patient organizations, and close cooperation with regulatory authorities to ensure compliance with rigorous standards for patient safety.
By leveraging their expertise in gene therapy and strategic partnerships, Rocket Pharmaceuticals seeks to develop life-changing treatments for patients suffering from rare genetic disorders and improve their quality of life.