VYGR
- Voyager Therapeutics, Inc.
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Overview
Company Summary
Voyager Therapeutics, Inc. is a clinical-stage gene therapy company that focuses on developing transformative treatments for severe neurological diseases. The company utilizes the power of gene therapy to target the underlying genetic causes of these disorders and provide potential long-term solutions.
Voyager Therapeutics primarily focuses on developing therapies for genetic diseases that affect the central nervous system, such as Parkinson's disease, Huntington's disease, and amyotrophic lateral sclerosis (ALS). These diseases are characterized by the progressive degeneration of certain cells or regions within the brain, leading to debilitating symptoms.
The company's approach involves using adeno-associated virus (AAV) vectors to deliver therapeutic genes to specific target cells within the central nervous system. These genes are designed to either replace a defective gene, introduce a therapeutic protein, or modulate gene expression to mitigate the disease's progression.
Voyager Therapeutics' gene therapy platform leverages cutting-edge technology and extensive research to design and optimize AAV vectors that can safely and efficiently deliver genes to the affected areas of the brain or spinal cord. By doing so, the company aims to address the underlying causes of these neurological diseases, offering potentially transformative therapies that may slow down or even halt disease progression.
As a clinical-stage company, Voyager Therapeutics is actively conducting preclinical and clinical trials to assess the safety and efficacy of its gene therapies. These trials involve collaborations with leading academic institutions, clinical experts, and industry partners.
The ultimate goal of Voyager Therapeutics is to leverage the potential of gene therapy to provide patients suffering from severe neurological diseases with much-needed therapeutic options that can improve their quality of life and potentially alter the course of their conditions.