Voyager Therapeutics, Inc., incorporated on June 19, 2013, is a clinical-stage gene therapy company. The Company focuses on developing treatments for patients suffering from severe diseases of the central nervous system (CNS). The Company's pipeline consists of programs for CNS indications, including advanced Parkinson's disease; a monogenic form of amyotrophic lateral sclerosis (ALS); Huntington's disease; Friedreich's ataxia; frontotemporal dementia/Alzheimer's disease, and severe chronic pain. The Company's clinical candidate, VY-AADC01, is an adeno-associated virus (AAV) gene therapy product candidate, for the treatment of advanced Parkinson's disease. VY-AADC01 consists of the AAV2 capsid, which has been used in multiple AAV gene therapy clinical trials for various diseases, and the cytomegalovirus promoter that drives expression of the aromatic L-amino acid decarboxylase (AADC) transgene. VY-AADC01 is intended to deliver the AADC gene directly into the putamen. AADC01 is being evaluated in an open-label, Phase 1b clinical trial for advanced Parkinson's disease. Its pipeline also includes VY-SOD101, VY-FXN01, VY-HTT01, VY-TAU01 and VY-NAV01.
The Company's other preclinical programs target severe CNS indications where loss or abnormal expression of a specific gene has been identified as the cause of the disease. VY-SOD101 is for the treatment of Amyotropic Lateral Sclerosis (ALS). VY-FXN01 is indicated for Friedreich's ataxia. VY-HTT01 is for Huntington's disease. VY-SOD101, VY-FXN01 and VY-HTT01 are in preclinical development. VY-TAU01 is for the treatment of Frontotemporal Dementia/Alzheimer's Disease. VY-NAV01 is its severe, chronic pain program. VY-NAV01 is in the research stage. It is are additional severe neurological diseases that could be treated using AAV gene therapy through application of either a gene replacement or a gene knockdown approach.
The Company competes with bluebird bio, Inc., Abeona Therapeutics, Inc., Adverum Biotechnologies, Inc., Agilis Biotherapeutics, LLC, Applied Genetic Technologies Corporation, Asklepios BioPharmaceutical, Inc., Audentes Therapeutics, Inc., AveXis, Inc., Pfizer Inc., Dimension Therapeutics, Inc., GenSight Biologics SA, MeiraGTx Ltd., NightstaRx Ltd, REGENXBio Inc., uniQure NV and Spark Therapeutics, Inc.
75 Sidney St
The company trades on NASDAQ exchange. The company headquarter is located in Massachusetts, MA. The company was established in the 2013.| Ticker | VYGR |
| Long Name | Voyager Therapeutics, Inc. |
| Short Name | Voyager Therapeutics |