Directed To Deliver
Voyager is advancing early-stage programs powered by proprietary, next-generation AAV capsids to deliver innovative gene therapies using various payloads. The pipeline supports the Company’s strategic vision for developing best-in-class treatments by focusing on validated targets, following efficient pathways to preclinical and clinical proof of concept, and seeking opportunities to provide meaningful therapeutic benefit to patients in areas of significant unmet need.
Voyager is developing novel and complementary approaches to disrupt the progression of tau pathology to address tauopathies including Alzheimer’s disease. We believe our passive-immunity therapeutic and vectorized anti-tau antibodies are highly differentiated from competitive approaches by offering improved targeting of specific regions of the tau protein that could offer an improved clinical profile compared to first generation approaches.
Up to 10% of Parkinson’s disease (PD) patients have a GBA1 mutation, which increases the risk of PD approximately 20-fold. Voyager is leveraging gene replacement technology to address PD with GBA1 mutations. Our approach is designed for delivery of GBA1 via intravenous AAV dosing to enable widespread distribution to multiple affected brain regions and avoid the need for more invasive approaches.
Superoxide dismutase 1 (SOD1) mutations cause toxic gain of function in forms of familial amyotrophic lateral sclerosis (ALS), a devastating neurodegenerative disease with minimally effective treatments. Voyager’s SOD1 knockdown program aims to address ALS caused by SOD1 mutations by combining a highly potent siRNA construct with a CNS-tropic, BBB penetrant capsid identified from Voyager’s TRACER platform, allowing for intravenous AAV dosing to enable broad CNS knockdown of SOD1 and slow of the decline of functional ability.
*Programs named according to target and lead indication.
Voyager is partnering with Neurocrine Biosciences on a preclinical Friedreich’s Ataxia (FA) program and two undisclosed discovery programs. Voyager has an option to co-develop/co-commercialize the FA program in the U.S. or to grant Neurocrine global commercial rights.
Here we have gathered resources for patients and caregivers looking to better understand the diseases we seek to treat. If you are a patient or caregiver, please feel free to contact Voyager with questions: email@example.com