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 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.
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.
*Allele-specific mHTT + MSH3 gene silencing for HD; Tau gene silencing for Alzheimer’s; vHER2 antibody for brain metastases.
**After the Phase 1 readout, Voyager has the option to either: (1) co-develop and co-commercialize with Neurocrine in the U.S. under a 60/40 cost- and profit-sharing arrangement (Neurocrine/Voyager), or (2) grant Neurocrine Biosciences full U.S. commercial rights in exchange for milestone payments and royalties based on U.S. sales.
*** After the Phase 1 readout, Voyager has the option to either: (1) co-develop and co-commercialize with Neurocrine Biosciences in the U.S. under a 50/50 cost- and profit-sharing arrangement, or (2) grant Neurocrine Biosciences full U.S. commercial rights in exchange for milestone payments and royalties based on U.S. sales.
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