Pipeline
Advancing transformative medicines
for neurological diseases.
Neurologic diseases – which are estimated to impact more than 1 billion people globally – represent a significant burden on those who suffer from them, on their families and communities, and on the healthcare system.
Thanks to significant advances in human genetics and genomics, we now understand the molecular causes of many neurological diseases and know how to target them.
Our pipeline includes medicines targeting the genetics underlying multiple neurological diseases.
MECHANISM / INDICATION
Research
IND-Enabling
Phase I/II
Pivotal
Approved
WHOLLY-OWNED PIPELINE
+ VY7523 (Anti-tau Antibody) / Alzheimer’s Disease
+ VY1706 (Tau Silencing Gene Therapy) (siRNA) / Alzheimer’s Disease
+ APOE Gene Therapy / Alzheimer’s Disease
ALPL-VYGR-NeuroShuttle / Undisclosed
Research
IND-Enabling
Phase I
Phase II
Phase III
OPT-IN RIGHTS
NBIB-‘223 (FXN Gene Therapy) / Friedreich’s Ataxia
Neurocrine (VYGR has option for 40% US rights)
GBA1 Gene Therapy / Gaucher Disease / Parkinson’s Disease
Neurocrine (VYGR has option for 50% US rights)
TDP-43 Small Molecule / ALS / FTD
Transition Bio (VYGR has option for 100% WW rights)
LICENSES & COLLABORATIONS
Three Gene Therapies / Undisclosed
Neurocrine
2 in IND-enabling; 1 undisclosed
Three Gene Therapies / HD, SMA, Undisclosed
Novartis
Undisclosed
One Gene Therapy / Undisclosed
Alexion
Undisclosed
Patient Resources
One of Voyager’s core values is “Patients First.” This means we act with urgency and drive every decision with the knowledge that patients are waiting for us.
Photo: Klaus C., living with ALS, 2023
Partners
VY7523
anti-tau antibody for Alzheimer’s disease
Indication: Alzheimer’s disease
Modality: monoclonal antibody
Target: tau protein. Designed to target unique C-terminal epitope and block the spread of pathological tau, which is closely correlated with disease progression and cognitive decline in Alzheimer’s disease.
Key Data: At AAIC 2022, Voyager presented data demonstrating that an anti-tau antibody delivered intravenously inhibited the spread of pathological tau by >70% in a mouse seeding model. In a single ascending dose study in healthy volunteers, data on VY7523 demonstrated safety, tolerability, and dose-proportional pharmacokinetics as presented at CTAD 2025. Also at CTAD 2025, Voyager presented the study design of the multiple ascending dose intravenous dose study.
Status: Multiple ascending dose (MAD) clinical trial is fully enrolled with tau PET imaging data expected in H2 2026.
VY1706
tau silencing gene therapy program
Indication: Alzheimer’s disease
Modality: gene therapy: novel TRACER-derived capsid with vectorized anti-tau siRNA
Target: intracellular tau
Key Data: At ASGCT 2025, Voyager presented data that a single IV dose of 1.3E13 vg/kg in NHP resulted in: Tau mRNA reduced 44-73%; Tau protein reduced 27-55%; Tau protein reduced 41-57% in neurons; broad distribution across brain; and ~30-fold liver de-targeting.
Status: GLP toxicology study ongoing. Expected to achieve first-in-human dosing in H2 2026.
Apolipoprotein E (APOE) gene therapy program
Indication: Alzheimer’s disease (AD)
Modality: gene therapy: novel TRACER-derived capsid with bifunctional payload designed to decrease expression of APOE in carriers of the variant APOE4, which has been strongly linked with a higher risk of developing AD, while delivering the variant APOE2, which has been associated with a lower risk of developing AD
Status: Early data presentation at Society for NeuroScience in Q4 2025