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
+ SOD1 Silencing Gene Therapy (siRNA) / ALS
+ Tau Silencing Gene Therapy (siRNA) / Alzheimer’s Disease
Neurocrine (VYGR has 50% co/co option)
Neurocrine (VYGR has 40% co/co option)
Five Gene Therapy Programs / Undisclosed Diseases
Alexion, AstraZeneca Rare Disease License
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
anti-tau antibody for Alzheimer’s disease
Modality: monoclonal antibody
Target: tau protein. Designed to block the spread of pathological tau, which is closely correlated with disease progression and cognitive decline in Alzheimer’s disease.
Key Data: We believe VY-TAU01 is differentiated from other anti-tau antibodies based on the epitope it targets, which is located in the C-terminal rather than the N-terminal, mid-domain, or microtubule binding region (MTBR) region of the tau protein. 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.
Status: IND submission expected in the first half of 2024.
Alzheimer’s gene therapy early research programs
Tau knock-down program
Indication: Alzheimer’s disease
Modality: gene therapy: novel TRACER-derived capsid with vectorized anti-tau siRNA
Target: intracellular tau
Status: IND anticipated in 2026
Vectorized anti-amyloid antibody program
Modality: gene therapy: novel TRACER-derived capsid with vectorized anti-A beta amyloid antibody
Target: amyloid plaques
Status: Early preclinical research
gene therapy for Amyotrophic Lateral Sclerosis (ALS)
Modality: gene therapy: novel TRACER-derived capsid with vectorized anti-SOD1 siRNA
Target: superoxide dismutase 1 (SOD1); mutations in this gene can cause toxic gain of function.
Key Data: At ASGCT 2022, Voyager presented data demonstrating that an intravenously delivered, gene therapy approach to targeting SOD1 ALS resulted in robust SOD1 knockdown and significant improvements in survival in mice.
Status: IND submission expected in mid-2025.