Voyager Chooses Tau Silencing Gene Therapy for Alzheimer's

Voyager Therapeutics, Inc. has designated VY1706 as its primary candidate for the tau silencing gene therapy program aimed at treating Alzheimer's disease. This biotechnology firm plans to submit an IND application to the FDA and a CTA to Health Canada by 2026.

VY1706 incorporates an effective siRNA construct that reduces tau expression, combined with an IV-administered TRACER™ capsid capable of penetrating the blood-brain barrier. In studies with non-human primates, a single dose led to a 50% to 73% reduction in tau mRNA levels across the cerebral cortex, including areas affected during Alzheimer’s disease progression. Voyager intends to share these findings at an upcoming scientific event.

Toby Ferguson, Voyager's Chief Medical Officer, emphasized the importance of targeting tau in Alzheimer's treatment. He noted ongoing research, including a clinical trial of the VY7523 anti-tau antibody, reinforces their dedication. Ferguson mentioned data from third-party sources suggesting that both tau antibodies and tau knockdown strategies can influence tau accumulation in human brains, which might be associated with clinical benefits. He affirmed the company's commitment to advancing both tau antibody and gene therapy programs to achieve human proof-of-concept.

Alzheimer’s disease is a progressive neurological condition affecting an estimated 7 million people in the U.S. and up to 416 million globally. It typically begins with memory loss and can lead to reduced independence, communication issues, behavioral problems like paranoia and anxiety, and physical control loss. In 2023, the cost of caring for individuals with Alzheimer's and other dementias in the U.S. was estimated at $345 billion.

Voyager’s TRACER™ AAV capsid discovery platform, which uses RNA-based screening, has been instrumental in the rapid identification of novel capsids for gene therapy. The platform has enabled the creation of several capsid families that can cross the blood-brain barrier and target various CNS regions and cell types. Cross-species preclinical studies in rodents and non-human primates have shown that intravenous delivery of TRACER-generated capsids leads to widespread CNS expression at low doses, allowing Voyager to select multiple candidates for gene therapy programs targeting neurological diseases.

Voyager Therapeutics is focused on utilizing human genetics to change the course of neurological diseases. Their pipeline includes treatments for Alzheimer’s disease, amyotrophic lateral sclerosis (ALS), Parkinson’s disease, and other CNS disorders. Many of these programs stem from the TRACER™ AAV capsid discovery platform, which identifies capsids and receptors that may enable significant brain penetration for genetic medications administered intravenously. Voyager's programs are a mix of wholly-owned initiatives and partnerships with organizations like Alexion, AstraZeneca Rare Disease; Novartis Pharma AG; Neurocrine Biosciences, Inc.; and Sangamo Therapeutics, Inc.