Voyager Adds Fourth Alzheimer's Program to Pipeline, Expanding Tau, Amyloid, and APOE Approach

Voyager Therapeutics has added a new Alzheimer's disease program to its pipeline, targeting apolipoprotein E (APOE) with a bifunctional payload. This program utilizes the company's TRACER capsid technology to reduce APOE4 and increase APOE2 expression. The addition brings Voyager's Alzheimer's franchise to four wholly-owned assets, including VY7523, VY1706, a vectorized anti-amyloid beta gene therapy, and the new APOE program. Voyager plans to present early data on this program in 2025.

Voyager Therapeutics, Inc. (Nasdaq: VYGR) has announced the addition of a new Alzheimer's disease (AD) program to its pipeline, focusing on the APOE gene. This program leverages the company's proprietary TRACER capsid technology to deliver a bifunctional payload designed to reduce APOE4 expression while increasing APOE2 expression in carriers of the high-risk APOE4 variant. This new asset brings Voyager's Alzheimer's franchise to four wholly-owned programs, including VY7523, VY1706, a vectorized anti-amyloid beta gene therapy, and the new APOE program [1].

The new APOE program utilizes a single intravenous (IV) injection of a TRACER capsid carrying a bifunctional vector to achieve significant reductions in endogenous APOE4 in key AD-relevant brain regions of APOE4 knock-in mice. This approach aims to maintain overall APOE levels by increasing the expression of the APOE2 isoform, which has been associated with a lower risk of developing AD [1].

Voyager anticipates presenting early data on this program at an upcoming scientific meeting in 2025. The company's Chief Executive Officer, Alfred W. Sandrock, Jr., M.D., Ph.D., highlighted the importance of this addition to the Alzheimer's franchise, stating, "We believe each of these approaches will have an important role to play in the treatment of Alzheimer’s disease, particularly as the field begins to understand how best to sequence and combine treatments to improve outcomes for patients" [1].

Voyager's Alzheimer's franchise now includes:
- VY7523, a pathologic-specific anti-tau antibody in a multiple ascending dose (MAD) clinical trial with initial tau positron emission tomography (PET) data expected in the second half of 2026.
- VY1706, an IV-delivered tau silencing gene therapy with up to 73% knockdown of tau mRNA in non-human primates following a single IV dose and advancing towards IND in 2026.
- A vectorized anti-Aβ antibody gene therapy with over 15-fold greater brain-to-plasma ratio compared to a passively administered antibody over 4 weeks in a murine model.
- The new APOE gene therapy program [1].

The TRACER™ capsid discovery platform, developed by Voyager, is a broadly applicable, RNA-based screening platform that enables rapid discovery of novel AAV capsids for gene therapy. Voyager has leveraged TRACER to create multiple families of novel capsids that can cross the blood-brain barrier and transduce a broad range of CNS regions and cell types following intravenous delivery [1].

Voyager Therapeutics is dedicated to leveraging human genetics to modify the course of and ultimately cure neurological diseases. The company's pipeline includes programs for Alzheimer's disease, Friedreich's ataxia, Parkinson's disease, amyotrophic lateral sclerosis (ALS), and multiple other diseases of the central nervous system [1].

References:
[1] https://www.biospace.com/press-releases/voyager-adds-fourth-wholly-owned-alzheimers-disease-program-to-pipeline-complementing-existing-tau-and-amyloid-assets-with-new-apoe-approach