Voyager Therapeutics Expands AD Franchise with APOE Modulation Program

Voyager Therapeutics expands its Alzheimer's disease franchise with a new program that modulates the expression of apolipoprotein E, the strongest genetic risk factor for AD. The program uses a proprietary TRACER capsid to deliver a bifunctional payload that decreases APOE4 expression while increasing APOE2 expression. Voyager anticipates presenting early data on this program at an upcoming scientific meeting in 2025.

Voyager Therapeutics, Inc. (Nasdaq: VYGR), a biotechnology company dedicated to leveraging genetics to treat neurological diseases, has continued to expand its Alzheimer's disease (AD) franchise with the introduction of a new program that modulates the expression of apolipoprotein E (APOE). The program uses a proprietary intravenous (IV)-delivered, blood-brain barrier (BBB)-penetrant TRACER capsid to deliver a bifunctional payload designed to decrease expression of APOE in carriers of the high-risk APOE4 variant while delivering the protective APOE2 variant [1].

The new APOE gene therapy program is part of Voyager’s broader strategy to target the three most-promising targets in AD: tau, amyloid, and APOE. The company aims to sequence and combine these treatments to improve therapeutic outcomes for patients. Voyager anticipates presenting early data on this program at an upcoming scientific meeting in 2025 [1].

Voyager’s AD franchise now includes:

1. VY7523: A pathologic-specific anti-tau antibody currently in a multiple ascending dose (MAD) clinical trial in AD patients, with initial tau positron emission tomography (PET) data expected in the second half of 2026.
2. VY1706: An IV-delivered tau silencing gene therapy that has shown up to 73% knockdown of tau mRNA in non-human primates following a single IV dose and is advancing towards an Investigational New Drug (IND) application in 2026.
3. Vectorized anti-Aβ antibody gene therapy: Demonstrated over 15-fold greater brain-to-plasma ratio after a single IV dose compared to a passively administered antibody over 4 weeks in a murine model.
4. APOE gene therapy program: Designed to knock down APOE in APOE4 carriers while delivering APOE2 and maintaining total APOE levels.

The TRACER™ Capsid Discovery Platform, developed by Voyager, is a RNA-based screening platform that enables rapid discovery of novel AAV capsids for gene therapy. The platform has been used to generate multiple families of 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 a biotechnology company focused on leveraging the power of human genetics to modify the course of and ultimately cure neurological diseases. Its 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. The company’s strategic partnerships with organizations such as Alexion, AstraZeneca Rare Disease, Novartis Pharma AG, and Neurocrine Biosciences, Inc., further enhance its capabilities in developing innovative treatments for neurological disorders [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