Voyager Expands Alzheimer’s Pipeline with Dual-Action IV Gene Therapy Targeting APOE4 And APOE2

Voyager Therapeutics, Inc., a biotech company focused on genetic approaches to neurological diseases, has expanded its Alzheimer’s disease (AD) pipeline with a new wholly-owned therapeutic program targeting apolipoprotein E (APOE). APOE is the strongest known genetic risk factor for AD, with the APOE4 variant increasing disease susceptibility, while APOE2 is linked with protective effects. Voyager’s new approach aims to reduce APOE4 expression and deliver APOE2 using its proprietary TRACER capsid platform via intravenous (IV) administration.

“The Voyager team is leveraging our deep expertise in Alzheimer’s disease biology and drug development to advance multiple programs against what we believe to be the three most-promising targets: tau, amyloid, and APOE. 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. We look forward to near-term data on some of these targets expected from third parties, which we expect will continue to inform our Alzheimer’s disease franchise and approach,” said Alfred W. Sandrock, Jr., M.D., Ph.D., Chief Executive Officer of Voyager.

The novel program utilizes a single bifunctional vector delivered through a TRACER capsid capable of crossing the blood-brain barrier. Preclinical models in APOE4 knock-in mice showed promising results: a single IV dose both lowered APOE4 expression and elevated APOE2 levels in AD-relevant regions of the brain, maintaining total APOE balance. This dual-action mechanism has the potential to reshape genetic risk profiles associated with Alzheimer’s disease.