VectorY Doses First Patient with AAV-Based TDP-43 Therapy in Phase 1/2 PIONEER-ALS Trial

Amsterdam, The Netherlands, and Boston, MA — February 9, 2026 -VectorY Therapeutics has dosed the first patient in its Phase 1/2 PIONEER-ALS clinical trial, advancing its investigational AAV-based gene therapy VTx-002 into the clinic for amyotrophic lateral sclerosis (ALS).

The first administration took place at the Sean M. Healey & AMG Center for ALS at Mass General Brigham. The study represents the first clinical evaluation of an AAV-delivered antibody therapy designed to broadly target TDP-43 pathology, a key biological driver in the majority of ALS cases.

VTx-002 utilizes an adeno-associated virus (AAV) vector platform to enable sustained expression of a therapeutic antibody within the central nervous system. Unlike conventional therapies requiring repeated dosing, the AAV gene therapy approach is designed to provide long-term antibody production in the brain and spinal cord, continuously targeting mislocalized and aggregated TDP-43 protein.

ALS affects more than 5,000 newly diagnosed patients annually in the United States, with median survival of two to three years. Current treatments offer limited disease modification, underscoring interest in durable AAV-based gene therapy strategies aimed at underlying neurodegenerative mechanisms.

The multicenter, open-label PIONEER-ALS trial is expected to enroll 12 adult patients across the U.S., Europe and the U.K. The study will evaluate two dose levels of the AAV gene therapy, with primary endpoints focused on safety and tolerability. Secondary and exploratory endpoints include biomarkers such as neurofilament light chain (NfL), TDP-43 pathway markers, and clinical measures including respiratory function, muscle strength and survival.

VTx-002 has received FDA Fast Track designation, supporting expedited development of the AAV-based therapeutic candidate.

Beyond ALS, the company is advancing additional AAV vector programs targeting neurodegenerative disorders including frontotemporal dementia, Huntington’s disease and Alzheimer’s disease, reflecting broader momentum for AAV-mediated gene therapy in neurodegeneration.

While early-stage trials remain exploratory, first patient dosing marks a key milestone in the clinical translation of AAV-delivered antibody strategies designed for sustained CNS activity.