“What if someone very close to you were to get ALS — what would you do differently?”
Marcel, living with ALS
Questions like these remind us why our work matters. Every day, we remain relentlessly focused on people living with neurodegenerative diseases and their families, guided by their needs and driven to deliver meaningful innovation that can make a difference in their lives.
Olga Uspenskaya-Cadoz, M.D., Ph.D. Chief Medical Officer
At VectorY, we're working toward advancing science to reverse the course of neurodegenerative disease. To achieve this, we are committed to working closely with and learning from affected individuals, their families, and advocacy organizations.
Our first focus is in Amyotrophic Lateral Sclerosis (ALS), a devastating disease with limited available treatment options. In March 2026, the first participant living with ALS was dosed on PIONEER-ALS®, a Phase 1/2 study of VTx-002. More information on PIONEER-ALS® is available on clinicaltrials.gov.
Our scientists and clinicians are also working diligently to advance new treatments for Huntington’s disease, Alzheimer’s disease, and Frontotemporal Dementia (FTD). These potential treatments are being researched in the lab and we will provide updates as this research progresses.
About VTx-002
VTx-002 is a vectorized antibody designed to holistically target TDP-43 pathology in ALS.
VTx-002 is designed to teach the motor neuron cells to make a special antibody that can find and clear the toxic TDP-43 aggregates, or clumps and help bring TDP-43 back to where it belongs inside the cell.
Watch this video to learn more about how VTx-002 is designed to work.
Expanded Access Policy
If you or a loved one are seeking options beyond clinical trials, you can explore our Expanded Access Policy to understand how investigational treatments may be requested and considered.
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VTx-002 and the other VTx programs discussed on this page are investigational. They have not been approved by the U.S. Food and Drug Administration (FDA) or any other regulatory authority. The safety and efficacy of VTx-002 have not been established.
