VTx-002

What is the drug thought to do in the body?

In about 97% of people with MND, a protein called TDP-43 becomes faulty and forms abnormal clumps, which contributes to motor neuron damage. VTx-002 is a gene therapy that is designed to target these clumps of TDP-43 and kick start the body’s immune system to remove them from the neurones. The gene therapy delivers the instructions for our cells to make an antibody which sticks to the toxic clumps of TDP-43 and mark them for removal by the immune system. It is thought that a single treatment of this gene therapy could mean that the body produces this antibody for years. Previous research has shown that VTx-002 may prevent death of motor neurones and reduce decline in motor function in a mouse model of MND.

Phase 1/2 - PIONEER-ALS

Current status: Recruiting

Primary objective: To investigate the safety of VTX-002.

PIONEER-ALS is the first in-human, randomised study which is investigating the safety and tolerability of VTX-002 in people living with MND. The study will test two doses of VTX-002 in 12 people with MND, with participants being randomly assigned to a dose when they are enrolled into the study. Participants will be given a single injection of the gene therapy into large fluid-filled space at the base of the brain. They will then be followed up through clinic visits, remote monitoring through calls, tests and assessments to monitor the ongoing effects of the drug. Find out more on clinicaltrials.gov.  This trial is recruiting in the US and Europe.

Last updated: 12/02/2026