TRCN-1023 (FUNCtion ALS)

What is the drug thought to do in the body?

TRCN-1023 is a gene therapy which targets the protein UNC13A, whose function is lost in people with MND. UNC13A is important for communication between neurones, which is essential for the nervous system to work properly. When UNC13A loses its function neurones cannot send signals effectively, which can lead to neurone damage. The function in UNC13A is lost because TDP-43 normally controls the production of  UNC13A. In 97% of people with ALS, TDP-43 is in the wrong place in the cell. This means it cannot do its job properly, and therefore UNC13Aisn’t made correctly and can’t function normally. TRCN-1023 is thought to restore the production and function of UNC13A. 

Phase 1/2 - 

Current status: In preparation in the UK

Primary objective: To investigate the safety of TRCN-1023.

This Phase 1/2 trial is a randomised and double-blind study which is investigating the safety and tolerability TRCN-1023 in people living with MND. The study will also test different doses of TRCN-1023 to determine the best dose to use in future trials. Participants will receive a single injection of either the study drug or a placebo (dummy drug) into the fluid around the spine. This will be decided at random, and participants will be monitored and assessed over a 24-week period.  Find out more on the MND Clinical Studies Group website or on a clinicaltrials.gov. 

Last updated: 27/07/2026