Developing a new gene therapy
Original source
Developing a new gene therapy (opens in a new tab)Related topics
Trace Neuroscience is developing TRCN-1023, an antisense oligonucleotide designed to restore the UNC13A protein’s levels and function. The company reported that the therapy worked as designed and was safe and well tolerated in laboratory cells and animals. A phase 1/2 international trial called FUNCtion ALS is planned.
Why this matters
The therapy is intended to address faulty UNC13A linked to TDP-43 changes in MND. So far, the evidence is from cells and animals, so it does not yet show that TRCN-1023 is safe or effective in people or change treatment for anyone with MND.
Limitations and context
This report comes from an organisation’s account of a presentation by the company developing the therapy, not from a published human clinical trial. The reported results are preclinical, and the planned phase 1/2 trial still needs to test safety and effectiveness in people. The source does not provide detailed study methods or results.