What does the drug do?

WVE-004 is an investigational gene therapy being developed by Wave Life Sciences, and is specifically designed for MND caused by a mutation in the C9orf72 gene. WVE-004 uses an approach known as ‘antisense’, where the drug directly interferes with the faulty instructions for making a protein.

The C9orf72 gene contains three different sets of instructions (RNA) to make the C9orf72 protein and these three RNA are called V1, V2 and V3. In some forms of MND, mutations in the C9orf72 gene cause the V1 and V3 instructions to be faulty and this leads to the production of toxic proteins which build up in the neurons. WVE-004 targets the faulty V1 and V3 RNA and signal to the cell that they need to be destroyed, leaving the V2 instructions functional so that healthy version of the C9orf72 protein can still be made.

Phase 1b/2a - FOCUS-C9

The Phase 1/2a study aims to assess the safety and tolerability of different doses of WVE-004 in those with MND. The multi-centre study is taking place at several sites across nine different countries, including the UK. This trial aims to recruit 42 participants and will involve participants being randomly assigned to receive either the placebo or one of four different doses of WVE-004 for a total of 24 weeks. You can find out more about the trial here.

The trial is currently recruiting in the UK and Republic of Ireland at the following sites:

Latest News

April 2022 - Interim results show some reduction in key biomarker Poly(GP). Further results expected in 2022. Find out more here.

July 2021 - The WVE-004 trial opened for recruitment


Want to find out more about WVE-004? Check out the resources below:

Blog posts:

Virtual Symposium 2021: Update on clinical trials

Last updated: 05/08/2022