On 4th June 2025, the Biotechnology company Design Therapeutics announced that they have dosed the first person with Friedreich’s ataxia (FA) in their phase 1 clinical trial of their drug DT-216P2. The study will assess the safety and effectiveness of DT-216P2 in treating FA. The drug is also being tested in healthy volunteers. This trial is taking place in Australia.
FA is caused by a change to the frataxin gene. Genes are made up of the A, T, G and C building blocks. People with FA have a high number of GAA repeats in their frataxin gene, compared to people that do not have FA. DT-216 is a GeneTAC™ molecule which targets the GAA repeats, restoring the function of the gene. The frataxin gene is used to create a message called frataxin messenger RNA (or mRNA). Design Therapeutics hope that DT-216P2 will therefore increase the amount of frataxin mRNA in people with FA.Â
Click here to read their press release.
This trial follows positive results from the US phase 1 trial of an earlier formulation of the drug, which enrolled 29 people with FA. Read the results from that trial here.
Update 28th April 2026
On 28th April 2025 Design Therapeutics provided a first quarter 2026 update via press release. In summary, patients with FA continue to be dosed in the RESTORE-FA trial over 4 or 12 week treatment periods. This trial is taking place in Australia. The study is evaluating safety, how the body processes the drug, DT-216P2, (pharmacokinetics) and biomarker endpoints (such as the amount of frataxin mRNA and protein levels in the blood and in muscle biopsy samples). They are also looking at the modified Friedreich Ataxia Rating Scale (mFARS), Upright Stability Score, and PROMIS fatigue scale as exploratory endpoints.
It is anticipated that an update on the effect of DT-216P2 on the levels of frataxin in the body will be provided in the second half of 2026.
Read more about the trial here.
