On 4th June, 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.
