DS-5141 is a therapy being developed by the Japanese company Daiichi Sankyo for the treatment of Duchenne muscular dystrophy (DMD). The investigational drug received a Japanese SAKIGAKE designation in April 2017 that gives it priority for clinical trial consultation and review for the purpose of making the drug available to patients in Japan ahead of the rest of the world.

DS-5141 belongs to a group of treatments known as gene therapy, which aim to repair damages in the patient’s genetic material.

How does DS-5141 for DMD work?

DMD is characterized by the absence of dystrophin, a vital protein for correct muscle function. In DMD patients, the gene that caries the instructions for creating the dystrophin protein is altered, or mutated. When the cell’s reading machinery encounters the mutation, it immediately stops reading and, as a result, dystrophin is not formed.

DS-5141 works by binding to the mutation site, which makes the cell’s reading machinery skip the error and continue reading the gene farther ahead. The protein that is formed is incomplete since it lacks the portion of the information the drug is linked to, but a functional form of dystrophin is still formed.

This gene therapy might help DMD patients improve muscular function by lessening the effects of the genetic mutation. DS-5141 probably won’t improve all the symptoms of the disease, but will make the symptoms less severe.

Studies of DS-5141

A Phase 1/2 clinical trial to evaluate the safety, tolerability, effectiveness, pharmacokinetics (how the drug is processed in the body), and determine the dosage for future studies of DS-5141 is under way in Japan (NCT02667483).

DMD patients participating in the study are given subcutaneous (beneath the skin) injections of DS-5141 and are followed for up for 12 weeks to assess the occurrence of adverse events and the drug’s period of activity, as well as the maximum dosage.

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