Entrada's Duchenne drug shown to improve muscle function in early trial

Entrada Therapeutics

07/05/2026 12:03

May 7 (Reuters) - Entrada Therapeutics TRDA.O said on Thursday its drug improved a key measure of muscle function in children with Duchenne muscular dystrophy, a rare and fatal muscle-wasting disease, in an early-to-mid stage trial.

The Boston-based company tested the drug, ENTR-601-44, in ambulatory patients aged six to 17 who have a form of Duchenne caused by a gene mutation that can be treated by skipping exon 44.

Duchenne is an inherited condition that mostly affects boys and causes muscles to weaken steadily over time. Children typically lose the ability to walk in their early teens and later develop heart and breathing problems.

Entrada said patients treated with the drug showed a meaningful improvement in how fast they could rise from the floor to a standing position, a widely used test of muscle strength that doctors use to track disease progression and predict when a patient might lose the ability to walk.

The improvement was statistically significant and more than three times the threshold considered clinically meaningful, Entrada said.

Treated patients also showed an increase in dystrophin, a key muscle protein missing in people with Duchenne, by about 2.4 percentage points from a baseline of 4%.

The first group of patients in the trial showed the treatment was generally safe and well tolerated, with no serious side effects and no patients stopping treatment, Entrada said.

The company said less of the drug reached the bloodstream in children than expected compared to adults. It has begun dosing a second group of patients at double the original dose and results are expected by the end of 2026.

 (Reporting by Kamal Choudhury in Bengaluru; Editing by Shailesh Kuber)

 ((Kamal.Choudhury@thomsonreuters.com;))