Solid Biosciences has issued an update on IGNITE DMD, the Phase I/II clinical study with its microdystrophin gene therapy, SGT-001.

The most recent child dosed in the study has experienced a serious adverse event (SAE), and thankfully, he is now improving. He is being closely followed by his care team. Solid is working hard to better understand this event and will continue to monitor this patient with Dr Barry Byrne and his exceptional team at the University of Florida. The FDA has placed the trial on hold.

The DRF team’s thoughts and wishes are with the boy and his family at this time. We are extremely grateful to the Solid team for all they continue to do – for their efforts in overcoming the challenges faced in this brave and critical work, and their continued determination to tackle this complex condition.

To date, six patients have been dosed with SGT-001. This includes three patients in the first dose cohort at the 5E13 vg/kg dose. These patients are all doing well. In February of this year Solid reported initial data from this first cohort – and announced its plan to increase the dose to 2E14 vg/kg. Three patients have now been dosed in this second cohort. The first two of these patients are also doing well.

Click here to read Solid’s letter to the Duchenne community.

The Duchenne Research Fund is proud to have helped seed fund Solid’s gene therapy program together with Duchenne UK and Joining Jack, with a joint investment of $5 million in 2014.