The firm's stock price dropped 27 percent on Tuesday following the news that the young man died from acute liver failure after getting Elevidys.

Precision Medicine Online's 2024 survey shows biomarker test expansion but low clinical trial enrollment and long wait times for advanced therapies.

The firm will assess whether adding its CD33-targeted radiotherapy Actimab-A to PD-1 inhibitors will benefit patients more than PD-1 inhibitors alone.

The firm will use the funds to develop its lead candidate in primary hyperoxaluria type 1 and other pipeline products in liver and CNS disorders.