The agency is considering further regulatory action after a second Duchenne patient died from acute liver failure less than two months after receiving the gene therapy.

The firm will evaluate VS-7375 as both monotherapy and in combination with Eli Lilly's EGFR inhibitor Erbitux in the study.

The firm is evaluating two dose levels of its nectin-4-targeted ADC plus Merck's Keytruda in a range of solid tumors.

The firm hopes to overcome common dose-limiting toxicities with its PSMA-targeted radiotherapy while boosting potency versus competitors.

In a Phase I study, previously treated pediatric SMA patients exhibited reductions in a marker of neurodegeneration after salanersen treatment.