The therapy, which delivers COL7A1 transgenes by eyedrop, was used previously to treat a patient under a compassionate use protocol.

The company said the therapy is the first allogenic therapy developed with its Tmod technology to be tested in humans.

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.

UCLA-led investigators say additional research is needed into why guideline-recommended genetic testing remains low among ...

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

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.

For selected applicants, the agency plans to cut review times down to one to two months and provide "tumor board-style" ...

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

NEW YORK – Nuvalent is preparing to submit a rolling new drug application to the US Food and Drug Administration, seeking approval for its ROS1 inhibitor zidesamtinib as a treatment for patients with ...