The firm said at the JP Morgan Healthcare Conference that it wants zorevunersen to be approved as a disease-modifying ...

The firm at the JP Morgan Healthcare Conference additionally expects a regulatory decision on its gene therapy RGX-121 for Hunter syndrome next month.

Researchers will use a quantitative continuous scoring platform to measure patients' tumors for TROP2 expression in the trial.

Genethon has licensed to AskBio a component of a preclinical gene therapy approach for treating Pompe disease.

The firm hopes to become a commercial-stage company this year, if the KIT inhibitor bezuclastinib is approved by the FDA as expected.