Gene therapy programs remained under intense regulatory review. Regenxbio’s Hunter syndrome asset faced a second FDA clinical hold after asymptomatic spine MRI masses were found in five enrolled patients, reinforcing how AAV-related safety signals continue to reverberate across development timelines. Regeneron also halted an early-phase monoclonal antibody eye therapy trial (TITAN) amid an unfavorable risk-versus-benefit assessment following a safety event, with the company reporting that the underlying cause had not been identified at the time of termination. Separately, Capricor’s deramiocel review timeline moved, with the FDA extending review and seeking additional data after an advisory committee vote, demonstrating how label and indication refinement can affect PDUFA clocks even when the core program is progressing.