The February–March 2026 period involved a mix of regulatory recalibration, late-stage clinical readouts, and continued expansion of platform-enabled cell and gene therapy pipelines. Among the most closely watched developments was the ongoing tension between uniQure and the US FDA over the evidentiary pathway for AMT-130 in Huntington's disease, which became a focal point for broader questions around regulatory flexibility in rare diseases. This dynamic was further amplified by the announcement that Vinay Prasad, director of the FDA's Center for Biologics Evaluation and Research (CBER), will depart the agency in April, introducing additional uncertainty into an already evolving regulatory landscape. Alongside these developments, neuromuscular disease remained a major focus, with multiple Duchenne muscular dystrophy programs reporting interim datasets and advancing regulatory interactions. Beyond therapeutic progress, the period also highlighted growing attention to manufacturing enablement, including AI-supported process development and rising expectations around scalable, commercial-ready production.
Abigail Pinchbeck (Wed,) studied this question.