The ML4 Foundation announced FDA clearance of an IND for an experimental gene therapy targeting mucolipidosis type IV (ML4), enabling the first U.S. clinical trial for the ultra-rare inherited neurological disease. The program is aimed at patients with MLIV, affecting fewer than 100 known children worldwide. The IND clearance is a gating event for U.S. patient access and trial initiation, shifting the project from preclinical and regulatory preparation into clinical execution. It also serves as a signal for the pace of translational planning in ultra-rare neurology gene therapy. For the biotech community, the clearance highlights how orphan indications and established regulatory pathways can accelerate early clinical entry when assets demonstrate sufficient quality and preclinical rationale. It also adds to the small but expanding set of ML4-focused development programs moving through U.S. clinical regulatory steps.