Clinical DevelopmentCyclodextrin-based therapies are showing promise in treating rare neurological disorders, with positive early-stage trial results indicating potential for significant breakthroughs.
Regulatory MilestoneThe company's lead clinical candidate for treating Niemann-Pick Disease Type C has been granted Fast Track designation by the FDA, accelerating the potential path to market.
Strategic PartnershipsThe recent merger with Applied Molecular Transport is poised to bolster the company's financial strength and operational capabilities, enhancing its market position.