Sarepta Therapeutics presented data showing its experimental gene therapy increased expression of the missing gene in limb girdle muscular dystrophy (LGMD) 2E and said it plans to file for approval, aiming to deliver the first approved therapy for this ultra‑rare LGMD subtype. Company materials indicate the filing will leverage biomarker increases and early clinical signals in a small patient population. Separately, Allterum Therapeutics secured FDA clearance to begin a first‑in‑human Phase I study of 4A10, a CD127 (IL‑7Rα)‑targeting monoclonal antibody for relapsed or refractory acute lymphoblastic leukemia (ALL). The IND clearance sets the stage for dose‑escalation testing focused on safety and target engagement. Together these regulatory moves underscore continuing momentum and regulatory engagement for gene‑ and antibody‑based programs in rare and hematologic diseases.