Sarepta Therapeutics reported data showing its experimental gene therapy raised levels of the missing protein in limb‑girdle muscular dystrophy (LGMD) 2E and said it will seek regulatory approval—potentially the first approved therapy for an LGMD subtype. Sarepta’s submission would mark a landmark rare‑disease regulatory filing if accepted. Separately, Chiesi Global Rare Diseases committed up to $115 million to collaborate with Arbor Biotechnologies on in vivo CRISPR‑based editing for primary hyperoxaluria type 1, expanding Chiesi’s rare disease strategy into genetic medicines. Both moves underline ongoing investment in genetic medicines for ultra‑rare disorders and increased industry appetite for in‑vivo delivery solutions.