Regeneron reported positive Phase 3 results for an siRNA-based drug licensed from Alnylam, targeting an under-the-radar rare autoimmune condition. This success positions Regeneron to pursue FDA approval early next year. Concurrently, the company’s cemdisiran showed symptom control in generalized myasthenia gravis, though combination therapies had mixed efficacy. These advances underscore Regeneron's growing footprint in neuromuscular rare disease therapeutics.