Regeneron achieved a pivotal phase 3 win with garetosmab for fibrodysplasia ossificans progressiva (FOP), an ultra-rare bone disease, reducing aberrant bone growth significantly and gearing up for FDA filing. Roivant and Priovant reported Phase 3 success for brepocitinib in dermatomyositis, planning regulatory submission. UC San Diego and Yale teams discovered safer small nuclear RNA base editing as an alternative to CRISPR, offering potential new gene therapies. These developments underscore progress in targeted treatment approaches for rare, debilitating conditions.