Rare diseases pose a global burden to healthcare despite making up a very small fraction of the global disease landscape. Most treatment options for rare diseases focus on maintaining the disease symptoms, rather than a disease-modifying approach. To address this unmet need, RNA therapies are emerging as disease-modifying approaches addressing the genetic basis, which is the predominant cause of most rare diseases. Over the past ten years, RNA therapies have progressed from experimental to validated treatments, with several approvals for rare diseases demonstrating their potential. Notable approvals that show the efficacy of ASOs (Antisense Oligonucleotides) and siRNAs (Small Interfering RNAs) platforms and increase confidence in RNA drug development include Nusinersen for spinal muscular atrophy, Patisiran for hereditary transthyretin-mediated amyloidosis, Givosiran for acute hepatic porphyria, and Tofersen for superoxide dismutase 1 (SOD1)-associated amyotrophic lateral sclerosis. RNA therapies are emerging as a rapidly growing therapeutic modality in the rare disease landscape through better delivery methods, precision targeting technologies, and personalization. The analysis also addresses key questions like:

• Which unmet needs are creating growth opportunities for RNA therapies as disease-modifying treatments for rare diseases?
• What role do RNA therapies play in facilitating better delivery methods, precision targeting technologies, and personalization?
• What are the leading companies accelerating innovations for RNA therapeutics in the rare disease segment?