TTR and amyloidosis: More recently, advances have extended beyond these established platforms: as shown in Table 1, inclisiran has shown sustained lipid-lowering efficacy in long-term follow-up studies (ORION-4, 2025), eplontersen has demonstrated promise for transthyretin amyloidosis (Benson et al., 2023), and the first CRISPR-based RNA-guided editing therapy, exa-cel, was approved for sickle cell disease (U.S. Food and Drug Administration (FDA), 2023).