From Laboratory to Clinic: Translational Challenges, Lessons, and Future Strategies for microRNA Delivery Systems. (PubMed, Curr Drug Deliv) - Sep 26, 2026 - "This review critically evaluates representative clinical and advanced translational programs, including Miravirsen, MRX34, Cobomarsen, Remlarsen, RGLS4326, and farabursen (RGLS8429), to compare molecular designs, delivery approaches, development outcomes, and translational bottlenecks. Drawing on these lessons, we propose a Four-Pillar Framework integrating Delivery Systems, Immune Safety, CMC and Manufacturing Readiness, and biomarker-guided Clinical Design. Together, these principles support coordinated optimization across biological, pharmaceutical, and clinical dimensions rather than isolated improvement of individual delivery parameters."
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Baoyue Xu; Liwei Jia; Qianqian Liu; Xin Meng; Yanhua Zhu; Yu Zhong
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