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Advances in Delivery Technologies Opening New Avenues
One of the major bottlenecks limiting the clinical potential of RNAi therapeutics has been the lack of efficient and safe delivery systems. The highly labile nature of nucleic acid drugs and challenges associated with their in vivo delivery to target sites requires innovative formulation and design strategies. Novel delivery technologies such as ligand-conjugated nanoparticles, exosome-mimetic vesicles and cell-penetrating peptides can enhance the cellular uptake and protection of RNAi payloads. Advancements are also being made in modulating biodistribution through tissue-targeting ligands and optimizing pharmacokinetic profiles. Recent delivery platforms enable targeting of RNAi molecules to specific organs, cell types and subcellular locations based on pathological needs. This high precision delivery promises to augment therapeutic indices while reducing adverse effects. As more biocompatible carriers successfully navigate clinical translation, these will facilitate the development of RNAi drugs for diverse pathological conditions including those traditionally considered difficult to drug.
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