The development of miRNA therapeutics has emerged as a promising yet challenging frontier in precision medicine. By enabling targeted regulation of gene expression, miRNA-based therapies hold significant potential for treating cancer, genetic disorders, and various other diseases. However, despite the entry of several miRNA-based drugs into clinical trials, only a few have progressed to significant human trials. This is due to the key challenges in high-throughput screening of targets, delivery insufficiencies, off-target effects, immune responses, and high costs. Nevertheless, advancements in delivery technologies, synthetic modifications, and biomarker-driven patient selection may enable miRNA therapies to carve out a niche in modern medicine. This chapter explores the trials and tribulations of miRNA therapy market, examining experimental and developmental setbacks, and regulatory hurdles, also highlights the evolving landscape of miRNA therapeutics and its future prospects.

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The Trials and Tribulations of the microRNA Therapy Market

  • Swetha Vaishnavi,
  • Satheesh Ellipilli

摘要

The development of miRNA therapeutics has emerged as a promising yet challenging frontier in precision medicine. By enabling targeted regulation of gene expression, miRNA-based therapies hold significant potential for treating cancer, genetic disorders, and various other diseases. However, despite the entry of several miRNA-based drugs into clinical trials, only a few have progressed to significant human trials. This is due to the key challenges in high-throughput screening of targets, delivery insufficiencies, off-target effects, immune responses, and high costs. Nevertheless, advancements in delivery technologies, synthetic modifications, and biomarker-driven patient selection may enable miRNA therapies to carve out a niche in modern medicine. This chapter explores the trials and tribulations of miRNA therapy market, examining experimental and developmental setbacks, and regulatory hurdles, also highlights the evolving landscape of miRNA therapeutics and its future prospects.