MicroRNA (miRNA)-based therapeutics have advanced from preclinical studies to early-phase clinical trials, highlighting their promise in oncology. Several miRNA mimics and inhibitors are currently under evaluation for cancers such as liver, lung, and hematological malignancies, aiming to restore tumor suppressor miRNA function or silence oncogenic miRNAs. Clinical outcomes so far demonstrate both therapeutic potential and challenges, including delivery efficiency, off-target effects, and immune responses. Parallel to clinical developments, a growing number of patents reflect the rapid innovation in miRNA drug design, delivery platforms, and combinatorial approaches with chemotherapy, radiotherapy, and immunotherapy. Patent filings also indicate significant global interest from academia, biotechnology firms, and pharmaceutical industries. Together, clinical trial progress and patent landscapes provide critical insights into the translational potential of miRNA therapy. This chapter reviews key clinical updates, patient trends, and future perspectives in miRNA-based cancer therapeutics, emphasizing opportunities and barriers for clinical application.

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Clinical Trials and Patent Updates of miRNA Therapy in Cancer

  • Rudraksh Mukherjee,
  • Abhi Tyagi,
  • Shikha Tiwari,
  • Aayushi Arora

摘要

MicroRNA (miRNA)-based therapeutics have advanced from preclinical studies to early-phase clinical trials, highlighting their promise in oncology. Several miRNA mimics and inhibitors are currently under evaluation for cancers such as liver, lung, and hematological malignancies, aiming to restore tumor suppressor miRNA function or silence oncogenic miRNAs. Clinical outcomes so far demonstrate both therapeutic potential and challenges, including delivery efficiency, off-target effects, and immune responses. Parallel to clinical developments, a growing number of patents reflect the rapid innovation in miRNA drug design, delivery platforms, and combinatorial approaches with chemotherapy, radiotherapy, and immunotherapy. Patent filings also indicate significant global interest from academia, biotechnology firms, and pharmaceutical industries. Together, clinical trial progress and patent landscapes provide critical insights into the translational potential of miRNA therapy. This chapter reviews key clinical updates, patient trends, and future perspectives in miRNA-based cancer therapeutics, emphasizing opportunities and barriers for clinical application.