MicroRNAs: The Future of Gene Expression Regulation

MicroRNAs (miRNAs) are small, non-coding RNA molecules that play a crucial role in regulating gene expression. Several companies are advancing candidates relying on miRNAs in 2024, with potential applications in disease diagnosis and treatment. Companies such as aceRNA Technologies, ARTHEx Biotech, and Transcode Therapeutics are developing innovative miRNA-based therapeutics for various diseases, including cancer, cardiovascular diseases, and neurodegenerative disorders.
  • In the next 6 months, we can expect to see the completion of phase 1/2 clinical trials for several miRNA-based therapeutics, including Transcode Therapeutics’ TTX-MC138 and ARTHEx Biotech’s ATX-01.
  • Within the next year, we anticipate the initiation of phase 3 clinical trials for some of these miRNA-based therapeutics, marking a significant step towards their potential approval and commercialization.
  • In the next 5 years, we expect to see the widespread adoption of miRNA-based therapeutics in the treatment of various diseases, with several companies achieving significant commercial success and expanding their product portfolios.
  • By the end of the next decade, miRNA-based therapeutics are likely to become a standard treatment option for many diseases, with ongoing research and development driving further innovation and improvement in their efficacy and safety.

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