Towards mRNA therapeutics 2.0.

Novel in vivo delivery systems and immune-silent mRNA formulations are advancing mRNA therapeutics 2.0, enabling repeated administration and targeted organ delivery.

• Why it matters: Overcoming barriers to mRNA drug development is crucial to unlock its full potential for treating rare and common diseases, beyond infectious diseases.
• What they did: The review analyzes clinical studies on mRNA-based therapies, including enzyme replacement, cancer immunotherapy, genome editing, and immune cell reprogramming, highlighting interdisciplinary technical advances.
• The result: These innovations may accelerate the development of mRNA therapeutics 2.0 by improving delivery, reducing immune responses, and enabling organ-specific targeting, thus broadening clinical applications.