From the RNA world to the clinic

Area of Science:

• Biochemistry
• Molecular Biology
• Therapeutics

Background:

• RNA molecules possess remarkable structural and functional versatility.
• This versatility has driven research into RNA-based therapeutic agents for diverse medical applications.

Purpose of the Study:

• To highlight the growing role and potential of RNA therapeutics in clinical practice.
• To summarize the current landscape of RNA-based drug development.

Main Methods:

• Review of ongoing clinical trials for various RNA therapeutic modalities.
• Analysis of patient enrollment and therapeutic targets in late-stage and early-stage development.

Main Results:

• Multiple RNA therapeutics, including messenger RNAs, small interfering RNAs, and aptamers, are in late-stage clinical trials.
• Hundreds of patients are participating in trials for cancer, renal and hepatic disorders, and ocular and cardiovascular diseases.

Conclusions:

• RNA therapeutics represent a rapidly advancing class of agents with significant clinical potential.
• Positive trial outcomes are expected to drive future development and adoption of RNA-based medicines.