Where should siRNAs go: applicable organs for siRNA drugs

Area of Science:

• Biotechnology
• Molecular Biology
• Pharmacology

Background:

• Small interfering RNAs (siRNAs) offer targeted gene regulation for therapeutic development.
• Efficient delivery of siRNAs to target organs remains a significant hurdle.
• Five siRNA drugs targeting liver cells were approved between 2018 and 2022.

Purpose of the Study:

• To review current siRNA drugs and clinical trial candidates targeting multiple organs.
• To discuss organ-specific advantages, disadvantages, and delivery strategies for siRNA therapeutics.
• To highlight progress in developing siRNA drugs for organs beyond the liver.

Main Methods:

• Literature review of approved siRNA drugs and ongoing clinical trials.
• Analysis of organ-specific characteristics influencing siRNA delivery.
• Discussion of strategies to overcome delivery barriers for various target organs.

Main Results:

• The liver, eye, and skin are preferred targets, with multiple siRNA candidates in late-stage clinical trials.
• Lungs, kidneys, and brain present greater delivery challenges, with fewer clinical trials underway.
• Organ-specific delivery strategies are crucial for advancing siRNA therapeutics.

Conclusions:

• Significant advancements have been made in siRNA drug development, with initial successes in liver-targeted therapies.
• Targeting organs beyond the liver requires overcoming specific biological barriers.
• Further research into organ-specific delivery strategies is essential for expanding the therapeutic potential of siRNAs.