Researchers at Tel Aviv University have developed an innovative gene therapy using locked nucleic acids (LNAs) encapsulated in lipid nanoparticles (LNPs) to treat inflammatory bowel diseases like Crohn's and ulcerative colitis.

This targeted delivery method overcomes past challenges of high-dose LNA therapies, ensuring precise delivery to the disease site and reducing harmful side effects.

The new approach significantly lowers the required dosage—by 30 times—compared to earlier methods with free LNA drugs, enhancing safety and efficacy.

The study focused on silencing the TNFα gene, which is crucial in inflammation, with the LNA molecules delivered directly to affected areas in mice, leading to notable improvements in systemic inflammation markers.

Led by Prof. Dan Peer, the research team, including Neubauer doctoral student Shahd Qassem and postdoctoral fellow Dr. Gonna Somu Naidu, collaborated with Roche Pharma on this project.

Prof. Peer highlighted that this method could pave the way for new LNA-based drugs, with plans to initiate clinical trials in humans soon.

The promising results in mice suggest potential applications for treating other diseases, such as rare genetic disorders, vascular diseases, and neurological conditions like Parkinson's and Huntington's.