RNAi Startups Innovate with New Delivery Methods for CNS and Genetic Diseases

RNA interference (RNAi) technology, which gained traction after its Nobel Prize-winning discovery in the 1990s, has made significant progress in recent years, especially with the approval of RNAi-based medicines for rare diseases and high cholesterol. Traditionally, these treatments have focused on liver-targeted therapies, but advancements in delivery methods are expanding their potential to target other tissues, such as the brain, lungs, and kidneys.

A new generation of biotech startups, including City Therapeutics, Judo Bio, Switch Therapeutics, and Aro Biotherapeutics, is capitalizing on these innovations. These companies are developing novel RNAi therapies using synthetic RNAs, ligands, and proteins to enhance delivery and efficacy across a broader range of diseases. Investors are increasingly excited about RNAi’s untapped potential, with these companies receiving substantial funding to explore new frontiers.

These developments are made possible by new delivery technologies, which enable RNAi drugs to bypass previous barriers to effective treatment. Notably, the use of ligands to target specific kidney cells and the integration of sensors to activate treatments in select cell types are paving the way for more precise and effective therapies. This has opened new opportunities in fields such as central nervous system disorders and genetic diseases like Pompe disease.

The rise of these RNAi startups reflects the growing recognition that the field’s potential extends far beyond the liver. Former Alnylam Pharmaceuticals CEO John Maraganore notes that large companies are often unable to explore new, risky innovations due to their size, creating space for smaller, more agile firms to lead the charge in RNAi’s next phase of development.