About microRNAs.

We believe that targeting pathways of human disease with antisense oligonucleotides targeting RNA biology represents a novel and potentially powerful therapeutic approach for multiple diseases.

We are beginning this work with therapies designed to target microRNA regulation of protein expression. MicroRNAs are small, naturally occurring, non-coding RNAs that function as important regulators of gene expression of messenger RNA (mRNA) and play a role in multiple cellular processes. They do this by binding specific mRNA and blocking translation, leading to control of gene expression and direct degradation of target mRNA.

These naturally occurring non-coding RNAs were first discovered in 1993, and have been identified in almost every mammalian species, including humans. This conservation across species supports the important function of these regulatory RNA elements in mammalian biology. The Nobel Prize in Physiology or Medicine was awarded jointly to Victor Ambros and Gary Ruvkun on October 7, 2024, for their discovery of microRNA and its role in post-transcriptional gene regulation.¹

MicroRNAs are integral in numerous biological processes, including the immune response, cell-cycle control, metabolism, viral replication, stem cell differentiation, and human development. There are about 2,600 predicted mature human microRNAs, 600 of which are considered to be well validated and provide a large space of novel target opportunities. Indeed, microRNA expression or function is significantly altered in numerous diseases including cancer and fibrosis, as well as in CNS, metabolic and inflammatory disorders.

We are currently focused on genetic kidney diseases, where we have evolved our foundational technology and demonstrated the ability to preferentially distribute our therapeutic molecules to the kidney in both preclinical models and in patients.

_{1. www.nobelprize.org}