We are developing tailored RNA-based corrective interventions to target the root causes of serious diseases. We take a comprehensive approach, leveraging insights from patient genetics, disease biology and pathology to determine the most beneficial way to modulate RNA, whether through up- or downregulation of specific genes, by using either a small molecule or an oligonucleotide-based approach, depending on patient needs. By combining our expertise in genetics and disease biology with our unique platforms that allow us to evaluate multiple modalities and chemistries at scale, we can uncover the ideal RNA-based corrective therapeutic to target the genetic roots of disease.

Our RNA Technology Platform

We use comprehensive high-throughput screening, bioinformatics and modeling to harness natural processes and modulate gene expression through two proprietary platforms to generate optimized small molecules, antisense oligonucleotides (ASOs) and small interfering RNAs (siRNAs).

Attack point discovery

Small molecule attack point discovery and evaluation

Certain regions of RNA transcripts are particularly suited for small molecule intervention. With our proprietary RNA toolbox, which integrates both in silico and wet lab technologies, we can pinpoint these ‘attack points’, enabling the modulation of RNA levels—either increasing or decreasing the expression of target genes.

SpliceOmics – small molecule discovery platform

Our proprietary bioinformatic tools enable us to quickly design and implement multiplexed high-throughput assays to assess identified ‘attack points.’ These next-generation sequencing-based assays offer exceptional sensitivity, facilitating the discovery of small molecules that modulate RNA output.

By combining our expertise in genetics and disease biology with our unique platforms that allow us to evaluate multiple modalities and chemistries at-scale we can uncover the ideal RNA-based corrective therapeutic to target the genetic roots of disease.