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Science

RAP Platform®

Reaching New Heights

Advances in gene modulation and therapeutic regulation of gene expression have unlocked new promise for diseases that were once considered untreatable.

We believe that we can do better.

Targeting regRNAs to selectively upregulate genes can provide a new way to treat a wide range of diseases and discover a new class of medicines.

Our RAP Platform® allows us to identify regRNAs and develop targeted antisense oligonucleotides (ASOs) designed to restore protein production and address genetic diseases at their root cause. Our approach is built on ASOs: a proven and clinically-validated therapeutic modality to target RNA that is supported by well-established delivery methods and chemistries that provide flexibility to design the right therapy for each disease.

A chromatin region is depicted with labels indicating the types of assays used to generate maps. These include measures of histone modification, chromatin accessibility, transcription factor (TF) and cofactor binding and DNA looping.

1 Map candidate regRNAs

  • Generate genomic data sets for cells & tissues
  • Identify regulatory regions using machine learning tools
  • Capture and sequence predicted regRNAs
  • Create proprietary catalogs containing tens of thousands of regRNAs across diverse cell and tissue types

2 Generate ASO leads

Screen regRNAs to rapidly identify leads that upregulate target genes

The process of ASO screening is depicted by showing a set of ASOs, highlighting one that binds to an image of a regRNA. This ASO is shown individually on the right at a potential lead.

3 Optimize lead candidates

Optimize chemistry and sequences for activity, pharmacology, & safety

Two versions of the same ASO sequence are depicted. The original lead from the above figure is on the left, and a chemistry modified version representing an optimized lead candidate is shown on the right.

Our Goal

Deliver precise, potent, and durable ASO therapeutics that can be designed to treat diseases where increased protein levels are needed.

Illustration depicting how antisense oligonucleotides (ASOs) modulate regulatory RNA at an active enhancer. At the top, a regRNA molecule connects an activator protein and a repressor protein, labeled as a “balance of activating and repressive factors at the enhancer.” Near the enhancer, an ASO labeled “CAMP4 ASO” is shown binding regRNA, with arrows indicating that this interaction shifts the complex so that more activator proteins bind the enhancer and fewer repressors are present. Text near the enhancer states that ASO binding to regRNA promotes activating factor binding to the enhancer, implying increased enhancer activity and downstream gene transcription.

The Starting Point

Our primary therapeutic focus is on diseases of the central nervous system (CNS), where there are numerous haploinsufficient diseases with no approved treatments. We are building a leading pipeline of CNS therapeutic candidates leveraging proven ASO delivery technology to target genes underlying these diseases.

Beyond CNS, our platform has broad potential across multiple tissue types. We have mapped pathways in the CNS, liver, heart, skeletal muscle, and immune system, revealing significant opportunities to address a wide range of diseases through targeted protein modulation.

Learn About Our Pipeline