CAMP4 Therapeutics Maps Thousands of Regulatory RNAs, Increases OTC Expression
CAMP4 mapped thousands of previously undiscovered regulatory RNAs using epigenomic mapping and regRNA Capture-seq, assembling the largest catalog to date in human liver cells. The company demonstrated antisense oligonucleotides that increased OTC gene expression in hepatocytes and plans to begin a Phase 1/2 trial of SYNGAP1 candidate CMP-002 in H2 2026.
1. Development of regRNA Capture-seq Platform
CAMP4 combined epigenomic mapping with a novel regRNA Capture-seq technique to achieve high-resolution profiling of regulatory RNAs across human cell types, constructing what it believes is the largest catalog of regRNAs to date by identifying thousands of previously undescribed molecules in primary liver cells.
2. Therapeutic Demonstration with OTC ASOs
The company designed antisense oligonucleotides targeting a liver enhancer-associated regRNA controlling the ornithine transcarbamylase gene, demonstrating increased OTC mRNA levels in human hepatocytes through regRNA structural modulation and displacement of transcriptional repressors, indicating a potential new treatment for OTC deficiency.
3. Pipeline Progress and Clinical Plan
Leveraging its platform, CAMP4 identified CMP-002, a lead candidate targeting a SYNGAP1 regRNA to upregulate protein expression, and expects to advance this program into a Phase 1/2 clinical trial in individuals with SYNGAP1 disorders in the second half of 2026.