• Using epigenomic mapping and regulatory RNA (regRNA) Capture-seq, CAMP4 has assembled what it believes to be the largest catalog of regRNAs ever compiled, uncovering thousands of previously undiscovered regRNAs in human cells
• New research enables the discovery of antisense oligonucleotides (ASOs) to increase expression of disease-relevant genes for therapeutic potential

CAMP4 Therapeutics Corporation (“CAMP4” or “the Company”) (Nasdaq: CAMP), a clinical-stage biopharmaceutical company developing a pipeline of regulatory RNA-targeting therapeutics designed to upregulate gene expression with the goal of restoring healthy protein levels to treat a broad range of genetic diseases, today announced the submission of a preprint to bioRxiv, an open-access server for life sciences research, titled, “Profiling and Targeting of Regulatory RNAs to Upregulate Gene Expression.”

The preprint describes CAMP4’s development of regulatory RNA (regRNA) Capture-seq methodology, a highly sensitive method for characterizing regRNAs, which have previously been difficult to detect using conventional sequencing methods due to their short-lived nature and low expression levels. In combination with epigenetic mapping, this technology uniquely enables the first step in CAMP4’s novel therapeutic approach to increasing expression of disease-relevant genes by targeting regRNAs with antisense oligonucleotides.

“Submitting this work to bioRxiv is a milestone that reflects years of rigorous science executed by a dedicated team, and we are thrilled to share it with the broader scientific community,” said Alla Sigova, Ph.D., Senior Vice President, Platform. “The application of this technology and the vast regRNA catalog we have assembled represent a meaningful advance in our ability to understand and harness this fundamental layer of gene regulation. Translating these insights into a potentially novel strategy for increasing gene expression speaks to the strength and versatility of our platform.”

Applicable across all human cell types, epigenomic mapping and regRNA Capture-seq enables high-resolution characterization of regRNAs at scale. Applying this technology to primary human liver cells, CAMP4 has constructed what it believes to be the largest catalog of regRNAs ever assembled in any human cell type, identifying thousands of previously undescribed molecules in the process.

To illustrate the therapeutic potential of mapping and targeting regRNAs, CAMP4 developed ASOs targeting a regRNA produced by an enhancer that controls the expression of the ornithine transcarbamylase (OTC) gene in the liver. Mutations in this gene cause OTC deficiency (OTCD), the most common urea cycle disorder and a life-threatening condition in which the body cannot properly process nitrogen, leading to toxic ammonia buildup in the blood. CAMP4 designed ASOs that increased OTC expression in human hepatocytes, suggesting a potentially novel therapeutic approach for diseases where increased expression of a functional gene copy may improve clinical outcomes. The preprint describes mechanistic studies on the ASOs’ ability to reshape regRNA structure, increase its levels within cells, and displace transcriptional repressors from the associated enhancer, providing new insights into how regRNA modulation regulates gene expression.

Building on this platform, CAMP4 has applied advanced regRNA mapping across additional cells of the central nervous system to identify CMP-002, its lead product candidate, which is designed to target a SYNGAP1 regRNA to increase protein levels. CAMP4 expects to advance CMP-002 into a Phase 1/2 clinical trial in individuals with SYNGAP1 as early as the second half of 2026.

Source – Globe Newswire