CAMP4 Therapeutics submitted a preprint to bioRxiv on April 28, 2026, titled “Profiling and Targeting of Regulatory RNAs to Upregulate Gene Expression.” The paper details the company’s proprietary Capture‑seq method for identifying regulatory RNAs and its use of epigenomic mapping to build what the authors describe as the largest catalog of regRNAs in human cells.
The study shows that antisense oligonucleotides (ASOs) can stabilize regulatory RNAs, thereby increasing transcription of target genes. In human hepatocyte cultures, ASOs directed at a regulatory RNA that controls the OTC gene raised OTC protein levels, demonstrating a potential therapeutic strategy for ornithine transcarbamylase deficiency.
CAMP4’s lead candidate, CMP‑002, targets a regulatory RNA that modulates the SYNGAP1 gene, a haploinsufficient disorder linked to neurodevelopmental disease. The company expects to initiate a Phase 1/2 clinical trial for CMP‑002 in the second half of 2026, marking a key milestone for validating its up‑regulation platform in haploinsufficient conditions.
The preprint underscores the progress of CAMP4’s RAP Platform® and reinforces the strategic partnership with GSK, which has provided an upfront payment and milestone‑based funding. The company’s IPO in October 2024 and a cash position that extends into 2028 give it the financial runway to pursue the clinical development of CMP‑002 and other pipeline candidates.
The preprint is a preliminary publication and has not yet undergone peer review, so the findings should be interpreted as early-stage scientific data pending formal validation.
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