Announcement • Apr 30
Camp4 Therapeutics Corporation Submits Preprint on Regulatory Rna Profiling and Gene Upregulation Camp4 Therapeutics Corporation 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. 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. Announcement • Apr 24
Camp4 Therapeutics Corporation, Annual General Meeting, Jun 10, 2026 Camp4 Therapeutics Corporation, Annual General Meeting, Jun 10, 2026. Announcement • Mar 24
CAMP4 Therapeutics Corporation Appoints Michael Maclean to Its Board of Directors CAMP4 Therapeutics Corporation announced the appointment of Michael MacLean to the Company’s Board of Directors. Mr. MacLean brings more than 35 years of strategic financial leadership in the biotechnology and life sciences industries, with extensive experience supporting the growth of innovative biotechnology companies advancing novel genetic medicines. Most recently, he served as Chief Financial Officer at Avidity Biosciences, joining prior to the company’s 2020 IPO and leading finance and business functions until its acquisition by Novartis in 2026. Prior to Avidity, Mr. MacLean served as Chief Financial Officer, Executive Vice President of Akcea Therapeutics, where he led the buildout of the company’s financial and commercial infrastructure. He also served as Chief Financial Officer and Executive Vice President of PureTech Health, and as Chief Accounting Officer of Biogen Inc., where he oversaw the company’s worldwide finance operations. He previously served as a board member and Chair of the Audit Committee at Verve Therapeutics, a company focused on genetic medicines in cardiovascular diseases, from 2021 to 2025. Mr. MacLean received his undergraduate degree from Boston College. 
