CO-5122

CO-5122 is an investigational antisense oligonucleotide (ASO) developed by Camp4 Therapeutics for the treatment of ornithine transcarbamylase (OTC) deficiency, a rare urea cycle disorder. Utilizing Camp4's proprietary RAP (RNA Actuator Protein) platform, CO-5122 is designed to target specific regulatory RNAs (regRNAs) that control the expression of the OTC gene. By binding to these regRNAs, the drug specifically upregulates the endogenous transcription and translation of OTC mRNA, leading to increased production of functional OTC enzyme. This mechanism aims to restore the urea cycle's ability to detoxify ammonia, thereby preventing hyperammonemia and its associated neurological complications in patients with OTCD.