Core Viewpoint - CAMP4 Therapeutics Corporation reported strong execution in Q1 2025, advancing its clinical studies and achieving significant milestones in its therapeutic pipeline [2][4]. Corporate Highlights - The Phase 1 clinical trial of CMP-CPS-001 for Urea Cycle Disorders (UCDs) is ongoing, with dosing completed in multiple ascending dose (MAD) cohort 3; safety, pharmacokinetic, and pharmacodynamic data are expected in Q4 2025 [4][5]. - A Clinical Trial Application (CTA) was successfully submitted in Europe for a Phase 1b clinical trial targeting female OTC heterozygotes [4][5]. - The company nominated CMP-SYNGAP-01 as a development candidate for SYNGAP1-related disorders, with GLP toxicology studies expected to begin in 2025 [4][6]. - CAMP4 will present at the American Society of Cell and Gene Therapy (ASGCT), showcasing data on increased SYNGAP1 protein levels from CMP-SYNGAP-01 and interim safety results from the Phase 1 study of CMP-CPS-001 [4][5]. Financial Results - As of March 31, 2025, cash, cash equivalents, and marketable securities totaled $49.3 million, down from $64.0 million as of December 31, 2024 [7]. - Research and development expenses for Q1 2025 were $10.1 million, an increase from $9.7 million in Q1 2024, primarily due to higher clinical and preclinical study costs [7]. - General and administrative expenses rose to $3.8 million in Q1 2025 from $3.1 million in Q1 2024, attributed to increased personnel-related and overhead expenses [8]. - The net loss for Q1 2025 was $12.4 million, slightly improved from a net loss of $12.5 million in Q1 2024 [8][14].

Core Insights - CAMP4 Therapeutics Corporation has appointed Doug E. Williams, Ph.D., and Murray Stewart, DM FRCP, to its Board of Directors to enhance its strategic guidance in drug development efforts [1][2] - The company focuses on developing regRNA-targeting antisense oligonucleotide (ASO) therapies aimed at upregulating gene expression to restore healthy protein levels [1][4] Company Overview - CAMP4 is a clinical-stage biotechnology company developing disease-modifying treatments for a range of genetic diseases where increasing healthy protein levels may provide therapeutic benefits [4] - The proprietary RAP Platform™ allows for targeted gene upregulation by mapping regRNAs, which are crucial in controlling gene expression [4] Leadership Expertise - Dr. Doug E. Williams has over 30 years in the biopharma industry and has contributed to the development of transformative drugs such as LEUKINE, ENBREL, and SPINRAZA [2][3] - Dr. Murray Stewart has extensive clinical development experience, having served as Chief Medical Officer at Rhythm Pharmaceuticals and held leadership roles at GlaxoSmithKline [3] Strategic Focus - The company aims to advance its lead clinical program for urea cycle disorders and preclinical program for SYNGAP1-related disorders while developing additional RNA-targeting medicines [2] - The focus is on addressing diseases where modest increases in protein levels can significantly impact patient lives [2]