WATERTOWN, Massachusetts, United States — October 27, 2026
CAMP4 Therapeutics Corporation, a clinical-stage biopharmaceutical company developing regulatory RNA-targeting therapeutics, announced that it has received Clinical Trial Application (CTA) approval from regulatory authorities and ethics committees in multiple European Union countries to initiate its global Phase 1/2 ASCEND clinical trial of CMP-002. The investigational antisense oligonucleotide (ASO) is being developed as a potential disease-modifying treatment for SYNGAP1-related disorder, a rare genetic neurological condition associated with intellectual disability, epilepsy and behavioral challenges. The European authorization follows earlier trial clearances in Australia and Argentina in July 2026 and authorization from the United Kingdom’s Medicines and Healthcare products Regulatory Agency in September 2026. CAMP4 remains on track to initiate the ASCEND trial and dose its first patient in the fourth quarter of 2026. The company aims to expand trial access internationally for families affected by a disorder for which no approved disease-modifying therapies are currently available.
EU Authorization Expands the Global ASCEND Clinical Trial
The latest CTA approval extends the planned geographic reach of ASCEND, CAMP4’s first-in-human Phase 1/2 study evaluating CMP-002 in patients with SYNGAP1-related disorder. The trial’s earlier regulatory milestones include clearances from Australia’s Therapeutic Goods Administration and local Human Research Ethics Committee, Argentina’s Administración Nacional de Medicamentos, Alimentos y Tecnología Médica, and the UK’s MHRA. According to CAMP4 President and Chief Executive Officer Josh Mandel-Brehm, the additional European approvals create opportunities for more families to participate closer to home. The company’s immediate priority is to begin enrolling participants and advance the clinical evaluation of CMP-002. As a Phase 1/2 investigation, ASCEND is intended to evaluate the investigational candidate in humans, with clinical testing needed to establish its safety, tolerability, dosing and potential therapeutic effects. The planned first-patient dosing remains targeted for Q4 2026.
CMP-002 Targets SYNGAP1 Gene Expression to Address the Underlying Disorder
SYNGAP1-related disorder is caused by mutations in the SYNGAP1 gene that result in haploinsufficiency, leaving affected individuals with approximately half the normal level of SYNGAP protein. The disorder affects the central nervous system and is associated with intellectual disability in all patients, epilepsy in approximately 85%, severe behavioral problems in around 70%, and sleep problems in approximately 60%. CAMP4 estimates that more than 10,000 individuals in the United States are affected. CMP-002 is designed to bind to a regulatory RNA associated with SYNGAP1 gene expression, with the goal of increasing gene activity and restoring SYNGAP protein toward near-normal levels. The candidate is administered intrathecally, delivering treatment into the fluid surrounding the spinal cord. Its development focuses on addressing the underlying protein deficiency rather than only managing individual symptoms. Clinical trials will need to determine whether the approach translates into meaningful benefits and an acceptable safety profile for patients.
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Preclinical Findings Support Continued Development of CMP-002
CAMP4 reported that CMP-002 has demonstrated dose-dependent increases in SYNGAP protein expression in patient-derived neurons. In preclinical models, the candidate reversed disease-related behavioral phenotypes in a humanized mouse model of SYNGAP1 haploinsufficiency and produced statistically significant improvements in seizure-related phenotypes and parameters in a chemically induced seizure mouse model. The company also reported broad brain distribution and significant SYNGAP protein upregulation in non-human primates. These findings provide a scientific rationale for progressing CMP-002 into clinical testing, but they do not establish efficacy or safety in humans. The program is based on CAMP4’s RAP Platform®, which maps regulatory RNAs and supports the development of antisense oligonucleotides designed to increase expression of genes associated with certain genetic disorders. The upcoming ASCEND trial will be an important step in determining whether this regulatory RNA-targeting strategy can safely increase SYNGAP protein levels and potentially deliver clinical benefits for people living with SYNGAP1-related disorder.
Source:CAMP4 Therapeutics Press release
