Cure VCP Disease

Cure VCP Disease is a U.S.-based nonprofit rare-disease organization focused on accelerating treatments and cures for disorders caused by pathogenic variants in the VCP gene, especially VCP-associated multisystem proteinopathy/IBMPFD. Rather than operating as a traditional drug developer, it functions as a patient advocacy and translational research organization that funds and coordinates preclinical tools, natural history studies, biomarker work, registry development, and clinical-trial readiness initiatives. Its research strategy spans small-molecule discovery, DNA/RNA modulation approaches, and gene-editing collaborations intended to enable future therapeutic programs. The organization also supports patients and clinicians through education, community building, and disease-management resources, while convening academic, industry, and patient stakeholders around VCP disease.