Copernicus Therapeutics is a clinical-stage biotechnology company in Cleveland, Ohio, developing nucleic acid therapeutics delivered through STELLAR, its non-viral gene delivery platform. Built on technologies developed at Case Western Reserve University, the company targets genetic disorders with programs in cystic fibrosis, eye disease, and brain disease. Its lead cystic fibrosis program completed a Phase 1/2 trial with results published in Human Gene Therapy, and the company holds FDA Orphan Drug designations for cystic fibrosis and Stargardt disease.
The STELLAR platform packages compacted DNA into nanoparticles for receptor-mediated delivery, designed for repeat dosing without the immune response that limits viral vectors. The company has received research funding from the NIH and the Foundation Fighting Blindness, including a NIDA-funded collaboration with Northeastern University developing a gene therapy approach for opioid use disorder.