GEB-101

GEB-101 is an investigational in vivo genome editing therapy developed by GenEditBio for the treatment of TGFBI corneal dystrophy, a group of inherited eye disorders caused by mutations in the TGFBI gene that result in abnormal protein accumulation within the cornea’s stromal layer. The therapy utilizes CRISPR-Cas ribonucleoprotein (RNP) technology to directly target and edit disease-causing mutations at a specific locus within the TGFBI gene. GEB-101 is formulated as an RNP complex encapsulated within GenEditBio’s proprietary engineered protein delivery vehicle (PDV), designed to enhance safety, precision, and localization of genome editing. It is administered via intrastromal injection and intended as a once-and-done curative approach. Preclinical studies demonstrated high safety with minimal off-target effects, and clinical evaluation began in 2025 with an open-label dose-escalation trial assessing tolerability when combined with standard phototherapeutic keratectomy[1][2][3][6][8].