GEM103

GEM103 is a recombinantly produced, full-length version of the human complement factor H (CFH) protein. It is designed as a precision medicine for the treatment of age-related macular degeneration (AMD), specifically targeting patients with genetically defined CFH loss-of-function variants that lead to complement dysregulation. Administered via intravitreal injection, GEM103 acts as a native complement modulator by restoring functional CFH levels in the eye, thereby regulating the alternative pathway of the complement system and inhibiting C3 activation and amplification. This approach aims to slow disease progression in patients with dry AMD and geographic atrophy (GA). Clinical studies have shown that GEM103 increases intraocular CFH levels, reduces biomarkers of complement activation, and has demonstrated safety and tolerability in early-phase trials[1][4][5][6][7].

Other names

recombinant human complement factor HrCFH