Therapy for Fuchs Endothelial Corneal Dystrophy

富克斯内皮性角膜营养不良（FECD）的特征是角膜内表面内皮细胞单层的进行性变性。细胞外基质在角膜基质和Descemets膜内皮层之间积聚，导致角膜水肿，视力下降。FECD进展缓慢，直到内皮层严重退化，患者才寻求治疗。移植是目前唯一的治疗方法。然而，供体角膜供应有限，手术并发症可能很严重，并且由于内皮细胞营养不良的移植有较高的长期失败率。一种更理想的治疗方法将完全避免移植的需要。

Fuchs endothelial corneal dystrophy (FECD) is marked by progressive degeneration of the monolayer of endothelial cells on the inner surface of the cornea. Extracellular matrix accumulates between the corneal stroma and the endothelial layer at Descemets membrane, leading to corneal edema, loss of optical quality, and decreased vision. FECD is slowly progressive, and patients do not seek treatment until the endothelial layer is badly degenerated. Transplantation is the only current treatment. However, donor corneas are in limited supply, surgical complications can be significant, and transplants due to endothelial dystrophy have a higher long-term failure rate. A more optimal therapy would avoid the need for transplantation altogether. Fibroblast growth factors (FGFs) have been shown to stimulate proliferation and migration of human corneal endothelial cells in vitro and have the potential to be regenerative therapies in vivo. However, the application of wild type FGFs as therapeutics is limited by poor stability and pharmacokinetics. The lead collaborators have developed an engineered fibroblast growth factor (eFGF) that has demonstrated improved stability and potency in preclinical studies. TRND scientists developed a production process for eFGF and completed IND-enabling studies. TRND support enabled the lead collaborators to conduct a Phase 1 clinical study and begin Phase 2 clinical trials.