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Print/PDF Repurposing FDA-Approved Drugs for the Treatment of Aniridia
Aniridia is a panocular disease causing progressive severe visual impairment and blindness. The disease is characterized by abnormal development of almost all eye structures, caused by dominantly inherited heterozygous mutations in primarily the PAX6 gene. Although aniridia is a developmental disorder, corneal opacification begins late in childhood, leaving a large time window for therapy. Unfortunately, there is no available treatment to prevent progressive visual loss.
UNMET NEED
One of the most disabling ocular symptoms is aniridia-related keratopathy (ARK), a progressive corneal opacification due to epithelial impairment, vascular and conjunctival pathologies. ARK is associated with a deficiency of the limbal epithelial stem cells (LSC) that reside at the corneal/conjunctival boundary and normally maintain a healthy corneal epithelium.
OUR SOLUTION
Repurposing three FDA-approved drugs to improve corneal transparency or prevent ARK due to PAX6 haploinsufficiency to be administrated locally via eye drops.
STATUS
Data Strongly support the restoration of defective limbal functions following treatments in cell cultures
INTELLECTUAL PROPERTY
PCT
REFERENCES
Dorot, O., Roux, L. N., Zennaro, L., Oved, K., Bremond-Gignac, D., Pichinuk, E., & Aberdam, D. (2021). The antipsychotropic drug Duloxetine rescues PAX6 haploinsufficiency of mutant limbal stem cells through inhibition of the MEK/ERK signaling pathway. The Ocular Surface, December, 6–8. https://doi.org/10.1016/j.jtos…
Oved, K., Zennaro, L., Dorot, O., Zerbib, J., Frank, E., Roux, L. N., Pichinuk, E., Aberdam, D., & Bremond-Gignac, D. (2021). Ritanserin, a potent serotonin 2A receptor antagonist, represses MEK/ERK signalling pathway to restore PAX6 production and function in aniridia-like cellular model. Biochemical and Biophysical Research Communications, 582, 100–104. https://doi.org/10.1016/j.bbrc…
