MDC-101

MDC-101 is an antisense oligonucleotide (ASO) therapeutic candidate developed by MediciBio for the treatment of Avellino Corneal Dystrophy (ACD), also known as Granular Corneal Dystrophy Type 2. The condition is caused by a specific mutation (R124H) in the TGFBI gene, which leads to the accumulation of protein deposits in the cornea and progressive vision impairment. MDC-101 is designed to selectively target and degrade the mutant TGFBI R124H mRNA, thereby reducing the production of the pathogenic protein while ideally sparing the wild-type allele. This precision medicine approach aims to halt or reverse the progression of corneal opacities. As of early 2024, the program is in preclinical development.