CCM-848

CCM-848 is a small molecule translational readthrough-inducing drug (TRID) being developed by CCM Protein Upregulation, a business unit of CCM Biosciences. The drug is designed to correct nonsense mutations (premature stop codons) in genes associated with cystic fibrosis and Duchenne muscular dystrophy. CCM-848 works by enabling the translational machinery to read through premature stop codons, allowing for the production of full-length, functional proteins such as the cystic fibrosis transmembrane conductance regulator (CFTR) and dystrophin. Acquired through exclusive licensing from the University of Palermo and the Italian Cystic Fibrosis Foundation, CCM-848 has demonstrated significantly lower cytotoxicity compared to ataluren in preclinical mouse and zebrafish models. It is currently undergoing IND-enabling studies for cystic fibrosis.