Drug intelligence / Profile preview

Codagen

Development stage
Preclinical
Lead developer
CCM Biosciences
Modality
Small Molecules
Administration
Oral
01

Overview

Codagen (comprising CCM-848, CCM-914, and CCM-930) is a suite of small-molecule translational readthrough inducing drugs (TRIDs) developed by CCM Biosciences for the treatment of genetic diseases caused by nonsense mutations (premature stop codons). These mutations, which account for approximately 10% of all genetically inherited diseases, lead to the production of truncated, non-functional proteins. Codagen compounds work by enabling the ribosome to read through these premature stop codons, thereby restoring the production of full-length, functional proteins. Currently in the IND-enabling stage, these candidates are being developed for indications such as Cystic Fibrosis and Duchenne Muscular Dystrophy. Preclinical studies have shown that Codagen candidates possess efficient readthrough activity with significantly lower cytotoxicity and better metabolic stability compared to the competitor drug Ataluren. The compounds were licensed from the University of Palermo and the Italian Cystic Fibrosis Foundation.

Other names
CCM-848 + CCM-914 + CCM-930
02

Targets

80S ribosome (Ribosome and ribosomal components)

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