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. 2024 Mar 10;16(4):870–884. doi: 10.1038/s44321-024-00048-8

Figure 6. Daclatasvir demonstrates strong preclinical efficacy in RDEB primary cells and an RDEB mouse model.

Figure 6

Endogenously-secreted matrix from primary RDEB patient fibroblasts can provide a model for medium-throughput screening of compounds for anti-fibrotic effect. Antivirals were identified as having a novel preventative effect on the dermal fibroblast matrix in RDEB. Daclatasvir, our hit compound of interest, inhibits fibrosis in vitro and in vivo through the modulation of the TGFβ pathway and impacting collagen in the matrix in an RDEB-specific mechanism. The RDEB mouse model experiences improved quality of life and longevity with daclatasvir treatment.