Category
Current Projects, Gene Therapy, Quality of Life Change
About This Project

Development of a topical gene-editing treatment to restore Collagen VII

Investment summary

The lead product in development, BrB-101, uses Branca Bunús’ non-viral polymer platform technology to deliver a gene editing treatment directly to open wounds of patients with Recessive Dystrophic Epidermolysis Bullosa (RDEB). This non-invasive approach aims to permanently remove the mutations (faults) which cause the skin fragility and restore functional Collagen VII.

Cure EB invested in Branca Bunús alongside Enterprise Ireland and other investors.

Scientific Summary

The lead product in development, BrB-101, is targeted to treat and excise disease causing mutations in exon 80 of the COL7A1 gene, leading to a restoration of patients’ own Collagen VII protein. Applied topically to open RDEB wounds, BrB-101 uses Branca Bunús’ patented polymer-based gene delivery platform technology to carry CRISPR-Cas to PAM sequences adjacent to Exon 80 mutations in the COL7A1 gene. This non-invasive approach can restore functional Collagen VII production, thus anchoring fibril formation and structural integrity to patients’ skin.