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Healiva, C4U Join Forces to Develop CRISPR-Cas3 Drugs for Rare Skin Conditions

NEW YORK – Two biotech firms, Healiva and C4U, on Monday said they will jointly develop CRISPR-Cas3-based drugs for epidermolysis bullosa (EB), a rare genetic disorder that causes the skin to be highly sensitive and easily blister.

Lugano, Switzerland-based Healiva develops therapeutics for acute and chronic wounds, and Osaka, Japan-based C4U develops rare disease therapeutics using its CRISPR-Cas3 gene-editing platform. In inking a strategic alliance, the two firms have agreed to codevelop a gene-editing therapy for EB through a staged development process that leverages Healiva's experience developing gene therapies and uses C4U's gene-editing platform for manufacturing the drugs.

EB refers to a group of rare skin conditions largely caused by inherited genetic mutations. The most common type, EB simplex, is most often due to mutations in the KRT5 or KRT14 genes, which normally produce proteins that make the outer layer of the skin tough and resilient.

This latest agreement makes Healiva and C4U players in the emerging cell and gene therapy market for inherited skin disorders. In May, the US Food and Drug Administration approved the first topical gene therapy, Krystal Biotech's Vyjuvek (beremagene geperpavec-svdt), for treating wounds related to dystrophic EB, a form of the disorder caused by mutations in the COL7A1 gene.

Abeona Therapeutics in August also said it will submit a biologics license application with the FDA for its investigational autologous cell therapy EB-101 as a treatment to heal wounds on patients with recessive dystrophic epidermolysis bullosa caused by a lack of the COL7A1 protein.