Healiva and C4U working together to combat rare disease

Healiva from Lugano in the canton of Ticino and C4U, a biotechnology company based in the Japanese city of Osaka, will from now on be working together. The partners intend to develop therapeutic solutions on the basis of the CRISPR-Cas3 gene scissors to help in the fight against the rare disease epidermolysis bullosa (EB).

This genetic skin disease, which leads to painful blisters forming on the skin at the slightest rubbing or scratching, primarily affects young children. EB affects approximately 20 out of 1 million newborns in the USA, where between 25,000 and 50,000 children live with this debilitating disease.

“The collaboration aims to provide answers and hope to the individuals affected by EB, a devastating skin condition that renders the skin of the affected children as fragile as butterfly wings”, as Valerio M. Ferrari, Chairman of the Bioseutica Group and co-founder of Healiva, explains in a press release. It is for this reason that the youngsters impacted by this disease are often known as “butterfly children”.

The partnership between Healiva and C4U will focus on the development of CRISPR-Cas3 based gene therapy for EB, the press release states. For this, C4U will bring its proprietary gene scissors technology to the table. This is essentially a new generation of gene editing. With its CRISPR-Cas3 platform, C4U aims to develop alternative and complete cures for genetic diseases. As Dr. Priyanka Dutta Passecker, co-founder & CEO of Healiva, underlines in the press release, this partnership integrates “gene therapy prospects with our comprehensive cell therapy portfolio, leveraging our expertise in autologous (Epidex) and allogenic therapy technologies and the capabilities of our manufacturing infrastructure”. ce/mm