Healiva and C4U Corporation: Alliance for Epidermolysis bullosa gene therapy

Epidermolysis bullosa is a rare genetic skin disorder characterized by highly sensitive skin that blisters and erodes with minimal friction or trauma, such as rubbing or scratching. This condition predominantly affects young children, causing them to experience severe pain and a substantial reduction in their quality of life due to the constant formation of blisters in response to minor injuries. EB affects approximately 20 out of every 1 million newborns in the United States where 25,000 to 50,000 young children are living with this debilitating condition.

“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,” said Valerio M. Ferrari, Bioseutica Group Chairman & Co-Founder of Healiva.

The collaborative endeavor between Healiva and C4U will focus on the development of CRISPR-Cas3 based gene therapy for EB. In this collaboration the parties will work together to develop a gene therapy for EB with a staged development process to maximize speed and approval to perform clinical studies. By combining their complementary expertise, resources, and shared vision, both entities will strategically collaborate to enhance the quality of life for young children globally affected worldwide by this rare skin condition.

“By utilizing its proprietary CRISPR-Cas3 technology, a new generation of gene editing technology, C4U’s focus is to develop alternative and complete cures to genetic diseases. Skin diseases, especially EB, are relevant targets for the CRISPR-Cas3 platform, and we believe that we will bring a bright future to EB patients throughout the world,” said Akimitsu Hirai, President & CEO of C4U.

"This collaboration 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," articulated Dr. Priyanka Dutta Passecker, Co-Founder & CEO of Healiva.