Descripción del proyecto
Each year, >250 babies are born without a thymus. These babies have a life expectancy of 2-3 years. The only cure is transplantation of thymus tissue, but this has a 30% mortality rate and costs almost >€2.5M. We developed i-Thymus, a revolutionary cure based on the patient’s own cells leveraging innovative gene therapy to generate thymus organoids that can be implanted minimally invasively in the patient (Fig. 1). We have already shown this works in functional proof of concept in vivo. With this EIC Accelerator, we will finalise development to enter the clinic for a Phase I/II trial, to enter the EU market with a pharma partner by 2028. After rapid market entry with ODD for complete DiGeorge syndrome, we will leverage this clinical success and our first revenues to develop i-Thymus for unmet needs of hundreds of thousands of other patients, starting with AML patients who have lost thymus function after high-dose chemotherapy for stem cell transplantation.