Genespire

Genespire is a biotechnology company based in Milan, Italy, focused on developing innovative gene therapies for severe genetic diseases.

Founded in March 2020 as a spin-out from the San Raffaele Telethon Institute for Gene Therapy, Genespire aims to create "off-the-shelf" therapies that can be administered directly into a patient's bloodstream. This approach simplifies treatment by eliminating the need for complex cell extraction and re-infusion procedures. The company utilizes its proprietary Immune Shielded Lentiviral Vectors (ISLVs) to deliver therapeutic genes directly to the liver, ensuring long-term expression and benefits for patients. Genespire is currently advancing its lead candidate, GENE202, targeting methylmalonic acidaemia, with plans to initiate clinical trials by the end of 2026. The company is dedicated to addressing high unmet medical needs in inherited metabolic diseases and primary immunodeficiencies, making gene therapy more accessible for both pediatric and adult patients.