Genespire raises €46.6 million in a Series B round to advance its first pediatric in-vivo gene therapy into the clinic

MMA has onset in early infancy, is associated with significant mortality and morbidity, and is characterized by severe clinical manifestations including muscular weakness, seizures, developmental delays and organ damage. Currently, there are no disease-modifying treatments for MMA. GENE202 is a pioneering off-the-shelf gene therapy which harnesses the company’s Immune Shielded Lentiviral Vector (ISLV) platform. ISLVs are designed to be used intravenously, enabling the patient's liver to produce the therapy throughout its lifetime. This unique mode of action makes Genespire’s approach optimally suited to address pediatric patients affected by genetic diseases who currently face the most pressing unmet medical needs, while also benefitting adult patients. The ISLV technology was developed by Genespire’s scientific co-founders and gene therapy leaders, Pr. Luigi Naldini and Dr. Alessio Cantore, at the San Raffaele Telethon Institute for Gene Therapy (SR-Tiget).

This Series B financing round will also strengthen the Company’s pipeline by fostering discovery and preclinical work on candidate products targeting several additional genetic diseases.