Genespire Srl is a biotechnology company based in Milan, Italy, developing off-the-shelf gene therapies for children and adults with genetic diseases. It was founded in March 2020 as a spin-off of the San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), co-founded by Prof. Luigi Naldini, Dr. Alessio Cantore, Fondazione Telethon and Ospedale San Raffaele. Its platform uses immune shielded lentiviral vectors (ISLVs), designed to be administered intravenously so that the patient's liver produces the therapy throughout life.

The lead candidate, GENE202, targets methylmalonic acidemia (MMA), a rare metabolic disorder with no approved disease-modifying treatments. Preclinical data were presented at the ASGCT Annual Meeting in 2024 and 2025, and durable preclinical efficacy in mouse models was reported in July 2026. GENE202 received FDA and EC orphan drug designation in January 2026, and the company planned to take it to the clinic by the end of 2026. An international observational study, InforMMA, began enrolling MMA patients in August 2026. The company's pipeline also covers primary immunodeficiencies and other inherited metabolic diseases.

Genespire raised a €16M Series A led by Sofinnova Partners in April 2020 and a €46.6M (~$52M) Series B in September 2024, co-led by Sofinnova Partners, XGEN Venture and CDP Venture Capital, with Indaco SGR participating. Karen Aiach-Pignet was appointed Chief Executive Officer in September 2024, and the company's website lists Lucia Faccio, a partner at Sofinnova Partners, as chief executive officer since 2025.