A privately held Italian company has raised $52 million to progress development of an in vivo gene therapy to treat methylmalonic acidemia (MMA), a genetic disorder that is manifest in early infancy. Genespire Srl is a 2020 spin-out of the San Raffaele Telethon Institute for Gene Therapy, a cell and gene therapy research institute. The funds will support development of the company’s lead product, GENE202, up to a clinical Phase 1/2 trial.
