Leptogen

Leptogen is a preclinical-stage biotechnology company dedicated to developing novel treatments for Amyotrophic Lateral Sclerosis (ALS) and other neurodegenerative disorders. A spin-out from the University of Sheffield’s Institute for Translational Neuroscience (SITraN), the company’s research centers on the STMN2 (Stathmin-2) gene, which is essential for axonal repair and stability. In most ALS cases, the TDP-43 protein fails to regulate RNA correctly, leading to a depletion of STMN2; Leptogen aims to restore these levels using advanced gene therapy techniques. By addressing this fundamental pathological mechanism, the company seeks to preserve motor neuron function and improve outcomes for patients with TDP-43-linked diseases.