OTL-205

**OTL-205** is an investigational **ex vivo autologous hematopoietic stem cell gene therapy** being developed for **amyotrophic lateral sclerosis**. Publicly available company and pipeline materials identify it as a **preclinical** Orchard Therapeutics neurodegenerative disease program developed in collaboration with the **University of Padua** and Professor Alessandra Biffi, with Orchard having disclosed a sponsored research agreement with the University of Padua and an exclusive option with Boston Children's Hospital related to the program. Limited public detail is available on the precise transgene construct and final product composition, but secondary pipeline information describes the approach as genetically modifying microglia to reduce **NADPH oxidase 2** activity, consistent with a microglia-directed, hematopoietic stem cell-based neuroinflammation-modulating strategy for ALS.