PR 004

PR 004 is an investigational, single-dose, adeno-associated virus (AAV) gene therapy being developed by Prevail Therapeutics, now a subsidiary of Eli Lilly and Company, for the treatment of specific synucleinopathies, a group of neurodegenerative disorders characterized by pathological aggregation of alpha‑synuclein in the central nervous system. It is described as a glucosylceramidase replacement gene transfer therapy, aiming to deliver a functional copy of the relevant gene to restore lysosomal enzyme activity and thereby reduce alpha‑synuclein pathology and modify disease progression, building on Prevail’s AAV9-based platform used for other neurodegenerative indications.[3][4][6][7]