LY3884961

LY3884961 (also known as PR001) is an investigational gene therapy developed for the treatment of Parkinson's disease with GBA1 mutations and Gaucher disease, including both neuronopathic and non-neuronopathic forms[1][2][5][8]. The therapy uses an adeno-associated virus serotype 9 (AAV9) vector to deliver a functional copy of the human GBA1 gene directly into the central nervous system via intracisternal injection[2][3][8]. The GBA1 gene encodes glucocerebrosidase, a lysosomal enzyme essential for glycosphingolipid metabolism. Mutations in this gene lead to reduced enzyme activity, resulting in substrate accumulation that contributes to Gaucher disease and increases risk for Parkinson’s disease. By restoring normal glucocerebrosidase levels, LY3884961 aims to reduce pathological substrate buildup and improve neurological function[2][5][8]. Preclinical studies have shown increased enzyme activity, reduced glycolipid accumulation, improved motor function in animal models, and decreased insoluble α-synuclein—a key component of Lewy bodies implicated in Parkinson’s pathology[2].

Other names

LY3884961LY-3884961LY 3884961PR001APR-001APR 001AAAV9.CBA.GBA1.AAAV-9.CBA.GBA1.AAAV 9.CBA.GBA1.A