Spur Therapeutics selects gene therapy development candidate for Parkinson’s disease with GBA1 mutations
Dec. 4, 2024
Spur Therapeutics Ltd. has selected SPR-301 as lead development candidate from its gene therapy program for a genetically defined subset of Parkinson’s disease characterized by mutations in the GBA1 gene. The mutations cause a deficiency in the enzyme glucocerebrosidase (GCase), leading to the accumulation of α-synuclein and subsequent death of neuronal cells that are hallmarks of Parkinson’s disease.