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.
Spur Therapeutics Ltd., formerly Freeline Therapeutics, has announced new data from its GBA1 Parkinson’s disease research program. In a subset of Parkinson’s disease patients with mutations in the GBA1 gene, such mutations lead to a deficiency of the glucocerebrosidase (GCase) enzyme and the accumulation of harmful substrates.
Quoted biotech investor Syncona Ltd. is still struggling with the fallout from one of the worst bear markets on record, reporting its net asset value fell from £1.25 billion (US$1.58 billion) to £1.24 billion in its 2023-24 financial results on June 20. That is despite a restructuring toward more mature companies, with 70% of the portfolio now invested in clinical-stage assets.
