Freeline Therapeutics announced plans to leverage knowledge gained from its FLT201 gene therapy program in Gaucher disease to advance a similar candidate to treat people whose Parkinson’s is tied to mutations in the GBA1 gene. “Our GBA1-linked Parkinson’s disease program is a natural extension of our…

Having longer-than-normal fatty molecules and an inactive enzyme called cathepsin-B in nerve cells’ recycling centers may increase the risk of developing Parkinson’s disease in people with mutations in the GBA1 gene, the disease’s most common genetic risk factor. This is the hypothesis generated by a recent study, titled “Pathological…

Gain Therapeutics announced that preclinical studies with two of its compounds, GT-02287 and GT-02329, showed they could reduce the alpha-synuclein aggregates that characterize Parkinson’s and increase glucocerebrosidase protein levels in cell models of Parkinson’s and Gaucher disease. GT-02287 and GT-02329 are known as STARs, which stands for structurally targeted…

Vanqua Bio has raised $85 million in funding to accelerate the development of its small molecule technology platform for treating neurodegenerative disorders such as Parkinson’s disease. Led by Omega Funds, the Series B financing will advance the research of Dimitri Krainc, MD, PhD, of Northwestern University, an…

Two investigational small molecules known as GT-02287 and GT-02329 lowered toxic protein levels in patient-derived cell models of Parkinson’s disease associated with a mutation in GBA1, their developer, Gain Therapeutics, announced. Gain is planning additional preclinical studies this year that could support applications to regulatory agencies allowing the compounds to…

Increased levels of certain lipids in the brain may be an early sign of Parkinson’s disease (PD), and could help identify patients at risk, as well as provide opportunities for early treatment, a study has found. The study, “Glycosphingolipid levels and glucocerebrosidase activity are altered in…

Mutations of genes responsible for breaking down and recycling cell waste products may promote Parkinson’s disease, a study shows. Scientists have known for some time that deficiencies in these lysosomal storage genes cause Gaucher disease and other disorders. The study, “Excessive burden of lysosomal storage disorder gene…

Researchers identified a gene that makes cognitive problems much more likely in people with Parkinson’s disease. The findings not only help to explain why some patients develop memory problems, but may also support trials leading to a more personalized treatment approaches. The study, “Specifically neuropathic Gaucher’s mutations accelerate cognitive…

While investigating links to Gaucher disease — a rare genetic condition with strong ties to Parkinson’s — researchers collaborating across three National Institutes of Health (NIH) centers reported finding a drug molecule potentially capable of treating Parkinson’s by reducing the clusters of aggregated α-synuclein inside cells. In doing so, the research…