Potential ASO Therapies for Patients With GBA Mutations Ready for Testing

Q-State Biosciences has developed a set of optimized antisense oligonucleotides (ASOs) that may serve as potential therapies for Parkinson’s disease patients with specific mutations, the company announced. These ASO candidates are designed for patients carrying mutations in the GBA gene, a key genetic risk factor for the disease’s development. Their…

Molecule Delivered in Small Fatty Vesicles May Be Potential Parkinson’s Therapy, Mouse Study Shows

Delivering an alpha-synuclein-targeting molecule, called ASO4, in tiny fatty vesicles reduced toxic alpha-synuclein clumps and dopaminergic neuron loss — two main hallmarks of Parkinson’s disease — and lessened motor impairments in a mouse model of the disease. Results of the study add to previous findings suggesting that these…

Silverstein Foundation, Q-State Launch Company Aiming to Treat Parkinson’s Linked to GBA Mutations

The Silverstein Foundation for Parkinson’s with GBA together with Q-State Biosciences have launched Chamishi Therapeutics, a company aiming to develop innovative treatments for Parkinson’s disease and other neurodegenerative diseases. Chamishi will initially focus on developing antisense oligonucleotide (ASO) therapies for Parkinson’s patients carrying mutations in the…