Potential ASO Therapies for Patients With GBA Mutations Ready for Testing

Potential ASO Therapies for Patients With GBA Mutations Ready for Testing
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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 development is the first milestone reached under an agreement between Q-State and Chamishi Therapeutics, a company it launched together with the Silverstein Foundation for Parkinson’s with GBA. Chamishi focuses on creating treatments for Parkinson’s and other neurodegenerative diseases.

With this milestone realized, Q-State is entitled to a pre-specified grant of equity in Chamishi.

“We are very pleased with the progress achieved during our collaboration with Q-State and look forward to advancing this program towards studies in humans,” Jim Sullivan, CEO of Chamishi, said in a press release.

The GBA gene has the instructions to produce the enzyme beta-glucocerebrosidase (GCase), which is important for the digestion and recycling of different types of molecules. This occurs in cellular structures called lysosomes.

GBA mutations that impair the normal activity of the GCase enzyme lead to toxic waste accumulating inside cells, and subsequently to inflammation. Mutations in the GBA gene are present in 7%–10% of all Parkinson’s cases. Aging itself is also associated with impaired lysosome function.

ASOs are small molecules that bind to RNA, the molecule that carries the instructions from DNA to make proteins, to correct genetic defects and rescue the production of a particular protein. Designed using Q-State’s proprietary technology, the newly optimized ASOs target a molecular player in neuroinflammation. The target’s identity was not disclosed.

Enzymes are globular proteins that act as catalysts of specific reactions within cells.

“Selectively targeting components of the immune system within the CNS [central nervous system, which includes brain and spinal cord] represents an important therapeutic approach to addressing the needs of patients with devastating neurological diseases,” said Sullivan.

Added Paul Medeiros, the CEO of Q-State: “This collaboration demonstrates the successful application of Q-State’s advanced technology-derived discovery capabilities in a challenging disease domain within the CNS. We are hopeful that these efforts may provide a favorable foundation for advancing new therapeutics for patients with GBA-Parkinson’s and related neurodegenerative disorders.”

Patricia holds a Ph.D. in Cell Biology from University Nova de Lisboa, and has served as an author on several research projects and fellowships, as well as major grant applications for European Agencies. She has also served as a PhD student research assistant at the Department of Microbiology & Immunology, Columbia University, New York.
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José is a science news writer with a PhD in Neuroscience from Universidade of Porto, in Portugal. He has studied Biochemistry also at Universidade do Porto and was a postdoctoral associate at Weill Cornell Medicine, in New York, and at The University of Western Ontario, in London, Ontario. His work ranged from the association of central cardiovascular and pain control to the neurobiological basis of hypertension, and the molecular pathways driving Alzheimer’s disease.
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Patricia holds a Ph.D. in Cell Biology from University Nova de Lisboa, and has served as an author on several research projects and fellowships, as well as major grant applications for European Agencies. She has also served as a PhD student research assistant at the Department of Microbiology & Immunology, Columbia University, New York.
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