CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Q-State Biosciences (“Q-State”), a discovery technology and therapeutics company advancing programs for the treatment of central nervous system disorders, today announced a pipeline update introducing three advancing antisense oligonucleotide (ASO) discovery programs targeting rare neurodegenerative disorders. These disorders, each associated with high unmet medical need and a lack of approved disease-modifying therapeutics, include a specific monogenic subtype of ALS, a form of spinocerebellar ataxia (SCA), and dentatorubral-pallidoluysian atrophy (DRPLA). Each of these programs is powered by the company’s fully integrated platform that enables unique computational, artificial intelligence/machine learning-driven insights into central nervous system (CNS) disorders and candidate therapeutics.

“Q-State’s discovery technologies have wide-ranging utility across an array of CNS and other disorders, and we are excited to broaden our therapeutic development progress in several fields within neurodegeneration,” said Paul Medeiros, President and CEO of Q-State Biosciences.

Newly Identified Discovery Programs

• ALS Program: targeting an undisclosed monogenic form associated with rapid disease progression. Q-State has identified early ASO candidates that have achieved significant and potent target transcript reduction in patient cells.

• Spinocerebellar Ataxia (SCA) Program: targeting an undisclosed genetic form characterized by dysarthria, nystagmus, and ataxia of gait due to cerebellar dysfunction. Identified ASOs are well tolerated, demonstrate significant target transcript reduction, and reverse in vivo pathological features in animal models.

• Dentatorubral-Pallidoluysian Atrophy (DRPLA) Program: targeting a severe, progressive triplet repeat expansion disorder often characterized by ataxia, choreoathetosis, progressive dementia and cognitive decline. Q-State has identified early ASO candidates that have achieved significant and potent target transcript reduction in patient cells.

About Q-State Biosciences

Q-State Biosciences is a discovery technology and therapeutics company that applies a proprietary multi-discipline platform to discover genetically targeted therapeutics for neurodegeneration, epilepsy, pain and other disorders of the CNS. By integrating advanced human neuronal models, custom determinative measurement engineering and powerful AI/machine learning, we unlock unique insights into the biological complexity of the brain, its associated disease states and the creation of transformational medicines. For more information, please visit www.qstatebio.com.

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