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Alpha-G Therapeutics is a preclinical-stage biotechnology company focused on developing next-generation enzyme replacement therapies (ERTs) for lysosomal storage disorders with significant central nervous system (CNS) involvement. The company utilizes its proprietary Alpha-G platform to engineer enzymes capable of crossing the blood-brain barrier, addressing a critical unmet need in the treatment of neuronopathic Gaucher disease (nGD) and GBA-associated Parkinson's disease. By delivering functional glucocerebrosidase (GBA1) directly to the brain, Alpha-G aims to halt neurodegeneration and improve clinical outcomes for patients with these devastating conditions. The company's approach represents a significant advancement over traditional ERTs, which are typically excluded from the CNS by the blood-brain barrier.
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