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AVP-6 is a synthetic peptide therapeutic being developed by Phoenix Nest Inc. for the treatment of Sanfilippo syndrome (Mucopolysaccharidosis type III or MPS III) Types A, B, C, and D. Administered intranasally for nose-to-brain delivery, AVP-6 is designed to modulate neuronal synaptic transmission and correct synaptic dysfunction, which is a hallmark of this neurodegenerative lysosomal storage disorder. Unlike enzyme replacement or gene therapies that target specific genetic defects, AVP-6 employs a disease-agnostic approach to address the neurological symptoms of the condition. It is currently in the discovery phase of development.
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