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NXL-003 is a preclinical in vivo cell therapy candidate developed by NeuExcell Therapeutics for the treatment of Huntington's Disease, Amyotrophic Lateral Sclerosis (ALS), and Traumatic Brain Injury (TBI). The therapy utilizes NeuExcell's proprietary Astrocyte-to-Neuron (ATN) platform, which leverages adeno-associated virus (AAV) vectors to deliver neural transcription factors directly to the brain. These factors reprogram endogenous reactive astrocytes—which typically proliferate following brain injury or neurodegeneration—into functional, integrated neurons. By regenerating lost neuronal populations and repairing damaged neural circuits, NXL-003 aims to provide a regenerative approach to treating severe neurological disorders where traditional therapies have failed to reverse disease progression.
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