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BREN02 (hEN1) is a recombinant human Engrailed-1 (EN1) protein therapy being developed by BrainEver for the treatment of Amyotrophic Lateral Sclerosis (ALS). EN1 is a homeoprotein transcription factor that is essential for the survival and maintenance of alpha-motoneurons in the spinal cord. The therapeutic approach involves the delivery of the recombinant EN1 protein, which utilizes the unique ability of homeoproteins to be internalized by cells and translocate to the nucleus. Once inside the target neurons, BREN02 regulates gene expression to provide neuroprotective and restorative effects, aiming to slow the progression of neurodegeneration and preserve motor function in patients with ALS.
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