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AP-101 is a recombinant human monoclonal antibody designed to target and bind specifically to misfolded forms of the superoxide dismutase 1 (SOD1) protein. Misfolding of SOD1 is implicated in both familial and sporadic forms of amyotrophic lateral sclerosis (ALS), also known as motor neuron disease (MND). By binding to these aberrant proteins, AP-101 aims to promote their removal via phagocytosis, potentially slowing neuronal damage and disease progression. The drug is administered intravenously and has shown promising preclinical results in slowing disease progression. AP-101 was developed using Neurimmune’s Reverse Translational Medicine technology platform and is currently being evaluated in Phase 2 clinical trials for ALS/MND[2][3][4][5][6][7].
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