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ACP-ADA is a preclinical enzyme replacement therapy (ERT) developed by Avixgen for the treatment of Severe Combined Immunodeficiency due to Adenosine Deaminase deficiency (ADA-SCID). The therapeutic consists of the adenosine deaminase (ADA) enzyme conjugated to Avixgen's proprietary ACP™ (Avixgen Cell Penetrating) technology. Unlike conventional ERTs that primarily clear toxic metabolites from the extracellular space, the ACP™ conjugation is designed to facilitate the intracellular delivery of the ADA enzyme, potentially offering a more comprehensive restoration of immune function by addressing the metabolic defect within the cells themselves.
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