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SDP1001 is a preclinical-stage therapeutic candidate developed by Scindy Pharmaceutical for the treatment of Propionic Acidemia (PA). PA is a rare, life-threatening autosomal recessive metabolic disorder caused by a deficiency of the enzyme propionyl-CoA carboxylase (PCC), which leads to the accumulation of toxic metabolites such as propionyl-CoA and methylcitrate. While the specific modality of SDP1001 has not been publicly disclosed by the sponsor, Scindy Pharmaceutical specializes in developing next-generation enzyme replacement therapies (ERT) and mRNA-based treatments for rare metabolic diseases. The program is currently in the discovery/preclinical phase, aiming to restore enzymatic function and prevent the metabolic crises associated with the disease.
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