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SBC-105
- Development stage
- Preclinical
- Lead developer
- Alexion Pharmaceuticals
- Modality
- Replacement Enzymes → Therapeutic Enzymes → Recombinant Proteins and Enzymes
01
Overview
SBC-105 is an enzyme replacement therapy developed by Synageva BioPharma (later acquired by Alexion Pharmaceuticals) for rare disorders of calcification, with its primary indication being Generalized Arterial Calcification of Infancy (GACI). It is designed to replace the deficient ectonucleotide pyrophosphatase/phosphodiesterase-1 (ENPP1) enzyme, which in healthy individuals generates pyrophosphate (PPi), a potent inhibitor of tissue calcification. GACI is caused by inherited mutations in the ENPP1 gene, leading to severe calcification of arteries, particularly affecting infants and children. The drug was in preclinical development.
02
Targets
ENPP1
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