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Orph-001

Development stage: Preclinical
Lead developer: Orphazyme
Modality: Recombinant Proteins and Enzymes
Administration: Intravenous

Overview

Orph-001 is a recombinant human heat-shock protein 70 (HSP70) developed by the Danish biopharmaceutical company Orphazyme. It is designed to treat lysosomal storage diseases by leveraging HSP70 chaperone biology, which originated from discoveries at the Danish Cancer Society. The drug aims to address the significant unmet medical need in these conditions by assisting in protein folding and function.

Targets

Bis(monoacylglycero)phosphate-dependent lysosomal enzyme complexHSPA1A (HSP70-1A)

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