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Orphan Technologies

Ownership: Public · TVTX
Development stage: Other

Overview

The lead program, OT-58, is a PEGylated recombinant enzyme replacement therapy for classical homocystinuria (HCU), which in Phase 1/2 demonstrated significant homocysteine reduction in preclinical and early clinical data. The company was acquired by Retrophin (now Travere Therapeutics) in 2020 before advancing to late-stage development[1][2].

Therapeutic areas: Metabolic DisordersRare Diseases
Modalities: Enzyme replacement therapyRecombinant proteinPEGylated protein
Industry: Biotech

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