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Inozyme Pharma

Boston, Massachusetts, United Statesinozyme.com ↗

Ownership: Public · INZY
Employees: ~67
Development stage: Phase 2

Overview

Positive interim results in infants and young children with ENPP1 Deficiency showed improvements in survival, heart function, and stabilization or reduction in ectopic calcification and hypophosphatemia. Enrollment is complete in ENERGY 3 pivotal trial in pediatric patients with ENPP1 Deficiency, with topline data expected in early 2026. The company has received regulatory guidance from FDA and EMA for the planned ASPIRE pivotal trial for ABCC6 Deficiency in children.[6]

Therapeutic areas: RheumatologyRare DiseasesNephrology
Modalities: Enzyme Replacement Therapy (ERT)Gene therapy
Industry: Biotech

Drug pipeline

1 asset

INZ-701

Phase 2

Indications: ENPP1 Deficiency, ABCC6 Deficiency, Calciphylaxis

Partnerships

Yale University

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