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ECUR 506

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Drug Profile

ECUR 506

Alternative Names: ECUR-506; GTP-506; iECURE-OTC

Latest Information Update: 16 Jan 2025

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At a glance

  • Originator iECURE
  • Class Gene therapies
  • Mechanism of Action Gene transference; Ornithine carbamoyltransferase replacements
  • Orphan Drug Status

    Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

    Yes - Ornithine carbamoyltransferase deficiency disease
  • New Molecular Entity No

Highest Development Phases

  • Phase I/II Ornithine carbamoyltransferase deficiency disease

Most Recent Events

  • 09 Jan 2025 Efficacy and adverse events data from the phase I/II OTC-HOPE trial in Ornithine carbamoyltransferase deficiency disease released by iECURE
  • 08 May 2024 ECUR 506 receives Fast Track designation for Ornithine carbamoyltransferase deficiency disease [IV,Infusion] in USA
  • 30 Apr 2024 Phase-I/II clinical trials in Ornithine carbamoyltransferase deficiency disease (In infants, In neonates) in Australia, Spain (IV) (NCT06255782)

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