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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: 13 Jan 2026

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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

  • 07 Jan 2026 ECUR 506 receives an Innovation Passport under the United Kingdom’s Innovative Licensing and Access Pathway (ILAP)
  • 07 Jan 2026 ECUR 506 receives Regenerative Medicine Advanced Therapy (RMAT) status for Ornithine carbamoyltransferase deficiency disease in USA
  • 07 Jan 2026 iECURE announces intention to submit BLA to the US FDA for Ornithine carbamoyltransferase deficiency disease

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