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Iduronicrin genleukocel-T - Immusoft

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Drug Profile

Iduronicrin genleukocel-T - Immusoft

Alternative Names: ISP-001; Mucopolysaccharidosis type I therapy - Immusoft

Latest Information Update: 30 Oct 2025

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At a glance

  • Originator Immusoft Corporation
  • Developer Immusoft
  • Class Cell therapies; Gene therapies
  • Mechanism of Action Iduronidase replacements
  • Orphan Drug Status

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

    Yes - Mucopolysaccharidosis I
  • New Molecular Entity No

Highest Development Phases

  • Phase I/II Mucopolysaccharidosis I

Most Recent Events

  • 30 Oct 2025 Phase-I/II clinical trials in Mucopolysaccharidosis I (In the elderly, In adults) in USA (Parenteral)
  • 21 Oct 2025 Iduronicrin genleukocel-T - Immusoft receives Fast Track designation for Mucopolysaccharidosis I [Parenteral] (In the elderly, In adults) in USA
  • 23 Sep 2024 Efficacy, adverse events and pharmacodynamics data from a phase I trial in Mucopolysaccharidosis I released by Immusoft

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