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EGT 201

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Drug Profile

EGT 201

Alternative Names: AAV9 alpha N-acetylglucosaminidase gene therapy - Esteve/UAB; EGT-201

Latest Information Update: 28 Feb 2025

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At a glance

  • Originator Universitat Autonoma de Barcelona
  • Developer ESTEVE; Universitat Autonoma de Barcelona
  • Class Gene therapies
  • Mechanism of Action Alpha N acetyl D glucosaminidase replacements; Gene transference
  • Orphan Drug Status

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

    Yes - Mucopolysaccharidosis III
  • New Molecular Entity No

Highest Development Phases

  • No development reported Mucopolysaccharidosis III

Most Recent Events

  • 28 Feb 2025 No recent reports of development identified for preclinical development in Mucopolysaccharidosis-III in Spain (Parenteral)
  • 10 Feb 2023 EGT 201 is still in preclinical phase for Mucopolysaccharidosis-III in Spain (Parenteral) (ESTEVE pipeline; February 2023)
  • 28 Jun 2022 No recent reports of development identified for preclinical development in Mucopolysaccharidosis-III in Spain (Parenteral)

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