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GCB 002

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Drug Profile

GCB 002

Alternative Names: GCB-002

Latest Information Update: 10 Jan 2025

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At a glance

  • Originator Genecombio
  • Class Gene therapies
  • Mechanism of Action Methyl-CpG-binding protein 2 gene replacements
  • Orphan Drug Status

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

    No
  • New Molecular Entity No

Highest Development Phases

  • Preclinical Rett syndrome

Most Recent Events

  • 18 Dec 2024 Preclinical trials in Rett syndrome in China (Intrathecal)
  • 18 Dec 2024 Genecombio plans a clinical trial for Rett Syndrome (In children), in December 2024 (Intrathecal, Injection) (NCT06739434)

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