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Research programme: Krabbe disease therapeutics - Gain Therapeutics

Drug Profile

Research programme: Krabbe disease therapeutics - Gain Therapeutics

Alternative Names: GALC programme - Gain Therapeutics; Krabbe disease therapeutics - Gain Therapeutics

Latest Information Update: 18 Apr 2024

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At a glance

  • Originator Gain Therapeutics
  • Class Neuroprotectants; Pharmacological chaperones; Small molecules
  • Mechanism of Action Enzyme stimulants; Galactosylceramidase modulators
  • Orphan Drug Status

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

  • New Molecular Entity Yes

Highest Development Phases

  • Preclinical Globoid cell leukodystrophy

Most Recent Events

  • 18 Apr 2024 Preclinical trials in Globoid cell leukodystrophy in USA (PO) (Gain Therapeutics Pipeline, April 2024)
  • 15 Dec 2021 Gain Therapeutics plans to initiate animal proof of concept study in Globoid cell leukodystrophy in 2022
  • 15 Dec 2021 Gain Therapeutics intends to file IND application for Globoid cell leukodystrophy in 2023
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