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GT 02287

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Drug Profile

GT 02287

Alternative Names: GT-02287

Latest Information Update: 28 Oct 2025

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At a glance

  • Originator Gain Therapeutics
  • Class Antidementias; Antineoplastics; Antiparkinsonians; Neuroprotectants; Pharmacological chaperones; Small molecules
  • Mechanism of Action Galactosylceramidase modulators
  • Orphan Drug Status

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

    No
  • New Molecular Entity Yes

Highest Development Phases

  • Phase I Parkinson's disease
  • Preclinical Alpha 1-antitrypsin deficiency; Globoid cell leukodystrophy; GM1 gangliosidosis; Solid tumours
  • Research Dementia
  • No development reported Gaucher's disease

Most Recent Events

  • 28 Oct 2025 No recent reports of development identified for preclinical development in Gaucher's-disease in USA (PO, Pill)
  • 06 Oct 2025 Efficacy and pharmacokinetics data from a phase I trial in Parkinson's disease released by Gain Therapeutics
  • 30 Jun 2025 Adverse events data from a phase Ib trial in Parkinson's disease released by Gain Therapeutics

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