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GT 02287

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Drug Profile

GT 02287

Alternative Names: GT-02287

Latest Information Update: 03 Jul 2025

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At a glance

  • Originator Gain Therapeutics
  • Class Antidementias; Antineoplastics; Antiparkinsonians; Neuroprotectants; Pharmacological chaperones; Small molecules
  • Mechanism of Action Alpha 1-antitrypsin modulators; Galactosidase modulators; 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; Gaucher's disease; Globoid cell leukodystrophy; GM1 gangliosidosis; Solid tumours
  • Research Dementia

Most Recent Events

  • 30 Jun 2025 Adverse events data from a phase Ib trial in Parkinson's disease released by Gain Therapeutics
  • 30 Jun 2025 Gain Therapeutics plans a phase II trial for Parkinson's disease in early 2026
  • 11 Apr 2025 Interim pharmacodynamics data from a preclinical studies in Parkinson’s disease released by Gain Therapeutics

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