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GT 02287

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Drug Profile

GT 02287

Alternative Names: GT-02287

Latest Information Update: 26 Dec 2024

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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; Glucosylceramidase stimulants
  • 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

  • 23 Dec 2024 Gain Therapeutics initiates enrolment in a phase Ib trial for Parkinson's disease in Australia (NCT06732180)
  • 13 Dec 2024 Gain Therapeutics plans a phase I trial for Parkinson's disease in Australia (PO) in January 2025 (NCT06732180)
  • 22 Nov 2024 Gain Therapeutics plans pre-IND meeting with the USFDA by the end of 2024

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