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GT 00513

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Drug Profile

GT 00513

Alternative Names: GT-00513

Latest Information Update: 12 Jan 2024

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At a glance

  • Originator Gain Therapeutics
  • Class Small molecules
  • Mechanism of Action Beta-galactosidase expression 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

  • Preclinical GM1 gangliosidosis

Most Recent Events

  • 01 Dec 2023 Preclinical trials in GM1-Gangliosidoses in Switzerland (PO)

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