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Tx 01

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Drug Profile

Tx 01

Alternative Names: Tx-01-Theranexus

Latest Information Update: 12 Feb 2026

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At a glance

  • Originator Theranexus
  • Class Small molecules
  • Mechanism of Action Undefined mechanism
  • 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

  • Research Gaucher's disease; Niemann-Pick disease type C

Most Recent Events

  • 10 Feb 2026 Tx 01 licensed to Biocodex in USA and Canada
  • 17 Dec 2024 Tx 01 licensed to Exeltis in European Union, Latin America, United Kingdom and Middle East
  • 17 Dec 2024 Early research in Gaucher's disease in France (unspecified route)

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