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BTB 201

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Drug Profile

BTB 201

Alternative Names: BTB-201

Latest Information Update: 22 Jul 2025

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At a glance

  • Originator BTB Therapeutics
  • Class Small molecules
  • Mechanism of Action RNA splicing 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 Fabry's disease

Most Recent Events

  • 17 Jul 2025 Preclinical trials in Fabry's disease in Japan (unspecified route) prior to July 2025 (BTB Therapeutics pipeline, July 2025)

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