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DT 216

Drug Profile

DT 216

Alternative Names: DT 216P2; DT-216; Syn TEF1

Latest Information Update: 26 Mar 2024

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At a glance

  • Originator Design Therapeutics
  • Class Small molecules
  • Mechanism of Action Frataxin expression stimulants; Genetic transcription modulators
  • Orphan Drug Status

    Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

  • New Molecular Entity Yes

Highest Development Phases

  • Phase I Friedreich's ataxia

Most Recent Events

  • 19 Mar 2024 Design Therapeutics plans a clinical trial for Friedreich ataxia in 2025
  • 31 Dec 2023 Design Therapeutics has patent pending applications and two pending Patent Cooperation Treaty applications for "compositions of matter and methods for the treatment of FA" in the US, European Union and multiple countries worldwide
  • 15 Aug 2023 Pharmacokinetics and adverse events data from a phase I trial in Friedreich Ataxia released by Design Therapeutics
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