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PPL 001

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Drug Profile

PPL 001

Alternative Names: PPL-001

Latest Information Update: 05 Nov 2024

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At a glance

  • Originator Papillon Therapeutics
  • Class Gene therapies; Haematopoietic stem cells therapies
  • Mechanism of Action Frataxin expression stimulants
  • Orphan Drug Status

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

    Yes - Friedreich's ataxia
  • New Molecular Entity No

Highest Development Phases

  • Preclinical Friedreich's ataxia

Most Recent Events

  • 15 Oct 2024 PPL 001 receives rare pediatric disease designation in USA for Friedreich's ataxia
  • 17 Sep 2024 Preclinical trials in Friedreich's ataxia in USA (Parenteral) prior to September 2024 (Papillon Therapeutics pipeline; September 2024)
  • 17 Sep 2024 PPL 001 receives Orphan Drug status for Friedreich's ataxia in USA

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