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MRT 5201

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Drug Profile

MRT 5201

Alternative Names: MRT-5201

Latest Information Update: 17 Sep 2021

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At a glance

  • Originator Translate Bio
  • Class RNA
  • Mechanism of Action Ornithine carbamoyltransferase expression stimulants
  • Orphan Drug Status

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

    Yes - Ornithine carbamoyltransferase deficiency disease
  • New Molecular Entity Yes

Highest Development Phases

  • Discontinued Ornithine carbamoyltransferase deficiency disease

Most Recent Events

  • 14 Sep 2021 Translate Bio has been acquired by Sanofi
  • 12 Sep 2019 Translate Bio withdraws a phase I/II trial in Ornithine transcarbamylase deficiency as the program was discontinued (IV) (NCT03767270)
  • 09 Sep 2019 Discontinued - Preclinical for Ornithine carbamoyltransferase deficiency disease in USA (IV)

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