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Homocystinuria gene therapy - Tome Biosciences

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Drug Profile

Homocystinuria gene therapy - Tome Biosciences

Latest Information Update: 05 Aug 2024

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At a glance

  • Originator Tome Biosciences
  • Class Gene therapies
  • Mechanism of Action Cystathionine beta-Synthase replacements; Gene transference
  • Orphan Drug Status

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

    No
  • New Molecular Entity No

Highest Development Phases

  • Research Homocystinuria

Most Recent Events

  • 02 Aug 2024 Early research in Homocystinuria in USA (Parenteral) (Tome Biosciences pipeline, August 2024)

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