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Research programme: cystinuria targeting adeno-associated virus gene therapy - BridgeBio Pharma

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Research programme: cystinuria targeting adeno-associated virus gene therapy - BridgeBio Pharma

Latest Information Update: 06 Sep 2023

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At a glance

  • Originator BridgeBio Pharma
  • Class Gene therapies; Urologics
  • Mechanism of Action 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 Cystinuria

Most Recent Events

  • 27 Jun 2023 Early research in Cystinuria in USA (Parenteral) (BridgeBio Pharma Pipeline, June 2023)

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