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PBGENE PH1

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Drug Profile

PBGENE PH1

Alternative Names: HAO1 gene editing therapeutic; PBGENE-PH1

Latest Information Update: 28 Mar 2025

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At a glance

  • Originator Precision Biosciences
  • Class Endodeoxyribonucleases; Gene therapies; Urologics
  • Mechanism of Action Gene modulators
  • 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

  • No development reported Primary hyperoxaluria

Most Recent Events

  • 28 Mar 2025 No recent reports of development identified for preclinical development in Primary-hyperoxaluria in USA (Parenteral)
  • 16 May 2022 Pharmacodynamics data from a preclinical studies inPrimary hyperoxaluria released by Precision Biosciences
  • 09 Sep 2021 Precision Bioscience intends to submit IND or CTA application in 2023

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