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PBFT 02

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Drug Profile

PBFT 02

Alternative Names: AAV-Granulin Gene Therapy; AAV1-GRN vector; PBFT-02

Latest Information Update: 27 Jun 2025

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At a glance

  • Originator University of Pennsylvania
  • Developer Passage Bio
  • Class Antidementias; Gene therapies
  • Mechanism of Action Gene transference; Progranulin protein replacements
  • Orphan Drug Status

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

    Yes - Frontotemporal dementia
  • New Molecular Entity No

Highest Development Phases

  • Phase I/II Frontotemporal dementia
  • Preclinical Amyotrophic lateral sclerosis; CNS disorders

Most Recent Events

  • 27 Jun 2025 9445146- updated KDM, AE, PK, TT, HE added
  • 23 Jun 2025 Interim efficacy, adverse events, pharmacokinetics data from phase I/II clinical trials in Frontotemporal dementia released by Passage Bio
  • 04 Mar 2025 Passage Bio plans to seek regulatory feedback on pivotal trial design for Frontotemporal Dementia in 1H 2026

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