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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: 28 Apr 2026

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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

  • 20 Apr 2026 Interim efficacy, adverse events, pharmacodynamics data from the phase-I/II upliFT-D trial in Frontotemporal dementia released by Passage Bio
  • 03 Mar 2026 Phase-I/II clinical trials in Frontotemporal dementia (In the elderly, In adults) in Australia (Intracisternal)
  • 27 Jul 2025 Adverse events data from a phase I/II upliFT-D trial in Frontotemporal dementia presented at the Alzheimer's Association International Conference 2025 (AAIC-2025)

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