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Drug Profile


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

Latest Information Update: 22 Jul 2024

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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

Most Recent Events

  • 16 Jul 2024 Passage Bio plans to submit revised trial protocol to US FDA for phase-I/II upliFT-D trial for Frontotemporal dementia and expects to initiate dosing of FTD-C9orf72 patients in 1H 2025
  • 16 Jul 2024 Passage Bio receives positive feedback from the US FDA in Type C meeting for the protocol amendment for phase-I/II upliFT-D trial in Frontotemporal dementia
  • 04 Mar 2024 Phase-I/II clinical trials in Frontotemporal dementia (In adults, In the elderly) in European Union (Intracisternal) (NCT04747431)
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