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PR 001

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Drug Profile

PR 001

Alternative Names: GBA1 Gene Therapy - Eli Lilly and Company; LY-3884961; PR-001; PR001A

Latest Information Update: 19 Feb 2025

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At a glance

  • Originator Prevail Therapeutics
  • Class Antiparkinsonians; Gene therapies; Neuroprotectants
  • Mechanism of Action Gene transference; Glucosylceramidase replacements
  • Orphan Drug Status

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

    Yes - Gaucher's disease
  • New Molecular Entity No

Highest Development Phases

  • Phase II Gaucher's disease; Parkinson's disease

Most Recent Events

  • 20 Dec 2022 Phase-I/II clinical trials in Gaucher's disease (In adults) in USA (IV) (NCT05487599)
  • 04 Aug 2022 Prevail Therapeutics and Eli Lilly plans a phase-I/II PROCEED trial for Gaucher's disease (In adults) in USA (IV, infusion) in August 2022 (NCT05487599)
  • 01 May 2022 US FDA accepts an IND application to study PR 001 for Type 1 Gaucher disease in a phase I/II clinical trial in the US

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