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LX 1004

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Drug Profile

LX 1004

Alternative Names: LX-1004

Latest Information Update: 21 Oct 2022

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At a glance

  • Originator Weill Cornell Medicine
  • Developer LEXEO Therapeutics; Weill Cornell Medicine
  • Class Gene therapies; Neuroprotectants
  • Mechanism of Action Gene transference
  • Orphan Drug Status

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

    Yes - Neuronal ceroid lipofuscinosis
  • New Molecular Entity No

Highest Development Phases

  • Phase I/II Neuronal ceroid lipofuscinosis

Most Recent Events

  • 18 Oct 2022 LEXEO Therapeutics plans an end of phase I/II meeting with US FDA in December 2022
  • 18 Oct 2022 LX 1004 receives Orphan Drug status for Neuronal ceroid lipofuscinosis by European Commission
  • 18 Oct 2022 Adverse events data from a phase I/II trial in Neuronal ceroid lipofuscinosis released by LEXEO Therapeutics

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