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TTX 181

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Drug Profile

TTX 181

Alternative Names: AAV9.hCLN2; RGX-181; TTX-181

Latest Information Update: 12 Nov 2025

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At a glance

  • Originator REGENXBIO
  • Developer Tern Therapeutics
  • Class Gene therapies; Neuroprotectants
  • Mechanism of Action Gene transference; Tripeptidyl-peptidase 1 replacements
  • 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
  • Available For Licensing Yes

Highest Development Phases

  • Clinical Phase Unknown Neuronal ceroid lipofuscinosis

Most Recent Events

  • 08 Oct 2025 Preclinical trials in Neuronal ceroid lipofuscinosis in USA before October 2025 (Intracisternal) (Tern Therapeutics pipeline, October 2025)
  • 27 Aug 2024 RGX-181 licensed to Tern Therapeutics worldwide before August 2024
  • 13 Nov 2023 RGX 181 is available for licensing as of 09 Nov 2023. https://www.regenxbio.com/therapeutic-programs/

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