Drug Profile

TTX 181

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

Latest Information Update: 08 Oct 2025

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At a glance

Originator REGENXBIO
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

Phase I Neuronal ceroid lipofuscinosis

Most Recent Events

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/
08 Sep 2023 Phase-I clinical trials in Neuronal ceroid lipofuscinosis (In children) in Europe, USA (Intracisternal) before September 2023

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