Drug Profile

RGX 181

Alternative Names: AAV9.hCLN2; RGX-181

Latest Information Update: 25 Sep 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

Discontinued Neuronal ceroid lipofuscinosis

Most Recent Events

25 Sep 2025 Discontinued - Clinical-Phase-Unknown for Neuronal ceroid lipofuscinosis (In children) in Brazil (Intracisternal)
25 Sep 2025 Discontinued - Phase-I for Neuronal ceroid lipofuscinosis (In children) in Europe (Intracisternal)
25 Sep 2025 Discontinued - Phase-I for Neuronal ceroid lipofuscinosis (In children) in USA (Intracisternal)

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