Drug Profile

RGX 381

Alternative Names: RGX-381

Latest Information Update: 13 Nov 2023

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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/II Neuronal ceroid lipofuscinosis

Most Recent Events

13 Nov 2023 RGX 381 is available for licensing as of 09 Nov 2023. https://www.regenxbio.com/who-we-are/
17 May 2023 Phase-I/II clinical trials in Neuronal ceroid lipofuscinosis (In children, In infants, Treatment-experienced) in United Kingdom (Intraocular) (NCT05791864)
31 Dec 2022 REGENXBIO has patent protection for RGX 381 in USA

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