Drug Profile

RGX 111

Alternative Names: RGX-111

Latest Information Update: 24 Jan 2025

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At a glance

Originator REGENXBIO
Class Gene therapies
Mechanism of Action Gene transference; Iduronidase replacements

Orphan Drug Status

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

Yes - Mucopolysaccharidosis I
New Molecular Entity No
Available For Licensing Yes

Highest Development Phases

Phase I/II Mucopolysaccharidosis I

Most Recent Events

14 Jan 2025 RGX 111 licensed to Nippon Shinyaku in US and Asia including Japan
13 Nov 2023 RGX 111 is available for licensing as of 09 Nov 2023. https://www.regenxbio.com/therapeutic-programs/
24 Feb 2023 Updated efficacy, safety and pharmacodynamic data from a phase I/II trial in Mucopolysaccharidosis I released by REGENXBIO and presented at the 19th Annual WORLDSymposium

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