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Drug Profile

JR 479

Alternative Names: BBB-penetrating βHexosaminidase A (rDNA origin) - JCR Pharmaceuticals; JR-479; β-Hexosaminidase A - JCR Pharmaceuticals

Latest Information Update: 06 Apr 2022

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At a glance

Originator JCR Pharmaceuticals
Class Hexosaminidases; Recombinant proteins
Mechanism of Action Hexosaminidase A replacements

Orphan Drug Status

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

No
New Molecular Entity Yes
Available For Licensing Yes

Highest Development Phases

Preclinical GM2 gangliosidoses

Most Recent Events

22 Mar 2022 JR 479 is available for licensing as of 22 Mar 2022. https://congress.jcrpharm.co.jp/
18 Mar 2022 Preclinical trials in GM2 gangliosidoses in Japan (IV) as of March 2022
18 Mar 2022 JCR Pharmaceuticals plans clinical trials in GM2 gangliosidosis within three years by 2025

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