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Drug Profile

GM1 gangliosidosis targeting gene therapy - JCR Pharmaceuticals

Latest Information Update: 03 Dec 2024

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At a glance

Originator JCR Pharmaceuticals
Class Antibodies; Gene therapies
Mechanism of Action Gene transference; GLB1 protein 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

Highest Development Phases

Preclinical GM1 gangliosidosis

Most Recent Events

05 Sep 2024 Preclinical trials in GM1-gangliosidosis in Japan (Parenteral)
05 Sep 2024 Pharmacodynamics data from a preclinical study in GM1 gangliosidosis released by JCR Pharmaceuticals

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