HG 204
Alternative Names: AAV-hfCas13Y-gMECP2; HG-204Latest Information Update: 07 Oct 2024
At a glance
- Originator HuidaGene Therapeutics
- Class Gene therapies
- Mechanism of Action Gene silencing; Methyl-CpG-binding protein 2 expression inhibitors
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Orphan Drug Status
Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.
- New Molecular Entity No
Highest Development Phases
- Preclinical Lubs X-linked mental retardation syndrome; Neurodegenerative disorders
Most Recent Events
- 26 Sep 2024 HuidaGene Therapeutics plans the HERO clinical trial for Lubs X linked mental retardation syndrome (Treatment-experienced, In Adolescents, In children) in November 2024 (Intracerebral, Injection) (NCT06615206)
- 29 May 2024 HG 204 receives Orphan Drug status for Lubs X-linked mental retardation syndrome in European Union, prior to May 2024
- 31 Oct 2023 Preclinical trials in Lubs X-linked mental retardation syndrome in China (Parenteral)