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HuidaGene Therapeutics Receives European Medicines Agency Orphan Designation for HG204 to Treat MECP2 Duplication Syndrome

2024.05.06 09:00
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·  The preclinical studies in the humanized MECP2 transgenic mice showed motor function restoration, social behavior rescue, and lifespan extension after a single injection of HG204.

·  HG204 has also already been granted orphan drug designation and rare pediatric disease designation by the U.S. FDA in the treatment of MECP2 duplication syndrome

SHANGHAI and CLINTON (NJ), May 6, 2024 – HuidaGene Therapeutics (“HuidaGene”), a global clinical-stage biotechnology company developing potentially curative genomic medicines, today announced that the European Medicines Agency (EMA)’s Committee for Orphan Medicinal Products (COMP) has issued a positive opinion on the company’s application of orphan designation for HG204, a novel CRISPR/Cas13Y RNA-editing therapy for the treatment of MECP2 duplication syndrome (MDS). The orphan designation from EMA supplements the ODD and rare pediatric disease designation (RPDD) previously granted by the U.S. FDA in October 2023.

“The EMA’s orphan designation for the Company’s HG204 to treat MECP2 duplication syndrome is an important step forward in the development of novel CRISPR RNA-editing therapy in this disease,” said Alvin Luk, Ph.D., M.B.A., C.C.R.A., Co-founder, CEO, and Head of Medical of HuidaGene. “Although there is currently no clinical trial evaluating MDS, this designation is a sign of recognition for MDS and raises hopes for children and families affected by this fatal neurodevelopmental disorder. The MDS community has waited far too long for a potentially curative therapy that addresses the underlying cause of this devastating rare disease with a huge unmet medical need. We are committed to bringing novel CRISPR-based genomic medicines to patients beyond China and the United States.”

To qualify for orphan designation in the European Union, an investigational medicine must be intended to treat a life-threatening or chronically debilitating condition affecting fewer than five in 10,000 people in the European Union, based on data that supports that the investigational medicine may produce clinically significant benefits over existing treatments. Orphan designation provides companies with certain benefits and incentives, including reduced regulatory fees, access to centralized authorization procedure and research grants, clinical protocol assistance, a simplified approval process, and up to 10 years of market exclusivity in the European Union if approved.


About MECP2 Duplication Syndrome

Methyl-CpG binding protein 2 (MeCP2) duplication syndrome (MDS) is a rare, fatal childhood neurodevelopmental disorder. MDS,  which is caused by the duplication of the MECP2 gene, manifests predominantly in boys and is characterized by infantile hypotonia, developmental delay, intellectual disability, anxiety, loss of motor skills and speech, epilepsy, recurrent respiratory tract infections and shortened lifespan, with death often occurring before the age of 25 years. There are no approved disease-modifying therapies for MDS, and current treatments for MDS include symptom management and supportive care.

About HG204

HG204 (AAV-hfCas13Y-gMECP2) is developed using a single adeno-associated viral (AAV) vector delivering a novel CRISPR/high-fidelity Cas13Y (hfCas13Y) and gRNAs targeting MECP2 (gMECP2).  HG204 is stably expressed in brain regions of mice and non-human primates where the pathologic changes of MDS mainly occur. One extensive preclinical study using HG204 in the humanized MDS mouse model demonstrated a significant reduction of MeCP2 protein and reversal of its disease features.

About HuidaGene
HuidaGene utilizes its proprietary HG-PRECISE® platform to discover, engineer, and develop CRISPR-based genomic medicine. The Company is advancing clinical programs of HG004 in RPE65-associated inherited retinal disease (granted ODD and RPDD), HG202 CRISPR RNA-editing in neovascular age-related macular degeneration, and the preclinical pipeline, including HG204 CRISPR RNA-editing in neurodevelopmental disease of MECP2 duplication syndrome (granted ODD and RPDD by U.S. FDA and ODD by EMA), HG302 CRISPR DNA-editing for Duchenne muscular dystrophy (granted ODD and RPDD), and HG303 CRISPR DNA-editing for Amyotrophic Lateral Sclerosis (ALS). HuidaGene’s extensive intellectual property portfolio positions it as a leader in unleashing the full potential of genome medicines for neurology and ophthalmology. Learn more at or on LinkedIn.