Drugs / HG204
Trials 1
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| — | NCT06615206 | Oct 2024 → Oct 2026 expected | syndrome caused by partial chromosomal duplication | HuidaGene Therapeutics Co., Ltd. | Recruiting | No outcome recorded |
News releases announcing trial results or a regulatory action · 5
| Date | Issuer | Release |
|---|---|---|
| 2025-04-11 | HuidaGene Therapeutics Co., Ltd. | Results HuidaGene at CRISPR MEDiCiNE 2025: A Celebration of Progress, Promise, and Patients huidagene.com ↗
HG204 (hfCas13Y), the world's first RNA-editing therapy for MECP2 duplication syndrome (MDS), showed meaningful cognitive, motor, and emotional improvements in children — a significant breakthrough in tackling a devastating pediatric neurodevelopmental disorder. |
| 2024-12-12 | HuidaGene Therapeutics Co., Ltd. | Results HuidaGene Therapeutics Announces Landmark Publication in Nature Neuroscience Highlighting HG204’s Potential as First RNA-editing Therapy for MECP2 Duplication Syndrom huidagene.com ↗
HuidaGene Therapeutics (“HuidaGene”), a global clinical-stage biotechnology company advancing programmable genome medicine, announced that its groundbreaking research results on HG204, a CRISPR RNA-editing therapy, has been published in Nature Neuroscience. |
| 2024-04-22 | HuidaGene Therapeutics Co., Ltd. | Results HuidaGene Orally Presents Data Highlighting Strength of Ophthalmology Portfolio at the 2024 Association for Research in Visual and Ophthalmology Annual Meeting huidagene.com ↗
Oral presentation of HG004 gene augmentation therapy restores the function of RPE-photoreceptor cells in the Rpe65-/- murine model and demonstrates significant improvement of visual and retinal functions in LCA2 patients. |
| 2023-10-31 | HuidaGene Therapeutics Co., Ltd. | Regulatory U.S. FDA Awards Both Rare Pediatric Disease and Orphan Drug Designations to HG204, a CRISPR RNA-editing Therapy, for the Treatment of MECP2 Duplication Syndrome huidagene.com ↗ |
| 2023-09-14 | HuidaGene Therapeutics Co., Ltd. | Results HuidaGene Showcases Gene-Editing Based Medicine for Neurological Disease at Japan Society of Gene and Cell Therapy 2023 Annual Meeting huidagene.com ↗
HG204 CRISPR/Cas13 RNA-editing significantly rescues motor functions and prolongs lifespan in MDS mouse model and effectively knocks down MECP2 mRNA level in non-human primates |
All press releases naming this drug 6 releases
Evidence & citations 5 cited values
Every value below carries the sentence it was read from. 1 source stands behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | HG204 | ClinicalTrials.gov intervention name — accepted as the source's own label NCT06615206 ↗ |
| Action | Inhibit | “to target and knock down MECP2 mRNA in the brain” NCT06615206 ↗ |
| Modality | CRISPR / gene editing | “HG204 is a CRISPR RNA-editing therapy” NCT06615206 ↗ |
| Route | Other | “a single intracerebroventricular injection of HG204” NCT06615206 ↗ |
| Target | MECP2 | “to target and knock down MECP2 mRNA in the brain” NCT06615206 ↗ |