Drugs / HG004
Trials 2
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 01 trial | ||||||
| Phase 0 | NCT06088992 | Jan 2023 → Oct 2024 overdue | Leber congenital amaurosis | Xinhua Hospital, Shanghai Jiao Tong University School of Medicine | Active not recruiting | No outcome recorded |
| Phase 1/21 trial | ||||||
| Phase 1/2 | NCT05906953 | Oct 2023 → Dec 2025 overdue | Leber congenital amaurosis, RPE65-related recessive retinopathy | HuidaGene Therapeutics Co., Ltd. | Recruiting | No outcome recorded |
News releases announcing trial results or a regulatory action · 5
| Date | Issuer | Release |
|---|---|---|
| 2025-01-08 | HuidaGene Therapeutics Co., Ltd. | Regulatory European Medicines Agency Issues Positive Opinion on HuidaGene's Triple-Designation HG004 Orphan Designation Application huidagene.com ↗
HuidaGene Therapeutics (“HuidaGene”), a global clinical-stage biotechnology company advancing programmable genome medicine, is pleased to announce that the European Medicines Agency (EMA) for Committee for Orphan Medicinal Products (COMP) has issued a positive opinion on the orphan designation application of HG004 gene therapy for inherited retinal dystrophy caused by dysfunction in the RPE65 gene. |
| 2024-09-02 | HuidaGene Therapeutics Co., Ltd. | Results HuidaGene to Highlight Latest Advances in Retina Portfolio at the European Society of Retina Specialists (EURETINA) 2024 Annual Meeting huidagene.com ↗
Our ‘LIGHT-I’ first-in-human clinical study (NCT06088992) of HG004 for inherited blindness shows a superior safety profile and visual improvement than the approved LUXTURNA. |
| 2023-08-07 | HuidaGene Therapeutics Co., Ltd. | Regulatory HuidaGene Therapeutics Receives FDA Rare Pediatric Disease Designation for HG004 to Treat Inherited Blindness huidagene.com ↗
HuidaGene Therapeutics (辉大基因，“HuidaGene”), a clinical-stage biotechnology company focused on developing CRISPR-based programmable genomic medicines, today announced the U.S. Food and Drug Administration (FDA) had granted Rare Pediatric Disease Designation (RPDD) to HG004 for the treatment of inherited retinal disease caused by RPE65 mutations(RPE65-IRDs). |
| 2023-04-18 | HuidaGene Therapeutics Co., Ltd. | Regulatory HuidaGene Announces IND Approval Of The First China Multinational Master Protocol of HG004 By China NMPA huidagene.com ↗ |
| 2023-02-14 | HuidaGene Therapeutics Co., Ltd. | Results HuidaGene Announces Completion of First Patient Enrollment in its Ophthalmic Gene Therapy Candidate HG004 IIT Clinical Trials huidagene.com ↗
Within 2 weeks after low-dose HG004 administration, this subject had a good safety profile and no serious adverse events, ocular adverse reactions (no intraocular inflammation was observed), significant drug-related adverse events, or clinically significant immune reactions were observed. |
All press releases naming this drug 7 releases
Evidence & citations 3 cited values
Every value below carries the sentence it was read from. 2 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | HG004 | ClinicalTrials.gov intervention name — accepted as the source's own label NCT06088992 ↗ |
| Modality | Gene therapy (AAV / viral vector) | “HG004 as gene therapy” NCT06088992 ↗ |
| Route | Other | “Once unilateralsubretinal injection” NCT06088992 ↗ |