Drugs / HG302
Trials 1
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 0 | NCT06594094 | Nov 2024 → Dec 2025 | Duchenne muscular dystrophy | HuidaGene Therapeutics Co., Ltd. | Completed | No outcome recorded |
News releases announcing trial results or a regulatory action · 7
| Date | Issuer | Release |
|---|---|---|
| 2026-08-05 | HuidaGene Therapeutics Co., Ltd. | Results HuidaGene Therapeutics Provides an Update on the HG302-01 First-in-Human Trial huidagene.com ↗
Based on the available clinical and scientific evidence, the participant developed acute respiratory distress syndrome in the setting of severe complement and cytokine activation following high-dose systemic administration of an adeno-associated virus vector. |
| 2025-04-11 | HuidaGene Therapeutics Co., Ltd. | Results HuidaGene at CRISPR MEDiCiNE 2025: A Celebration of Progress, Promise, and Patients huidagene.com ↗
HG302 (hfCas12Max) for Duchenne muscular dystrophy (DMD) demonstrated strong safety with no SAEs, DLT, or major toxicities |
| 2024-09-24 | HuidaGene Therapeutics Co., Ltd. | Results HuidaGene Presents Late-Breaking Data at World Muscle Society 2024 Demonstrating HG302 CRISPR/hfCas12Max DNA-Editing Therapy for Duchenne Muscular Dystrophy to Support M.U.S.C.L.E. Clinical Trial huidagene.com ↗
Systemic delivery of HG302 in humanized DMD mice efficiently restored dystrophin expression and rescued motor function, including rotarod and grip strength tests, to wild-type levels. |
| 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 ↗ |
| 2024-01-23 | HuidaGene Therapeutics Co., Ltd. | Regulatory HuidaGene Receives Orphan Drug Designation from FDA for HG302 for the Potential Treatment of Duchenne Muscular Dystrophy after Receiving Rare Pediatric Drug Designation huidagene.com ↗
HuidaGene Therapeutics (“HuidaGene”), a global clinical-stage biotechnology company developing potentially curative genomic medicines, has been granted Orphan Drug Designation (ODD) by the U.S. FDA for HG302 to treat Duchenne muscular dystrophy (DMD). |
| 2023-12-19 | HuidaGene Therapeutics Co., Ltd. | Regulatory HuidaGene Announces Rare Pediatric Drug Designation Granted to HG302, A Novel CRISPR DNA-editing Therapy, for the Treatment of Duchenne Muscular Dystrophy huidagene.com ↗
HuidaGene Therapeutics (辉大基因“HuidaGene”) today announced the U.S. FDA granted Rare Pediatric Drug Designation (RPDD) to HG302, a novel CRISPR-Cas12 DNA-editing therapy, to treat Duchenne muscular dystrophy (DMD), affecting 1 in ~5,000 newborn boys. |
| 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 ↗
HG302 CRISPR/Cas12 DNA-editing therapy sufficiently edits and induces exon51 reframing/skipping in DMD gene |
All press releases naming this drug 8 releases
Evidence & citations 3 cited values
Every value below carries the sentence it was read from. 1 source stands behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | HG302 | ClinicalTrials.gov intervention name — accepted as the source's own label NCT06594094 ↗ |
| Action | Restore | “Preclinical studies have shown that a single intravenous injection of HG302 significantly restores dystrophin protein expression in muscle fibers and rescues their muscle...” NCT06594094 ↗ |
| Target | DMD | “HG302 uses a single AAV vector to deliver the CRISPR/hfCas12Max DNA editing system in the human DMD exon 51 splice donor site.” NCT06594094 ↗ |