Drugs / EXG001-307
Trials 3
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 02 trials | ||||||
| Phase 0 | NCT06888661 | Mar 2025 → Mar 2026 overdue | spinal muscular atrophy | Guangzhou Jiayin Biotech Ltd | Enrolling by invitation | No outcome recorded |
| Phase 0 | NCT06576388 | Jan 2022 → Sep 2023 | spinal muscular atrophy, type 1 | Guangzhou Jiayin Biotech Ltd | Completed | No outcome recorded |
| Phase 1/21 trial | ||||||
| Phase 1/2 | NCT05614531 | Feb 2023 → Aug 2025 overdue | spinal muscular atrophy, type 1 | Guangzhou Jiayin Biotech Ltd | Enrolling by invitation | No outcome recorded |
News releases announcing trial results or a regulatory action · 1
| Date | Issuer | Release |
|---|---|---|
| 2025-05-14 | Exegenesis Bio | Results Exegenesis Bio to Present Data on Next Generation BBB-penetrating AAV Capsids & Intravitreal nAMD Gene Therapy at the American Society of Gene and Cell Therapy Meeting exegenesisbio.com ↗
Next-Generation AAV -Based Gene Therapy for Spinal Muscular Atrophy: Safety and Efficacy of EXG001-307 in Clinical Trials |
All press releases naming this drug 1 release
Evidence & citations 3 cited values
Every value below carries the sentence it was read from. 4 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | EXG001-307 | ClinicalTrials.gov intervention name — accepted as the source's own label NCT06888661 ↗ |
| Modality | Gene therapy (AAV / viral vector) | “EXG001-307 is an AAV9-based gene therapy vector” PMID 40458203 ↗ Apr 2025 |
| Route | Intravenous | “The purpose of this trial is to evaluate safety and efficacy of intravenous delivery of EXG001-307” NCT05614531 ↗ |