Drugs / BOMTABEGAGENE BAVOPARVOVEC
Trials 1 · a red edge is where a trial was stopped
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 1/2 | NCT03952637 | Aug 2019 → Jan 2028 expected | GM1 gangliosidosis | National Human Genome Research Institute (NHGRI) | Recruiting | No outcome recorded |
Evidence & citations 7 cited values
Every value below carries the sentence it was read from. 2 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | BOMTABEGAGENE BAVOPARVOVEC | ChEMBL registry synonym — accepted as the source's own label CHEMBL4802255 ↗ |
| Known as | AAV9-GLB1 | ClinicalTrials.gov intervention name — accepted as the source's own label NCT03952637 ↗ |
| Known as | AXO-AAV-GM1 | ChEMBL registry synonym — accepted as the source's own label CHEMBL4802255 ↗ |
| Action | Restore | “Intravenous administration of a gene transfer vector to deliver a normal copy of the GLB1 gene to the CNS could potentially provide an effective treatment for GM1 gangliosidosis.” NCT03952637 ↗ |
| Modality | Gene therapy (AAV / viral vector) | “Intravenous administration of a gene transfer vector to deliver a normal copy of the GLB1 gene to the CNS could potentially provide an effective treatment for GM1 gangliosidosis.” NCT03952637 ↗ |
| Route | Intravenous | “AAV9-GLB1 vector following intravenous infusion” NCT03952637 ↗ |
| Target | GLB1 | “Intravenous administration of a gene transfer vector to deliver a normal copy of the GLB1 gene to the CNS could potentially provide an effective treatment for GM1 gangliosidosis.” NCT03952637 ↗ |