Drugs / PBGM01
Trials 1
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 1/2 | NCT04713475 | Mar 2021 → Feb 2028 expected | GM1 gangliosidosis type 1, GM1 gangliosidosis type 2 | Gemma Biotherapeutics | Active not recruiting | No outcome recorded |
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 | PBGM01 | ClinicalTrials.gov intervention name — accepted as the source's own label NCT04713475 ↗ |
| Action | Restore | “intended to deliver a functional copy of the GLB1 gene to the brain and peripheral tissues” NCT04713475 ↗ |
| Modality | Gene therapy (AAV / viral vector) | “PBGM01 is an adeno-associated viral vector serotype hu68 carrying GLB1” NCT04713475 ↗ |
| Route | Other | “formulated as a solution for injection into the cisterna magna” NCT04713475 ↗ |
| Target | GLB1 | “PBGM01 is a gene therapy for GM1 gangliosidosis intended to deliver a functional copy of the GLB1 gene” NCT04713475 ↗ |