Drugs / PBKR03
Trials 1
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 1/2 | NCT04771416 | Feb 2022 → Jan 2027 | Krabbe disease | Gemma Biotherapeutics | Suspended | No outcome recorded Stop: Business |
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 | PBKR03 | ClinicalTrials.gov intervention name — accepted as the source's own label NCT04771416 ↗ |
| Action | Restore | “intended to deliver a functional copy of the GALC gene” NCT04771416 ↗ |
| Modality | Gene therapy (AAV / viral vector) | “PBKR03 is an adeno-associated viral vector serotype Hu68 carrying the gene encoding for human galactosylceramidase, GALC, formulated as a solution for injection into the cisterna magna.” NCT04771416 ↗ |
| Route | Other | “via intra cisterna magna” NCT04771416 ↗ |
| Target | GALC | “intended to deliver a functional copy of the GALC gene” NCT04771416 ↗ |