Drugs / LIXMABEGAGENE RELDUPARVOVEC
Trials 1
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 1/2 | NCT04273269 | May 2021 → May 2023 | GM1 gangliosidosis | LYSOGENE | Terminated | No outcome recorded |
Evidence & citations 5 cited values
Every value below carries the sentence it was read from. 2 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | LIXMABEGAGENE RELDUPARVOVEC | ChEMBL registry synonym — accepted as the source's own label CHEMBL5095132 ↗ |
| Known as | LYS-GM101 | ClinicalTrials.gov intervention name — accepted as the source's own label NCT04273269 ↗ |
| Action | Restore | “intended to deliver a functional copy of the GLB1 gene” NCT04273269 ↗ |
| Route | Intrathecal | “participants with infantile GM1 gangliosidosis will receive a single dose of LYS-GM101 by intracisternal injection” NCT04273269 ↗ |
| Target | GLB1 | “LYS-GM101 is a gene therapy for GM1 gangliosidosis intended to deliver a functional copy of the GLB1 gene to the central nervous system.” NCT04273269 ↗ |