Drug Information
| Drug General Information | Top | |||
|---|---|---|---|---|
| Drug ID |
DY8D2C
|
|||
| Drug Name |
LYS-GM-101
|
|||
| Drug Type |
Gene therapy
|
|||
| Indication | GM1 gangliosidosis [ICD-11: 5C56.00] | Phase 1/2 | [1] | |
| Company |
Lysogene
|
|||
| Target and Pathway | Top | |||
|---|---|---|---|---|
| Target(s) | Beta-galactosidase (GLB1) | Target Info | . | [1] |
| References | Top | |||
|---|---|---|---|---|
| REF 1 | ClinicalTrials.gov (NCT04273269) An Open-Label Adaptive-Design Study of Intracisternal Adenoassociated Viral Vector Serotype rh.10 Carrying the Human beta-Galactosidase cDNA for Treatment of GM1 Gangliosidosis. U.S.National Institutes of Health. | |||
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