| English Name | Research Code | Highest Development Stage | Company | Indication | Clinical Trial |
|---|---|---|---|---|---|
| Bomtabegagene bavoparvovec | AAV9-GLB1; AXO-AAV-GM1 | Phase 2 Clinical | University Of Massachusetts Medical School | Gangliosidoses | Details |
| Gaxilose | Phase 1 Clinical | Lactose Intolerance | Details |