DJ
Therapeutic Areas
Rare Therapeutics Pipeline
| Drug | Indication | Phase |
|---|---|---|
| GM1 Gangliosidosis (GM1) Gene Therapy | GM1 Gangliosidosis | Clinical-stage |
| Krabbe Disease Gene Therapy | Krabbe Disease | Clinical-stage |
| Metachromatic Leukodystrophy (MLD) Gene Therapy | Metachromatic Leukodystrophy | Clinical-stage |