Small moleculeOligonucleotideGene therapy / editingAntibody / biologicUndisclosedRepurposingPossibly inactiveClick a chip for details
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| Modality | Preclinical | IND | Phase 1 | Phase 2 | Phase 3+ | Approved |
|---|---|---|---|---|---|---|
| mRNA-upregulating small molecules | ||||||
| mRNA-upregulating ASOs | ||||||
| PKD protein correctors | ||||||
| Signaling modulators | ||||||
| Read-through correctors | ||||||
| Gene therapy | ||||||
| Gene editing | ||||||
| Antibody / biologic | ||||||
| Undisclosed | ||||||
| Repurposing |