Our Pipeline
| Program | Indication | Gene | Technology | Discovery | IND-Enabling | Phase 1 | Phase 2 | Phase 3 |
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| NP3011 | Sanfilippo Syndrome Type A (MPSIIIA) |
SGSH | ERT (ICV) |
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| NP3021 | GM1 Gangliosidosis | GLB1 | ERT (ICV) |
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| NP3022 | Morquio B Syndrome (MPSIVB) |
GLB1 | ERT (IV) |
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| NP3031 | Krabbe Disease | GALC | ERT (ICV) |
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| - | SMA | SMN1 | - |
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| - | Glutaric aciduria type 1 | GCDH | - |
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| NP1031 | Prader-Willi Syndrome | - | Oxytocin |
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| Program | Indication | Technology | Discovery | IND-Enabling | Phase 1 | Phase 2 | Phase 3 |
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| NP1116 | Prostate Cancer Central Precocious Puberty |
GnRH Agonist |
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