Target organ/Cells | indications (Gene of Interest) | Gene size | Est. Prevalence (US + EU) | Discovery | Optimization | IND-Enabling | Clinical |
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Eye/Retina | Stargardt (ABCA4) | 6.8 KB | 60,000 |
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Usher Syndrome (USH2A) | 15.6 KB | 16,500 |
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Retinitis Pigmentosa 25 (EYS) | 9.5 KB | 4,500 |
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LCA10 (CEP290) | 7.4 kB | 3,600 |
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Wet AMD (VEGF Trap) * | N/A | 5M+ |
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Lung | Cystic Fibrosis (CFTR) | 4.4 kB | 105,000 (WW) |
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Ear | Multiple | --- | --- |
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Brain/CNS | Multiple | --- | --- |
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CAR-T | Oncology*, AutoImmune* | --- | --- |
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