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眼科类疾病
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Therapeutic Area / Indication
RPE65 突变相关视网膜疾病(RPE65 -IRD)
Strategy
基因替代
Commercial Rights
全球
Discovery
Optimization
IND Enabling
IIT / Phasel
Phase Il / Ill
Learn More
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Therapeutic Area / Indication
年龄相关性黄斑变性(AMD)
Strategy
RNA编辑
Commercial Rights
全球
Discovery
Optimization
IND Enabling
IIT / Phasel
Phase Il / Ill
Learn More
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Therapeutic Area / Indication
视网膜色素变性(RP)
Strategy
DNA编辑
Commercial Rights
全球
Discovery
Optimization
IND Enabling
IIT / Phasel
Phase Il / Ill
Learn More
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神经类疾病
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Therapeutic Area / Indication
MECP2 重复综合症(MDS)
Strategy
RNA编辑
Commercial Rights
全球
Discovery
Optimization
IND Enabling
IIT / Phasel
Phase Il / Ill
Learn More
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Therapeutic Area / Indication
亨廷顿舞蹈症(HD)
Strategy
DNA编辑
Commercial Rights
全球
Discovery
Optimization
IND Enabling
IIT / Phasel
Phase Il / Ill
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Therapeutic Area / Indication
渐冻症(ALS)
Strategy
DNA编辑
Commercial Rights
全球
Discovery
Optimization
IND Enabling
IIT / Phasel
Phase Il / Ill
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神经肌肉类疾病
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Therapeutic Area / Indication
杜氏肌营养不良(DMD)
Strategy
DNA编辑
Commercial Rights
全球
Discovery
Optimization
IND Enabling
IIT / Phasel
Phase Il / Ill
Learn More
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