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Neurology
Ophthalmology
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Neurology
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Therapeutic Area / Indication
MECP2 Duplication Syndrome(MDS)
Strategy
RNA editing
Commercial Rights
Global
Discovery
Optimization
IND Enabling
IIT / Phasel
Phase Il / Ill
Learn More
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Therapeutic Area / Indication
Huntington's Disease (HD)
Strategy
DNA editing
Commercial Rights
Global
Discovery
Optimization
IND Enabling
IIT / Phasel
Phase Il / Ill
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Therapeutic Area / Indication
Amyotrophic Lateral Sclerosis (ALS)
Strategy
DNA editing
Commercial Rights
Global
Discovery
Optimization
IND Enabling
IIT / Phasel
Phase Il / Ill
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Ophthalmology
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Therapeutic Area / Indication
RPE65 Mutation-Associated Inherited Retinal Dystrophies (RPE65-IRDs)
Strategy
Gene Replacement
Commercial Rights
Global
Discovery
Optimization
IND Enabling
IIT / Phasel
Phase Il / Ill
Learn More
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Therapeutic Area / Indication
Neovascular Age-related Macular Degeneration (nAMD)
Strategy
RNA editing
Commercial Rights
Global
Discovery
Optimization
IND Enabling
IIT / Phasel
Phase Il / Ill
Learn More
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Therapeutic Area / Indication
RHO-Associated Retinitis Pigmentosa (RHO-adRP)
Strategy
DNA editing
Commercial Rights
Global
Discovery
Optimization
IND Enabling
IIT / Phasel
Phase Il / Ill
Learn More
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Neuromuscular
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Therapeutic Area / Indication
Duchenne Muscular Dystrophy (DMD)
Strategy
DNA editing
Commercial Rights
Global
Discovery
Optimization
IND Enabling
IIT / Phasel
Phase Il / Ill
Learn More
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