About the Program
This therapeutic approach utilizes specific pieces of RNA, called micro-RNA, that are delivered in an AAV virus to the brain, aimed to bind to the UBE3A-ATS and activate the silent paternal copy of the UBE3A gene in neurons.
- FAST Pillar
- Pillar 2: Turn on Dad’s UBE3A
- Investigational Drug Name
- ETX201
- Company Website
- Encoded Therapeutics
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