About the Program
CRISPR-GNDM® is an epigenome editing technology that combines dCas9, which lacks the cutting enzyme activity of traditional CRISPR, with an epigenome editor/modulator and a guide that defines the target site for modulating. CRISPR-GNDM® is packaged in a single AAV to potentially restore paternal UBE3A expression.
- FAST Pillar
- Pillar 2: Turn on Dad’s UBE3A
- Investigational Drug Name
- MDL-206
- Company Website
- Modalis