Shots:
- The US FDA has approved IND application of MVX-220 for the treatment of Angelman Syndrome (AS) to initiate P-I/II (ASCEND-AS) trial, with first pts dosing expected in H2’25
- The P-I/II trial will evaluate the safety, tolerability, & effectiveness of MVX-220 in both adult & pediatric pts with multiple AS genotypes, incl. rare types like uniparental disomy & imprinting center defects
- MVX-220 is a hu68AAV gene therapy designed to deliver the human UBE3A gene to brain neurons via a single intra-cisterna magna (ICM) injection, showing restored UBE3A protein expression & improved symptoms in the AS mouse model
Ref: PRNewswire | Image: MavriX Bio
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