MavriX Bio Doses First Patient in ASCEND-AS Trial
Slice of Healthcare Team
11/10/2025
Key highlights
- First patient dosed in ASCEND-AS trial
- MVX-220 targets UBE3A gene restoration
- FDA grants Orphan Drug Designation for MVX-220
- Collaboration with FAST and AS2Bio
- Potential breakthrough for Angelman syndrome treatment
Source: PR Newswire
Notable Quotes
“ Dosing our first participant in the ASCEND-AS trial is a critical step in evaluating the potential of gene targeted therapies for AS. ”
Jennifer Panagoulias, COO at MavriX Bio
“ This trial represents a new era in Angelman syndrome research. ”
Dr. Elizabeth Berry-Kravis, MD, PhD at Rush University Medical Center
Why this matters
The ASCEND-AS trial is a groundbreaking step in the treatment of Angelman syndrome, potentially offering hope to thousands affected by this rare genetic disorder. This development is particularly significant for the healthcare sector.
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