Drug Profile

MVX 220

Alternative Names: MVX-220

Latest Information Update: 14 Nov 2025

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At a glance

Originator University of Pennsylvania
Developer MavriX Bio
Class Gene therapies
Mechanism of Action Gene transference; UBE3A protein replacements

Orphan Drug Status

Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

Yes - Angelman syndrome
New Molecular Entity No

Highest Development Phases

Phase I/II Angelman syndrome

Most Recent Events

06 Nov 2025 MVX 220 receives Orphan Drug status for Angelman syndrome in USA
23 Oct 2025 Phase-I/II clinical trials in Angelman syndrome (In children, In adults, In adolescents) in USA (Intracisternal) (NCT07181837)
22 Sep 2025 MVX 220 receives Fast Track designation for Angelman syndrome [Intracisternal] in USA

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