Clinical Trials
ASCEND-AS is a phase 1/​2 study of the safety and efficacy of MVX-220, an investigational gene therapy, in participants with Angelman syndrome (AS)
MavriX Bio joined the Foundation for Angelman Syndrome Therapeutics (FAST) and the Angelman Syndrome Foundation (ASF) for an Angelman Community Webinar on Thursday, October 9, 2025 to provide a summary of the trial design, enrollment criteria, study procedures, etc. for the ASCEND-AS study. A recording of this webinar can be accessed through FAST or ASF.
Clinical Trial Participation
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Information about the study sites and eligibility criteria for the ASCEND-AS study is posted on www.clinicaltrials.gov (see NCT07181837).
Expanded Access Policy
At MavriX Bio, our mission is to bring potentially life-changing therapies to individuals living with AS. We approach our work every day with great urgency and commitment to the individuals and families who suffer from this devastating disorder for which there are no treatment options.
What is Expanded Access?
Expanded access—also known as compassionate use—is a potential pathway for patients with serious or life-threatening conditions to gain access to investigational therapies outside of a clinical trial, when no comparable or satisfactory alternative therapy options are available. These programs are subject to regulatory oversight and require certain criteria to be met to ensure patient safety and appropriate use of the investigational product.
MavriX Bio’s Position on Expanded Access
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We understand that patients and families facing serious or life-threatening conditions may seek access to investigational therapies outside of clinical trials. At this time, MavriX Bio is not offering an expanded access program for MVX-220.
We believe that the most efficient and scientifically rigorous way to assess the potential of MVX-220 is through clinical studies designed to generate the data required for regulatory review and potential future approval.
Future Updates
MavriX Bio will continue to evaluate our access policies as our development program progresses. If our position on expanded access changes in the future, we will provide updates on this page.