Ultragenyx GTX-102 Aspire trial: community webinar recording
Following Ultragenyx’s announcement that its Phase 3 Aspire trial of GTX-102 did not meet its primary or key secondary endpoints, the Angelman Syndrome Foundation (ASF) and FAST hosted a community webinar with Ultragenyx. The webinar provided an opportunity for the Angelman community to hear from Ultragenyx about the trial results and ask questions. Watch the […]
MavriX Bio announced that the FDA Rare Pediatric Disease Designation
MavriX Bio announced that the FDA has granted Rare Pediatric Disease Designation (RPDD) to its investigational gene therapy, MVX-220. Rare Pediatric Disease Designation is granted by the FDA to therapies intended to treat serious or life-threatening diseases that primarily affect individuals from birth through 18 years of age and affect fewer than 200,000 people in […]
Ionis shares update on CHAMPION Phase 3 clinical trial
Ionis has shared an important update with the Angelman syndrome community about the design of CHAMPION, its Phase 3 clinical trial evaluating ION582 (obudanersen). The study will include children and adults with Angelman syndrome caused by uniparental disomy (UPD) or an imprinting centre defect (ICD). Ionis is actively preparing to begin the CHAMPION study. The […]
Ionis completes enrolment in pivotal cohort of the Phase 3 REVEAL study evaluating obudanersen in Angelman syndrome
“Guided by input from the Angelman community, REVEAL was intentionally designed to evaluate obudanersen across a broad range of people living with Angelman syndrome, reflecting the real-world diversity of this condition. We are deeply grateful to the entire Angelman syndrome community, especially the participants and their families, whose partnership made this achievement possible.” – Holly […]
Oak Hill Bio has shared a community letter to the Angelman syndrome community
Oak Hill Bio has shared a community letter to the Angelman syndrome community with an important update on the next steps of the development of rugonersen. They are actively preparing to initiate the Phase 3 study in the middle of 2026. Eligibility criteria are available on clinicaltrials.gov; additional details, including study locations, will be updated as […]
MavriX Bio Announces FDA Clearance of IND Application to Initiate First-in-Human Study of Gene Therapy for Angelman Syndrome
MavriX Bio, a clinical-stage biotechnology company focused on the development of transformative genetic therapies for Angelman syndrome (AS), today announced that the U.S. Food and Drug Administration (FDA) has cleared its Investigational New Drug (IND) application for MVX-220, an investigational adeno-associated virus (AAV) gene therapy for the treatment of AS. Read Press Release Read […]
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Phase 3 Aspire trial of gene therapy GTX-102 doses 1st patient
New trial, Aurora, to test GTX-102 in other Angelman genotypes, age groups
Exciting News: Positive Interim Phase 1/2 Results
We are thrilled to share that Ultragenyx has released promising interim findings from their Phase 1/2 clinical trial involving patients living with the deletion genotype of Angelman syndrome.