GTX-102 (apazunersen) is an investigational antisense oligonucleotide therapy delivered intrathecally for Angelman syndrome. It targets the UBE3A antisense transcript to reactivate paternal UBE3A expression. The program has advanced into Phase 3 development with FDA Breakthrough Therapy Designation.
Opus Genetics reported positive early data from its BEST1 gene therapy program and expects FDA action on its presbyopia treatment in October 2026. Ultragenyx announced 2025 revenue of $673 million and initiated a strategic restructuring plan targeting profitability in 2027.
Ultragenyx Pharmaceutical confronts multiple shareholder class action lawsuits over Phase III trial disclosures while reporting positive UX111 gene therapy data and implementing a 10% workforce reduction.
Ultragenyx reported Q4 2025 revenues of $207 million, up 25% year-over-year, while announcing a 10% workforce reduction and resubmission of its UX111 gene therapy application following positive long-term clinical data.
| NCT ID | Title | Status | Phase |
|---|---|---|---|
| NCT07157254 |
A Safety and Efficacy Study of GTX-102 in Subjects With Deletion- or Nondeletion-type Angelman Syndrome (AS) |
RECRUITING | PHASE2 |
| NCT06617429 |
Phase 3 Efficacy and Safety Study of GTX-102 in Pediatric Subjects With Angelman Syndrome (AS) |
ACTIVE_NOT_RECRUITING | PHASE3 |
| NCT06415344 |
Long-term Extension of GTX-102 in Angelman Syndrome |
ENROLLING_BY_INVITATION | PHASE3 |
| NCT04259281 |
A Study of the Safety and Tolerability of GTX-102 in Children With Angelman Syndrome |
COMPLETED | PHASE1/PHASE2 |
