Ultragenyx Pharmaceutical, Inc. ((RARE)) announced an update on their ongoing clinical study.
Elevate Your Investing Strategy:
- Take advantage of TipRanks Premium at 50% off! Unlock powerful investing tools, advanced data, and expert analyst insights to help you invest with confidence.
Ultragenyx Pharmaceutical, Inc. is conducting a Phase 2 clinical study titled A Safety and Efficacy Study of GTX-102 in Subjects With Deletion- or Nondeletion-type Angelman Syndrome (AS). The primary objective of this study is to assess the safety and efficacy of GTX-102, a treatment aimed at individuals with Angelman Syndrome, a rare genetic disorder. This study is significant as it explores potential therapeutic options for a condition with limited treatment avenues.
The intervention being tested is GTX-102, an antisense oligonucleotide administered via intrathecal injection. It is designed to target and treat various genotypes of Angelman Syndrome, with the aim of achieving the desired therapeutic dose over time.
The study follows a randomized, parallel intervention model with no masking, focusing on treatment as its primary purpose. Participants are divided into different subprotocols based on age and genotype, receiving increasing doses of GTX-102 every three months.
The study began on August 21, 2025, with the latest update submitted on September 26, 2025. These dates are crucial as they mark the progression and current status of the study, which is actively recruiting participants.
This clinical update could potentially influence Ultragenyx’s stock performance positively, as successful results may enhance investor confidence and market positioning. In the broader industry context, advancements in treating Angelman Syndrome could set new benchmarks for competitors.
The study is ongoing, with further details available on the ClinicalTrials portal.