Ultragenyx Pharmaceutical, Inc. ((RARE)) announced an update on their ongoing clinical study.
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Ultragenyx Pharmaceutical Inc. is conducting a follow-up study titled ‘A Long-term Follow-up Study of Patients With MPS IIIA From Gene Therapy Clinical Trials Involving the Administration of ABO-102 (scAAV9.U1a.hSGSH).’ The study aims to evaluate the safety, tolerability, and efficacy of UX111 (formerly ABO-102) in patients with Mucopolysaccharidosis IIIA (MPS IIIA), a rare genetic disorder. This research is significant as it could lead to improved treatments for this debilitating condition.
The study involves two cohorts: Cohort A, which includes participants receiving adjuvant immunomodulatory therapy, and Cohort B, which involves participants not receiving any investigational product. The primary goal is to monitor long-term outcomes in patients who have previously participated in trials involving UX111.
The study is interventional, non-randomized, and follows a parallel intervention model without masking. Its primary purpose is to gather additional data on the long-term effects of the gene therapy.
The study began on April 20, 2020, with the latest update submitted on October 16, 2025. These dates are crucial as they indicate the study’s progression and ongoing nature, which is important for tracking its development and potential outcomes.
For investors, this study update from Ultragenyx could influence stock performance and investor sentiment, particularly if the results show positive long-term benefits of UX111. This could position Ultragenyx favorably against competitors in the rare disease treatment market.
The study is currently ongoing, and further details can be accessed on the ClinicalTrials portal.