Abeona Therapeutics Inc ((ABEO)) announced an update on their ongoing clinical study.
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Abeona Therapeutics Inc, in collaboration with Ultragenyx Pharmaceutical Inc, is conducting a Phase I/II/III clinical trial titled ‘Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH for Mucopolysaccharidosis (MPS) IIIA’. The study aims to evaluate the efficacy and safety of the gene therapy UX111 for treating MPS IIIA, a rare genetic disorder. This trial is significant as it explores a potential treatment for a condition with limited therapeutic options.
The intervention being tested is UX111, a gene therapy delivered via a single intravenous injection. It involves a self-complementary adeno-associated virus carrying the human SGSH gene, intended to address the underlying cause of MPS IIIA. Additional immunomodulatory therapies may be administered to manage immune responses.
The study follows a non-randomized, sequential intervention model with no masking, focusing on treatment. Participants are divided into cohorts receiving varying doses of UX111, with some receiving additional immunomodulatory therapies.
The trial began on April 25, 2016, with the most recent update submitted on July 16, 2025. These dates are crucial for tracking the study’s progress and ensuring transparency in reporting.
This update could positively impact Abeona Therapeutics’ stock performance by enhancing investor confidence in the company’s innovative approaches to rare diseases. As the study progresses, it may influence market dynamics, especially in the competitive gene therapy sector.
The study is ongoing, with further details available on the ClinicalTrials portal.