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Experimental MPS 1 Therapy Shows Biomarker Activity, Improved Function
REGENXBIO presented results from its ongoing Phase I/II trial of RGX-111, an investigational one-time gene therapy for people with Mucopolysaccharidosis Type I (MPS I). The data, presented at the 18th annual WORLDSymposium, represented six patients diagnosed with severe MPS I who were treated with RGX-111.
Mucopolysaccharidosis Type I is a rare, genetic condition that can affect many parts of the body. The condition stems from a mutation in the IDUA gene which provides instructions for producing an enzyme that is involved in the breakdown of large sugar molecules. In people with MPS I, the gene reduces or completely eliminates the enzymes needed for the breakdown of large sugar molecules, leading to the accumulation of these molecules in cells called lysosomes, whose function is to digest and recycle molecules. The lysosomes become enlarged, causing significant issues in multiple organs including decline in central nervous system (CNS) functioning.
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