Global Lysosomal Storage Diseases Therapeutics Market - Regional Analysis
Among regions, North America is estimated to hold a dominant position in the global lysosomal storage diseases therapeutics market over the forecast period. This is due to the increasing research and development activirties for treatment of lysosomal storage disease. For instance, on February 9, 2022, Regenxbio, a U.S. based biotechnology company presents additional positive interim data from phase I/II trial of RGX-121 for the treatment of Hunter Syndrome at 18th Annual WORLD Symposium 2022. The gene therapy was found to be well tolerated with no drug related serious adverse events across three dose level. Dose-dependent reductions in Cerebrospinal fluid biomarkers continued neurodevelopmental after RGX-121 administration and evidence of systemic enzyme expression and biomarker activity was observed. They are further planning expansion using commercial-scale Current good manufacturing practices (cGMP) material in 2022.
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