- In July 2021, Denali Therapeutics Inc. reported promising results from their clinical study evaluating ETV: IDS, an investigational brain-penetrant enzyme replacement therapy for treating the peripheral manifestations of Hunter Syndrome. These results are set to be presented at the 16th International Symposium on MPS and Related Diseases
- In December 2023, Orchard Therapeutics has been granted priority review by the US Food and Drug Administration (FDA) for its gene therapy OTL-203 targeting Hurler Syndrome. This designation provides several expedited drug development benefits, designed to accelerate the progress of treatments for serious conditions. Orchard is advancing a hematopoietic stem cell (HSC) gene therapy to address Hurler Syndrome, a rare lysosomal storage disorder also known as mucopolysaccharidosis type I
