Orchard Therapeutics has presented additional positive data from a proof-of-concept study of OTL-203 in MPS-IH at the European Society of Gene and Cell Therapy (ETSG) 2023.
The gene therapy company announced a range of interim clinical outcomes, in addition to the previously reported neurological and skeletal results, from its ongoing proof-of-concept (PoC) study of OTL-203, a hematopoietic stem cell (HSC) gene therapy in development for the treatment of the Hurler subtype of mucopolysaccharidosis type I (MPS-IH).
The data were presented at the European Society of Gene and Cell Therapy (ESGCT) 30th Annual Congress taking place October 24-27th, 2023, in Brussels.
MPS-I is a rare, inherited neurometabolic disease caused by a deficiency of the alpha-L-iduronidase (IDUA) lysosomal enzyme resulting in the accumulation of glycosaminoglycans (GAGs) in multiple organs, including the eyes, ears, heart, as well as the musculoskeletal and central nervous systems.
It is estimated to occur globally in approximately 1 in 100,000 live births. Approximately 60% of children born with MPS-I have the most severe subtype, MPS-IH, also called Hurler syndrome, and rarely live past the age of 10 when untreated.
Current treatment options for MPS-IH include allogeneic hematopoietic stem cell transplant (HSCT) and chronic enzyme replacement therapy (ERT), neither of which effectively address the broad range of clinical manifestations of the disease.
“These positive data presented at ESGCT add to the growing body of evidence underscoring the potential of a one-time HSC gene therapy to correct a range of disease manifestations not effectively addressed by the current standard of care,” said Leslie Meltzer, Ph.D., Chief Medical Officer of Orchard Therapeutics. “The complications associated with MPS-IH involve multiple organ systems and have an adverse impact on patients’ quality of life. We continue to be encouraged by these results from our proof-of-concept study and look forward to initiating our global registrational trial later this year.”
In the single-center PoC study, eight patients diagnosed with MPS-IH were treated at Ospedale San Raffaele in Milan, Italy with investigational OTL-203 between July 2018 and December 2019. Interim results published in The New England Journal of Medicine showed all patients had stable cognitive performance post-treatment.
In addition, all participants had progressed along expected growth percentiles of healthy children and exhibited longitudinal growth that was considered within the normal range adjusted for age and gender.
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