ORTX - Orchard Therapeutics updates gene therapy data in metabolic disorders

2023-05-19 09:54:47 ET

British biotech Orchard Therapeutics ( NASDAQ: ORTX ) on Friday announced new clinical data for its hematopoietic stem cell (HSC) gene therapy candidates in metabolic disorders Mucopolysaccharidosis type I (MPS-I) and Mucopolysaccharidosis type IIIA (MPS-IIIA).

The updated readout shows new skeletal data for OTL-203, the investigational therapy for neurometabolic disease MPS-I, at a median follow-up of 3.78 years in eight proof-of-concept trial patients.

According to new data, the patients who had previously achieved the primary endpoint for a measure called supraphysiologic blood alpha-L-iduronidase demonstrated longitudinal growth in line with healthy children adjusted for age and gender.

Meanwhile, the readout for OTL-201, the investigational therapy for metabolic disease MPS-IIIA, indicates neurocognitive data for all five patients from the ongoing proof-of-concept study.

Highlights of updated results at a median follow-up of 2.5 years indicate that four out of five patients continued to gain cognitive skills in line with healthy children.

Concurrently, Orchard ( ORTX ) also disclosed pre-clinical data for its HSC gene therapy OTL-204, highlighting its impact on biomarkers, neuroinflammation, and microglial function in neuronal disorder, frontotemporal dementia (FTD).

The readouts will be part of a series of presentations selected for the ongoing 26th Annual Meeting of the American Society of Gene and Cell Therapy.

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• Orchard Therapeutics: MLD Opportunity Could Be Larger Than Initial Estimates

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