Orchard Therapeutics Inc ORTX announced multiple clinical and preclinical updates from its portfolio of investigational hematopoietic stem cell (HSC) gene therapies in neurometabolic and neurodegenerative disorders.
The data were presented at the American Society of Gene and Cell Therapy Annual Meeting.
The oral presentation of OTL-203 in mucopolysaccharidosis Type IH (MPS-IH) showcased new skeletal data for all eight proof-of-concept trial patients with a median follow-up of 3.78 years, ranging from 3.14 to 4.58 years, compared to a median of two years reported in the November 2021 New England Journal of Medicine publication.
As previously reported, all eight participants achieved the primary endpoint of supraphysiologic blood alpha-L-iduronidase (IDUA) activity. Growth velocity, cognition and motor function post-treatment were collected as secondary and exploratory endpoints.
Treatment with OTL-203 was generally well tolerated and demonstrated extensive metabolic correction over four years after treatment.
The company announced updated neurocognitive data for OTL-201 in MPS-IIIA, showing that with extended follow-up, four out of five patients continued to gain cognitive skills in line with development in healthy children.
Two patients were able to progress to a more advanced cognitive test.
Evidence of developmental gains was also observed in treated patients.
The company also shared preclinical data from OTL-204 in the progranulin form of frontotemporal dementia (GRN-FTD).
Data from in vivo studies indicate effective GRN protein delivery to the CNS of knockout mice transplanted with gene-modified HSCs.
Price Action: ORTX shares are up 7.47% at $5.47 on the last check Friday.
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