Orchard Therapeutics Announces Positive Clinical and Preclinical Data in Programs Targeting Neurometabolic and CNS Disorders at ASGCT
Retrieved on:
Friday, May 19, 2023
Kaufman, UNIPD, Prefrontal cortex, Cerebrospinal fluid, Plasma, Motion, Frasier, Protein, Thalamus, Acquisition, Patient, Atidarsagene autotemcel, Randomized controlled trial, The New England Journal of Medicine, Cell, Disease, IDUA, UOM, Enzyme replacement therapy, Phenotype, DSM-IV codes, Tissue, University, Hip, Gene, Orchard, GRN, Dementia, San Raffaele Hospital, Growth, HSC, Cognition, Natural history, Kyphosis, Biomarker, MPS, Șaeș, Frontotemporal dementia, Webcast, Mortality, Therapy, Central nervous system, Doctor of Philosophy, Speech, HSCT, CNS, EDT, Health, MRI, SGSH, Dysplasia, Society, FTD, Finnish heritage disease, Hippocampus, Medical imaging, Pharmaceutical industry, Vaccine, Medical device, Nursing, Dentistry
BOSTON and LONDON, May 19, 2023 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today announced multiple clinical and preclinical updates from its portfolio of investigational hematopoietic stem cell (HSC) gene therapies in neurometabolic and neurodegenerative disorders. The data are being featured in several oral presentations at the ongoing 26th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT) in Los Angeles.
Key Points:
- The data are being featured in several oral presentations at the ongoing 26th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT) in Los Angeles.
- At baseline, most patients presented with severe joint range of motion impairment, severe acetabular (hip) dysplasia and varying degrees of dorso-lumbar kyphosis.
- Treatment with OTL-203 was generally well tolerated and demonstrated extensive metabolic correction over four years after treatment.
- Data from in vivo studies indicate effective GRN protein delivery to the CNS of knockout mice transplanted with gene-modified HSCs.