ORTX - Orchard Therapeutics announces interim data for OTL-203 in mucopolysaccharidosis

Orchard Therapeutics (ORTX) climbed ~14.3% yesterday after the company announced new data from its hematopoietic stem cell (“HSC”) gene therapies undergoing development for neurodegenerative disorders.The interim data included the clinical proof-of-concept results for OTL-203 for MPS-I (Mucopolysaccharidosis Type I). The Hurler subtype of MPS-I has limited treatment options. Its symptoms include neurocognitive impairment and skeletal deformities that lead to impaired growth in the first decade of life.Eight patients with MPS-IH have been treated with OTL-203 in the proof-of-concept study and follow-up in all patients has reached at least 12 months as of November 2020.In the study conducted at San Raffaele Telethon Institute for Gene Therapy (SR-Tiget) in Milan, Italy, OTL-203 has been generally well-tolerated with patents’ biomarker data indicating a rapid and sustained metabolic correction, the company said.Based on the Bayley scale, the new clinical results (n=8) indicate stable cognitive performance post-treatment in all patients with follow-up ranging from 6

For further details see:

Orchard Therapeutics announces interim data for OTL-203 in mucopolysaccharidosis