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Orchard Therapeutics' MPS-I Gene Therapy Shows Encouraging Action In Early Study, Shares Spike

  • Orchard Therapeutics Plc (NASDAQ: ORTX) gains in premarket in reaction to positive interim data from a proof-of-concept study evaluating its gene therapy OTL-203 in Mucopolysaccharidosis type I (MPS-I). All eight patients treated with OTL-203 showed stable cognitive function, motor function, and growth within the normal range at multiple data points post-treatment.
  • OTL-203 treatment demonstrated rapid and sustained metabolic correction with all patients achieving supra-physiological IDUA expression in dried blood spot samples at 12 months (a primary efficacy endpoint).
  • FDA and European Medicines Agency (EMA) have accepted Orchard's request for parallel scientific advice to seek feedback in advance of initiating a registrational study for OTL-203 by this year-end.
  • The company also announced encouraging preliminary biomarker data for OTL-201 in three Mucopolysaccharidosis type IIIA (MPS-IIIA) patients, showing promising tolerability, engraftment, and biomarker data over the initial three-month follow-up period. Recently, a fourth patient was enrolled in the study. The enrollment could complete (n=5) this year, and additional interim data expected later this year.
  • IR webinar is scheduled today at 4:30 p.m. ET to review OTL-203 and OTL-201 data.
  • Last year in December, the company received its first product approval for Libmeldy in Europe for eligible patients with early-onset metachromatic leukodystrophy.
  • Price Action: ORTX jumped 10% at $8.31 in premarket trading on the last check Tuesday.

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Posted-In: European Medicines Agency (EMA) phase 1Biotech News Small Cap FDA General

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