WebOTL-203 for MPS-IH: As part of ongoing interactions with regulators, Orchard is incorporating recent feedback related to study design and clinical endpoints into a revised global registrational protocol, with study initiation now expected to occur in 2024. WebMar 2, 2024 · Treatment with OTL-203 has been generally well-tolerated with a safety profile consistent with the selected conditioning regimen. An oral presentation featuring supportive biomarker data from the first three patients in the ongoing OTL-201 POC study for mucopolysaccharidosis type IIIA (MPS-IIIA, or Sanfilippo syndrome) was also featured.
Orchard Therapeutics Announces FDA Clearance of IND Application for OTL …
WebJan 5, 2024 · About OTL-203 OTL-203 is an investigational hematopoietic stem cell gene therapy being developed for the treatment of MPS-IH. It uses a modified virus to insert a … WebMay 16, 2024 · OTL-203 is an ex vivo autologous hematopoietic stem cell gene therapy being studied for the treatment of MPS-I. Orchard was granted an exclusive worldwide … datong active antenna
Orchard Therapeutics Announces New England Journal of
WebFeb 10, 2024 · OTL-203 uses hematopoietic stem cells taken from the patient, then genetically modified using a lentiviral vector to express the IDUA gene, before being reinfused. RGX-111, meanwhile, uses an adeno-associated viral vector to deliver the gene directly to the brain, getting around a central problem with Aldurazyme, which cannot … WebMar 4, 2024 · Based on these proof-of-concept data, Orchard is planning the launch of a registration-enabling study for OTL-203 in MPS-IH by the end of 2024. The company will be meeting with the FDA and European Medicines Agency (EMA) before the end of the year to identify an optimal path forward for OTL-203. WebJan 5, 2024 · About OTL-203 OTL-203 is an investigational hematopoietic stem cell gene therapy being developed for the treatment of MPS-IH. It uses a modified virus to insert a … datong anf filter