OTL-201

DB17652ExperimentalGene Therapies

OTL-201 is an investigational hematopoietic stem cell (HSC) gene therapy being developed by Orchard Therapeutics for the treatment of mucopolysaccharidosis type IIIA (MPS-IIIA). It consists of a lentiviral vector to... It consists of a lentiviral vector to insert a functional copy of the human N-Sulfoglucosamine Sulfohydrolase (SGSH) gene into autologous CD34+ hematopoietic stem cells.

Code names: OTL-201

What you can answer from here — as of April 02, 2025

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Which companies are running trials of OTL-201, in which indications and phases, and which of those programs are still active?

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