Modified Autologous Hematopoietic Stem Cells | Type IIIA | Phase I-II | Orchard Therapeutics

March 9, 2020

Trial Information

This study is the first in-human clinical trial to explore the safety, tolerability, and clinical efficacy of ex vivo gene therapy (autologous CD34+ cells transduced with a lentiviral vector containing the human SGSH gene) in MPSIIIA patients.

This study aims to recruit 3 to 5 patients with MPS IIIA who satisfy the inclusion and exclusion criteria and provide full consent, between 3 months and 24 months of age. The investigational medicinal product (IMP) will be a cell-based gene therapy that uses genetically modified autologous CD34+ haematopoietic stem cells transduced with a lentiviral vector containing the human SGSH gene. Patients will be followed up for a minimum of 3 years after gene therapy.

This trial has one location, in the United Kingdom.Read this clinical trial’s information on, which includes enrollment criteria, trial locations, and study contact information.

Trial Updates

June 2021

Orchard Therapeutics announced that it has fully enrolled 5 patients in its proof of concept study. Read more.

February 2021

Orchard Therapeutics announced “supportive initial OTL-201 biomarker data for mucopolysaccharidosis type IIIA (MPS-IIIA or Sanfilippo syndrome type A) in three patients.” Read more.

April 2020

Orchard Therapeutics announced that the first patient had been dosed with the open-label, proof-of-concept investigational study of OTL-201, an ex vivo autologous hematopoietic stem cell (HSC) gene therapy for the treatment of mucopolysaccharidosis type IIIA (MPS-IIIA).

Page last updated June 29, 2021

Related Posts