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OTL-201
- Development stage
- Phase 2
- Lead developer
- Orchard Therapeutics
- Modality
- Peripheral Blood HSCs → Hematopoietic Stem Cells → Adult Stem Cells → Stem Cell Therapies → Cell Therapies, Gene Therapies, Bone Marrow HSCs → Hematopoietic Stem Cells → Adult Stem Cells → Stem Cell Therapies → Cell Therapies
- Administration
- Intravenous
01
Overview
OTL-201 is an investigational ex vivo autologous hematopoietic stem cell (HSC) gene therapy developed for the treatment of mucopolysaccharidosis type IIIA (MPS-IIIA, also known as Sanfilippo syndrome type A), a rare and life-threatening metabolic disorder with no approved therapies. The therapy involves collecting a patient’s own CD34+ HSCs, which are then genetically modified outside the body using a lentiviral vector encoding a codon-optimized human N-sulfoglucosamine sulfohydrolase (SGSH) gene under the control of either PGK or CD11b promoters. These modified cells are infused back into the patient after myeloablative conditioning. The expressed SGSH enzyme is intended to restore deficient enzymatic activity in patients, reducing pathological accumulation of heparan sulfate and potentially improving neurocognitive outcomes[1][2][3][4][5][6][7][8].
- Other names
- cryopreserved autologous CD34+ cells transduced with a lentiviral vector containing the human SGSH gene
02
Targets
SGSH (N-sulphoglucosamine sulphohydrolase)
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