Orchard Therapeutics Reports OTL-201 Initial Clinical Data in Sanfilippo Syndrome Type A (MPS-IIIA)
BOSTON and LONDON,
“The data presented yesterday represents encouraging progress for patients and families living with MPS-IIIA, a progressive, life-threatening metabolic disease with no approved treatment options,” said Professor
Initial Study Results
- The treatment was generally well-tolerated with no treatment related adverse events or serious adverse events to date.
- Evidence of hematological engraftment as suggested by recovery of neutrophils and platelets post myeloablative conditioning.
- N-sulphoglucosamine sulphohydrolase (SGSH) enzyme expression reached supra-physiological levels in plasma, total leukocytes and multiple cell subpopulations, including CD3+ and CD15+ cells, within 3 months of receiving OTL-201.
- Reduction of urinary heparan sulfate from 60.8 mg/mmol creatinine at baseline to the normal range by three months post-treatment with gene therapy.
Three patients have been treated in the ongoing proof-of-concept study, which is being sponsored and conducted by
About OTL-201 and MPS-IIIA
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Director, Investor Relations
Manager, Corporate Communications
1 Lavery, C., Hendriksz,
Source: Orchard Therapeutics (Europe) Limited