FDA Clearance Granted to Orchard Therapeutics

FDA Clearance Granted to Orchard Therapeutics

Global gene therapy leader, Orchard Therapeutics (Nasdaq: ORTX), has received FDA clearance for its Investigational New Drug application for a hematopoietic stem cell (HSC) gene therapy.

OTL-203 is being developed for the treatment of the Hurler subtype of mucopolysaccharidosis type I (MPS-IH).

Highlights

• Orchard’s proof-of-concept trial demonstrated that treatment with a single administration of OTL-203 has the potential to address a range of multisystemic manifestations of MPS-IH.
• The study is a multi-center, randomized, active controlled clinical trial of 40 patients.
• OTL-023 uses a modified virus to insert a functional copy of the alpha-L-iduronidase (IDUA) gene into a patient’s cells.

MPS-IH is a devastating rare, inherited neurometabolic disease that causes severe neurocognitive impairment, skeletal deformities, cardiovascular and pulmonary complications and impaired motor function.

OTL-203 has the potential to be an urgently needed new treatment option for the MPS-IH community.