Gene Therapy Pipeline Update: Orchard Therapeutics Presents Data on Metachromatic Leukodystrophy

Orchard Therapeutics, a commercial-stage biopharmaceutical company, announced initial results from a clinical trial (NCT03392987) with a cryopreserved formulation of OTL-200, gene therapy in development for the treatment of metachromatic leukodystrophy (MLD). Get the details of the results here.

What is OTL-200: OTL-200 is autologous CD34+ cells transduced with a lentiviral vector containing human arylsulfatase A (ARSA) complementary deoxyribonucleic acid (cDNA) used for the treatment of MLD.

What is metachromatic leukodystrophy (MLD): MLD is an autosomal recessive lysosomal storage disorder (LSD) characterized by severe and progressive demyelination affecting the central and peripheral nervous system. This condition is called metachromatic leukodystrophy because when viewed under a microscope, sulfatide accumulation in cells appears as granules that are colored differently than other cellular material (metachromatic).

What is the prevalence of metachromatic leukodystrophy (MLD): The true prevalence rate of MLD is unknown, but is estimated to be between 1 in 40,000 and 1 in 160,000. The Navajo also have a higher prevalence rate of 1 in every 2,500 people. In certain populations in the Middle East, these numbers may be even higher.

Since the drug is being developed for a rare disease, has it received any designations: The drug has received two designations:

• Orphan Drug Designation (2007, EMA, MLD)
• Rare Pediatric Disease Designation (USFDA, 2018, MLD)

What is the overall gene therapy pipeline like for metachromatic leukodystrophy (MLD): As per a recent report by Roots Analysis, there are not many gene therapies that are currently being developed for MLD.

To get more insights about the gene therapy pipeline, check out the detailed report here.