Orchard Therapeutics is a fairly new gene therapy company that has a pipeline more appropriate for a bigger, more established biotech.

“People are surprised when they hear that actually Orchard has only been around for four years but we have one of the most advanced pipelines of gene therapy medicines in the world,” states Mark Rothera, CEO of the company, adding, “we have six clinical stage programs and we have three of those that we’re looking to file in the next two and a half years.”

The focus of Orchard Therapeutics is in ex vivo autologous hematopoietic stem cell gene therapy. Rothera noted, “that’s quite a mouthful but essentially what we’re taking is the hematopoietic stem cells or blood stem cells from patients, we’re inserting a working copy of the defective gene into those stem cells and then we’re infusing those back into patients and when they graft into the bone marrow that provides the opportunity for lifelong correction of the disease.”

Three programs they have in the clinical stage are metachromatic leukodystrophy (MLD), adenosine deaminase severe combined immunodeficiency (ADA-SCID), and Wiskott-Aldrich syndrome. All three conditions are severe, and often deadly conditions, that the company hopes can be cured by the use of their gene therapy programs.

For more information, visit https://www.orchard-tx.com/

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