Orchard’s pickings

How Orchard cell therapies could best competitors in ADA-SCID, MPS IIIA

Orchard Therapeutics Ltd.’s autologous stem cell therapies could improve upon more advanced cell and gene therapies that also were designed to produce durable responses by restoring production of missing enzymes in rare genetic diseases.

In severe combined immunodeficiency due to adenosine deaminase deficiency (ADA-SCID), Orchard thinks the vector it uses could give it an edge over GlaxoSmithKline plc’s Strimvelis, which is approved for the indication in Europe.

Orchard’s unnamed lead program uses an EFS lentiviral vector to transduce autologous CD34+ hematopoietic stem cells ex vivo

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