Orchard Therapeutics is a clinical-stage biotechnology company dedicated to bringing transformative autologous ex-vivo gene therapies to patients with rare diseases. The biotech has now received rare pediatric disease designation for OTL-101, a gene therapy for the treatment of the rare disease ADA-SCID from the FDA.
ADA-SCID or the “bubble baby” disease is a rare inherited disorder of the immune system, caused by mutations in the adenosine deaminase encoding gene, resulting in a severe deficiency in white blood cells and life-threatening infections.
Orchard’s gene therapy, OTL-101, follows a procedure wherein hematopoietic stem cells from the patient are harvested, genetically engineered outside of the body and reinfused, as cure for adenosine deaminase severe combined immunodeficiency (ADA-SCID).
The therapy tested in 40 patients so far, has shown promising results since the start of a Phase I/II trial last December.
Orchard is competing with GSK in the ADA-SCID market. GSK’s Strimvelis got an EU approval mid last year, and has since treated only one patient. The price tag, €594,000 — refundable if the therapy does not work — could account for the slow adoption.
Currently, the only option for children in the US with this disease is enzyme replacement therapy, which still leaves them at high risk of life-threatening infections.
