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July 24, 2017

Orchard Therapeutics Announces That OTL-101 Has Received a Rare Paediatric Disease Designation

London, UK — Orchard Therapeutics Limited (“Orchard”), a clinical-stage biotechnology company dedicated to bringing transformative autologous ex-vivo gene therapies to patients with rare diseases of high unmet medical need is delighted to announce today that the US Food and Drug Administration (FDA) granted a Rare Paediatric Disease Designation to OTL-101, its lead programme for the treatment of adenosine deaminase severe combined immunodeficiency, commonly known as ADA-SCID or “bubble baby” disease. OTL-101 is developed in collaboration with the University of California, Los Angeles (“UCLA”) and University College London / Great Ormond Street Hospital (“UCL” and “GOSH”).

ADA-SCID is a rare inherited disorder of the immune system. ADA-SCID is caused by mutations in the gene encoding for the enzyme adenosine deaminase, which result in a severe deficiency in white blood cells and life-threatening infections. In the absence of treatment, ADA-SCID is fatal within the first year of life.

To be granted Rare Paediatric Disease Designation, a drug must be designed for the treatment of a serious or life-threatening disease which affects less than 200,000 patients in the United States and which primarily includes patients aged between 0 and 18 years.

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