ARTICLE | Clinical News
Orchard's ADA-SCID candidate gets rare pediatric disease designation
August 18, 2017 4:29 PM UTC
FDA granted rare pediatric disease designation to OTL-101 (EFS lentiviral gene therapy) from Orchard Therapeutics Ltd. (London, U.K.) to treat adenosine deaminase (ADA) severe combined immunodeficiency (ADA-SCID). The product also has Orphan Drug and breakthrough therapy designations...
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