BioCentury
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...

BCIQ Company Profiles

Orchard Therapeutics plc

BCIQ Target Profiles

Adenosine deaminase (ADA)