The first attempts at human gene therapy were made almost 10 years ago in children with a form of severe combined immunodeficiency (SCID) caused by defects in adenosine deaminase (ADA), a purine ...

European regulatory bodies have given a nod to one gene therapy product of primary immunodeficiency (PID) to cure ADA SCID, essentially, Strimvelis. Interestingly, SCID was the first disease known ...

The London-based company is developing the gene therapy for adenosine deaminase severe combined immunodeficiency, commonly known as ADA-SCID or “bubble baby” disease. OTL-101 is similar to GSK ...

Great Ormond Street Hospital (GOSH) in London is piloting the approach with a lentiviral gene therapy for the rare genetic disorder ADA-SCID, which causes children to be born without a properly ...