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The treatment, which Orchard designated OTL-101, addresses severe combined immunodeficiency due to adenosine deaminase deficiency, or ADA-SCID.

The disease at issue is severe combined immunodeficiency due to adenosine deaminase deficiency, or ADA-SCID.

As of last year, according to Scientific American, the FDA had approved nine gene therapies, which for the most part target rare physiological disorders, such as adenosine deaminase deficiency and lipoprotein lipase deficiency.

Gene therapy for immunodeficiency due to adenosine deaminase deficiency.

Prof Bobby Gaspar, from Great Ormond Street Hospital in London is working on a Medical Research Council trial using gene therapy as a treatment for adenosine deaminase deficiency - which also leads to immune problems.