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A rare, aggressive form in children results from a mutation in a gene for a metabolic enzyme called adenosine deaminase 2.

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.

To make a more refined tool, Beverly Mok, a graduate student in Liu’s lab, attached the toxin-derived deaminase from Mougous’s lab to a TALE, a protein that can enter mitochondria and, like gRNA, leads the complex to the target.