, a longtime UCLA professor and researcher, is leading development of the one-time gene-therapy treatment.after treating 50 patients who have a type of severe combined immunodeficiency, or SCID, called ADA-SCID. All 50 kids were still alive at least two years after treatment, and over 95% required no additional treatment.on Kohn's therapy, returning the license to him earlier this year after failing to apply for regulatory approval.
"Right now, we are buying time," Paola Andrea Fernández de Soto Abdul-Rahim, the mother of a child with ADA-SCID, told Insider in May."We cannot wait forever." Kohn said UCLA and University College London, which control the intellectual property for this therapy, are talking with several commercial-stage gene-therapy companies about licensing the therapy out.
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