[2] OTL-103 is based on the lentiviral vector technology licensed from GlaxoSmithKline by Orchard Therapeutics in 2020.
[1] In 2019, the Food and Drug Administration granted OTL-103 Regenerative Medicine Advanced Therapy status.
[3][4] In the same year, the first clinical trial using OTL-103 for severe cases of Wiskott–Aldrich syndrome began at the San Raffaele Hospital in Milan, Italy.
[7] The cells migrate to the bone marrow, where they produce functional copies of Wiskott–Aldrich syndrome protein.
This would mitigate the symptoms of Wiskott-Aldrich syndrome, such as frequent infections, autoimmune disorders and cancers.