OTL-103

Content sourced from Wikipedia, licensed under CC BY-SA 3.0.

OTL-103 (GSK-2696275) is a gene therapy for Wiskott–Aldrich syndrome (WAS), a rare immune system disorder caused by mutations in the WAS gene that makes the WASp protein. It is being developed by Orchard Therapeutics together with GlaxoSmithKline (GSK).

The treatment uses the patient’s own blood stem cells (CD34+ cells) collected from bone marrow or blood, which are genetically modified with a lentiviral vector to produce a functional WASp, and then returned to the patient. The edited cells go to the bone marrow and start making WASp, aiming to reduce problems like frequent infections, autoimmune issues, and cancer risk.

OTL-103 is in Phase I/II clinical trials, with an expected completion around October 2025. The therapy uses lentiviral vector technology licensed from GSK to Orchard in 2020. In 2019, the FDA gave OTL-103 Regenerative Medicine Advanced Therapy (RMAT) designation. The first clinical trial for severe WAS began in 2019 at the San Raffaele Hospital in Milan, Italy. Orchard had planned to file a Biologics License Application with the FDA in 2021, but the filing was postponed to 2022 due to the COVID-19 pandemic.